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5 Levels of Substance Use and Abuse

What are the Different Levels of Substance Abuse?

Do you or a loved one use drug substances? Use the following categories to determine the seriousness of your drug use situation:

1. Experimental Use: trying substance once or twice.

2. Recreational Use: using a substance socially, only with friends or at a party.

3. Circumstantial Use: using a substance to escape or avoid personal problems or life circumstances OR using a substance to get through a challenging situation (e.g.: taking caffeine-laced pills to stay awake to study for exams).

4. Intensified Use: increasing over a long period of time. This describes the regular user who experiences on-going physical symptoms because of their drug use (sore throat, cough, runny nose, nosebleeds, weight loss, irritability, etc.).

5. Compulsive Use: True addicts who can’t voluntarily give up their substance abuse. They can no longer choose not to use, and suffer significant life and heath disruption because of their addiction.

If you or a loved one have a substance abuse problem, or if you think you’re becoming and addict, contact us at (844) 543-3242 . We can help.

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Phases of Drug Use: From Experimentation to a Substance Use Disorder

In 2016, roughly 7.4 million  people over the age of 11 were affected by substance misuse or addiction. Everyone with a substance use disorder has a unique story about how they began to get involved with drugs. While many people with addiction can relate to each other, it’s important to remember that everyone’s experience is personal. Still, most people’s addiction spurred from several phases ranging from their first introduction to their spiral into dependency. Examining the different periods of drug use that eventually lead to addiction can help people understand where recreational drug use ends and where a dangerous substance use disorder begins.

• Introduction

A lot of people experiment with drugs or alcohol before reaching adulthood. The natural curiosity and rebellion of teenagers and young people usually cause the initiation into trying substances for the first time. With the help of some added peer pressure and desire to fit in, young people are most often at the most significant risk of exposure in early life. Because adolescents still don’t have fully developed prefrontal lobes in their brains, their judgment and impulse control is often shaky, making them vulnerable for potential misuse or addiction. A survey conducted by the Substance Abuse and Mental Health Services Administration (SAMHSA) showed that 2.8 million people above the age of 12 used an illicit drug for the first time in 2013. Every day, about 4,220 people under the age of 18 will use drugs and alcohol for the first time.

However, even people who abstained from an early introduction to substance use can still experience this stage later on in life. As adults, we deal with many stressful situations on the job, at home, and even personal struggles with mental health. This can prompt some people to self-medicate with drugs or alcohol, especially when they are emotionally compromised. Even those who are not dealing with personal issues may want to fit in with co-workers, friends, or even family members who may be misusing drugs recreationally. After this first introduction to a substance, people generally will decide to experiment further or decide against it.

• Experimentation

Upon the initial first try, drug use can quickly become experimental, occurring more frequently. Some people start this by only using drugs in a specific situation, like a party or during events like concerts. This exploratory phase is usually a more social matter that involves drugs as a way to relax or have some fun. At this point, people generally don’t seem to think about these substances too much other than right before the time they plan to use them. Cravings generally do not exist at this phase, but the anticipation of the social event of using the drug may become a factor. At this time people will decide to consciously consume drugs, understanding the potential consequences, or they will take the drugs impulsively, without much pre-planning. Still, their thoughts and concerns aren’t centered on their drug use, and will only take part when it is convenient.

• Recreational Use

Recreational drug use is more common than most people realize. Some use drugs to “party,” while others use them to unwind. When someone is regularly buying and ingesting drugs, it qualifies as recreational use that can be as frequent as every weekend or several times a week. At this stage, people are more aware of their intake and usually have a ritual surrounding their methods of obtaining and preparing the drugs. Sometimes they may miss work or school due to the after-effects of using the drugs. This drug use often goes hand-in-hand with people who are looking for drugs to escape their situations or cope with other issues. Others may use drugs regularly because they feel that it makes their social interactions much more exciting and enjoyable. At this point, they prefer to use drugs socially rather than stay sober.

When someone enters this phase, their drug use is considered to be risky and has become a problem. People that are misusing substances will sometimes have run-ins with dangerous situations like DWIs or other negative legal consequences. Their performance at work or school will be dwindling, and their relationships with loved ones are negatively impacted by their frequently unpredictable behavior. When someone is misusing drugs, they are beyond the point of regular recreational use and are walking the fine line between risky use and a substance use disorder. This is often the stage where people will first be approached by someone concerned about their frequent intoxication.

Drug dependency can be split into three parts: tolerance, physical dependence, and psychological dependence. When someone builds tolerance, they will require more and more of the drug they are misusing to achieve the desired effect. When a physical dependence occurs, it means that the person will go through withdrawal responses when they aren’t able to ingest their drug of choice. These withdrawals can range from light cravings to severe symptoms that can leave the user in significant pain. Psychological dependence happens when cravings are not only controlled by painful or bothersome withdrawals, but by the mental drive to use drugs. Some people who prefer to be ‘high’ constantly or under the influence are not able to face simple day-to-day tasks without their drug and require it to function. These three steps are cumulative and usually progress within several weeks or months of each other depending on the frequency of use.

• Substance Use Disorder (SUD)

When someone’s drug use has spiraled out of control, they will experience the following symptoms to fit the SUD criteria:

• They cannot control their use
• They continue to use despite drastically negative consequences
• They cannot function daily without their drug
• They have abandoned personal relationships and hobbies
• They have issues with the law
• Their health and life is in danger

Addiction is a disease that has developed from these long phases of drug use, carefully rewiring the brain’s reward pathway to constantly prioritize drug use over all else. Even when this person has attempted to cease their drug use, they have been unsuccessful and experienced dangerous relapses, returning to their previous behaviors. Usually, at this phase, people rarely can feel the high they once desired and continue to use the drug to keep withdrawal at bay.

The last stage of the substance use disorder progression is treatment , remembering however that treatment can begin at any phase of drug use. There are several treatment options for people who are faced with addiction that involve medication, behavioral therapy, and other counseling to help get them on the road to recovery.

Sources: https://www.stanfordchildrens.org/en/topic/default?id=stages-of-substance-abuse-1-3060

https://www.drugs.ie/drugs_info/about_drugs/the_nature_and_states_of_drug_use/

Related Posts About The Phases Of Drug Use

• How Drug Addiction Affects Relationships
• Can Boredom Lead to a Drug Addiction?
• Medical Consequences of Substance Use Disorders
• Signs of Pill Addiction

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The Clinical Team at Health Care Resource Centers is our team of physicians and medical directors within the organization. HCRC is a CARF accredited organization and has been providing addiction treatment services for over 32 years in the New England area.

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Patterns of use

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Drug use can be legal or illegal, high or low risk and beneficial or problematic depending on the circumstances.

Controlled use is considered sufficiently temperate to avoid harmful intoxication or dangerous use and the development of problematic dependence on a substance or substances.

Hazardous use refers drug use that puts ones safety and health in jeopardy.  This can include:

• Using at excessive level of a substance over a defined period
• Using a combination of substances which can interact dangerously
• Using substances in an unsafe manner such as sharing injecting equipment 

The UN (1997) also makes a distinction between first use and subsequent use.

First use is always experimental in the sense that a person is experiencing the effects of the drug for the first time.

Experimentation may involve only a single exposure to a drug’s effects. It may also involve several exposures, most often over the short term. Through experimentation a person might decide that the drug does not suit them and choose not to continue. Others who decide to continue using will adopt a pattern of use that works for them. People drug use patterns often change with time and circumstance and as tolerance to drug effects develop. Some people’s pattern of use develops beyond their control.

A range of substance use patterns, drawn from a number of sources (YSAS, 2001; Denning et al., 2004; Shafer, 1973), are represented in the following ‘continuum of usage patterns’. 

Continuum of Usage Patterns Non Use People choose not to use drugs for a number of reasons.  Some of these reasons relate to religion, culture or health.  In rare cases even non-use can be a very risky choice when drug use is required for “medical/therapeutic” purposes. First Use Experimental use: All people who use substances will go through a period of experimentation.  This process will determine how and if a person will use the drug subsequently (YSAS 2002). Subsequent Use Occasional use: This form of drug use is often circumstantial.  For example, a person may only drink alcohol at social functions or may only take paracetamol when they have a headache.  The more problematic form of occasional use would be binge style use, which can be quite intensive in short periods or bursts.  This too, is most often circumstantial (YSAS 2002). Regular social/recreational use: A person who uses drugs on a regular basis may or may not experience problems. Social/recreational use is likely to feature the least opportunity for harm. Regular, controlled users of drugs are not considered to be dependent on their drug of choice as they have the ability to moderate or change their use as circumstances dictate (YSAS 2002). Regular intensive use: There is no standardised method of categorising use as intensive or heavy. It depends on a range of factors including health and the norms of one’s group or culture.  People can consume large amounts of substances on a regular basis in a controlled way, without developing dependence. Alternatively this form of regular use can be hazardous and meet the criteria for ‘substance abuse’ (see DSM IV and ICD-10 classification) Dependent use: Some people come to depend or rely on one or more drugs in order to function and manage their circumstances.  People can be dependent on a drug and manage without problems occurring or engaging in high-risk behaviour.  If a person’s drug use does begin to impinge on other areas of their life such as family or work and the person is unable to change or moderate their use, they would be thought of as being dependent on that drug (see DSM IV and ICD-10 classification)

Figure 1 demonstrates how subsequent use will vary depending on the circumstances of each individual.  People may move from one type of use to another. 

Classifying substance dependence and abuse

The World Health Organisation International Classification of Diseases (ICD10) and the Diagnostic Statistical Manual (DSM) IV provide a complimentary set of criteria for defining substance dependence and abuse. 

