Policy Brief: A Modern System for Approving the Cures of the Future

Published April 30, 2019

The world should be entering a golden age of medicine, but the Food and Drug Administration’s (FDA) antiquated process for certifying the safety and efficacy of new treatments could significantly delay that future, limiting innovation and unnecessarily harming millions of patients in the process.

All Americans desire access to drugs that are safe and effective, but they also want timely and affordable drugs. However, a report by the Tufts Center for the Study of Drug Development found it takes on average more than a decade—at a total cost of $2.9 billion (including the cost of failed drugs)—to bring a new drug from research labs to market, where patients can access these life-saving or life-improving treatments. Much of those resources are consumed by years of FDA-mandated tests allegedly meant to certify the efficacy of medicines that have already been determined to be safe. 

While FDA bureaucrats slow-walk the approval of new pharmaceuticals, millions of Americans suffer or even die waiting for promising treatments to be made available. Many of those patients left in anguish rightly ask, “Isn’t there a better way?” The answer is unequivocally, “Yes!” 

The Free to Choose Medicine (FTCM) model would offer the developers of new drugs that have passed safety tests and at least one efficacy trial the option of making them available to patients. Patients have a right to take responsibility, in consultation with their doctors, for their own health and to accept informed risk in order to preserve or enhance their own lives by accessing such new drugs and biologics. Currently, only the few thousand patients who are permitted to participate in drug trials have that opportunity. 

FTCM would also create a system for determining efficacy that, at minimum, would supplement FDA’s use of statistical analyses generated using data from randomized, controlled clinical trials. Under FTCM, a Tradeoff Evaluation Drug Database (TEDD) would be created. Information about patients, including their genetic data, relevant biomarkers, and clinical treatment results from the use of drugs on FTCM tracks, would be logged into TEDD, with patient privacy strictly guarded. This database would be accessible to the public and researchers alike and would give patients and physicians a fuller understanding of all their treatment options in a way that is currently impossible to achieve.

In the past decade, real-world data, which are used as evidence to determine the efficacy of medical treatments, have increasingly been used to gain insights of practical value. Relying on such observational data is now recognized, even by FDA, as the cutting edge in medical research. On the issue, FTCM was ahead of its time, highlighting the importance of such data as early as 2007.

This Policy Brief argues an FTCM approach, by collecting real-world data on the effects of new drugs, would provide more patients with access to potentially life-saving drugs, and it would do so much faster and more efficiently than the current FDA system. By providing observational data in real time to drug developers, FTCM would allow less-efficacious drugs to fail faster, weeding out poorly performing drugs in a shorter period and revealing more quickly which drugs are likely to work well. 

By expediating these processes, the overall cost and time associated with drug development would be reduced markedly. This would create more opportunities—especially for smaller, innovative researchers—to develop breakthrough drugs and methods by which to demonstrate their efficacy.