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FDA and the Voice of the PatientsThe FDA approved the Pipeline Project’s proposal to expand the agency’s Patient Consultant Program to include Parkinson’s disease. Parkinson patient advocates now are incorporating the patient perspective into the FDA drug review regulatory process. As patient consultants in the pre-approval, clinical trial phase of Parkinson’s drug development, they advise the FDA and drug sponsors on topics such as clinical trial design, endpoint determination, expanded access, protocol development, and clinical trial patient recruitment strategies. The program is modeled after the successful Cancer Drug Development Patient Consultant Program initiated in 2001, and a similar one involving HIV/AIDS patient advocates. “Parkinson’s is a good candidate to lead other neurological conditions in this pilot effort because of the active community advocating for enhanced scientific research and new therapy development,” said Perry Cohen, Ph.D., who has served as an FDA patient representative since the Deep Brain Stimulation Advisory Panel in March, 2000. (5) The FDA has named the following Parkinson’s disease patient advocates to serve as special consultants on the development of new Parkinson's disease therapies:
Involving patients in the design of studies to evaluate new therapies accelerates the process of bringing more effective treatments to market, and ensures measurement of a broader range of clinical outcomes in weighing the risk/benefit tradeoffs of an investigational treatment. For example, non-motor Parkinson’s symptoms were not given much attention until an October 2003 survey by people with Parkinson’s (PWP). The survey, The Impact of Parkinson's Disease on Quality of Life, revealed that loss of energy and pain were two of the top three most disabling symptoms. (1) This discovery prompted health care professionals to start asking patients which PD symptoms were most troubling, resulting in a new focus on autonomic functions (e.g. swallowing. digestion, and cognitive and psychological factors, including memory and mood changes). Decisions about what to measure, how to administer treatment and trial length are made during the design phase, with little opportunity to adjust the protocol based on feedback from trial participants. Often questions arise during study design concerning what patient participants will or will not do, with assumptions made that can greatly affect outcomes. Patient advisors bring the patient perspective to questions such as, “Does the proposed clinical trial provide adequate disclosure of risk information to patients in a manner that is understandable?” Documenting the safety and efficacy of new treatments is a slow process. The two to four years added to repeat a flawed study may mean the difference in disability for those with chronic illnesses, and/or even life and death. Patient consultants have been hired as Special Government Employees and are subject to the FDA's conflict of interest and confidentiality regulations that govern each FDA employee, consultant, and advisor. Parkinson’s disease patient advocates chosen to serve as patient consultants have been receiving training 1) at FDA's Rockville, Maryland facilities, at FDA expense, 2) by telephone lectures from FDA staff along with cancer patient consultants and 3) in joint training sessions with PD industry consultants. The FDA will arrange for patient consultants to participate via telephone in FDA in-house meetings and meetings with sponsors. Pipeline Project Director Perry Cohen has consulted with FDA staff in OSHI on the coordination of our company advisor training-development with the FDA patient consultant efforts.
For further information regarding the application process, selection
criteria, and training of PD-FDA patient consultants visit the FDA
Office of Special Health Issues at
http://www.fda.gov/oashi/cancer/pconback.html,
or contact JoAnn Minor or David Banks at 301-827-4460 Patient Reps’ Unique Insights Enhance FDA Decision-MakingThe Patient Representative Program recruits and trains advocates to serve on FDA advisory committees, providing “insight on issues, problems, and/or questions pertinent to the viewpoint of patients and family members living with a specific serious or life-threatening disease.” (4) This input “contributes to the quality of the FDA’s regulatory decision-making and lends credibility to the product review process.” (1) “When it comes to approving a drug’s use, you want to prolong someone’s life, make it better, or both,” said Steven Hirschfeld, M.D., Ph.D., a medical officer in the FDA's division of oncology drug products. “The hard part is figuring out what and how you can determine what 'better' means. No one else will have the experience and perspective of a patient familiar with a disease to determine if a benefit is meaningful or if the burden is severe.” (3) Patient representatives and care-givers, and industry and consumer advocates work alongside physician-scientists, statisticians, epidemiologists, nutritionists, and toxicologists, discussing unique aspects of safety, effectiveness, or clinical development of a particular product. Advocates’ experience as patients enable them to voice “real world” concerns of the potential recipient of new drugs, biologics (e.g. vaccines), and medical devices under review. (1) “Access to a growing range of products offers opportunities for advancing public health and improving people's lives, but also …creates greater potential risks for people who use the products.” (1) To keep up with the challenges FDA experts face when evaluating cutting edge scientific medical developments, the agency hires outside advisers as "special government employees” to serve on technical and scientific advisory committees, which have a prominent role in the product approval stage. Committee recommendations are non-binding. Final regulatory decision rests with the FDA. Sources
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Copyright © 2004-2006 Parkinson Pipeline Project. All rights reserved. Revised: 06/24/08. |