ICD-10: F1x.2 Dependence syndrome A definite diagnosis of dependence should usually be made only if three or more of the following have been present together at some time during the previous year: (a) a strong desire or sense of compulsion to take the substance; (b) difficulties in controlling substance-taking behaviour in terms of its onset, termination, or levels of use;                     (c) a physiological withdrawal state (see F1x.3 and F1x.4) when substance use has ceased or been reduced, as evidenced by: the characteristic withdrawal syndrome for the substance; or use of the same (or a closely related) substance with the intention of relieving or avoiding withdrawal symptoms; (d) evidence of tolerance, such that increased doses of the psychoactive substances are required in order to achieve effects originally produced by lower doses (clear examples of this are found in alcohol- and opiate-dependent individuals who may take daily doses sufficient to incapacitate or kill nontolerant users); (e) progressive neglect of alternative pleasures or interests because of psychoactive substance use, increased amount of time necessary to obtain or take the substance or to recover from its effects; (f) persisting with substance use despite clear evidence of overtly harmful consequences, such as harm to the liver through excessive drinking, depressive mood states consequent to periods of heavy substance use, or drug-related impairment of cognitive functioning; efforts should be made to determine that the user was actually, or could be expected to be, aware of the nature and extent of the harm. Narrowing of the personal repertoire of patterns of psychoactive substance use has also been described as a characteristic feature (e.g. a tendency to drink alcoholic drinks in the same way on weekdays and weekends, regardless of social constraints that determine appropriate drinking behaviour). It is an essential characteristic of the dependence syndrome that either psychoactive substance taking or a desire to take a particular substance should be present; the subjective awareness of compulsion to use drugs is most commonly seen during attempts to stop or control substance use. This diagnostic requirement would exclude, for instance, surgical patients given opioid drugs for the relief of pain, who may show signs of an opioid withdrawal state when drugs are not given but who have no desire to continue taking drugs. http://www.who.int/substance_abuse/terminology/ICD10ClinicalDiagnosis.pdf

DSM‐IV‐TR Criteria for Substance Dependence A maladaptive pattern of substance use, leading to clinically significant impairment or distress, as manifested by three (or more) of the following, occurring at any time in the same 12‐month period (emphasis ours): 1. Tolerance, as defined by either of the following: A need for markedly increased amounts of the substance to achieve intoxication or desired effect or Markedly diminished effect with continued use of the same amount of the substance 2. Withdrawal, as manifested by either of the following: The characteristic withdrawal syndrome for the substance or The same (or a closely related) substance is taken to relieve or avoid withdrawal symptoms 3. The substance is often taken in larger amounts or over a longer period than was intended 4. There is a persistent desire or unsuccessful efforts to cut down or control substance use 5. A great deal of time is spent on activities necessary to obtain the substance (e.g., visiting multiple doctors or driving long distances), use the substance (e.g., chain‐smoking), or recover from its effects 6. Important social, occupational, or recreational activities are given up or reduced because of substance use 7. The substance use is continued despite knowledge of having a persistent physical or psychological problem that is likely to have been caused or exacerbated by the substance (e.g., current cocaine use despite recognition of cocaine‐induced depression, or continued drinking despite recognition that an ulcer was made worse by alcohol consumption) Criteria for Substance Abuse A maladaptive pattern of substance use leading to clinically significant impairment or distress, as manifested by one (or more) of the following, occurring within a 12‐month period: Recurrent substance use resulting in a failure to fulfil major role obligations at work, school, or home (e.g., repeated absences or poor work performance related to substance use; substance‐related absences, suspensions, or expulsions from school; neglect of children or household Recurrent substance use in situations in which it is physically hazardous (e.g., driving an automobile or operating a machine when impaired by substance use) Recurrent substance‐related legal problems (e.g., arrests for substance‐related disorderly conduct) Continued substance use despite having persistent or recurrent social or interpersonal problems caused or exacerbated by the effects of the substance (e.g., arguments with spouse about consequence of intoxication, physical fights) http://www.ncbi.nlm.nih.gov/books/NBK64247/

Ways to Access Experimental Cancer Drugs

The most common and preferred way to access an experimental drug is through a clinical trial.

An experimental drug is one that has been tested in the lab and with animals and approved for testing in people by the U.S. Food and Drug Administration (FDA). But, such a drug can’t yet be advertised, sold, or prescribed. Experimental drugs may also be called “ investigational drugs.”

Experimental drugs may be available through clinical trials , expanded access , or right to try.

Clinical trials

The most common and preferred way to access an experimental drug is through a clinical trial . Clinical trials are research studies that involve people. Trials are carefully designed to answer scientific questions about the new drug. People who take part are fully informed and carefully monitored. The data that are collected during a clinical trial help researchers learn how the drug behaves in people, if it is safe and effective, or how it compares to standard treatments .

See Clinical Trials Information for Patients and Caregivers to learn more about clinical trials and how to find one for which you may be eligible.

Expanded access (compassionate use)

Expanded access, which is also called compassionate use , is the use of an experimental drug outside of clinical trials to treat people with serious or life-threatening diseases. It is regulated by the FDA. If you meet certain criteria, you might be able to obtain an experimental drug through expanded access. To qualify, you must

• have an illness that is life-threatening
• have no standard treatments available
• not be eligible for a clinical trial

How to obtain a drug through expanded access

The only way to obtain a drug through expanded access is with the help of your doctor. Your doctor will need to

• Ask the company that is developing and testing the experimental drug if it is willing to provide it for you. The company may not be willing to provide the drug if it believes that doing so is against its interests or if there isn’t enough drug available.
• Apply to the FDA, if the drug company agrees to supply the drug. Your doctor should refer to Expanded Access: Information for Physicians on the FDA site for complete information.

How your safety is protected if you obtain a drug through expanded access

Several measures help protect your safety if you are treated with a drug through expanded access.

• An Institutional Review Board or its representative must approve the use.
• The FDA must approve the use.
• You will go through an informed consent process to ensure that you understand the potential risks and benefits of taking the experimental drug.

Right to try

Right to try, like expanded access, allows the use of an experimental drug outside of clinical trials to treat people with serious or life-threatening diseases. But unlike expanded access, it is not regulated by the FDA. 

If you meet certain criteria, you might be able to obtain an experimental drug through right to try. These criteria include

• You have an illness that is life-threatening.
• There are no standard treatments available for you.
• You are not eligible for a clinical trial of the drug.

The drug that you are seeking must meet certain criteria, as well. These criteria include

• It has already been studied in a phase 1 clinical trial. 
• It has not been approved by the FDA for any use.
• The company that owns it has applied for FDA approval or is running a clinical trial to further study the drug before seeking approval. 

How to obtain a drug through right to try

The only way to obtain a drug through right to try is with the help of your doctor. Your doctor will need to consult with the company that is developing and testing the experimental drug to find out

• if the drug is eligible for use through right to try
• if the company is willing to provide the drug, which it is not required to do

How your safety is protected if you obtain a drug through right to try

Before you are treated with a drug through right to try, you will go through an informed consent process to ensure that you understand the potential risks and benefits of taking the experimental drug.  

Deciding on treatment with an experimental drug

Your doctor is in the best position to help you understand all your treatment options.  

As you think about your options, keep in mind that there may be unknown serious side effects with experimental drugs. Also, cost may be an issue. Federal law requires most insurance plans to cover routine patient care costs related to taking part in a clinical trial. But your insurance may not cover the cost of an experimental treatment received outside of a trial.

Our list of Questions to Ask Your Doctor about Clinical Trials may help you talk with your doctor about taking part in a clinical trial.

• Open access
• Published: 23 June 2020

“Right-to-Try” experimental drugs: an overview

• Vijay Mahant 1  

Journal of Translational Medicine volume  18 , Article number:  253 ( 2020 ) Cite this article

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The “Right-to-Try” experimental drugs act passed by Donald Trump in 2018 provides an opportunity of early access to experimental drugs for the treatment of life-threatening diseases and a potential boon to many young and under-capitalized biotechnology or pharmaceutical companies. The pros and cons of experimental drugs, including a number of “cutting edge” scientific, clinical, and a number of synergistic approaches such as artificial intelligence, machine learning, big data, data refineries, electronic health records, data driven clinical decisions and risk mitigation are reviewed.

Over a century ago, most medications were the “wild west” with alleged therapeutic benefits. Most such medications sold to the public offered negligible or no evidence-based therapeutic efficacy and safety to the patient at large, except the “placebo benefit”—and hope—at the best. The drugs were unregulated and sold to the public in the USA and many parts of the world. To protect the public against the sale of misbranded, mislabeled and/or adulterated foods and drugs, the US in 1906 enacted [ 1 ] the “Pure Food and Drug Act” and this resulted in the establishment of the federal agency, the Food and Drug Administration (FDA).

Historical overview

The “Right-to-Try” experimental drugs, however, originated [ 2 , 3 , 4 ] from the Abigail Alliance for Better Access to Developmental Drugs versus von Eschenbach. Due to Abigail’s high expression of EGFR, her oncologist recommended Abigail for her terminal head and neck cancer to try an investigational EGFR-targeted drug, C222 (Erbitux), which was then undergoing clinical trial for the treatment of colorectal cancer. Due to her ineligibility to participate in the clinical trials and denial by the FDA, Abigail’s father, Frank Burroughs sued the FDA in 2003 for access to the experimental drug, Erbitux, on the pretext that an investigational drug by terminally ill patients after phase I approval was a constitutional right. Abigail’s tragic story was one of the primary precursors and a “catalyst” that inspired patients and non-patients, including advocacy groups for access to unapproved therapies by the FDA.

In May 2018, President Donald Trump signed the “Right-to-Try” Act [ 5 ]. The legislation overcame many of the regulatory barriers, limited the risks to the sponsor while implementation of the act inherently burdened the sponsor. The “Right-to-Try” legislation is in essence a derivative of the Expanded Access Programs (EAPs). Advocates such as patients, families, friends and advocacy groups of the “Right-to-Try” legislation argue that the legislation is in line within the pre-existing framework of EAPs and that the legislation: (i) provides a “streamlined” avenue for making eligible drugs available to eligible patients with no other options; (ii) it increases patient’s engagement; (iii) it is a patient’s journey of self-actualization; (iv) it empowers the patient about his or her own health, well-being and quality of life; (v) it provides optimism and access to novel interventions with potential therapeutic benefits that may prolong life and improve quality of life; and (vi) the patient can be treated in the USA with valuable family time, more comfort and fewer risks than being treated overseas. The critics, on the other hand, argue: (i) there is an inherent safety risk that may potentially cause more harm to the patient or even death than the benefit because the experimental drug did not undergo rigorous testing; (ii) there is a lack of oversight by the FDA, except posting of the consolidated annual summary report; (iii) the patient in most cases has limited understanding of the informed consent due to complexity and confusion of the medical terminology used in the consent form; (iv) there are therapeutic misconceptions combined with high expectations and optimism by the patient; (v) there is potentially a considerable financial burden by the patient or the patient’s family because payors currently do not provide coverage and deny hospice care; (vi) there is a potential loss of trust in the regulatory agency, the sponsor and the health care provider; and (vii) there is a liability “immunity” for the health-care provider, including the drug sponsor for potential negative outcomes of the treatment unless the medical provider and the sponsor were engaged in “gross negligence, reckless or “wilful misconduct.”

