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Accelerating Rare Disease Treatments: Increased Transparency and Patient Involvement

This act aims to streamline the approval process for rare disease drugs by increasing the transparency of the Food and Drug Administration (FDA) and ensuring greater involvement of patients and experts. This means citizens suffering from rare conditions can expect faster access to new therapies and better understanding of their needs.
Key points
The FDA will annually publish reports on the progress of rare disease drug approvals, increasing transparency.
Comparative studies of drug approval processes in the US and the European Union will be conducted to identify better solutions.
Public meetings will be held for patients and experts to provide input on rare disease treatments.
The FDA will more extensively incorporate patient and physician perspectives into the evaluation process for rare disease drugs.
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Additional Information
Print number: 117_HR_6888
Sponsor: Rep. Tonko, Paul [D-NY-20]
Process start date: 2022-03-01