21st Century Cures Act and revitalizing the biopharma industry
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The nearly unanimous enactment of the 21st Century Cures Act by both the House and Senate potentially heralds a new era of global Healthcare leadership by the Unites States. America’s pharmaceutical and biotech sectors have been stagnant throughout the past presidential administration This is partly because of structural impediments to streamline drug approval and in part because the drugs, devices, and biologics required for the next century simply cannot be approved with the procedures that served us in the last century.

Nearly all Americans and nearly all on both sides of the aisle recognize that the Life Sciences Industry is one of the major economic engines of America and one of the key sources of job creation for Americans at nearly all educational levels.

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While there are industries we once led that may never return to U.S. shores, the U.S. loss of life science sector employment share is a self-inflicted wound that we can and must heal. The economic engine that drives that healing is a revitalized U.S. Food and Drug Administration and its partnership with the National Institutes of Health and other branches of the Department of Health and Human Services.

Over the past eight years of the Obama administration, we have lived through an environment where one promising drug after another has failed in early stage research and development (R&D), not because our industry has gotten stupider but because the science has gotten harder, the clinical targets more elusive, and the path to approval ever more tortuous.

Because of this stagnation, the shareholders of one major pharma company after another have backed off on R&D investment. In the most extreme market distortions, rapacious acquirers often domiciled offshore have affected hostile takeovers of US pharmaceutical companies, fired entire R&D enterprises, and harvested the profits from in-line drugs with remaining patent life. The practice is little different from the rampage of the dark ages Vandals through Europe, burning the villages, eating the crops, and consuming the seed grain in the process.

Take Alzheimer’s as an example. The decade between the first appearance of structural changes in the brain and subsequent measurable changes in cognition is a decade during which no pharmaceutical company can afford to be engaged in clinical trials. Unless FDA is finally able to approve drugs based on intermediate endpoints, such as subtle neurocognitive changes in people with possible early disease and subtle changes on imaging, biopharma companies are locked into five to 10 year long clinical trials with return-on-investment cycles that shareholders just can’t support.

The flip side, of course, is that companies that do strike a partnership with FDA must be willing to keep their promises to continue monitoring those on the drug after approval is granted and to share detailed data about long-term effects. Thus, both FDA and Industry must learn some new approaches, if the promise of the Act is to be fulfilled.

The good news is that success in mastering new approaches has been demonstrated in cancer. Concerted leadership at FDA combined with new innovation in industry have led to an environment where approvals for critical unmet needs have been streamlined. With reduced time to market and increased chance of success, the investment community has responded with enthusiasm, making increasing amounts of capital available for first-in-class, lifesaving cures. Now it’s time to apply those lessons learned to areas of drug development that still rely on the methods of the last century.ne

With regard to the structural needs, the US Food and Drug Administration needs more than 1,000 more regulatory scientists, Physicians, Nurses, PhD’s, and other senior scientific personnel than it has today. Of those, more than 600 are needed in the FDA Center for Drugs.

These positions are vacant today because FDA needs a salary structure and personnel policy that supports the recruitment, hiring, and retention of those scientists, if we are to have any hope of revitalizing our nation’s pharma R&D pipeline.

The 21st Century Cures Act provides the structural and financial support that FDA requires to fill its ranks and it’s critical that FDA not be caught up on any potential federal hiring freeze . Without shoring up FDA’s structure and filling its ranks, it doesn’t matter what policy direction we enact — drug approvals, both innovative and generic will grind to a halt.

Taxpayers and policymakers alike should realize that the majority of these unfilled positions are supported not by tax dollars but, rather, by user fees that are willingly paid by industry to support the process of drug approval. Thus, the American taxpayer saves nothing by keeping these positions vacant and the American economy, particularly the biotech sector suffers.

At the same time, FDA needs policy leadership that convinces the rank and file scientists of the need to support drug approval based on intermediate endpoints  — biomarkers — that may show structural improvement before true clinical improvement is seen.

That’s the intent of the Accelerated Approval provision of the Act, which is intended to bolster FDA’s ability to review and approve medications for the most threatening diseases, such as Cancer, CNS diseases, and others that are most likely to kill Americans, but where old fashioned clinical endpoints (such as memory loss in Alzheimer’s) are simply too difficult to measure and take too long to ascertain.

Attention on biomarkers and intermediate endpoints alone will not solve the problem, however. FDA must increasingly adopt risk-based regulation where clinical studies and drug approvals are not unduly slowed down by theoretical toxicity concerns that are unrelated to observed clinical risks, but which have the potential to add years of delay and hundreds of millions of dollars of cost to every product in the current drug pipeline.

If a clinical trial will involve the exposure of 200 patients to an existing, well-known pharmaceutical substance, it makes no sense to require the sponsor of that clinical trial to perform an extra million dollars in animal experiments in order to look for some new one in ten thousand theoretical toxicity prior to proving the basic efficacy of the drug substance. Such risks are better monitored through ongoing post market surveillance using electronic systems capable of population-wide safety monitoring.

With the revitalization of top level FDA leadership, it’s equally important to spread the message throughout the organization that FDA’s role is to approve safe and effective drugs, devices, and biologics, and can only accomplish this by being an engaged partner in the drug approval process. Protection of the public is paramount, but without 21st Century Cures, there will be less of a public to protect.

Congress has done a remarkable job of passing groundbreaking, bipartisan legislation. Now the incoming administration must support one of the Nation’s most critical regulatory agencies in its implementation.

Jonathan C. Javitt, MD, MPH is a Senior Fellow in the National Security Health Policy Center of the Potomac Institute for Policy Studies, an Adjunct Professor of the Johns Hopkins School of Medicine, and an Alumnus of Merit of the Harvard School of Public Health. He has served in a number of Presidentially-appointed capacities in the Executive Office of the President of the Clinton and Bush Administrations. Today he is Founder and CEO of NeuroRx, Inc., a Biopharma company that is developing the first oral treatment for suicidal depression.


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