Feb. 25, 2022

Speedy Rare Disease Drug Reviews

Speedy Rare Disease Drug Reviews

As Rare Disease Month comes to a close, we take a look at a major piece of legislation that impacts the FDA, and consequently, patients with rare diseases. 

With the Prescription Drug User Fee Act (PDUFA) up for renewal in Congress, much is at stake for the rare disease community. This act 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. And speaking of the FDA, the agency finally has a new Commissioner, Dr. Robert Califf. How will he shape the agency? 

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 Dr. Califf will bring to the patient community. 

And to conclude rare disease month, Kate Pecora speaks with patient advocate Yolanda Bermudez. Hear her story as she discusses how her rare disease changed her cancer journey. 


Terry Wilcox, Executive Director, Patients Rising

Dr. Robert Goldberg, “Dr. Bob,” Co-Founder and Vice President of the Center for Medicine in the Public Interest

Kate Pecora, Field Correspondent


Peter Pitts, Co-founder and President of the Center for Medicine in the Public Interest 

Yolanda Bermudez, Patient Advocate 

Annika Wojtowicz, Patient Correspondent 

Jearlean Taylor, Patient Correspondent 


Prescription Drug User Fee Amendments | FDA

PDUFA Renewal and the FDA: What Do Patients Need to Know?

Twitter | PatientsRiseNow Health Subcommittee Hearing on PDUFA

Robert M. Califf M.D | FDA Commissioner

Peter Pitts | CMPI

The Affordable Insulin Now Act 

Creating a promising pathway for faster access to new drugs — and a Califf confirmation

Raphael G. Warnock

Need help?

The successful patient is one who can get what they need when they need it. We all know insurance slows us down, so why not take matters into your own hands? Our Navigator is an online tool that allows you to search a massive network of health-related resources using your zip code so you get local results. Get proactive and become a more successful patient right now at PatientsRisingConcierge.org

Have a question or comment about the show, or want to suggest a show topic or share your story as a patient correspondent?

Drop us a line: podcast@patientsrising.org

The views and opinions expressed herein are those of the guest(s)/ author(s) and do not reflect the official policy or position of Patients Rising.