The US government has prioritized Pfizer’s Meaningful Use and Transparency (MUT) Act over the development of orphan drugs for rare diseases. The MUT Act, aimed at improving healthcare, has pushed back the rarity drug development and reimbursement. This may make it more challenging to find cures for rare diseases.
The latest US budget deal includes a short-term fix for the Pharmaceutical Benefits Manager (PBM) reform, which helps provide prescriptions to patients. While a step in the right direction, it does not address the needs of patients with rare diseases.
The PBM reform lacks specific provisions for rare diseases, which could hinder the development of new treatments. This is concerning, as rare diseases affect more than 30 million Americans, and there are no FDA-approved treatments for many of them.
Experts emphasize the need for federal policies that incentivize, not hinder, the development of orphan drugs for rare diseases. It is crucial for the US government to prioritize the needs of patients with rare diseases and provide the necessary support for the development of new treatments.