Bill > HR6888

This bill, the Helping Experts Accelerate Rare Treatments Act of 2022, aims to improve the treatment of rare diseases and conditions. The key provisions include: 1. Requiring the FDA to submit an annual report to Congress summarizing its activities related to designating and approving drugs for rare diseases, including the number of applications received, accepted, and approved, as well as the extent to which the FDA consults with external experts on rare disease topics. 2. Directing the Comptroller General to conduct a study on the European Union's process for evaluating the safety and efficacy of drugs for rare diseases, and to make recommendations for changes to the FDA's processes to facilitate the development of and access to treatments for rare diseases. 3. Requiring the Secretary of Health and Human Services to convene public meetings to solicit input from stakeholders on approaches to increasing and improving engagement with rare disease patients, patient groups, and experts to better understand patient burden, treatment options, and side effects. 4. Allowing the FDA to consult with external experts on the science of small population studies, which is important for rare disease research. 5. Directing the Comptroller General to study the FDA's use of mechanisms and tools to incorporate patient and clinician perspectives in its processes for approving or licensing drugs for rare diseases. Overall, the bill aims to improve the FDA's engagement with rare disease stakeholders and enhance the data and expertise available for the development and review of treatments for rare diseases and conditions.

Business and Industry, Health and Social Services

https://www.congress.gov/bill/117th-congress/house-bill/6888/all-info