The FDA “Must Have” Regulatory Science

By Margaret Anderson, Executive Director, FasterCures
"…One thing is clear: if we are to solve the most-pressing public health problems we face today, we need new approaches, new collaborations and new ways to take advantage of 21st century technologies," said FDA Commissioner Margaret Hamburg last week in a speech at the National Press Club that outlined the agency’s vision for advancing regulatory science.

These days it seems, each time anyone – government official, patient advocate, legislator – says anything about the FDA, it often starts with, “The FDA needs…” That’s because this agency, tasked with regulating nearly 25 percent of consumer spending in America, needs so much just to be able to complete its enormous tasks at hand: ensuring safe and effective products, and promoting public health. We all know and publicly acknowledge the gravity of the need. Let’s act and begin addressing it.

At FasterCures, we are committed to improving health outcomes, a better quality of life, and greater productivity for all by ensuring that effective treatment options are accessible and available when disease strikes. But we cannot accelerate medical progress if we do not have a robust, scientifically sound FDA. The real potential of scientific breakthroughs and cutting edge medical solutions to improve the lives of patients, even cure disease, will not be actualized without appropriately resourced review and regulatory processes.

In the report Commissioner Hamburg released last week, Advancing Regulatory Science for Public Health, the FDA outlined its priorities for its new effort, articulating what a focused agenda and targeted investment in regulatory science may bring. We all know too well the consequences of inaction. Here are just a couple of examples from the report where regulatory science can truly impact the research and development paradigm.

• Accelerating Delivery of New Medical Treatments to Patients. The report states that we can modernize product development and develop new tools, standards, assays, disease models and science-based pathways to improve the speed, efficiency, predictability, capacity and quality of the entire process, from development to evaluation to manufacturing.
  
  For example, we can have more adaptive clinical trials like the I-SPY 2 Trial launched in March 2010. This innovative trial design individually targets drugs in development to the biology of each woman’s tumor using specific genetic or biological markers, known as “biomarkers.” This allows scientists to efficiently test the most promising products in women with higher risk, rapidly growing breast cancers, and more quickly eliminating ineffective treatments and drugs.
• Enhancing Safety and Health Through Informatics. FDA houses the largest known repository of clinical data — unique, high-quality data on the safety, efficacy and performance of drugs, biologics and devices, both before and after approval. But the FDA lacks the hardware and software necessary to translate this data into valuable information that could help address key questions.
  
  For example, with the appropriate informatics framework in place, we would be able to look at 15 studies of HIV drugs at once to analyze which products are effective for which patient, and detect a new or rare safety risk by capturing safety information from millions of medical records in months instead of years.

This regulatory science agenda is matched with an implementation plan that requires broad-based support and adoption by all sectors engaged in the medical research enterprise. The four-part strategic framework includes:

• Leadership, coordination, strategic planning, and transparency to support science and innovation
• Support for mission-critical applied research, both at FDA and collaboratively
• Support for scientific excellence, professional development, and a learning organization
• Recruitment and retention of outstanding scientists

Too often, we hear of the FDA at times of crisis – product recalls, safety alerts, black box warnings. And too often, we take for granted the systems that need to be in place to ensure the safety we rightfully expect is guaranteed, and that the products we use work as promised. A recent Research!America poll found majority of Americans to be confident in the U.S. safety review system and believe that FDA’s most important job is protecting the safety of the American public.

FDA has outlined its future “must do” list. Now it’s time to provide FDA with the resources it needs to do its job and to do it well. The billions of dollars of public and private investment in medical research can only reach its full potential if we have an FDA that’s fully equipped to carry the baton of innovation from the research community over the regulatory “finish line” and into the hands of patients.