How to Evaluate a Clinical Trial

Alan Franciscus

Editor-in-Chief, HCV Advocate Website

Observational study:  a study to observe study participants to evaluate certain outcomes.  For instance, an observational study may evaluate the effect of HCV treatment on quality of life, how being cured (or not cured) affects an individual’s perceived stigma, etc.,

Interventional study:  an experimental study to test a new medicine to treat a certain condition.

This article discusses clinical trials with an emphasis on interventional clinical studies.

Institutional Review Board (IRB)
Every clinical trial in the United States must be approved and monitored by an Institutional Review Board (IRB).  The IRB evaluates the study to make sure that it is ethical and that clinical trial participant is not harmed by the drug under study.  Every study participant must read, understand and sign a consent form.  The consent form lists the possible benefits and the potential dangers or health risk to a study participant. Patients should ask questions and be as actively involved as much as possible in the clinical trial process.  A clinical trial coordinator is assigned to each clinical trial.  The study coordinator will follow the participant throughout the study.  It is the responsibility of the coordinator to make sure the patient understands the potential risks and benefits of the clinical study.

Getting support:
When a patient meets with the medical provider or the study coordinator, it is important to plan ahead and write down questions. Ask a friend or relative to accompany you to hear the responses, and write down the discussion to review later. If the patient is young, bring a babysitter so parents/caregivers can speak privately, without distractions.

Questions patients should ask before enrolling in a clinical trial:

  • What is the drug(s) being tested?
  • Is the study drug being tested to find out if it is better than the standard treatment for hepatitis C?  This could be higher cure rates, lower side effects, shorter treatment duration, etc.
  • Is there a placebo drug in study?  If so, what are the chances of receiving the study drugs versus the placebo?  The person who receives the placebo drug should ask if they will be offered the study drug at the end of the trial.
  • What are the possible risks and side effects?
  • What are the possible benefits of the experimental drug?
  • Is it possible that the HCV antiviral drug may cause viral resistance or cross-resistance to another HCV antiviral medication?
  • Will the experimental drug interfere with other medications such as HIV or hepatitis B medications?
  • How will the treatment affect a person’s quality of life?
  • Will researchers cover the cost of the drug and doctor’s visits? What costs are not covered?
  • What follow-up care is required?
  • What determines if the drug is successful?
  • Will results of the trial be provided to participants?
  • Will a specialist or a primary care provider be in charge of a patient’s care?  Will my physician be in contact about my care?
  • Who is the study sponsor—is it a pharmaceutical company, the National Institutes of Health, a university or other sponsor?

Randomized trials risks and benefits:
Participating in a randomized trial means that some patients will receive a placebo pill (a sugar pill that provides no benefit), some patients will receive the current standard treatment for hepatitis C, and others may receive the new experimental drug.  A computer program will ‘randomly’ assign patients to the different treatments.

 

Different Phases of a Clinical Trial

Pre-clinical studies:  A drug is tested in test tubes or on animals to evaluate the potential toxicities and drug exposure levels—that is how much of the drug in the body/blood.  If the medicine shows promise, the medication will advance into the next clinical phase.

Phase I: The drug will be tested in healthy people.  The main goal is to find out how the drug works in the body, the safety of the drug, and the dose of the drug that is safe and effective.   The experimental drug can also be given to people who have the condition the drug is being developed to treat.

Phase II:  In this phase, the study drug is tested in individuals who have the medical condition. The safety of the drug and the side effects are evaluated.  In some phase II studies, the new investigational drug may be compared to a placebo drug or the standard treatment of the condition.  The effectiveness of the drug is evaluated—that is, does it cure hepatitis C?

Phase III:  The results of phase I and II studies are used to design the phase III study—drug dose(s), how long to treat someone, etc. The drug can be compared to the current standard treatment to find out if the study drug is superior or as effective as the current drug therapy.  During a phase III study, patients are treated with the new drug, the current standard treatment, or a placebo.  If the phase III results are successful, the pharmaceutical company will apply to the Food and Drug Administration (FDA) to market the drug to treat the condition tested.

Phase IV:  These are post-marketing studies.  The FDA may require that the pharmaceutical company conduct additional studies after approving a medication.  The post-marketing studies can be about treating sub-populations with the same condition that may not have been included in the original studies or for the treatment of a different condition.

Finding a Clinical Trial

Listed below are resources that can refer a patient to a clinical trial:

  • U.S National Institutes of Health’s www.clinicaltrials.gov registry lists clinical trials throughout the world.  It is a very good site to find a clinical trial for any condition.  However, it can be overwhelming to try to find a study that is recruiting patients for a certain medical condition
  • Medical providers may refer patients to a clinical trial in their area
  • University hospitals frequently have clinical trials
  • HCV Advocate Hepatitis C Clinical Trials Blog is designed to simplify the process by listing current hepatitis C clinical trials

It is important to remember that clinical trials are conducted to find out if a medication is safe and effective.  Every attempt is made to safeguard the trial participants who enroll in a clinical trial. However, there is a chance that the drugs being tested, especially in the earlier phases, could produce severe side effects.  Although rare there is a possibility of death.  This is why is it important to weigh the possible benefits against the potential risks in the decision-making process, especially with interventional studies.

Related publications:

Making Sense of Hepatitis C Research and Medical Literature – Lucinda K. Porter, RN

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