The Patient Advocate Perspective on Preparing for FDA Advisory Committee Presentations
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D ~ Information R Juumal. Vol. 34, pp. 969-973. Zoo0 Printed in he USA. All rights reserved.
THE PATIENT ADVOCATE PERSPECTIVE ON PREPARING FOR FDA ADVISORY COMMITTEE PRESENTATIONS ABBEYS. MEYERS President, National Organization for Rare Disorders (NORD). New Fairfield, Connecticut
This article highlights the author S experiences as a patient advocate serving on the Food and Drug Administration (FDA) Biological Response Modifiers Committee. It provides an overview of the National Organization for Rare Disorders and the scope of the orphan disease problem. The author explains the process of serving on an advisory committee, and highlights the important role of patient advocates in protecting the patient S best interests. Key Words: Orphan drugs; FDA advisory committees; National Organization for Rare Disorders; Patients
I WAS THE CONSUMER representative on the FDA Biological Response Modifiers Advisory Committee during the past three years, was recently appointed as an FDA consumer consultant for the Center for Biologics Evaluation and Review, and serve on the Xenotransplantation Subcommittee. I have appeared as a consumer to testify to other FDA advisory committees when I felt strongly about specific issues related to certain orphan drugs. In this regard, it seems to me that the advisory committee system is one of the rare demonstrations of democracy in action because it provides an opportunity for ordinary people to express their opinions directly to our government and to scientists who may sometimes forget that the scientific data, including all of their charts and graphs, are really composed of people, human beings
Presented at the DIA 35* Annual Meeting, June 27July 1, 1999, Baltimore. Maryland. Reprint address: Abbey S. Meyers, President, National Organization for Rare Disorders (NORD), 100 Rt. 37, PO Box 8923, New Fairfield, (JTO6812. E-mail: [email protected].
who sacrifice a precious part of their lives when they agree to participate in clinical trials. The National Organization for Rare Disorders (NORD) is a nonprofit federation of approximately 140 voluntary health agencies dedicated to the identification, treatment, and cure of an estimated 6000 rare “orphan diseases.” We are the consumer organization that came together to advocate for the Orphan Drug Act, which was passed into law in 1983. Since that time, we have monitored the implementation of the law as well as the pharmaceutical industry’s participation in orphan drug development. We have also supported international efforts resulting in orphan drug legislation in Singapore, Australia, Japan, and currently the European Union. Our goal is to enlist the scientific and corporate resources of all industrialized countries of the world in order to forge an international effort to alleviate the consequences of rare diseases. The scope of the orphan disease problem is immense: In the United States there are more than 6000 orphan diseases affecting an
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Abbey S. Meye
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