Congress Introduces Bill to Speed up Rare Disease Treatment Access

Congress has recently introduced a new bill that would have a significant impact on the rare disease community if it passes. The legislation, called the Speeding Therapy Access Today (STAT) Act, was introduced in both the House and Senate. In the House, G. K. Butterfield (D-NC) and Gus Bilirakis (R-FL) sponsored the bill; on the Senate side, Amy Klobuchar (D-MN) and Roger Wicker (R-MS) backed the bill.

The law follows over a year of collaboration between these lawmakers and partners in the rare disease community. In many rare diseases, prompt and timely treatment can be essential in producing the best outcomes, the goal of this law was to find areas where access to treatment could be accelerated.

The STAT Act

If passed, the STAT Act would establish the Rare Disease Center of Excellence at the US Food and Drug Administration (FDA). It would serve as the agency’s coordinating office with relevant stakeholders. The Rare Disease Center of Excellence is based off of the success found with the Oncology Center of Excellence, which was first established in 2017. The center has improved cancer therapy development and provides a tried and true model. The establishment of this center would improve access to treatment and accelerate treatment development by:

  1. Optimizing interagency coordination of rare disease experts;
  2. Advancing science-based regulatory policy;
  3. Targeting new development for the rarest diseases;
  4. And ensuring that the target patient population receive access to newly approved therapies.

The STAT Act would also lead to the creation of a Rare Disease and Condition Drug Advisory Committee, chaired by the director of the Center of Excellence. This committee will provide input on clinical trial design, drug repurposing, biomarkers, and regulatory pathways. The committee will advise the Health and Human Services Secretary, who is responsible for appointing its 15 members. Membership is intended to reflect a variety of interested parties, such as patients and scientists. 

Development for ultra rare disease would be facilitated by the Accelerating Lifesaving Therapies in Treating Ultra-Rare Disease Entities (ALTITUDE) program, which would make recommendations regarding manufacturing, regulations, and trial design to accelerate therapy development.

This is just part of what the STAT Act has to offer. To learn more about this promising piece of legislation, click here

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