On this episode of the podcast, Terry and Bob discuss accelerated drug review for rare diseases. This topic coincides with Rare Disease Day on February 28. With the Prescription Drug User Fee Act (PDUFA) up for renewal in Congress, much is at stake for the rare disease community. This legislation expedites the drug reviewal process for new treatments, including those for patients with rare and unmet medical needs. Without it, drug reviews would slow down dramatically. 

Terry and Bob highlight how some legislators want to use PDUFA as a legislative “ornament,” something pretty to attach their pet legislation to in hopes of passing it. For instance, they point to Sen. Mike Braun’s Promising Pathways Act, which would require the FDA to prioritize an accelerated pathway for drugs. Terry and Bob explain that this legislation is good in principle but may be redundant in terms of existing accelerated approval law. 

To give listeners an inside look, Terry and Bob speak with past FDA Associate Commissioner Peter Pitts, who explains the importance of PDUFA and what new FDA Commission Dr. Robert Califf will bring to the patient community. Peter highlights how Dr. Califf can improve the accelerated approval pathway to make it more effective as well as fix clinical trial design to make it more inclusive and equitable. They discuss alternatives to drug price legislation in the stalled Build Back Better legislation, including a standalone bill proposed by Sen. Raphael Warnock that caps the price of insulin.

To conclude rare disease month, Kate Pecora speaks with patient advocate Yolanda Bermudez. She tells her story of how her rare disease cancer journey changed her life. She highlights how thankful she is for her care team. She explains her body mutation that doesn’t stop cancer. She discusses how she will manage this condition day to day. And she calls on patients to have a proactive mentality about their own care. 

Numerous rare disease advocates conclude the show by telling their stories of their healthcare system challenges and advocacy efforts. 

Listen to Episode 8: Speedy Rare Disease Drug Reviews