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FDA Public Hearing: Medical Device User Fee Program September 14, 2010. Diane Edquist Dorman Vice President. National Organization for Rare Disorders…. Serve the 30 million men, women and children in the U.S. affected by the 7,000 known rare diseases Education and outreach
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FDA Public Hearing:Medical Device User Fee ProgramSeptember 14, 2010 Diane Edquist Dorman Vice President
National Organization for Rare Disorders… Serve the 30 million men, women and children in the U.S. affected by the 7,000 known rare diseases Education and outreach Liaison with U.S. Congress, federal agencies, biopharmaceutical and medical device industries, patient organizations, medical societies
NORD’s Continuing Commitment to the FDA… • Office of Orphan Product Development • Science of Small Clinical Trials • Orphan Designation Workshops • Joint FDA/NIH/NORD Task Force • Rare Diseases Office in OND/CDER • FDA/NIH/Duke Univ./NORD: Accelerating Rare Disease Therapeutics Development: Research-Investigator Workshop
Alliance for a Stronger FDA… • FY 08 $238 million • FY 09 $280 million • FY10 $315 million • FY 11 Alliance request $385 million • FY 11 Presidents request $326 million • FY 11 Senate request $326 million
Humanitarian Use Devices… • “A HUD is approved for marketing through an HDE application filed in accordance with the requirements of this final rule. An HDE application is a PMA application that is not required to contain clinical data demonstrating “effectiveness” as “reasonable assurance based upon valid scientific evidence, that in a significant portion of the target population, the use of the device for its intended uses and conditions of use, when accompanied by adequate directions for use and warnings against unsafe use, will provide clinically significant results. 21 CFR Parts 20 and 814, Medical Devices, HUD, Final Rule Oct. 24 1996
Humanitarian Use Devices… • Treat or diagnose disease/condition that affects fewer than 4,000 people in the U.S. • Device would not be available unless exemption permitted • Benefit outweighs risk 21 CFR Parts 20 and 814, Medical Devices, HUD, Final Rule Oct. 24 1996
Humanitarian Use Device… • HUD cannot be sold for an amount exceeding cost of R&D, manufacturing & distribution costs • Can only be used in facilities that have established IRBs to supervise clinical testing
Challenges Facing Development of Medical Devices for Rare Diseases… • Lack of natural history & incidence data • Patients geographically dispersed • Statistical challenges with small studies • Few knowledgeable investigators, physicians/surgeons
Challenges Facing Development of Medical Devices for Rare Diseases… • Limited reimbursement - HUDs considered “investigational” • Lack of incentives for HUDs • No tax credits • No profits (except for pediatric HUDs)
Next Steps… • Increase funding for the Orphan Products Research Grants Program • Increase investment in the pediatric device consortia program • Carefully weight risk vs. benefit • Increased cooperation between all centers • Increase flexibility & transparency
Next Steps… • Allow profit for ALL humanitarian devices • Address the “experimental” issue • Increase funding for the Orphan Products Research Grants Program • Increase investment in the pediatric device consortia program
Vertical Expandable Prosthetic Titanium Rib(VEPTR)… -IOM Report, Safe Medical Devices for children, 2005
Contact Information… Diane Edquist DormanVice President, Public PolicyNational Organization for Rare Disorders (NORD) 1779 Massachusetts Avenue, NW, Suite 500 Washington, DC 20036 Office/(202) 588-5700 Email/ddorman@rarediseases.org http://www. Rarediseases.org