Some of the major inherent limitations and often overlooked about the “Right-to-Try” experimental drugs are: (i) patient’s vulnerability due to lack of oversight by the FDA; (ii) there is a lack of clinical study protocol, including the lack of sufficient statistical power to detect the intended effect(s); (iii) the information is collected in a “piece-meal”; and (iv) a lack of systematic reporting about efficacy and the safety of the experimental drug that may potentially result in limited information for the health and safety of the public. Some of these issues may be addressed and resolved by utilizing EHR systems. EHR systems are maintained by health care providers and health care organizations for delivering patient care. EHR systems can thus easily lend themselves for integrating real-time electronic health care information about the patient across multiple health care providers.

One of the most important considerations and occasionally overlooked is comorbidity; it is quite common among cancer patients and it can potentially affect the treatment outcome, worsen the adverse effects due to polypharmacy and may even shorten the life. The prognosis of patients with comorbidity is often poor survival combined with poor quality of life and higher financial costs. The use of experimental drugs in patients with morbidity may thus be risky or limited unless the experimental drug has gone through further rigorous testing and/or the experimental drug is paired synergistically with an FDA-approved drug and appropriate tools are employed for monitoring efficacy and safety of the therapy. Treatment of metastatic cancer patients with comorbidity using experimental drugs would be even more challenging and riskier with many implications that may warrant further considerations that are in the best interest of the patient’s health, well-being and life, including additional financial burden.

The “landscape, the ecosystem and the dynamics”

The implications of ethics, law, regulations, government policies, constitutional rights by terminal ill patients, patient advocacy groups, including stakeholders about the pros and cons echoed through the “ecosystem” of early access to investigational drugs. Under the “Right-to-Try” legislation, the eligibility to participate include: (i) the patient must have been diagnosed with a debilitating or life-threatening disease; (ii) the patient must have failed all standard of care treatments; (iii) the experimental or the investigational drug must have completed at least phase 1 trial; (iv) the patient must have signed an informed consent [ 6 ]; and (vi) the pharmaceutical company must be able to provide the experimental drug to the patient. Due to the potentially negative outcome about the therapeutic efficacy combined with the safety issues, most sponsors developing medications for life-threatening diseases have had reservations about participating in expanded access or the “Right-to-Try” programs. To de-risk the potential negative outcomes and the implications combined with an opportunity to target a much larger number of patients than to a few eligible patients under the early access programs, the sponsors’ primary goal has been to have full FDA approval of the drug. The drug approval process, of course, is lengthy and highly risky due to potential clinical trial failures along each step of the approval process. It is an expensive process—currently, estimated to be between US$ 1.9–2.5 billion [ 7 , 8 ].

On the other hand, China for almost two decades approved a number of experimental drugs. From 2003 to 2005, the SFDA approved H101, experimental oncolytic viral therapy for head and neck cancer, an angiogenic Endostar inhibitor for treating non-small cell lung cancer and Gendicine for treating head and neck cancer [ 9 , 10 , 11 ]. However, in December, 2019, the Drug Administration Law (DAL) by the SFDA came into effect [ 12 ]. The DAL is likely to have an impact on experimental drugs because it addresses several issues such as public health concerns, drug innovation, drug safety and drug accessibility. In the “wake” of the recent epidemics such as Ebola, MERS caused by coronavirus (MERS-CoV) and more recently the outbreak of the coronavirus, SARS-CoV2 in 2019–2020, the use of “emergency drugs and compassionate use of experiential drugs” may warrant further considerations and the options available in an epidemic or a pandemic. Gilead Sciences’ experimental drug (at the time of writing), remdesivir, is one of the most promising drug candidates that may be effective against SARS-CoV2. The drug exhibits broad-spectrum antiviral activity against a number of RNA viruses including the Ebola virus by interfering with the viral polymerase enzyme. Remdesivir is currently being used as an “emergency experimental drug” on compassionate basis while randomized controlled studies are underway [ 13 ].

The risks and benefits of phase 1 oncology trials from 1991 to 2002 have been reviewed [ 14 ] involving a total of 460 trials and 11,935 participants. The participants were tested for toxicity and 10,402 participants were assessed for therapeutic efficacy. The overall response rate was reported to be 10.6% with considerable variations among trials. The classic phase 1 trials using single investigational chemotherapeutic drug represented 20% of the trials with a response rate of 4.4%. On the other hand, the trials that included at least one FDA-approved anticancer drug consisted of 46.3% of the trials and the response rate was 17.8%. The overall death due to toxicity was found to be 0.49%. The above study demonstrated the value and merits such as higher efficacy and safety due to a lower death rate if the investigational drug is combined with at least one FDA-approved anticancer drug. Some of the most recently phase 1/II completed studies (at the time of writing) are exemplified in Table  1 [ 15 ] and the drugs are potential experimental drug candidates.

Integrative synergistic approaches

Some of the most emerging and promising tools being developed and increasingly being used in the health care-related sectors include data warehouse, that is a repository of historical data from data warehouses to data refineries for refining the crude data dubbed as the “oil of the digital era” into valuable data all the way from research to clinical use [ 16 , 17 ]. Because the data in the raw form is enormous, complex, lacks structure and standardization combined with interoperability issues, compliance issues and ethical challenges, the data refineries are envisioned to bridge the gap for refining and distilling the data on its journey from research to clinical utility for the benefits of the patients, including the stakeholders.

To navigate the costly and complex landscape of therapeutic drugs from basic research to clinical use hinges on integrating multi-disciplinary approaches. Because of different goals of the stakeholders, it has been historically challenging to strike a common chord that resonates across the whole ecosystem. Over the last few years, there has been a paradigm shift due to many factors such as the high cost of drug development, lengthy approval process, closer collaborations between academia and industries, integration of emerging technologies such as digital health, telehealth and wearables, gene editing, including big data, funding, education, and changes in government policies. The health benefits of panomics (genomics, proteomics, transcriptomics, metabolomics, epigenomics, ionomics and microbiomics) and the increasing use of panomics in personalizing medicine are emerging and promising in the treatment of diseases such as cancer, cardiovascular and gastrointestinal disorders [ 18 , 19 ]. The integral role of “gut” microbiome in health and in treating many diseases, including cancer is beginning to emerge as demonstrated by immune checkpoint inhibitor therapy [ 20 , 21 ]. While panomics addresses many of the precision medicine treatment benefits, it falls short in addressing issues such as the multi-morbidity, impact of the disease on the patients’ lives, their adaptability to the disease or other existing diseases, their family, their social life and their community life. Personomics is thus envisioned to bridge the gap between panomics and the patients’ personal or an individual’s circumstances [ 22 ]. This “echoes” with the words of Sir William Osler: “the good physician treats the disease; the great physician treats the patient who has the disease” [ 23 ].

The Precision Medicine Initiative (PMI) launched in 2015 [ 24 ] has been building to a “crescendo” and its impact on drug development, clinical trials and in personalizing the treatment for therapeutic efficacy, maximum safety, higher durable response, longevity and higher quality of life is emerging. Over the last few years, the FDA has emphasized the use of real-world data (RWD) and real-world evidence (RWE) to modernize clinical trials, an advancement made possible by the 21st Century Cures Act [ 25 , 26 ]. With real-world data and real-evidence, researchers will be able to go beyond the scope of traditional trials, transition to a “hybrid” trial that is dynamic, providing insights from information collected in clinical care. As an example, the FDA in April 2019 approved a supplemental New Drug Application based on data extracted from EHR and post-marketing reports of the real-world use of Pfizer’s drug, IBRANCE (Palbociclib) to expand the indication for in combination with Fulvestrant to include men with hormone receptor positive (HR+), human EGFR 2 negative (HER2−) advanced or metastatic breast cancer, for the treatment of breast cancer in men [ 27 , 28 ]. The former FDA Commissioner, Dr. Scott Gottlieb stated [ 29 ]: “the EHRs and other data sources, paired with advances in machine learning, will be crucial for architecting the next generation of successful clinical trials.

To address many of the challenges of implementing genomics medicine for routine use, the NIH funded IGNITE Network with the goals of integrating genomic data into EHR [ 30 ]. The IGNITE Network deploys plethoric “tools” for “Point-of-Care Decisions”, genetic markers for disease risk prediction including prevention, tools about family history data, pharmacogenomics data and refinement of disease diagnosis. Similarly, IBM Watson Health in collaboration with Brigham and Women Hospital and Vanderbilt University Medical Center has been pursuing the use of artificial intelligence for supporting precision medicine, to enhance patient safety, to nurture health equity, to expand and improve EHR usability [ 31 ]. Furthermore, the Watson Studio and Watson Knowledge Catalog has the data refinery tool for processing and transforming large amounts of raw data into valuable and clinical useful information for analytics. Several governments across the world, organizations, academia and institutes have created open access networks such as the Cancer Biomedical Informatics Grid (caBIG) and the Cancer Translational Research Informatics Grid (caTrip) for the caBIG project with a focus and a mission about driving translational research and improving the patient outcome by linking network of researchers, patients and physicians [ 32 ]. Similarly government and non-government sponsored programs have been established and they have been mushrooming globally such as the ICPerMed and the ECMC in the UK that support biotech and pharmaceutical companies to develop drugs in oncology through strategic partnerships [ 33 ]. Examples of programs in the USA include: the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH), the NCI-MATCH, a precision medicine cancer treatment clinical trial that is co-led by National Cancer Institute (NCI) and the ECOG-ACRIN Cancer Research Group. In the NCI-MATCH trial, patients received therapy based on the genetic changes found in their tumor as exemplified by the results from Arm H of the study demonstrated that treatment with a “cocktail” of dabrafenib and trametinib, designed to target cancers that have specific BRAF gene mutations, was effective in a trial of 35 patients having 17 distinct tumor types [ 34 ]. Most recently, the studies [ 35 ] published by the Pan-cancer Analysis of Whole Genome (PCAWG) consortium involving whole genome sequencing of 2658 cancer genomes demonstrated new information about cancer drivers from 38 tumor types and identified potentially new targets for precision medicine.

