Rare Disease Day: 

On February 27, 2023, FDA hosted a virtual Rare Disease Day the purpose of which was to explore patient and patient advocate engagement in supporting the development of products for rare diseases.  During the meeting, FDA outlined the common challenges in developing products for rare diseases, including poorly understood natural history, small patient populations, ethical issues associated with conducting clinical trials in pediatric patients, phenotypic/genotypic diversity within a rare disease, difficulty identifying outcome measures and biomarkers to establish clinically meaningful benefits, and business-related challenges associated with economically infeasible indications.

FDA also discussed Project Catalyst, a program under the Oncology Center for Excellence which is intended to provide innovation and development support to small organizations (e.g., academia, start-ups).  Their educational initiative, entitled Oncology Regulatory Expertise and Early Guidance (OREEG) is a great place for organizations to obtain product-type advice to inform product-specific development.  The program includes the ability to direct specific questions to FDA experts as well as participate in Accelerator Innovation Discussion (AID) meetings prior to any formal meetings with FDA.

Of note, FDA has observed that most companies in the rare disease space are lacking regulatory experience and strongly recommended that they partner with regulatory and development consulting organizations, like Facet, that can help bridge the science-regulation gap.

Diagnostic radiopharmaceuticals now deemed “drugs” by Congress: 

Congress passed an amendment to the Food, Drug, and Cosmetics Act (FD&C Act or FDCA) within the omnibus appropriations bill that passed on December 23, 2022.  The Genus decision and regulation of contrast agents and radiopharmaceutical drugs was included in this amendment and is now final.  Specifically, any contrast agent, radioactive drug, or OTC monograph drug shall be deemed to be a drug under section 201(g) and not a device under section 201(h).  The term “contrast agent” is defined as a product that is used in conjunction with a medical imaging device and meets the definition of a diagnostic radiopharmaceutical (21 CFR 315.2 and 601.31) or an agent that improves the visualization of structure or function within the body by increasing the relative difference in signal intensity within the target tissue, structure, or fluid.