Building a 21st Century FDA: Getting new treatments out of the pipeline and into the hands of patients requires more than just good research

By Gillian Parrish, Manager, Alliance Development and Communications

Every day, hundreds of thousands of lives are indefinitely on hold – patients waiting for effective treatment options to become available, families waiting for their loved ones to start having a better quality of life, the list goes on. At a standstill – and that’s if they’re lucky – because research that promises to curb their disease has yet to translate into new drugs or devices they can utilize.

Often, when a new molecular entity or biomarker is discovered that has promising implications for disease treatment, it gets waylaid for years in a needless web of outdated regulatory processes that are under-resourced and ill-equipped for modern scientific review. According to a report by Nature Reviews Drug Discovery, the number of new molecular entities approved by the FDA has dropped steeply in the last 15 years – from 53 in 1996 to 19 in 2009, over a period when the budget for National Institutes of Health (NIH) nearly doubled. And most patients living with serious illness can’t wait the 10-15 years it takes to move a new discovery through the current development, testing, and regulatory review process.

While increasing funding for NIH research is a critical step in finding cures, it’s only part of the equation. The remaining part depends on improving the regulatory process, building capacity—both financial and scientific—at the Food and Drug Administration (FDA) so it can carry the baton of innovation from the research community across the regulatory “finish line” to patients. One vehicle for speeding review and access is the Prescription Drug User Fee Act (PDUFA), which is due for its fifth Congressional reauthorization in 2012.

The advocacy community’s role in reauthorizing an effective PDUFA was the focus of a panel hosted by BIO and PhRMA last week. Panelists Marc Boutin (National Health Council), Marcie Bough (American Pharmacists Association) and Jeff Allen (Friends of Cancer Research) called upon advocates to step up and be disruptive, working collaboratively with each other and with industry to speed product reviews and ensure the right balance between safety and access. Margaret Anderson (FasterCures) who moderated the discussion summarized the key themes at the end of the briefing:

• Making sure the patient perspective is heard throughout the reauthorization process
• Applauding FDA’s efforts to be more transparent, and taking them up on their willingness to have a two-way dialogue
• Working together across sectors and diseases to find and leverage commonalities like:
  - Improving REMS – Creating a standard Risk Evaluation and Mitigation Strategies (REMS) template, involving patients in the evaluation process, and establishing clearer metrics
  - Incorporating Regulatory Science – Though user fees aren’t typically applied to scientific programs, reviewing drugs is a complex process that requires a complex way of thinking, and process science should be incorporated into PDUFA to increase efficiencies
  - Advancing Public Education – Helping patients and consumers understand what FDA can do for them, and mobilizing them to act
  - Balancing Fees and Appropriations – The appropriated base of human drug review has not kept pace with FDA’s workload (currently ~65% is paid for by user fees); a consistent, multi-year funding approach is necessary to operate a modern-scientifically based regulatory program