The exploitation of such tools for the “Right-to-Try” experimental drugs in treating life threatening diseases such as cancer will most likely favor the outcome and mitigate the negative outcomes associated with the clinical use of experimental drugs. The outcome using experimental drug has the potential to be more favorable if combined (“cock-tail”) with an FDA-approved drug. Some of the other approaches include implementation of programs and policies that incorporate the interests of patients such as education, understanding of risks, second opinion about the therapy, expectations and costs due to potential complications, education of physicians about precision medicine and emerging tools, dosing, the use of EHR, systematic reporting of results that may be important for public health and safety. The sponsor should provide transparency and immediate notifications to the physician including the FDA about safety issues during the drug development stage and any manufacturing or supply issues. Due to the complexity of the informed consent form and lack of oversight by the FDA, it is important that an independent or a neutral body such as an IRB or an ethics committee is engaged in reviewing the consent procedures. The positive outcome of the treatment results will thus be a potential boon to young biotech and pharmaceutical companies facing the “valley of death” syndrome, struggling to raise funding or looking for partnerships or trying to build trust and credibility. Furthermore, a positive outcome has the potential to spawn new ventures or opportunities such as veterinary oncology and “outpatient” clinics.

It is envisioned that the use of new tools encompassing electronic medical records, personalized medicine, data refineries, artificial intelligence and machine learning, further testing of drugs, including adjuvant therapies or “cocktail” of drugs will favor the outcome of experimental drugs and may pave the way for indication expansion as exemplified by IBRANCE. Furthermore, the use of such tools are expected to: (i) accelerate drug development time; (ii) reduce drug development cost; (iii) lower the cost of drugs; (iv) improve the durable response; (v) reduce adverse effects such as hepato-and cardio-toxicity; (vi) improve longevity and quality of life of the patient.

The progress made on several fronts in healthcare and the concerted efforts by the stakeholders, including the integral role of agencies such as World Health Organization (WHO) and Global Health Council (GHC) over the last few decades for the treatment of diseases, patient and public engagements, the role of healthcare practitioners, the role of education, data ownership, data sharing, transparency, privacy, ethics, standardization across the multi-industries, regulations, compliance, funding of programs, payment by medical insurance companies, including global policy development and implementation currently present limited opportunities and many challenges for the “Right-to-Try” experimental drugs for the treatment of life-threatening diseases. The “Right-to-Try” experimental drug is nevertheless a major “milestone” along the journey and its full impact on treating life-threatening diseases such as cancer and infectious diseases such as COVID-19 remain to be seen. One of the biggest impacts of emergency use of experimental drugs and compassionate drugs or “repurposing” of drugs is unfolding during the current coronavirus pandemic crisis.

Availability of data and materials

Data sharing is not applicable to this article because no data sets were generated and/or analyzed for the study.

Abbreviations

Experimental Cancer Medicine Center

Epidermal growth factor receptor

Electronic health record

Human epidermal growth factor receptor 2

Institutional review board

Middle East respiratory syndrome

Ribonucleic acid

State Food and Drug Administration (China)

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Mahant, V. “Right-to-Try” experimental drugs: an overview. J Transl Med 18 , 253 (2020). https://doi.org/10.1186/s12967-020-02427-4

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1 Drug Use and Misuse Overview

Introduction.

People of all ages are impacted by the use and misuse of drugs.  With use and misuse we have seen an increase in medical costs, loss of school and work productivity and impacts on individuals, families and communities. Understanding the implications drugs can have on all of these concerns, will help you understanding the risks you take when it comes to drug and drug use. The impacts drugs can have make understanding them that much more important.

Learning Objectives

• Identify types of drugs and differentiate between legal and illegal substances
• Understand how drugs are taken and administered
• Identify drug users and why they use drugs
• Explain the difference between use, misuse, and dependence
• Demonstrate familiarity with drug use policies and laws and the impacts they have on legal and illegal use

Image 1.1 – Picture of Various Pills

Learning Content

Have you thought about what the word “drug” means? When most of us think of drugs we think of illegal substances, prescription and over-the-counter medications, tobacco and alcohol.

The World Health Organization (1994) says that “drug” is a “a term of varied usage:”

• medicine it refers to any substance with the potential to prevent or cure disease or enhance physical or mental welfare.
• pharmacology it refers to any chemical agent that alters the biochemical physiology processes of tissue or organisms.
• common usage the term often refers specifically to psychoactive drugs and often illicit drugs which have non-medical use in addition to any medical use.”

Most drugs carry the risk of affecting you in ways that can impact your ability to get along with others, to work, to cope with things going on in your life and to think rationally. The effects that drugs produce are also influenced by society’s perception of them. Think about people who use illegal drugs—are they viewed in the same way as people who are using legal drugs?

Drugs can alter thinking and judgement in a person and can lead to health risks associated with misuse, dependency and addiction.

People use drugs for many reasons. Individuals want to fit in, they want to feel better, they want to perform better at work, in school and in sports. Drugs may make you want to use them more. What may have started as a way to feel better or even as a way to escape can quickly turn into misuse because your brain and body need the drug to feel normal.

Drug use in the U.S. is pervasive. Drug use is a valid societal concern because of its effects on individuals, families and communities. Exploring drugs and drug misuse further will encourage a better understanding of drugs and the implications they can have.

Let’s start by thinking about sugar.  Sugar is not considered a drug and most foods containing sugar are not addictive. But sugar can have a similar impact on the brain as drugs do. Watch this video on How Sugar Affects the Brain to gain a perspective on how easily you can become addicted to sweets and treats, then ask yourself if I can become addicted to sugar can I become addicted to drugs? When considering how to define a drug, where do cultures draw the line? And why? Is food a drug?

Video 1.1 – How Sugar Affects the Brain

Types of Drugs

Table 1.1 – Commonly Used Drugs Chart

Critical Thinking

Starting with types of drugs, explore the chart above and review the information provided on these various drugs. Spend time reviewing the chart above, and identify 10 drugs you are interested in learning more about. Make notes on these drugs for your knowledge. Think about these drugs like you did about sugar. What are the risks with using the drug? What will be the implications of use if I use a drug like I might consume sugar? Is it possible to become addicted?

The important thing here is to understand there are risks with many things in life; however, use, misuse, and addiction are concerns that everyone should think about when it comes to drugs, drug misuse, and putting things into our body.

Legal drugs include over-the-counter medications, prescription drugs, alcohol, tobacco, caffeine and marijuana.

Illegal drugs are drugs that are not prescribed by a licensed medical professional and drugs that are identified under the Controlled Substances Act .

There are many groupings of drugs and they typically fall into one of these categories:

NSAIDS : Non-Steroidal Anti-Inflammatory Drugs like acetaminophen and ibuprofen

Analgesics : drugs that relieve pain

Antibiotics : drugs used to treat disease caused by bacteria

Antiseptics : best known as mouthwash; typically used to treat the oral cavity

Antiviral : drugs used to treat disease caused by viruses

Designer Drugs : synthetic version of controlled substances

Over-the-counter : drugs that can be purchased without a prescription from a medical professional; sold legally in drug stores and pharmacies

Prescription Drugs : drugs that are prescribed to a specific person by their medical care provider

Psychoactive Drugs : drugs that change brain function resulting in alterations in perception, mood and consciousness

Psychotherapeutic Drugs : drugs that are prescribed to help relieve symptoms associated with anxiety, depression and mental disorders

Psychotropic Drugs: drugs that alter chemical levels in the brain which impact mood and behavior

Recreational Drugs: typically known as psychoactive drugs; used to enhance recreation and experimentation

Check Your Knowledge

How drugs are taken/administered.

Images 1.2 – Pills in Hand

Image 1.3 – Syringes

There are three primary ways that drugs are taken into the body. Drugs are typically administered orally, by injection and through inhalation. Oral administration occurs through the mouth. Taking drugs orally can be difficult as the drug’s absorption in the gastrointestinal tract can cause the drug to take longer to work. With injection, that happens via a hypodermic syringe or IV into a vein, muscle or under the skin, so the impact of the drug is much more rapid and the effects of the use can be seen more quickly. Inhalation occurs through smoking or huffing. This type of use is rapid and very efficient, the effects of use are seen quickly. Other ways that drugs can get into the body include topically (via mucous membranes) and rectally or vaginally (via suppositories).

Uses of Drugs

Drugs can be thought of in many ways. Drugs are good, drugs are bad. Drugs are socially acceptable. Drugs are socially unacceptable. Drugs are conventional. Drugs are deviant. The effects that drugs produce are influenced by our perception of them as well as the perception society has about their use. When people consider drug use, they typically use drugs for one of these reasons:

• Medical : used to treat and cure illness, disease, or symptoms that individuals have
• Spiritual : used mostly in the hallucinogen, stimulant or sedative category of drugs; used in religious and spiritual settings
• Self-Help : typically used to improve something about the user, such as brain concentration and physical abilities
• Recreational : used to help enhance euphoric effects typically associated with use of psychoactive or other illegal drugs

Patterns of Drug Taking

Many people use drugs for many reasons. Often, one set of problems is exchanged for another, possibly leading to the individual not even knowing how a cycle of use happened. Understanding the patterns of drug taking can help you understand why people use drugs and give you insight into the risks and concerns associated with drug taking.

There are five patterns of drug taking.

The patterns of drug taking are experimental use, social-recreational use, circumstantial use, intensified use, and compulsive use. Looking at these further will give you the insight you need to understand the different types of drug taking.

Image 1.4 – Drug Store Sign at Night

Table 1.2 – Drug Taking Patterns and Explanations

Drug Misuse Risks and Concerns

Infographic 1.1 – The Cycle of Addiction

A substance-use disorder is defined as a medical illness in which the person is unable to stop using a drug even though it causes health and social problems in their life. Severe substance-use disorders are also known as addiction.

The infographic above helps explain how drug users can progress from drug use to addiction. This progression can happen over a short period of time or it can take many years. Misuse is defined as drug use that is illegal or not aligned with medical use guidelines. Most drug use happens in the experimental and social recreational drug-taking patterns. The hope is that drug use does not lead to misuse because the user recognizes the need to break their use patterns and or seek help. Misuse begins with recurrent drug use. Often individuals fail to follow through on obligations at work, home and school. They may use substances when they know the use can be physically hazardous. They often have legal problems associated with the drug use and continued social and interpersonal problems despite knowing their drug use is a concern. It is really hard to define what misuse is because it isn’t always nicely wrapped into one package.

