The Food and Drug Administration (FDA) is one of the most important federal agencies in America. The agency regulates approximately one-quarter of all consumer products in this country, and the products that FDA regulates make up a substantial portion of our economy. More importantly, the companies and manufacturers that FDA regulates produce a significant amount of high quality jobs for Americans.
However, FDA is also perhaps one of the most controversial agencies. Recent polls have shown that various scandals have taken a toll: About half of the U.S. public thinks the FDA is doing a bad job, and the perceptions are that drug companies put profits ahead of patients. To address these concerns, FDA recently published a 40-page strategic outline for how it can do a better job stimulating (not smothering) life sciences innovation in the U.S.
Titled “Driving Biomedical Innovation: Initiatives for Improving Products for Patients,” the blueprint addresses concerns about the sustainability of the medical product development pipeline, which is slowing down despite record investments in research and development.
“The Obama Administration is committed to encouraging the entrepreneurs and businesses that are modernizing and strengthening our health care system,” said HHS Secretary Kathleen Sebelius. “The innovation blueprint is another part of our effort to help businesses grow and keep Americans healthy.”
“America is at an important crossroads, where the science before us presents unprecedented opportunities to create new and better medical products and to promote better health for the public,” said Hamburg. “Our innovation blueprint highlights some of the initiatives FDA will be implementing to ensure that these opportunities are translated into safe and effective treatments that can help keep both American patients and American industry healthy and strong.”
Dr. Hamburg recognized the need to create an FDA-wide framework to address the changing scientific landscape. The blueprint launches the Innovation Initiative, identifying additional steps the agency can take immediately to address the most pressing concerns facing patients and industry. The report’s proposals stem from a review of FDA’s current policies and practices, as well as months of meetings with major stakeholders nationwide, including key industry leaders, small biotech, pharmaceutical and medical device company owners, members of the academic community, and patient groups. The blueprint focuses on implementing the following major actions:
Rebuilding FDA’s small business outreach services. FDA noted that it needs to strengthen its outreach to small businesses and and ensure that individuals with small business expertise can serve as a resource to others on FDA issues. To that end, FDA will establish a FDA Small Business Liaison program and a Young Entrepreneurs program, as well as establish a new partnership with the U.S. Small Business Administration. FDA said it would continue to work to address the needs of small medical product companies through efforts to make the review process easier and more transparent, as well as by providing information that can help businesses prepare for the final phases leading to product approval and marketing.
Building the infrastructure to drive and support personalized medicine. FDA said it will continue its leadership role in personalized medicine by building a system to support the development of these more personalized medicines, including investments in regulatory science and by clarifying agency policies.
Creating a rapid drug development pathway for targeted therapies. FDA will host a series of scientific meetings with a diverse group of stakeholders intended to achieve a common understanding of steps needed when an investigational drug being studied for a serious disease shows exceptional promise during the early stages of development.
Harnessing the potential of data mining and information sharing, while maintaining strong privacy protections. To harness the potential of information sharing and data mining, FDA is rebuilding its IT and data analytic capabilities and establishing science enclaves that will allow for the analysis of large, complex datasets while preserving patient privacy.
Increasing consistency and transparency in the medical device review process. FDA pointed to the recent improvements to medical device review process designed to encourage innovation within the medical device community, which included 25 actions that FDA’s medical device center will take this year to improve the predictability, consistency, and transparency of its premarket review programs. FDA emphasized that these actions are designed to attract investors to innovative companies, predict estimated regulatory costs, and bring safe and effective products to patients more quickly.
Training the next generation of innovators. Building on the success of FDA’s Fellowship program, FDA is designing a new Future Innovators Program that will bring practical regulatory science and policy training together to meet the scientific and technological demands of the 21st century. Under this competitive program, FDA will bring promising candidates into the agency for training and experience that will provide FDA with important outside expertise and perspective while equipping these innovators with highly marketable skills and experience.
Streamlining and reforming FDA regulations. FDA is taking proactive steps to reform its existing regulations. At the direction of the President and the Commissioner and based on public feedback, the agency is reviewing its current regulations to identify burdensome, unclear, obsolete, ineffective, or inefficient regulations.
Discussion
While FDA should be applauded for its efforts, it may take several years before industry begins to see any real differences, and with budgets remaining uncertain and politics always a factor, this plan still has a ways to go. Interestingly, in response to FDA’s report, some have called for other policy changes as well. One commentator noted that industry should support an increase in the FDA budget to bring the agency up to modern standards for things such as a modern database that can capture and analyze data on adverse drug reactions for marketed pharmaceuticals.
The author also suggested a voluntary ban on all direct-to-consumer TV advertising because it has “helped create the national expectation, through ads that exaggerate benefits and downplay risks that prescription drugs are like taking Honey Nut Cheerios.” Besides the point that this is a clear request to silence free commercial speech, protected under the First Amendment, and recently recognized in IMS v. Sorrell by the U.S. Supreme Court, the author clearly ignores the tremendous benefits that prescription drugs have given to Americans. Moreover, the author ignores the fact that 75% of prescriptions are generics. In addition, concerns about biased ads are misguided because the FDA, through its Division of Drug Marketing, Advertising and Communication (DDMAC), and the new “Bad Ad Program,” properly ensure that companies advertising are fair and balanced.
The author also suggested that FDA support legislation to ban advisory committee members with conflicts of interest. This suggestion is clearly contrary to FDA’s own recognition that such rules have created significant difficulty for the agency to fill advisory committee seats. In fact, both Commissioner Hamburg and CDER Director Janet Woodcock have come out recently, acknowledging the need to change conflict of interest rules. And now, Congress has legislation pending to correct these rules. Furthermore, the author ignores a study in JAMA from 2005, which concluded that a weak relationship between certain types of conflicts and voting behaviors on FDA advisory committees was detected, and that excluding advisory committee members and voting consultants with conflicts would not have altered the overall vote outcome at any meeting studied.
The author also suggested that the FDA fully disclose complete response letters, which the agency uses to “reject” a drug or device application. However, the author fails to recognize that contained within the complete response letters or confidential information and trade secret information. Companies already face significant regulatory hurdles and burdens when it comes to discovering a new product. This would create a further disincentive for companies because competitors could then potentially use the information FDA would disclose to the public about a failed product. Such a decision would give companies yet another reason to take product approval to other countries first, denying Americans the first access to lifesaving treatments.
Conclusion
Shortly after the FDA report was published, the National Venture Capital Association released results of a survey that said the FDA—plagued by long delays, unpredictable decisions, poor communication, and excessive caution in new product reviews—has become the No. 1 reason why 40 percent of U.S. venture capitalists have cut their investments in life sciences the past three years, and that the same percentage plan to continue cutting U.S. investment the next three years. Jobs have been lost, and even more will be shipped overseas unless something is done at the FDA, the investors said.
Dr. Hamburg noted in announcing the report that “there are some signs that the health of these industries may be at increasing risk, jeopardizing a critical piece of our economy and, more importantly, obstructing patients’ access to new therapies.” Nevertheless, she recognized that, “American pharmaceutical, biotech, and medical device companies have maintained a competitive advantage in the global marketplace and continue to represent three of the few U.S. manufacturing sectors that maintain a positive trade balance.”
Ultimately, to maintain this competitive advantage in the global marketplace and to keep life science investment and jobs in America, FDA will need to implement these proposals swiftly and efficiently, and continue to work with industry and stakeholders to constantly improve and adapt regulatory measures, while still maintaining the appropriate balance to ensure product safety and effectiveness.