Leleah Robinson, NORD’s Special Assistant to the Vice President of Public Policy, recently attended the FDA public workshops. This blog provides her insight into that event.
In July 2012, President Obama signed into law the Food and Drug Administration Safety and Innovation Act (FDASIA) which requires the FDA to hold at least one public meeting to “encourage and accelerate the development of new therapies for pediatric rare diseases”. FDASIA included reauthorization of the Prescription Drug User Fee Act (PDUFA) legislation and PDUFA V additionally obligates the agency to hold public meetings focusing on the advancement of drug development for rare diseases. The FDA held a three-day public workshop raising awareness about Complex Issues in Developing Drug and Biological Products for Rare Diseases January 6-7 and Complex Issues in Developing Medical Devices for Pediatric Patients Affected by Rare Diseases on January 8.
The Complex Issues in Rare Disease Drug Development workshop focused clinical trial design (endpoints, study, design, conduct and analysis), safety and dosing, and foundational science. Panelists stressed the importance of natural history data before a clinical trial, and standardizing outcome measures. However, a consensus was not determined about logistical questions related to conducting natural history studies such as:
The Complex Issues in Developing Medical Devices for Pediatric Patients Affected by Rare Diseases workshop focused on current approaches toward usage of medical devices for pediatric clinical practice, FDA’s action plans to address medical devices for pediatric clinical practice, clinical trial issues, needs assessment and approaches to advance pediatric device development. Medical devices are imperative toward the identification, accurate diagnosis, and treatment of a rare disease.
Currently, clinicians manipulate existing devices or use adult devices off-label in order to treat pediatric patients. Barriers contributing toward limited development of orphan devices include: small population size, high clinical trial costs, lack of financial incentives and a lengthy regulatory approval process. Several panelists provided recommendations to encourage medical innovation for pediatric medical devices:
FDA’s Office of Orphan Products Development (OOPD) is the home to humanitarian use device (HUD) designation and the Orphan Products Research Grant program, which are initiatives that address challenges with device development by advancing marketing approvals for rare disease products. The Orphan Products Research Grant program has partially funded over 50 products approved for marketing and HUD designations provide an alternative pathway for products to reach the market.
For more information from the three-day workshop, presentations can be found on FDA’s website:
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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