Substance addiction behaviors often include one or more of the following:

• loss of control over drug use
• continuation of use despite harm to oneself and others
• compulsive use and cravings that are often the sole focus of the user

The 4 C’s of addiction and the examples provided below will help you better understand addiction and addiction risks.

• Control , often looked at as loss of control by the user. The user might say this about themselves: “I try to drink only one day a week, but I end up drinking everyday.”
• Continued use  when the user keeps on using even though they know there are harmful consequences. The user might say this about themselves: “I know my alcohol use caused me to lose my job, but I cannot stop drinking alcohol.”
• Compulsion  when the user can do nothing but think about using. The user might say this about themselves: “No matter what I do, I cannot get alcohol out of my mind.  I think about it all the time.”
• Cravings  which cause the physical drive to use and keep using. The user might say this about their cravings: “No matter how much alcohol I drink, I still crave it all the time.” (Bettinardi-Angres, K. & Angres, D. H., 2010; SAMHSA, n. d.)

Think critically about this situation and what you might say to your partner if you decided to confront them about their use. Your partner is buying small amounts of liquor at gas stations and liquor stores. You discover the various charges on your bank statement along with empty “airplane size” bottles in the trash almost everyday. What might your partner be going through? What might be contributing to their compulsion and cravings for alcohol?

Substance use also involves tolerance, physical dependence and psychological dependence that often lead to addiction and addiction risks.

Tolerance occurs when repeated exposure to the same dose of the drug results in a lesser effect and the need to take larger amounts of the drug to get the same effect that the user expects. Physical dependence happens when the body has become adapted to the drug’s presence causing withdrawal to occur when use stops.  Typically the user experiences symptoms when the drug use stops, often leading the individual to go back to using because they cannot tolerate the symptoms.  Psychological dependence is a behavioral dependence that involves cravings, a high rate of use and relapse when use stops.  Often these three things happen in the cycle of addition and that addiction continues until it is broken or continued use never ends.

In order for a person to be considered addicted to a drug, depending on the user’s risks, three or more of the following need to occur:

• withdrawal when use of the drug stops
• taking the drug in large amounts or over a long period of time (keep in mind it can take many years for addiction to occur)
• unsuccessful efforts to quit despite the desire to quit
• lots of time spent getting the drug
• cessation of activities that once involved work, family, school, and fun
• continued use of the drug despite user knowing they have a problem (NIDA, 2018; WebMD., n. d.)

Those individuals who are likely to become addicted to drugs lack self awareness and tend to be negative, depressed, tense, risk-taking, impulsive, and in need for immediate gratification. Of course, there are other correlates of drug use like race, gender, education, personality, genetics, and characteristics that predict later initiation of drug use.

The nature of addiction is complex and difficult to understand (especially for nonusers) but what is important to know is that the sooner issues with addiction risk can be identified, the better. Long-term misuse leads to changes in the brain that affect memory, judgement, learning and decision making, having devastating consequences for the user.

History of Drugs

Image 1.5 – Drug History Timeline

Legal consequences for drug misuse can have lifelong implications for users. Understanding drug laws and policies about legal and illegal drugs can inform individuals about consequences associated with their behaviors related to drug use, misuse, and dependence.

Current approaches to drug regulation in the U.S. are linked to two major pieces of legislation, The Pure Food and Drug Act and The Harrison Act. The Pure Food and Drug Act of 1906 has been the foundation for laws surrounding pharmaceutical drugs.

The Harrison Act of 1914 was the foundation of the controlled-substance regulations. This act imposed taxes on the sale, importation, manufacturing, and distribution of opium and coca leaves. It was the first act to provide criminal laws to nonmedical drug use. The “warning label” on addictive medications is a result of this Act. This Act was an important step in the United States’ war on illegal drugs.

Other Laws and Acts include:

• Narcotic Control Act of 1965
• Drug Abuse Control Amendments of 1965
• Comprehensive Drug Abuse Prevention and Control Act of 1970
• Anti Drug Abuse Acts of 1986 and 1988

Watch this short video on What the FDA Regulates to better understand the role these acts have played in current regulation.

Video 1.2 – What Does the FDA Regulate?

Legal Issues and Drugs

The majority of federal drug convictions are obtained for drug trafficking. The majority of local and state arrests and convictions are for drug possession.

Depending on the state laws, the community and the criminal history of the person, state arrests for simple possession tend to be charged as misdemeanors and usually involve probation, a short term jail sentence or a fine.

These are some helpful terms to be familiar with when looking at legal issues and drugs:

• Controlled substances
• Distribution
• Trafficking
• Manufacturing

What to Know about Drug Laws and Legal Issues

Drug schedules.

Drugs are classified into 5 categories or schedules depending on the medical uses or the risk of dependency and misuse. The risk of misuse of a drug is a determining factor in a drug’s classified schedule. Schedule I drugs have high potential for misuse and schedule V drugs have the least risk of being misused. The Controlled Substances Act (CSA)  identifies and lists drugs and their schedule.

Each schedule is determined based on the risks of using the drug. Below is a short summary of each schedule.

Schedule I drugs are drugs that have a high potential for misuse and typically have no medical uses. One drug that has been highly discussed and legalized in some states that is still considered schedule I is marijuana.

Schedule II drugs have high potential for misuse and a high risk of physical and psychological dependence. Hydrocodone and Adderall are two drugs that fall into this schedule.

Schedule III drugs have moderate to low potential risk of physical and psychological dependence. Tylenol with codeine and anabolic steroids are in this schedule.

Schedule IV drugs have low potential for misuse and dependence. Xanax and Valium are two drugs that fall into this schedule.

Schedule V drugs have low potential for misuse. Quantities of these drugs are often monitored. Cough medicine with codeine and Lyrica are two drugs that fall into this schedule (DEA, 2021; U.S. DOJ, n. d.).

​​Examples of Drugs in Each Drug Schedule

Table 1.3 – Drug Schedule

Drug Policies

Work, school, and volunteer opportunities can impact policies regarding drug use.  It is important to understand what these policies are and the implications that can result if the policies are not adhered to. Below are two examples related to a higher education environment. One is the policy in place for students, the other for employees. No matter where you work or go to school or volunteer, be aware of the policies in place that you will need to adhere to or face consequences for actions you take.

Take a look at these two sources below as it relates to policies you might be asked to follow at work, school or when volunteering. Have you thought about your own behaviors as it relates to drugs and drug use? Are you prepared to adhere to the policies expected of you in these situations or the consequences you might face if you choose not to adhere to the policies?

University of Illinois Student Code, Policy on Drugs

University of Illinois Employee Drug Policy

Supplemental Resources

Centers for Disease Control and Prevention. (n.d.). Data and statistics .  https://www.cdc.gov/datastatistics/index.html

Drug Policy Alliance. (n.d.). Drug Policy Alliance [Homepage]. https://drugpolicy.org/

Drug Policy Alliance. (n.d.). Uprooting the Drug War [Homepage]. Retrieved July 7, 2021, from https://uprootingthedrugwar.org/

MedlinePlus. (n.d.). MedlinePlus –Health information from the National Library of Medicine [Homepage]. https://medlineplus.gov/

National Institute on Drug Abuse. (2020, August 20). Commonly used drug charts . U.S. Department of Health and Human Services, National Institute of Health. Retrieved July 23, 2021, from https://nida.nih.gov/drug-topics/commonly-used-drugs-charts

Office of National Drug Control Policy. (n.d.). Principles of modern drug policy [Archived webpage]. Executive Office of the President of the United States.   https://obamawhitehouse.archives.gov/ondcp/policy-and-research/principles-of-modern-drug-policy

PubMed Central. (n.d.). Home [Homepage]. https://www.ncbi.nlm.nih.gov/pmc/

Substance Abuse and Mental Health Services Administration. (2009). Substance abuse treatment: Addressing the specific needs of women. Treatment improvement protocol (TIP) series, no. 51. https://www.ncbi.nlm.nih.gov/books/NBK83252/

U. S. Food and Drug Administration. (n.d.). Drugs . https://www.fda.gov/drugs

The White House. (n. d.). Office of national drug control policy . https://www.whitehouse.gov/ondcp/

Bettinardi-Angres, K. & Angres, D. H. (2010). Understanding the disease of addiction. Journal of Nursing Regulation, 1(2), 31-37. https://doi.org/10.1016/S2155-8256(15)30348-3

Controlled Substance Act, 21 U.S.C. § 801 et seq. (1970). https://www.deadiversion.usdoj.gov/21cfr/21usc/811.htm

Moss-King, D . (2016, March) . Addiction psychology [Chart]. ResearchGate. Retrieved July 7, 2021, from https://www.researchgate.net/figure/The-cycle-of-addiction-The-cycle-of-addiction-shows-how-difficult-it-can-be-to-avoid_fig1_304627074

National Institute of Drug Abuse. (2018, June 6). Understanding drug use and addiction drug facts . U.S. Department of Health and Human Services, National Institutes of Health. https://nida.nih.gov/publications/drugfacts/understanding-drug-use-addiction

Substance Abuse and Mental Health Services Administration. (n.d). SAMHSA–Substance Abuse and Mental Health Services Administration [Homepage]. https://www.samhsa.gov/

Tanabe, J. (2019, June 11). Drug. In N ew world encyclopedia. Retrieved July 3, 2021, from https://www.newworldencyclopedia.org/p/index.php?title=Drug&oldid=1020575

U. S. Drug Enforcement Administration. (2021, July 23). Drug scheduling. Retrieved July 3, 2021, from https://www.dea.gov/drug-information/drug-scheduling

U.S. Food and Drug Administration. (2021, Jun 24). What does FDA regulate? [Video]. YouTube. https://www.youtube.com/watch?v=bMEFp8cl19c

WebMD. (n.d.). What is drug addiction? https://www.webmd.com/mental-health/addiction/drug-abuse-addiction#1

World Health Organization (1994). Lexicon of alcohol and drug terms . https://www​.apps.who.int/iris/handle/10665/39461

a drug which has been declared by state and federal law to be illegal for use or sale, but may be prescribed by a physician

a person is accused of selling, delivery or providing a controlled substance illegally

refers to the illegal sale and or distribution of a controlled substance; typically the amount of drugs found is the primary concern

cultivation or manufacture of a controlled substance via chemical processes or lab production or growing

most common drug charge at local levels; knowingly and intentionally possessing a controlled substance, having drugs without a prescription and the quantity of the possession are all factors in charging a person

Drug Use and Misuse Copyright © by University of Illinois Board of Trustees is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License , except where otherwise noted.

