Greater Integration of Patient Input in Regulatory Decision-Making

A Year into Implementation, Stakeholders See Great Promise in PDUFA V

10.10.13 | By John Castellani

In the last 20 years, the Prescription Drug User Fee Act (PDUFA) has resulted in timelier patient access to more than 1,500 new drugs and biologics, decreasing review times for these treatments by more than 60 percent.

PDUFA was reauthorized in July 2012 and went into effect on October 1, 2012.  It refocuses the program on its original intent— timely patient access to life-saving medicines.  In the past year, the Food and Drug Administration (FDA) has made progress in implementing some of the key components of PDUFA V, but financial constraints have delayed efforts to enhance the Agency’s regulatory science capacity. 

This week’s Conversation Forum asked thought leaders to reflect on FDA’s progress in the past year:

On the one-year anniversary of implementation of the Prescription Drug User Fee Act (PDUFA V), what element has shown the most promise?

Collaborative Engagement between FDA and Industry

Richard Pops, CEO of Alkermes, cites a new, collaborative tone between industry and FDA.  The collaborative engagement between FDA and industry, including frequent meetings, will help advance the shared goal of supporting innovation and getting valuable new medicines to patients as quickly as possible.  

Andrew Emmett of the Biotechnology Industry Organization (BIO) echoes this sentiment by focusing on robust, interactive scientific communication during drug development and enhanced communication and transparency during the review process to increase the likelihood of a first-cycle approval.  Fewer review cycles will reduce the time it takes to make safe and effective therapies available to patients.

Communication can be more effective when all parties speak the same language or, in this case, use data in a standard format to streamline the research process. Rebecca Kush of the Clinical Data Interchange Standards Consortium (CDISC) says the development of consistent research standards and terminology across specific therapeutic areas will help the Agency perform higher quality reviews on higher quality data more efficiently.

Patient-Focused Drug Development and Benefit-Risk Assessment 

As noted by Ryan Hohman of Friends of Cancer Research, PDUFA V is innovative and patient-focused as a result of true collaboration between FDA, patients, and the biopharmaceutical industry.  This engagement has continued in the Patient-Focused Drug Development (PFDD) initiative which seeks to incorporate patient perspective on the severity of disease and current treatment options in the regulatory review process. 

Marc Boutin of the National Health Council highlights the commitment to incorporate patients’ views into the regulatory process through a comprehensive benefit-risk framework and the PFDD initiative, both of which are based on the recognition that people with chronic conditions are their own experts. Sascha Haverfield, VP of Scientific and Regulatory Affairs at PhRMA, agrees that systematic consideration of the views of patients – their perception of the acceptable balance of known and possible benefits and risks – has the potential to add crucial perspective when drug developers and regulators are faced with difficult benefit-risk decisions.

Challenges of Sequestration 

There is universal agreement that we need a sufficiently-funded FDA in order to facilitate timely patient access to safe and effective new medicines.  Steven Grossman of the Alliance for a Stronger FDA notes the financial challenges facing FDA despite broad bipartisan support for the Agency. Sequestration and the federal government shutdown threaten FDA’s ability to advance its critical public health mission and full implementation of PDUFA V, which he called both transformational and evolutionary. 

PhRMA is committed to continue the multi-stakeholder discussion that helped to shape PDUFA V, and we thank our contributors to this week’s Conversations question.  I encourage you to share your viewpoint in the comments section.



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