Participating in a Clinical Trial

The integrity of any clinical trial depends upon mutual trust between clinical investigators and study subjects, be they healthy volunteers or patients. Such a partnership also requires that a potential study subject (or a person who is responsible for the subject’s welfare, such as a family member) is aware of and understands what it means to participate in a trial (See Box 2 below). Subjects should know the trial’s purpose. What are the goals that the investigators want to accomplish by conducting the trial and what are the hypotheses they are testing? Remember that hypothesis-testing is belief-testing; thus, were the results of the trial already known, there would be no scientific or ethical reason for conducting it in the first place.

Box 2

Participating in a Clinical Trial

  • Know its purpose, generally and specifically

           -hypothesis testing is belief testing

  • Know the logistics

           -travel

           -hospitalizations

           -procedures

  • Know the investigators

           -competency in field of research

  • Understand the consent form

           -procedures, risks, benefits

           -right to refuse/withdraw without penalty

           -compensation

  • Participation = Partnership = Mutual Trust

Other questions to ask before participating in a clinical trial include:

  • What are the requirements regarding travel to and from the treatment center?
  • How long will the trial last?
  • How long will your participation be and how will your medical care be affected after your involvement ends?
  • When will you be made aware of the trial’s overall results and the details of your own responses to treatment?
  • Is the study conducted in an outpatient setting or does it require one or more hospitalizations and, if so, for what periods of time?
  • What are the procedures involved in conducting the study, such as blood and urine tests, x-rays, other imaging studies, biopsies, etc.?  

Subjects should also know the background and experience of investigators relevant to the research undertaken by the trial, including their familiarity with the investigational treatment.

Most importantly, subjects should understand the Informed Consent Form (ICF) that is provided to all individuals who are considering participating in any form of human research. The ICF should outline the procedures, risks and benefits of participating in the study in language understandable to persons with no more than an 8th grade education. The ICF must acknowledge the subject’s right to refuse participation or to withdraw at any time after enrollment without penalty. It must also identify what compensation, if any, a subject would receive for their participation. Subjects enrolled in a Phase I clinical trial usually receive monetary compensation, whereas patients who participate in later stage trials usually are not compensated beyond routine travel expenses.

Protection of Subjects

Well-designed clinical trials include several features that safeguard the welfare of participants (See Box 3 below). Each institution that conducts human research must have at least one Institutional Review Board (IRB), comprised of both professional and lay members, to determine whether the study is ethical, minimizes risks to the participants and has an appropriately written ICF. An investigator must have IRB approval before enrolling any subject in a clinical trial. Studies conducted in a GCRC benefit further from two additional levels of protection. A GCRC Scientific Advisory Committee of clinicians and scientists who are expert in the particular area of research reviews the scientific merits of the trial and determines whether its hypotheses, study design and methods of data analysis are scientifically sound and likely to provide useful new information. A GCRC Research Subject Advocate (RSA) is an individual knowledgeable about both local and federal guidelines governing the protection of human subjects. The RSA is available to meet with subjects to address questions or concerns that may arise during the course of their participation in the trial.

Box 3

  • Safeguards and Ombudsmen
  • Institutional Review Board
  • GCRC Scientific Advisory Committee
  • GCRC Research Subject Advocate
  • Data Safety Monitoring Board
  • FDA

Any clinical trial that involves so-called vulnerable populations, including children, prisoners, or people with impaired intellectual capacity, or any studies that include the blinding of subjects and/or investigators requires the existence of a Data Safety Monitoring Board (DSMB). This Board consists of experts in both the disease under study and in the ways to analyze the results of the trial. A DSMB also usually includes an expert who understands the ethical issues that relate to the trial and a member of the lay community. DSMB members are advocates for the patients participating in a particular trial. Consequently, they can recommend that the study ends earlier than originally planned, either because it is not being conducted well by the investigators or because the test drug demonstrates important efficacy or toxicity that was not originally considered when planning the study. The appearance of either of these effects would prevent further evaluation of the drug in a trial that is blinded.

Another level of safety is provided by the FDA, which must receive continuous progress reports of a trial. The FDA may also require changes in or early stoppage of the study, based in part upon its ongoing review of potential risks to study participants.

Why Participate in a Clinical Trial?

In the U.S., and in all other developed countries, clinical trials are an absolutely essential part of the development and approval of new treatments for both common and rare diseases. Since the 1960s, when the U.S. Congress required that new drugs demonstrate not only safety but also efficacy before they could be approved, ethically and scientifically well-designed clinical trials have been the springboard from which new knowledge gained from medical research is translated into new therapies.

Although critical information can be obtained early in the assessment of potential new therapeutics by word-of-mouth or by open-label studies, these are very self-limiting in terms of advancing the field of new drug development. Patients who are in need of new treatments for their diseases may be confused by such claims that cannot be confirmed by their evaluation through clinical trials. Patients may also believe that “more is better,” meaning that if a little of a drug, vitamin, cofactor, etc. is beneficial, taking more of it can only help. Such a belief may not only be baseless on scientific grounds but could potentially be harmful, unless it is tested appropriately in a rigorously controlled trial.

Searching for Clinical Trials

The Internet provides rich sources of information regarding both diseases and ongoing clinical trials in which patients may consider participating. A Google search of a disease may identify trials targeted to that condition. Exceptional Parent frequently advertises clinical trials for special needs populations. CenterWatch (www.centerwatch.com) provides a large and frequently updated database of clinical trials, particularly for more common diseases, while the National Organization for Rare Disorders (www.Nord.com) and the Office of Rare Diseases at NIH (http://rarediseases.info.nih.gov) are excellent sources for information about clinical trials for rare disorders.

 

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