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Talking about drugs

Drug use can be tricky to talk about. It’s a personal and sometimes sensitive issue. Understanding drugs and why people might use them can help start an open and supportive conversation.

What is a drug?

A drug is any substance that, when taken or administered into the body, has a physiological effect.

A psychoactive or psychotropic drug affects mental processes and can influence a person’s mood, behaviour, cognition and perception.

The effects of any drug vary from person to person. How a drug affects a person can depend on their size, weight and health, also whether the person is used to taking the drug, and whether other drugs are in their system at the same time.

The effects will also depend on the amount taken. It can be hard to judge how much of an illegal drug has been taken, as they are uncontrolled, so quality and strength will vary from one batch to another.

People use drugs for many reasons; to relax, for enjoyment, to be part of a group, out of curiosity, as a coping mechanism or to minimise physical and/or psychological pain and trauma.

They use drugs for the benefits (perceived and/or experienced), not for the potential harm. This applies to both legal and illegal drugs.

Many Australians take at least one psychoactive drug on a regular basis - they might take medication (i.e. over-the-counter or via a prescription), drink alcohol, smoke tobacco or use an illegal drug. All drugs have the potential to cause harm. As use increases, so does the potential for harm.

These are some of the different categories of drug use. People may use drugs in one or several categories, and one stage will not inevitably lead to another.

• Experimental use is when a person tries a drug once or twice out of curiosity.
• Recreational use is when a person chooses to use a drug for enjoyment, particularly to enhance a mood or social occasion.
• Situational use is when a drug is used to cope with the demands of particular situations.
• Intensive use or ‘bingeing’ is when a person consumes a heavy amount of drugs over a short period of time, and/or uses continuously over a number of days or weeks.
• Dependent use is when a person becomes dependent on a drug after prolonged or heavy use over time. They feel a need to take the drug consistently in order to feel normal or to avoid uncomfortable withdrawal symptoms.

While drug use can be harmful it does not automatically lead to a dependence, many people only use drugs once or infrequently.

Text the Effects

Text a drug name to 0439 835 563* to receive drug information via text message *(standard call rates apply)

What to do in a crisis

Always call triple zero (000) if a drug overdose is known or suspected and remember that paramedics don’t need to involve the police. If someone overdoses or has an adverse reaction, it’s very important that they receive professional help as soon as possible. A quick response can save their life.

Having the conversation

Discussing alcohol and drugs with family members and friends is an opportunity to learn more about different types of drugs and their individual and social impact.

The Power of Words

Designed to support healthcare and other professionals working with people who use alcohol and other drugs to reduce stigma and improve health outcomes.

Many parents are concerned about alcohol as well as other drugs as their children grow up Young people are at greater risk of alcohol-related harm than adults.

Vaping’s a hot topic right now.

In fact, a lot’s being said about vaping and its effects - but there’s lots of conflicting and confusing information out there.

An overview of drugs and Australian law.

Impact of AOD on the workplace

Alcohol and other drugs (including prescribed, or over the counter medicine) can affect employee health and a person’s ability to work safely.

Seeking help

Alcohol and other drug treatment services aim to assist people with problems around their drug use. Their goal is to help them reduce or stop using alcohol and other drugs, or to use these substances in a way that reduces the harm to them, their family and the community.

Medicinal cannabis products

In Australia, medicinal cannabis products are used for a range of illnesses and health conditions.

Synthetic cannabinoids – the facts

The rise of synthetic cannabinoids in Australia is a growing public health concern.

Last updated: 06 Nov 2023

Drug + Alcohol Info

The must know information for workers supporting young people with AOD support needs.

Understanding Youth Drug & Alcohol Use

Different patterns of drug use.

Understanding that there are many different patterns of drug use is crucial to understanding how best to support a young person.

There is no typical drug and alcohol use pattern applicable to young people, and not all drug or alcohol use is problematic or requires specialist attention. Most young people experiment with alcohol and other drugs at some stage, and even though experimentation creates risks for adolescents, most go on to develop a pattern of use that is relatively harmless.

There are several patterns listed below that describe a young person’s drug or alcohol use.

EXPERIMENTAL USE

Describes a young person’s exploration of drugs or alcohol and the effects of each.

RECREATIONAL USE

Describes AOD use for the purpose of enhancing leisure. This pattern often occurs socially. An example may be the use of ecstasy twice-monthly when clubbing.

SITUATIONAL USE

Describes AOD use specific to a situation or context. For example, a young person may use cannabis heavily but only when visiting their father on weekends, or may only use amphetamines prior to school exams.

INTENSIVE USE

Associated with heavier, more frequent and/or more harmful/risky use. Use is considered intensive when it occurs in a range of circumstances and settings.

DEPENDENT USE

Describes when a young person finds it difficult to control or stop their use, uses more than they intend, or where use continues despite negative consequences. A young person with a dependent use pattern may also experience withdrawal symptoms when not using.

It’s important to note:

• Young people may have different patterns of use for different drugs simultaneously
• Young people don’t necessarily move through these patterns sequentially: recreational use doesn’t necessarily lead to situational or intensive use
• Young people’s patterns of use can move both ways
• Regular substance use doesn’t always lead to serious problems.

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Assessment of the Right-to-Try Law: The Pros and the Cons

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On May 30, 2018, the Right-To-Try bill, allowing the use of experimental, non-Food and Drug Administration (FDA)–approved drugs as a last resort for those unable to participate in clinical testing who have also exhausted all other treatment options, was signed into law ( 1 ). At the time this bill was signed, 38 states had passed similar Right-To-Try laws; this law is nationwide ( 1 ). The requirements for granting approval of experimental drug treatment under Right-To-Try laws are: (1) a terminally ill patient has exhausted all other treatment options and is ineligible to participate in clinical trials, (2) the experimental drug passes FDA phase 1 clinical testing, (3) the patients’ health-care provider or treating physician must recommend and approve the experimental treatment, (4) the pharmaceutical manufacturer must approve the drug for use as an experimental treatment, and (5) patients’ written informed consent is required ( 2 , 3 ). Although certain experimental drugs may improve some patients’ conditions, adverse health effects or no health improvement are more likely.

In the following text we weigh the pros and cons of the federal Right-To-Try law.

Limited Patient Understanding

One requirement before beginning experimental drug therapy is patient informed consent. Consent documents are often confusing, written in a way that is difficult to understand, and contain highly technical terms at great length, which can result in patients missing or misunderstanding the gist of the experiment ( 4 , 5 ). Similarly, there may be misunderstanding of experimental drugs and their likely success rates, as well as the potential of therapeutic misconception ( 6 ). Terminally ill patients who feel as if they are running out of time may be swayed by the false hope provided by an experimental procedure, which can outweigh potentially detrimental effects of the experimental drug and other unintended consequences.

Lowered Trust in the FDA

The FDA’s clinical trial and review process took more than 50 y to establish in order to ensure that pharmaceuticals are safe for their intended use ( 7 ). Although this highly regulated process can require up to 15 y for a drug to receive FDA approval, it is an effective method that weeds out most ineffective or dangerous drugs ( 8 ). Arguably, Right-To-Try laws are hypocritical because they rely on the FDA process of determining the toxicity and possible side effects of products in phase 1 testing, but this argument ignores the fact that drugs need to be rigorously tested to be deemed safe for widespread use ( 3 ). There is a need to protect patients’ trust in the clinical research enterprise.

Financial Consequences

Marginalized patients could be vulnerable to exploitation through Right-To-Try. The legislation does not contain incentives for insurance companies to cover experimental treatment costs and the potential outcomes of the treatment ( 2 , 9 ). Insurance companies in many states that passed Right-To-Try laws have denied hospice coverage, home health-care coverage, and even health-care coverage for at least 6 mo after treatment ( 2 , 9 ). This denial of coverage creates a financial burden left for the families, who often cannot pay.

Decreased Liability

One of the most problematic issues with the federal Right-To-Try legislation is provided immunity from liability for physicians and pharmaceutical companies ( 2 , 4 ). Without FDA oversight, pharmaceutical companies can push unsafe drugs on sick patients. Although it is presumed that primary care physicians have their patients’ best interest in mind, physicians can own pharmaceutical company stock and charge patient treatment fees ( 2 ). Pharmaceutical companies, for their part, need have no fear that any negative outcomes from experimental drugs will hurt the chances of the drug becoming FDA approved, as this legislation prohibits the FDA from including negative outcomes of experimental treatments in the data that are used to approve the pharmaceuticals ( 2 , 10 ). Lack of systematic reporting of adverse events may result in decreased information for the public and providers.

Safety Consequences

Nearly 70% of drugs pass phase I testing ( 11 ). However, most drugs that pass phase I will be deemed unsafe or ineffective, failing to pass subsequent stages ( 11 ). There is a low probability that experimental drugs tested in early-stage research will significantly benefit patients and a higher probability that side effects from experimental interventions could worsen their condition ( 2 , 12 ). Additionally, the FDA expanded access process has been revised to be more transparent and to take less time: patients can now complete the expanded access request form within an hour ( 13 ). Right-To-Try legislation has helped make undertested drugs more readily accessible by reducing FDA oversight, which was created to protect patients and consumers from unsafe pharmaceuticals. Another consideration is the growing epidemic of counterfeit medicine; another reason to protect the integrity and safety of the drug development process ( 14 ).

Experimental Drugs May Extend Life

In most cases, experimental treatments have not yet proven effective. However, they can potentially extend life ( 15 ). Compassionate use has been used in epidemics with a high death rate (e.g., the Ebola outbreak in 2013–2016) ( 16 , 17 ). Widely untested therapeutic vaccines were given to people living with Ebola in hopes of improving their condition ( 18 ). Given a setting of compassionate use, even unsuccessful treatment may help strengthen patients’ trust in health-care systems due to potentially positive health outcomes.

In Line with Expanded Access

The case for the terminally ill to try potential life-extending drugs has been addressed by the FDA ( 8 , 13 ). Dr. Scott Gottlieb, Commissioner of the FDA (May 2017–present), has stated that the FDA approves almost all expanded access requests: high urgency immediately or over the phone, and all other requests within a few days ( 13 ). The Right-To-Try legislation is very similar to the Expanded Access program in that it recognizes that a streamlined process is necessary if experimental drugs are to reach patients who may not have any other options.

