A New Progressive Era in Personalized Medicine

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  • 02/13/2012

From today's edition of the Washington Examiner:

America needs more breakthrough “progressive” medicines

A recent global cancer conference was abuzz with discussion of a potential breakthrough therapy in the treatment of Alveolar Soft Part Sarcoma, a rare form of cancer afflicting only about 100 patients per year in the U.S.

If ASPS is not eliminated through surgery, it is always fatal. A new therapy for ASPS, called cediranib, was found in early testing to melt away the tumors in up to 70 percent of treated patients.

Yet there is no development pathway for FDA approval for such small populations of patients, so the world's next Gleevec lies on a shelf, with fingers crossed that years from now, after larger, longer clinical trials in higher-prevalence cancers are performed with cediranib, ASPS patients might get access through indirect means.

Simply put, at the FDA, there is no official process for the approval of a product developed to attack a condition afflicting just 100 patients. A Grand Canyon exists between reality and hope in our new age of personalized medicine.

Enter Sen. Kay Hagan, D- NC. As a member of the Senate Health, Education, Labor and Pensions Committee, she has proposed legislation known as the TREAT Act, which calls for FDA reform that would fast-track drugs that show promising early data in treating deadly diseases.

The bill is an acknowledgement of the failings of the 1990's accelerated approval regulations, which rely on "surrogate endpoints" for a clinical trial to represent and reflect the endpoint that is usually used for a drug's approval.

The result is an anemic armamentarium for our nation's number-one killer, and completely empty medicine chests for many rare cancer types.

The TREAT Act reforms the accelerated approval pathway to allow state-of-the-art clinical endpoints that are "reasonably likely to predict clinical benefit" as the basis for approval for life-threatening diseases.

For cancer, this change could allow a return to the notion of simple, universal measures such as significant tumor death, or delay in disease progression, as the basis for rapid release of new products to desperately ill patients. Additional data following confirmatory studies would then allow progression to full approval.

Safeguards are included in the bill to remove the product from the market should it not live up to its initial promise. Also, as a humanitarian gesture for micro-populations with deadly disease, there is the creation of an "Exceptional Approval" pathway.

This would authorize approvals when the usual data "cannot ethically, feasibly or practicably be generated." This pathway would be a game-changer for U.S. rare disease populations of extraordinarily small numbers.

Thanks to Hagan, there can be a new way forward for development of therapies not just for rare cancers, but for thousands of life-threatening rare diseases where there are no realistic opportunities for drug development. Such reforms go beyond helping only the forgotten orphans.

By fostering innovation in new arenas of drug development, jobs are created and businesses prosper, and innovation accelerates the boundaries of medical knowledge and patient possibilities.

Now is the time to embrace a 21st century FDA -- and progressive approvals is a good place to start.

Peter Pitts, a former FDA Associate Commissioner, is president of the Center for Medicine in the Public Interest. Dr. Mark Thornton, a former FDA Medical Officer, is president of the National Organization Against Rare Cancers and is employed in the biotechnology industry.


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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