Accelerating Rare Disease Treatments: New Rules for Orphan Drugs
This act aims to streamline the approval process for drugs treating rare diseases, known as orphan drugs. It mandates annual reports on progress in this area and requires a study of European experiences. It also increases the involvement of patients and experts in the drug evaluation process, which should accelerate access to necessary therapies.
Key points
Annual public reports on the progress of orphan drug approvals.
Study of European methods for evaluating rare disease drugs to improve U.S. procedures.
Increased involvement of patients and experts in drug evaluation, especially for rare and genetically targeted diseases.
Expired
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