The U.S. health care system has both advantages as well as shortcomings when compared to systems in other developed countries. One area where U.S. is a leader without much competition is in finding new therapies for deadly or disabling diseases. This is primarily due to the significant monetary and human capital investment biopharmaceutical companies make in drug discovery and development.
Currently, the annual investment of biopharmaceutical companies is greater than $80 billion, and the biopharmaceutical industry has the highest percentage of its revenue invested in research and development compared to any other industry. This commitment has continued despite the fact that research and development is both expensive and time-consuming. It ends more often in failure than success as fewer than 12 percent of the candidate medicines that enter human testing make it to approval. To maintain and encourage U.S. leadership, Congress passed the Prescription Drug User Fee Act in 1992. At Pfizer, we like to say it’s one of the most important laws most people have never heard of. While a majority of the FDA’s overall budget comes from the U.S. government, PDUFA requires biopharmaceutical companies, such as Pfizer, to pay user fees that partially support the Food and Drug Administration’s review process for drug evaluation, inspection, and surveillance.
The biopharmaceutical sector is the most heavily regulated industry in the U.S., and the FDA plays a critical role in maintaining a strong regulatory framework that ensures medicines are safe and effective while encouraging innovation for new drug discovery and development. It is important for the FDA to have the resources to accomplish its many responsibilities and to keep up with the ever-increasing demand by patients and their families in desperate need of cures. This is why Congress is on track to reauthorize PDUFA, which is set to expire on Sept. 30 of this year.
Every five years, biopharmaceutical companies and the FDA sit down to negotiate the terms of the PDUFA agreement. In the latest round of negotiations, three policies emerged that will help make significant scientific and process improvements for developing and evaluating drugs in the U.S. First, FDA needs to further integrate the patient’s perspective into the pharmaceutical research and development process. Since 2012, the FDA has hosted twenty disease-focused public meetings in an effort to better understand patient perspectives on issues such as the burden of disease symptoms and the impact of the availability — or lack thereof — of treatment options. Patients’ views were also sought regarding benefit and risk tradeoffs related to potential new therapies. However, the practice of incorporating patient perspectives has not yet been fully amalgamated into the FDA’s formal review process. In the new version of PDUFA, there are provisions that will advance the science of patient input to further integrate that input into the regulatory decision-making process.
First, FDA needs to further integrate the patient’s perspective into the pharmaceutical research and development process. Since 2012, the FDA has hosted twenty disease-focused public meetings in an effort to better understand patient perspectives on issues such as the burden of disease symptoms and the impact of the availability — or lack thereof — of treatment options. Patients’ views were also sought regarding benefit and risk tradeoffs related to potential new therapies. However, the practice of incorporating patient perspectives has not yet been fully amalgamated into the FDA’s formal review process. In the new version of PDUFA, there are provisions that will advance the science of patient input to further integrate that input into the regulatory decision-making process.
Second, the FDA needs to expand the use of innovative clinical trial designs and further the integration of real world evidence and biomarkers into the approval process. The FDA has traditionally relied heavily on data gathered through randomized clinical trials (RCT) when approving new medicines or indications for new uses of older drugs. Although data gathered through RCTs benefits from internal validity (specific cause and effect based on trial criteria), it lacks external validity or, better said, the ability to apply the outcomes to other situations or types of patients in real-world settings. By enhancing FDA’s capacity to leverage novel adaptive clinical trial designs, encouraging new uses for real-world evidence to complement clinical research, and better utilizing biomarkers in drug development, the updated PDUFA could improve health outcomes for thousands of patients.
Third, the FDA needs to hire and maintain a work force that can keep up with the new advances in science and technology. Hiring practices at the FDA have become outdated, as it now takes several months to hire a new employee. This has contributed to a staffing shortfall of several hundred positions across the agency, impacting the approval process for new medicines. The latest version of PDUFA offers to improve the FDA’s staffing capabilities, to fill more than 200 current vacancies and add hundreds more experts to lead new programs to better integrate patient perspectives into the drug approvals process and modernize clinical trials. As science advances, the FDA needs to have the resources and expertise to evaluate new medicines quickly, while preserving its high standards for safety and efficacy.
Before PDUFA was passed in 1992, it took the FDA an average of two-and-a-half years to approve new medicines. Since the law was enacted, the average review time for new medicines has declined to less than 11 months which has helped bring over 1,500 safe, new and effective medications to patients. In addition, it has helped the U.S. maintain its leadership in bringing new cures to the market, as approximately 84 percent of all medicines in the world are first approved in the United States. Patients and families need new medicines that are safe and effective without any unnecessary delays. Making the FDA more efficient and effective is vital if the U.S. wants to continue to be the leader in delivering innovative new products to patients and families in need. Congress can do its part by approving PDUFA VI — one of the most important laws people have never heard of.
Robert Popovian is the vice president of Pfizer U.S. Government Relations. He has two decades of experience in the biopharmaceutical health care industry and has published and presented extensively on the impact of pharmaceuticals and health care policies on health care costs and clinical outcomes.
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