How Can We Fix the FDA? - Pharmaceutical Executive


How Can We Fix the FDA?

Pharmaceutical Executive

What are the Solutions?

How can we achieve a better balance between addressing medical needs and ensuring that new drugs are both safe and effective? A new mechanism is required, but what shape should it take? Accelerated approval offers a useful paradigm. First implemented in the 1980s to speed HIV drugs to market, this regulatory approach acknowledges that surrogate markers can reasonably predict drug efficacy, even without evidence from lengthy clinical trials. Based on success with HIV/AIDS, both Congress and cancer patient advocates demanded similar treatment for oncology drugs. Not surprisingly, the numbers of new treatments in development for cancer now far outstrip those for any other condition. This shows what the American innovation enterprise can accomplish when obstacles are lifted and the right incentives are in place.

Medical science has continued to advance on numerous fronts since the 1990s, to the point where genomics, molecular biology, and bioinformatics have provided an unprecedented understanding of the underlying biological mechanism and pathogenesis of serious diseases, in addition to HIV and cancer. It should now be possible to expand the accelerated approval pathway to include other diseases, applying strategies based on biomarkers or pharmacogenomics, predictive toxicology, clinical trial enrichment techniques, and novel clinical trial designs (such as adaptive clinical trials). As in the case of HIV and cancer, standards of safety would be preserved. In addition, drugs green-lighted in an accelerated approval pathway would be subject to more definitive clinical trials in the post-market setting, and approval would be withdrawn if these were not confirmatory.

It would be unreasonable to pin all the blame for regulatory dysfunction on FDA. Regulators are buffeted by political winds that blow hot or cold indiscriminately. Our society is at fault because we expect to live in a 'Neverland' where powerful medical interventions have no risks whatsoever. The media and our political leadership too often serve as echo chambers for our fears, and ubiquitous personal injury ads reinforce our society's insistence that no risk should go unpunished. Notwithstanding all this, most Americans would likely be shocked to learn that they have 20 or more times the risk of dying from a fall than from taking most approved medicines.

There are no secret weapons in the reform proposals I've seen that can help FDA combat misguided social expectations. Nor should regulators relax their guard on patient safety. However, the reform measures noted above might reassure investors in U.S. biotechnology and preserve this sector as the birthplace of medical breakthroughs. These measures emerged from task forces within the Biotechnology Industry Organization and were informed by lengthy discussion with a wide range of interested parties, including FDA officials, patient and pharmaceutical groups, and political leaders.

FDA has been responsive to calls for change in the past. The agency embraced accelerated approval for HIV and cancer, and was receptive to calls from the biotech industry on the need to create an ombudsman's office to serve as a liaison with drug sponsors. Robust biopharmaceutical innovation is critical to our personal health, as well as the economic health of our country. With so much riding on the outcome, it's time for all parties to leave their biases at the door and agree on innovations to optimize the regulatory process itself.

Ron Cohen is President and CEO of Acorda Therapeutics. He can be reached at


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