Policy/Position Papers

Patient Protection in Clinical Trials

Background
After World War II, the United States and most other industrialized countries established regulations governing the protection of human subjects in research. In September 1999, a young research subject died as a result of his participation in a gene therapy clinical trial. Since then, the adequacy of patient protections in clinical trials has been catapulted to the center of national debate.

Problem
Existing patient protection regulations are inadequate and have not been updated to address current ethical issues, new evolving scientific technologies, the changing ratio of privately-funded vs. publicly-funded research, and the dominance of multi-institution research projects. Current oversight of the patient protection system is the responsibility of local Institutional Review Boards (IRBs), and government enforcement of uniform federal rules has been minimal.

Policy Position

• The “Common Rule” – which is the code of conduct for human research involving federal funds – must be applied to all human subject research conducted in the United States.
  
  
• All human subjects must be given informed consent documents that are written in understandable language to ensure that patients are able to make an informed decision based on truthful and complete information. When patients are incapable of giving their consent, their legal guardian or family member should be afforded the same informed consent process with unbiased and accurate descriptions of possible risks and benefits.
  
  
• Federal law and regulations must be updated to incorporate the various technological advancements in medical research – including genetic testing, gene therapy, human tissue research, xenotransplantation, etc., which raise new scientific, ethical, psychological, financial and legal issues that have not heretofore been addressed.
  
  
• Clinical research projects must undergo independent and unbiased review and monitoring by an Institutional Review Board (“IRB”) to ensure ethical behavior and protections against undue harm and risk.
  
  
• IRBs must have increased resources and training in order to meet their growing responsibilities, monitor the conduct of research, and act as the first line of defense in patient protections.
  
  
• The federal government should adopt recommendations by the General Accounting Office and the Health and Human Services Inspector General’s Office that require the following:
  
  
  • Registration of all IRBs and all research projects involving human subjects;
    
    
  • Membership of non-scientific members and non- institutional members on
    
    
  • IRBs to reduce possible conflicts of interest;
    
    
  • Revealing to IRBs if a research project has been previously submitted and
    
    
  • rejected by other IRBs.

Whom We Represent
NORD is a federation of approximately 140 not-for-profit health organizations and thousands of individuals and medical professional serving people with rare diseases. As a voluntary health agency dedicated to helping people with rare disorders, NORD encourages and promotes clinical research and is working to ensure that all research volunteers have the greatest protections afforded to them.

For more information, contact: Diane E. Dorman, Senoir Director for Public Policy at (301) 421-0018 or via e-mail at ddorman@rarediseases.org.

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