Gives Patients More Options

Supporters of the Right-To-Try law argue that the law will not replace Expanded Access but will create another avenue to streamline the process for experimental treatments ( 3 ). Although the FDA receives around 1,000 requests annually for experimental drug therapies, many such therapies never make it to the FDA ( 12 , 13 ). Confusing online forms and multiple attachments can be barriers to getting experimental drugs approved for use, and by that point of approval patients’ conditions may have worsened significantly. Right-To-Try laws are intended to further streamline this process, allowing patients to work directly with doctors to acquire nonclinically tested drugs, bypassing the FDA and providing multiple options for all terminally ill patients to acquire potentially life-extending drugs. The federal Right-To-Try law intends to give people more control over their life/health. Even if experimental treatments are ineffective, the argument is that people should have a choice to at least try a drug that may produce medical benefits. Similarly, medical aid in dying allows patients to make important medical decisions toward the end of their life ( 10 ). Some argue that if patients have the right to end their life with medical aid in dying, they should also have the right to try to extend their lives with experimental drugs ( 19 ). Personal choice over what happens to one’s health near the end of life is aligned with the ethical principle of autonomy and can be empowering.

• FINAL THOUGHTS

One of the main principles the Right-To-Try law was built on ways to reduce pain and suffering. However, bypassing FDA regulations means the data that would normally be available for physicians and clinical researchers, such as dosage, drug administration, and side effects, will be limited. The potential for lowered trust in the clinical research enterprise, financial and safety consequences, decreased liability, and limited patient understanding clearly outweigh the potential benefits of the experimental drugs tested outside the FDA purview.

That being said, health-care systems are often paternalistic and may treat the disease rather than the patient. Academics are similar in how they choose research projects and participants. For patients to make a well-informed decision, they must be provided good, clear knowledge regarding the benefits and limitations of experimental treatments ( 19 ). These patients also must have enough information to assess the risks of these treatments, including full comprehension of the severity of possible side effects as well as financial costs. In complicated clinical research, proper explanation and understanding of these issues may take several pages of text, and key points may get lost in the shuffle. However, the autonomy of those at the end of life should be respected, and terminally ill individuals should be given the opportunity make well informed decisions. This is why we side with the patient to have more options via Right-To-Try.

No potential conflict of interest relevant to this article was reported.

• Acknowledgments

We thank Jo Gerrard for editorial support.

Published online Aug. 10, 2018.

• © 2018 by the Society of Nuclear Medicine and Molecular Imaging.
• Bateman-House A ,
• Robertson CT
• Carrieri D ,
• Peccatori FA ,
• Nijhawan LP ,
• Janodia MD ,
• Muddukrishna BS ,
• Christopher PP ,
• Appelbaum PS ,
• Maranda L ,
• Dabrowska A ,
• Matthews KRW
• 11. ↵ The drug development process: step 3—clinical research . U.S Food and Drug Administration website. https://www.fda.gov/ForPatients/Approvals/Drugs/ucm405622.htm . Updated January, 4, 2018. Accessed August 16, 2018 .
• 13. ↵ Examining patient access to investigational drugs . Scott Gottlieb, M.D., before the House Subcommittee on Health, Committee on Energy and Commerce Testimony. U.S Food and Drug Administration website. https://www.fda.gov/NewsEvents/Testimony/ucm578634.htm . Updated on October 3, 2017. Accessed August 16, 2018 .
• Fantasia HC ,
• Caplan AL ,
• Bateman-House A
• Folayan M ,
• Peterson K ,
• Darrow JJ ,
• Sarpatwari A ,
• Kesselheim AS
• Received for publication July 11, 2018.
• Accepted for publication July 18, 2018.

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• v.11(2); 2021

Experimental therapies under investigation for COVID-19

Reshma golamari.

a Assistant Professor of Medicine, Penn State Health Milton S. Hershey Medical Center, Hershey, PA, USA

Neeru Kapoor

b Microbiologist, Maulana Azad Medical College, New Delhi, India

Tanuja Devaraj

Nitasa sahu, courtney kramer.

c Penn State College of Medicine, Hershey, PA, USA

Coronavirus Disease 2019, caused by the virus, Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2), is a pandemic first discovered in Wuhan, China which has claimed over 1.7 million lives to date across the globe as of 24 December 2020. As the virus spreads across the world affecting millions of patients, there has been a massive movement to discover readily available and effective treatment options including vaccines. One of the limiting factors in treating the disease is its varied presentation and effect in patients, ranging from asymptomatic patients to those left in intensive care units, intubated and fighting for their lives. There are numerous clinical trials and small-scale studies underway to investigate potential treatment options. However, very few studies and drugs demonstrated efficacy while many more are under investigation, leaving care teams dependent on supportive care coupled with experimental treatment options. In this review, we summarize the various treatment options explored to treat COVID-19, discussing possible the mechanisms of fighting the virus.

Introduction

Of the seven coronaviruses known to infect humans, majority of them cause a mild upper respiratory disease [ 1 ]. Three exceptions to these are severe acute respiratory syndrome coronavirus (SARS-CoV) in 2003, middle east respiratory syndrome coronavirus (MERS) in 2012 and the current SARS CoV-2 virus in 2020. Both the SARS-CoV and MERS outbreak were treated with supportive therapy as the main stay treatment since no clear drug or vaccine emerged as a winner [ 2 ,( 3 ]. The novel coronavirus, known as SARS-CoV-2 is very closely related to SARS-CoV with a 79.6% sequence similarity [ 4 ]. Because of the sequence similarity, many of the drugs which were investigated during the prior pandemics, re-surfaced as potential treatment options. With the gravity of the outbreak, the R 0 being 2.24–3.58 [ 5 ,( 6 ], and disease claiming more lives when compared to the prior outbreaks, there has been tremendous push towards developing effective treatment options. It is known that SARS-CoV-2 uses the receptor angiotensin-converting enzyme-2 (ACE2) for entry and employs the serine protease TMPRSS2 for S protein priming [ 7 ] ( Figure 1 ). COVID-19 patients, in their early stages of contracting the illness, present with a decrease in both CD4 + and CD8 + T-cells subsets leading to impairment in the host’s immunity [ 8 ]. Thus, the use of anti-viral agents early in the disease course has shown to be beneficial in controlling the severity of COVID-19 [ 9 ].

Diaphragm representing various experimental drugs used in the treatment of COVID-19 and their mechanisms of action

The current management focuses on supportive care in the mild-moderate setting and fluid/ventilation management in the acute respiratory distress phase (ARDS) and extracorporeal membrane oxygenation (ECMO) phases [ 10 ,( 11 ]. The role of steroids in this disease has been debated as well. We chose to review many potential medications that have been studied in this regard ( Figure 1 ).

Treatment options

One of the earlier drugs that were studied during the evolution of this pandemic was Chloroquine/hydroxychloroquine. In this review, we discuss major potential treatments studied until December 2020.

Chloroquine, hydroxychloroquine

The mechanism of action of chloroquine is via inhibition of viral cell entry and cell fusion by increasing endosomal pH. It also impairs the glycosylation of ACE2 receptors which the SARS viruses use for cellular entry [ 12 ,( 13 ]. It has shown efficacy in in-vitro studies against SARS-CoV-2, however, failed to demonstrate efficacy as more information was gained during the course of the pandemic [ 14 ].

According to Gautret et al . azithromycin was added to hydroxychloroquine for treating 20 patients with severe COVID-19 [ 15 ]. The combination therapy showed significant viral load reduction/disappearance [ 15 ]. This was followed by Chen et al . where a dose of 400 mg hydroxychloroquine did not lead to nasal clearance in 30 patients [ 16 ]. Both of these studies were randomized control trials. However, another study by Molina et al . showed that the combination therapy did not result in viral clearance at 5 or 6 days of treatment [ 17 ]. A recent observational study showed that hydroxychloroquine administration was not associated with either a lower or an increased risk for intubation or death [ 18 ]. Hydroxychloroquine was also studied for post-exposure prophylaxis and it did not prevent illness after being exposed to confirmed COVID-19 infection when given after 4 days of exposure [ 19 ].

Based on a myriad of data proving that these medications did not fasten viral clearance or symptom improval, there has been a change of practice to not include them for routine COVID-19 treatment.

Remdesivir (RDV)

Remdesivir has shown some promise in the treatment of COVID-19. This drug targets viral RNA-dependent RNA polymerase leading to premature termination of viral RNA transcription [ 20 ,( 21 ]. Remdesivir has been shown to inhibit the activity of Orthocoronavirinae (such as pathogenic SARS-CoV and MERS) and has shown some efficacy in treating SARS-CoV-2 in vitro [ 13 , 22 , 23 ]. Since there is sequence of similarities between SARS-CoV and SARS-CoV-2, this was trialled in patients with COVID-19.

This drug was first utilized in the USA in January 2020 in the state of Washington, where the very first COVID-19 patient was treated with it on day 7 for pneumonia [ 24 ]. The patient cleared the virus from the respiratory tract by day 12. In a study containing 237 patients by Wang et al . in April 2020, patients admitted to hospital for severe COVID-19 who were treated with remdesivir did not have statistically significant clinical benefits when compared to placebo [ 25 ]. The primary end-point in this study was time to recovery within 28 days. However, it was important to note that the patients who received remdesivir showed faster time to clinical improvement. Remdesivir did not show any statistically significant improvement whether treated for 5 or 10 days duration in patients with severe COVID-19 who were not mechanically ventilated [ 26 ].

Compassionate use of remdesivir in severe COVID-19 patients led to clinical improvement in 68% patients which led to the utilization in multiple health care facilities all across the world [ 27 ]. For the first time, a randomized-controlled trial in 1063 patients revealed positive results in terms of shortening time to recovery in adult patients hospitalized with COVID-19 [ 28 ]. Based on this data, the Food and Drug Administration has approved remdesivir for use in hospitalized patients with COVID-19 and the most benefit may be observed in time to recovery, with remdesivir shortening the disease course.

More recently, remdesivir was one of the medications studied among three other to look at in-hospital mortality in a multi-country randomized controlled trial (Solidarity trial). In that study, remdesivir did not show any in-hospital mortality benefit [ 29 ]. This study led to changing recommendations from World Health Organization (WHO) recommending against the use of remdesivir in hospitalized patients.

Favipiravir

Favipiravir inhibits the RNA polymerase activity of the viral genome of RNA viruses leading to chain termination and chain elongation [ 30 ]. Favipiravir has shown in-vitro viral inhibition activity against SARS-CoV-2 which resulted in it being a potential treatment option [ 13 ].

