Accelerating Rare Disease Drug Development Through Expert and Patient Collaboration.
This law establishes a mandatory process for meetings involving medical experts, drug companies, and patient organizations to speed up the development of treatments for rare diseases. It increases transparency in drug approval, requiring the FDA to publicly explain how expert input was used in assessing the risks and benefits of new medications. Citizens with rare conditions may benefit from faster access to innovative therapies.
Key points
Mandatory, externally led meetings (minimum four annually) are introduced to streamline the development of drugs for rare diseases, involving patients and experts.
Increased transparency: The FDA must publicly disclose how input from these meetings influenced the decision to approve a new drug.
A permanent steering committee, including patient representatives, is established to ensure development efforts focus on the most urgent unmet therapeutic needs.
Expired
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