Dear Friends and Colleagues –
We wanted to alert you to a recently published notice regarding an upcoming public hearing at the FDA on the topic of Review and Regulation of Articles for the Treatment of Rare Diseases. The FDA is seeking responses on the following questions:
- Orphan drug marketing applications are reviewed under the same review process and statutory standards regarding demonstration of safety, effectiveness, and product quality as drugs for patients with non-orphan diseases or conditions. FDA is sensitive to the unique needs of patients with rare diseases as it makes approval decisions regarding the overall risk-benefit profile of therapies for the particular patient population for which they are being considered. Please comment on whether this practice has adequately addressed the needs of patients with rare diseases.
- FDA designates a medical device as an HUD (humanitarian use device) designed to treat or diagnose a rare disease—defined in this instance as a disease affecting or manifesting in fewer than 4,000 patients per year. Please comment on whether this practice has adequately addressed the needs of patients with rare diseases.
- Current regulations for the approval of an HUD through the HDE pathway require that the application have a ‘‘description of the device and a discussion of the scientific rationale for the use of the device for the rare disease or condition’’ and ‘‘an explanation of why the probable benefit to health from the use of the device outweighs the risk of injury or illness from its use, taking into account the probable risks and benefits of currently available devices or alternative forms of treatment’’ (21 CFR 814.102 and 814.104). Please comment if you believe that these standards remain appropriate for the approval of devices for rare diseases under the HDE mechanism…..
- Have current processes for rare disease stakeholders to communicate with FDA regarding rare disease article development been useful? How could these processes be improved?
The complete public hearing notice is attached (PDF). Comments are due May 31. FYI on this wonderful opportunity to provide constructive feeback to the agency.