We can improve and modernize the drug development process in the United States
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In recent days, there has been a lot of attention focused on the Food and Drug Administration’s (FDA’s) drug approval process. Last week, during his first address to a joint session of Congress, President Trump stated that “our slow and burdensome approval process at the Food and Drug Administration keeps too many advances…from reaching those in need.”

FDA advocates were quick to respond, citing considerable declines in FDA review times over the last several years, from 27 months in 1993 compared to 10 months in 2016. Why such a divergence? Because there is a fundamental and dramatic difference between the time and cost of drug approval and drug development.

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While it may take FDA months to review a new drug application, the bigger concern is the fact that it takes more than ten years and $2 billion to develop a new drug from a molecule to a medicine, so that it can be made available to patients in need.

 

And every stage in this process is governed by FDA regulations, including laboratory-based research, pre-clinical studies, and clinical trials testing in human subjects. This is followed by the review and approval process, which requires submission of voluminous documentation and data to determine efficacy and risks.

Advocates for the FDA rightly compliment the agency for admirable progress in improving review times, due in large part to the provision of resources through industry user fees. But concurrently the development time however has become longer and more expensive, due to the complexity of science and technology and greater risk of failure in the later stages of development. Larger clinical trials can take as long as seven to ten years and cost on average $1.5 billion.

There is broad and bipartisan consensus around the need to modernize this burdensome development process—not only to help the millions of Americans with diseases for which there is no cure, but also to lower the cost of drugs and improve our nation’s global competitiveness.

The FDA has been aware of the need for modernization to accelerate the pace by which innovative therapies can move from the laboratory to people — eliminating suffering and saving lives — while still assuring safety and effectiveness. It has several initiatives aimed at improvement, including the Critical Path Initiative and the use of new methods for clinical trials such as adaptive trial designs and Bayesian statistical methodologies.

However, such efforts have been inadequately resourced and the process of modernization is falling woefully behind the incredible pace of progress in biomedical research and the emerging demands of precision medicine.

Modernizing the science of evidence generation through the use of biomarkers, real-world evidence — or data reflecting the actual experience of patients, adaptive trial designs, and more robust post-market surveillance processes, will have a significant impact on the time and cost of development.

Increasing regulatory clarity, ensuring adequate scientific capacity, and modernizing regulatory frameworks for advanced therapies, such as regenerative medicine, will also result in vast improvements.

Thankfully, the 21st Century Cures Act — passed nearly unanimously in the House and Senate and signed in to law in December 2016 — contained several of these provisions. Such provisions were also included in the Bipartisan Policy Center’s 2015 report, Advancing Medical Innovation for Healthier Americans. Soon, Congress will have another opportunity to improve the drug development process and advance medical innovation, as it considers “must-pass” legislation re-authorizing user fees for drugs and devices this year.

Both the supporters of the FDA and the president are correct. The FDA is the world’s gold standard and made up of exceptionally talented people who — with the support of industry — have significantly reduced review times.

Yet they are struggling to modernize the drug development process and require the joint support of all stakeholders.

The president and Congress can immediately do their part by continuing to advance policies that promote innovation without compromising safety. They should nominate and confirm an FDA commissioner who both knows what needs to be done and has the experience and knowledge of the agency to know how to do it.

Industry is supportive of user fees and patient advocates, scientists, and academia are contributing their expertise and advice. Now we no longer need to debate, but rather get the job done. Millions of lives are depending on it.

Andrew von Eschenbach is former commissioner of the Food and Drug Administration, former director of the National Cancer Institute, and senior advisor to the Bipartisan Policy Center’s Advancing Medical Innovation Initiative. Janet Marchibroda serves as the director of health innovation at the Bipartisan Policy Center.


The views of contributors are their own and are not the views of The Hill.