The Risks and Benefits of Participating in a Clinical Trial

There are always certain risks to clinical trials. We want to give you a full perspective of participation in an experimental treatment, covering both the positive and the negative aspects.

Potential Benefits

Your participation contributes to medical knowledge of new therapies and helps hundreds of people who are waiting for treatments to appear.
You gain access to innovative treatments before they are widely available.
You have the support of a medical team that monitors your health closely.
You get free medical care and monitoring.

Potential Risks

There may be side effects to the experimental treatment. They may range from slight discomfort that goes away on its own to a serious condition that will require medical assistance. In rare cases, you may develop life-threatening complications.
The research may require more of your time and attention than the standard treatment would: you need to undergo testing more frequently, pay regular visits to the clinic, go through medical procedures, and adhere to complex drug regimens.
You may be assigned to get a placebo because comparing a new drug with a dummy drug allows researchers to assess its effectiveness more accurately and quickly. However, a placebo should not be used if withholding effective treatment may be harmful to you.

Before enrolling in a clinical trial, you should carefully consider all possible risks and benefits of participation. Someone from the research team will explain the details of the trial and answer any questions related to your rights and safety.

Scientific Supervision of the Clinical Trial Safety

Each clinical trial has scientific oversight that deals with any concerns regarding the safety of experimental treatment.

The Institutional Review Board (IRB) is an independent committee of scientists, doctors, and community members who ensure that clinical trials are ethical (research risks are minimized and reasonable in relation to anticipated benefits) and assure the protection of participants’ rights. The IRB approves and oversees most clinical trials in the United States.
The Office for Human Research Protections (OHRP) helps protect the rights and wellbeing of healthy volunteers participating in trials.
Data and Safety Monitoring Board (DSMB) reviews data from clinical trials to address safety concerns.
The Food and Drug Administration (FDA) reviews new drug applications from companies before clinical trials and verifies that the informed consent requirements have been complied with.

Role of FDA Review Team in Clinical Trials

An FDA review team consists of experts in different scientific fields. Each member has different responsibilities.

Project manager: is the primary contact with the sponsor as he/she coordinates activities of the team throughout the review process.
Medical officer: reviews all clinical trial information and data before, during, and after the research is completed.
Statistician: interprets clinical trial projects and data, and works closely with the medical officer to evaluate protocols, data about safety and effectiveness.
Pharmacologist: reviews non-clinical studies.
Pharmakineticist: focuses on the processes of drug’s absorption, distribution, metabolism, and excretion. He/she interprets blood level data at various time intervals from clinical trials to estimate drug dosage and administration schedules.
Chemist: evaluates the chemical compounds of a drug, analyzes how the drug was manufactured along with its stability, quality control, continuity, presence of impurities, etc.
Microbiologist: reviews the obtained data if the drug is an antimicrobial product to assess response across different classes of microbes.

The FDA review team has 30 days to review the risks of a clinical trial. Based on the safety assessment of the experimental treatment, the team of experts issues a decision.

Approval to start a clinical trial.
Clinical hold to delay or permanently stop the proposed trial.

There are 5 Reasons why FDA can Impose a Clinical Hold on Trial

Participants are exposed to unreasonable or significant risk.
Investigators are not qualified.
Materials meant for volunteer participants are misleading or incomplete.
Informed consent does not provide enough information to assess the risks associated with the trial.

Clinical holds are rare. Instead, the FDA provides comments to help companies improve the quality of their trials.

What is the Purpose of a Placebo in a Clinical Trial?

In clinical trials, a new drug is sometimes compared to a placebo. It is a substance that contains no active ingredient (a pacifier) and looks identical to the original medication. For ethical reasons, placebos are never used where proven effective treatments are available. The only exceptions are trials in which a new drug or placebo is added to the standard therapy (instead of replacing it!).

The placebo group helps researchers quickly measure the effectiveness and safety of a new drug. Besides, some diseases can cause weakness, nausea, loss of appetite, and other unpleasant symptoms on their own. Having a placebo group allows researchers to understand whether an unpleasant symptom is a side effect of a new drug or a manifestation of the disease under study.

In many trials, uneven allocation of patients to the placebo group is used. For example, the new drug group may have twice as many patients as the placebo group. Also, some clinical trials are designed in a way that allows transferring patients from the placebo group to the test drug group. It happens when a new drug has shown significant clinical advantage compared to a placebo.