On November 18, 2021, the Rare Disease Legislative Advocates (RDLA) held their monthly webinar. These webinars provide the rare disease community with updates regarding relevant legislation. As the year begins to wind down, several bills continue to wait in the wings. Several speakers joined the virtual program to provide updates on them:

• Cheryl Jaeger, Williams & Jensen, PLLC
• Sarah-Lloyd Stevenson, Faegre Drinker Consulting
• Jonathan Daniels, Cystic Fibrosis Foundation
• Jamie Sullivan, EveryLife Foundation for Rare Diseases

Cheryl started off the program by providing an update on the overall legislative landscape  as the end of 2021 approaches. One goal that Congress is aiming to complete before year’s end that is relevant to the rare community is the confirmation of a new commissioner for the US Food and Drug Administration (FDA). The nominee is Robert Califf, who previously served as commissioner under the Obama administration.

Sarah-Lloyd spoke about the Advanced Research Projects Agency for Health (ARPA-H). The ARPA model has been used in the past for defense and intelligence research as an engine for funding ‘high risk, high reward’ research projects. ARPA-H would take this model into the health and medicine field. This research would be focused on technologies that could cause system-wide breakthroughs in healthcare. In President Biden’s budget recommendations for next year, he called for a $9 billion increase for the NIH, which would include $6.5 billion specifically for establishing ARPA-H. There are still many points of contention, however.

Jonathan talked about the Pioneering Antimicrobial Subscriptions To End Up Surging Resistance Act of 2021 (PASTEUR Act). This legislation is of critical importance to the cystic fibrosis community as it aims to address the threat of antibiotic resistance. These patients often have to take antibiotics regularly, and their infections can develop resistance to these treatments. Antibiotic resistance is also a growing medical problem around the world that affects people far beyond just cystic fibrosis. 

This legislation aims to allocate $11 billion in funding towards the development of new approaches for treating infections. Specific provisions include:

1. Creating a committee that will identify critical need infections and characteristics for high-value antibiotics.
2. Implementation of a ‘critical need’ designation for products in development that meet certain criteria.
3. These products would be negotiated for contracts worth up to $3 billion that would last from 5-10 years. These would be paid in equal amounts each year, and government insurers would cover them and have full access.

This approach aims to address the issue from the market perspective, as most drug companies aren’t developing many new antibiotics (not considered very profitable). Overall, the law could have major implications for cystic fibrosis patients and other rare disease patients that have compromised immune systems. The PASTEUR Act is still in its early stages.

Jamie provided an update on Prescription Drug Use Fee Act Reauthorization (PDUFA). It was first passed in 1992 and included provisions such as:

1. Funding FDA activities through a fee paid by pharma companies.
2. New regulations, such as the Accelerated Approval structure.
3. Set target timelines for drug review by the FDA.

The law has been renewed five times, with the latest renewal set to expire in September 2022. With this renewal, several new goals relevant to the rare disease community have been proposed such as:

1. New pilot programs such as Split Real Time Application Review (STAR) and Rare Disease Endpoint Advancement (RARE).
2. Furthering Patient Focused Drug Development
3. Refining and advancing policies such as digital health technologies, Real-World Evidence, and trial design innovation. 

If you would like to learn more, you can check out the full webinar program here.