Tuesday, March 30, 2010

Science and Progress at the FDA

Realizing the Return on Investment from Biomedical Research
by Cecilia Arradaza, Communications Director, FasterCures

Speakers at a congressional briefing on March 24 said that in order to realize the full return on investment from biomedical research, the U.S. Food and Drug Administration (FDA) needs to be in a position to take the baton of innovation from the research community and turn it into medical products that are safe, effective, and accessible for patients who need it most. The briefing, Science and Progress at the FDA, was convened by Friends of Cancer Research and FasterCures/The Center for Accelerating Medical Solutions.

In her opening remarks, Rep. Rosa L. DeLauro (D-CT) said that the FDA must work in new ways to enhance science at the agency, recruit more top minds to formulate regulatory regimens to promote safety without stifling innovation, and adopt a multidisciplinary approach. She noted that for FDA to be the thriving center of scientific knowledge, it requires more than resources alone, its culture must change from within.

Regulatory Science Requires Resources and Collaboration

Jesse L. Goodman, MD, MPH, Chief Scientist and Deputy Commissioner for Science and Public Health (Acting) at FDA defined the premise of the FDA’s focus on regulatory science. He said that the science done by academia – often supported by NIH – gives us tremendous insight into basic mechanisms of disease while the work done by the biopharmaceutical industry focuses on getting products on the market. Regulatory science is applied science that aims to develop, assess and provide new, validated tools and approaches to better evaluate the utility of new medical products.

“At our very best, FDA can join with partners to develop tools that make the evaluation process sounder,” Goodman said. “The model of going it alone doesn’t work. The world of science and amount of information out there is just too huge. What we need to do is collaborate and engage with the scientific community.”

Echoing DeLauro’s point, he said that some of it is about resources, but some of it is about doing things in a more innovative way. He noted the need to reduce risk while maintaining patient benefit.

“Along with medical and scientific risks, we need to consider financial risk as well,” said Max Wallace, CEO of Accelerated Brain Cancer Cure. Wallace, who has also founded a number of biotechnology companies, said that capital is the rocket fuel that propels the process from discovery to product.

“Capital is rocket fuel without soul – it hates risk. If risk is doubled by time delays or failure to understand science or opaque non-collaborative environment, the capital will seek its level in other places,” he noted.

Regulatory Science to Benefit Patients

“So, how do these discussions about regulatory science translate into patient benefit?” asked briefing moderator Margaret Anderson, Executive Director of FasterCures.

John Marshall, M.D., Clinical Director of Oncology for Georgetown University Hospital and principal investigator of over 150 clinical trials provided real-world context. He said that the FDA sets the goal, the rules, the parameters that we are to aim for in clinical research: safety and efficacy.

Marshall said that by having the scientific capacity to determine the best models to measure safety and work and collaborate with researchers to design appropriate trials, the FDA can make the process more effective, and efficient. He noted that having the ability to tailor trials to a subset of patients that are known to respond to specific medical products paves the way to a better development system and realizes the promise of personalized medicine.

Ellen Sigal, Chair and Founder of Friends of Cancer Research said that patients have different risks and profiles. But consistent with all patients is the need for more information. Sigal said that “we can evaluate risk with the right data. We want to be able to make choices based on the data.”

Marshall later noted the importance of “building a system that allows buy-in from the patients.”

Regulatory science is the backbone that supports all other FDA activities. Improvements in regulatory science will support better assessment of drug and device safety, and create efficiencies in the development process.

Realizing Change

But, how do we make change happen? Goodman likened making the appropriate changes at FDA to building an airplane while it is flying. He noted that the FDA could be in a position to catalyze change.

Collaborative science is critical to make this happen. Efforts like the Critical Path Initiative and Reagan-Udall Foundation can help by bringing together large numbers of stakeholders to solve problems.

But the FDA needs resources to be able to engage early in the process and enable staff to make regulatory be a part of what they do. He said that the FDA is “very stressed and limited to even engage at the baseline level.”

According to Wallace, “those who are developing ideas are on the bleeding-edge of new technologies and they need dance partners at the FDA.” The agency should ramp up to attract the best scientific talent – that’s where change starts.

Goodman cautioned that it will take five to 10 years to build this new mindset at the FDA. He said that “in the long run, it’s a transformation of culture from just trying to keep up to one that’s a true outstanding scientific partner.”

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