Former Food and Drug Administration (FDA) Commissioner Andrew von Eschenbach, M.D., recently wrote an op-ed in the Wall Street Journal calling on Congress to give FDA necessary resources to bring the agency into the 21st century. In a world where science is advancing at an exponential pace, Eschenbach asserted that, “the FDA must be capable of ensuring that its reviewers know just as much about advances in emerging sciences as the creators of the products they regulate.”
Current FDA Commissioner Margaret Hamburg conceded to Congress in 2010 that “the FDA is relying on 20th century regulatory science to evaluate 21st century medical products.” As Eschenbach explained, “that's not the only problem.”
Unanticipated side effects of high-profile drugs like Vioxx have pushed the agency to require more data and larger clinical trials from companies to search for rare adverse events. The Tufts Center for the Study of Drug Development has reported that clinical trials from 2003-2006 were nearly 70% longer than those from 1999-2002. Longer (and more complicated) trials have led to skyrocketing drug-development costs. High costs discourage investment in much-needed new therapies for conditions like obesity, diabetes and heart disease.
Another problem Eschenbach sees is that FDA is also broken into Centers dealing with drugs, biologics and devices. “Yet increasingly, diagnostic devices will be paired with therapeutics.” And crucially, efforts to prevent diseases like Alzheimer's will require an entirely new approach to designing clinical trials—one that relies on "biomarkers" to rapidly measure a drug's effectiveness and safety in small, targeted groups of patients rather than in large, randomized clinical trials with thousands of patients that can take years to complete and analyze.
What will it take to realize the potential of the new medicine? Eschenbach noted that, “The United States has the world's most innovative drug and device companies and research universities, plus the unparalleled National Institutes of Health.” What's missing, however, is a “modernized FDA that can rapidly and efficiently bring new discoveries to patients.”
“The FDA is the regulatory gatekeeper for every drug and medical device sold in the U.S. But there is a growing recognition—at the agency, in the industry and among patients' groups—that it is at serious risk of falling behind its core responsibility of evaluating new medical products in a timely and predictable manner.”
Eschenbach correctly asserted that, “Without an FDA that is as innovative and sophisticated as the companies it regulates, patient health and U.S.-based innovation will suffer.”
Adding more pressure on the agency and Congress is the fact that other countries such as Israel, Singapore and China “are already preparing to leapfrog the U.S. for leadership of the global life-sciences industry. And investors and companies are shifting jobs and research abroad as part of an offshore strategy to cut costs and reach the market faster.”
According to the 2012 California Biomedical Industry Report (published by the California Health Institute, BayBio, and PricewaterhouseCoopers), about 80% of life-sciences CEOs surveyed didn't believe that the FDA regulatory approval process "is the best in the world," and 81% believed that "within five years, another country could conceivably recreate the ecosystem that has made the U.S. the leading biomedical region in the world"—threatening a mass exodus of jobs and R&D.
Although Congress has repeatedly expanded FDA's responsibilities over the years, Eschenbach asserted that Congress “has not ensured that the agency is keeping pace with the enormous scientific advances made since the human genome was decoded in 2000. Congress and the Obama administration need to make that a priority,” Esenbach maintained.
Bringing the FDA into the 21st century is critical because “the U.S. biomedical industry is one of the crown jewels of the American economy.” It employs 1.2 million people directly and over five million throughout its supply chain, with a total output of $519 billion in 2009, according to a 2011 Milken Institute report, “The Global Biomedical Industry: Preserving U.S. Leadership.” Many of the firms are among the world's most innovative: From 2001-2010, the report shows, U.S.-based companies produced nearly 60% of the world's new medicines, up from 42% the previous decade.
However, as Eschenbach rightly asserted, “U.S. firms won't continue to lead unless the FDA retains its role as the world's “gold standard” for evaluating new medical products.”
Consequently, the former FDA Commissioner noted that the opportunity to make improvements to FDA is here today, with Congress currently reconsidering the reauthorization of the Prescription Drug User Fee Act—the fifth reauthorization. The agency is also considering User Fees for generic drugs, biosimilars, and the reauthorization of medical device user fees. Eschenbach recognized that these reauthorizations “present a rare opportunity to examine the FDA's overall needs and performance.”
As a result, he asserted that, “Congress should not only set the fees and add resources for the agency but also modernize it from the bottom up.” This includes
- A comprehensive external review of the agency's regulatory processes; and
- Creating FDA pilot programs to bring promising therapies to patients more quickly by allowing them to be approved based on safety, with efficacy to be proven in later trials.
“Breakthrough technologies deserve a breakthrough in the way the FDA evaluates them.” Eschenbach used the example of regenerative medicine. If a company can grow cells that repair the retina in a lab, patients who've been blinded by macular degeneration shouldn't have to wait years while the FDA asks the company to complete laborious clinical trials proving efficacy. Instead, after proof of concept and safety testing, the product could be approved for marketing with every eligible patient entered in a registry so the company and the FDA can establish efficacy through post-market studies.
Ultimately, Eschenbach recognized that America stands on the cusp of a revolution in health care. “Advances in molecular medicine will allow us to develop powerful new treatments that can cure or even prevent diseases like Alzheimer's and cancer. Tomorrow's high-tech cures can also slash health-care costs and eliminate ineffective treatments.”
Accordingly, he maintained that, “By empowering the FDA to create new paradigms for evaluating the most promising innovations, Congress can ensure that the FDA serves as a bridge—not a barrier—to cutting-edge technologies.”