Frank Sasinowski, MS, MPH, JD Hymen Phelps & McNamara: Rare Disease Regulatory Transformation: 1983 AIDS Crisis to Present

WCG Talks Trials

Drug policy has transformed to bring transformative improvement to the way rare diseases are developed resulting in more FDA approved treatments.

This transformation came about because of collaboration across stakeholders: patient advocates, the FDA, Congress, drug developers, researchers, and legal specialists. Profound change came in the mid 1980’s when AIDS activists, Rare Disease Activists, and others found ways to use policy to accelerate development of FDA approved treatments.

Legal specialists Frank Sasinowski of Hymen Phelps and McNamara has always been a champion of the patient voice in legislative change, drug development policy, and FDA approvals.

In this WCG Patient Radio episode with WCG’s President of Patient Advocacy, Steve Smith, Mr. Sasinowski speaks of the transition from a time in the late 90s when patients were not even considered in drug policy. He speaks of formalization of the patient voice into legislation, the Rare Disease Congressional Caucus, Patient-Centric Drug Development program at the FDA, and numerous other transformative mechanisms. Change is evident as we now see many patient-focused roles within drug development companies, working with more sophisticated patient advocacy groups, and regulators.

During this 20 minute podcast, Mr. Sasinowski also discusses the balance between the need for speed of drug development, with safety, and the concept of “certainty.” How can we include, in the drug approval process, the concept of “risk vs. benefit” as seen by patients that are in need of a therapy very soon who are willing to take more risk.