Favipiravir was compared with lopinavir/ritonavir (LPV/RTV) for treatment and in comparison to LPV/RTV, favipiravir led to faster viral clearance times and significant improvement rate in chest imaging [ 31 ]. A prospective, multicenter, open-label, randomized trial in China comparing favipiravir for COVID-19 with umifenovir (Arbidol) which is used against influenza viruses in 240 patients, was recently reported. Preliminary results have shown that although favipivar improved time to relief for fever and cough, it did not improve overall clinical recovery rate when compared to Arbidiol [ 32 ]. The overall consensus is that there are no definite results to use this drug in routine care of COVID-19 patients.

Additionally, favipiravir is being studied in clinical trials in combination with interferon-α (ChiCTR2000029600) [ 33 ] or with baloxavir marboxil (ChiCTR2000029544) [ 34 ] in China. Phase 3 clinical trial is currently underway in India.

Interferons

Interferon β (IFNb)-1b was a treatment option during the MERS and SARS-CoV outbreaks [ 35 ,( 36 ]. This drug displayed in-vitro susceptibility and SARS-CoV-2 was found to be more sensitive to IFN-I treatment when compared to the SARS-CoV, making this potential option [ 37 ]. ACTT-3 is a phase 3 randomized-controlled clinical trial looking at outcomes with combination of interferon beta-1a and remdesivir compared to remdesivir alone in 1038 patients which is ongoing [ 38 ]. Although not peer-reviewed yet, IFNb was studied to increase discharge rate at 14 days and decreased mortality rate at 28 days in a study from Iran (IRCT20100228003449N28) [ 39 ]. Interferon was also studied as a part of Solidarity trial, it did not show any mortality benefit or decrease the duration of hospital stay [ 29 ]

Lopinavir/ritonavir

Protease inhibitors (PIs) are drugs which are widely used in chronic human immunodeficiency virus (HIV) infection. In-vitro, SARS-CoV was susceptible to lopinavir/ritonavir (LPV/RTV) combination [ 40 ]. Therefore, it was tested and was deemed susceptible to SARS-CoV-2 in vitro.

LPV/RTV treatment alone failed to improve the primary outcome of clinical improvement in severe COVID-19 neither did it provide a mortality benefit [ 40 ]. Per Baden et al . reported that the benefit of this drug may not have been observed since the patients were treated with these drugs late in the infection and the concentration of drug necessary to inhibit the virus in the pulmonary circulation may be higher than the serum level [ 41 ]. When tested as a part of the solidarity trial, it too did not show any in-hospital mortality benefit [ 29 ].

Tocilizumab and other interleukin inhibitors

Tocilizumab is a monoclonal antibody which is an interleukin-6 receptor inhibitor which reduces the severity of illness by dampening the cytokine release syndrome [ 42 ]. COVID-19 patients who are severely ill present with a cytokine storm which led to investigators looking at agents that neutralize the inflammatory factors. Tocilizumab given either intravenously or subcutaneously reduced the risk of invasive mechanical ventilation and death in a retrospective observational study done in 1351 patients [ 43 ]. Another observational study in 764 patients showed reduced mortality in patients who received Tocilizumab [ 44 ]. However, these are retrospective or observational studies. More recently, in a randomized double-blinded controlled trial, Tocilizumab did not prevent intubation or death in moderately hospitalized patients warning clinicians against its use in moderately-ill COVID-19 patients [ 45 ]

Interleukin-10 was also hypothesized in reducing COVID-19 mortality by blocking the pro-inflammatory function, however, studies are warranted to prove this finding [ 46 ].

Convalescent plasma

Another investigational method is the administration of convalescent plasma made from recovered patients in China. It was studied in prior pandemics – SARS, H1N1, and severe Ebola virus infections [ 47 ]. For the first time for COVID-19, Shen et al . reported this experimental treatment where neutralizing IgG antibodies were transfused to 5 patients who were in severe ARDS. They theorized that adding antibodies from the convalescent plasma helped clear the virus leading to improvement in symptoms, however, very small proportion of patients were tested in this study [ 48 ]. However, a recently published randomized controlled trial in 103 patients who were severely infected with COVID-19, plasma therapy did not result in a significant clinical improvement within 28 days [ 49 ]. Literature such as this, has discouraged clinicians from using plasma as a potential treatment of COVID-19

Corticosteroids

The use of steroids in COVID-19 have been controversial as well. A study by Wu et al . reported that methylprednisolone may be beneficial, leading to decreased risk of death in patients with ARDS [ 50 ]. Some studies have shown a beneficial effect with low dose prednisone in cancer patients with COVID-19 [ 51 ,( 52 ]. A metanalysis of one randomized-controlled trial and 22 cohort studies showed that glucocorticoid therapy reduced the duration of fever but did not affect the mortality, duration of hospitalization or lung inflammation absorption [ 53 ]. A recently published open-labelled trial which studied dexamethasone vs usual care showed 28-day mortality benefit in those patients receiving invasive mechanical ventilation or oxygen with dexamethasone (Recovery trial). However, the positive results only applied to patients receiving respiratory oxygen support [ 54 ].

Other Experimental Modalities

Monoclonal or polyclonal antibodies were used to treat infections such as SARS-CoV, MERS [ 55 ]. LY-CoV555 (bamlanivimab) was studied in patients with mild to moderate COVID-19 studies as a phase 2 clinical trial, it led to fewer hospitalization and lower symptom burden leading to FDA approval among outpatients [ 56 ]. REGN-COV2 is a cocktail containing two neutralizing antibodies studied among 275 outpatients in a 1–3 phase trial, it showed that it reduced viral load with greater efficacy among those with a high baseline viral loads or those who have not yet developed immunity [ 57 ]. It is also now FDA approved for non-hospitalized COVID-19 patients.

Baricitinib, a janus kinase inhibitor, has been studied as a potential treatment for COVID-19 by preventing virus infectivity via inhibition of clathrin-mediated endocytosis [ 58 ]. A randomized-controlled trial was done comparing remdesivir plus baricitinib vs usual care and the combination with baricitinib reduced time to recovery in patients with high-flow oxygen and non-invasive ventilation [ 59 ].

Type II transmembrane serine protease (TMPRSS2) is a host protease that activates the SARS-CoV protein in cell cultures which makes it an exciting treatment option [ 7 ]. The protease inhibitor camostat plus EST [[ 23 , 25 ] trans -epoxysuccinyl- l -leucylamindo-3-methylbutane ethyl ester], a cathepsin inhibitor effectively stopped the SARS-CoV entry into host cells [ 60 ]. A randomized-controlled trial is underway to test the efficacy of camostat for COVID-19 (NCT04321096) [ 61 ].

Mesenchymal stem cells have improved the pulmonary functions in a case report described in China [ 62 ]. On a large scale, stem cell therapy for COVID-19 is being investigated in China currently (NCT04288102) [ 63 ].

Medications for concomitant conditions

The most common comorbidities of COVID-19 are hypertension and diabetes [ 64 ]. Many of these patients are on ACE inhibitors or angiotensin receptor blockers (ARB) and SARS-CoV-2 uses the ACE2 receptors to enter a host cell [ 64 ]. The theory is that these medications cause upregulation of ACE2 receptor which facilitates the viral entry. This is a double-edged sword since the use of these medications may prevent cardiovascular morbidity with could potentially be caused by the virus. A randomized-controlled trial evaluating the safety of continuing vs discontinuing ACE/ARBs is underway [ 65 ].

Non-steroidal anti-inflammatory drugs (NSAIDs), such as ibuprofen, were hypothesized to increase ACE2 receptors thereby facilitating the viral entry [ 64 ]. However, FDA released a statement that there is no scientific data suggesting that NSAIDs would worsen COVID-19 symptoms [ 51 ,( 66 ].

Currently, as of 22 nd December, per WHO’s draft evaluation, there are 61 vaccines under clinical investigation and 172 under pre-clinical investigation [ 67 ]. Two vaccines have been approved for emergency use authorization by FDA.

Conclusions

Till date, very few vaccines or specific anti-SARS-CoV-2 drug regimens have been identified to treat or prevent COVID-19. None of the drugs that have been studied has emerged as a clear winner. Remdesivir appears to be the most promising agent, however, results have been conflicting and it’s use has shown to have no effect on mortality. The most benefit from remdesivir is that it hastens time to recovery. Dexamethasone may be beneficial in severe COVID-19 cases, including patients on high-flow oxygen and invasive ventilation. The treatment atleast for now mainly focuses on supportive care and preventive measures including social distancing, wearing masks and avoiding large group gatherings.

Abbreviations

SARS-CoV2- severe acute respiratory syndrome coronavirus-2

SARS- CoV- severe acute respiratory syndrome coronavirus

MERS- Middle East Respiratory Syndrome Coronavirus

ACE2- angiotensin converting enzyme 2

ARDS- acute respiratory distress phase

ECMO- extracorporeal membrane oxygenation

RSV- respiratory syncytial virus

WHO- world health organization

IFN- interferon

HIV- human immunodeficiency virus

LPV/RTV- lopinavir/ritonavir

RA- rheumatoid arthritis

SOFA- sequential organ failure assessment

ARB- angiotensin II receptor blockers

NSAIDs- non-steroidal anti-inflammatory drugs

SIC- sepsis-induced coagulopathy

FDA- food and drug administration

Sources of Funding

This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors.

Disclosure statement

The authors do not have financial relationships with any commercial entity that has an interest in the subject of the presented manuscript or other conflicts of interest to disclose.

Author Contributions

RG, NK, NS assisted in acquisition of data, drafting of the manuscript. TD, CK, RJ assisted in article concept and design, interpretation of data, revision of the manuscript and final approval. RJ, NS, RJ further assisted in revisions of the final manuscript.

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Expanded Access

Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. 

Expanded access may be appropriate when all the following apply:

• Patient has a serious or immediately life-threatening disease or condition.
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Investigational drugs, biologics or medical devices have not yet been approved or cleared by FDA and FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.

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For emergency requests and specific questions during normal business hours:

• Oncology drugs: 240-402-0004 or [email protected]
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For emergency requests for all medical products (drugs, biologics, and medical devices) contact FDA's Emergency Call Center at 866-300-4374.

Report from the 2018 external assessment of the Expanded Access Program