EditorialPolicy

Sequester Cuts May Delay Drug Approvals—And More

Science Translational Medicine  04 Sep 2013:
Vol. 5, Issue 201, pp. 201ed14
DOI: 10.1126/scitranslmed.3007383

Clifford A. Hudis

CREDIT: MEDIA SERVICES/ MSKCC

The U.S. Congress’s current mandatory budget cuts—sequestration—threaten unprecedented opportunities in all biomedical research and development. As a medical oncologist, these severe cuts raise the particular concern that translation of intricate knowledge about the molecular biology of cancer to new, targeted treatments will be impeded in part by an underfunded U.S. Food and Drug Administration (FDA). As of 16 August 2013, 7 of the 16 new drugs approved by FDA this year are for treating cancers (1). According to Richard Pazdur, director of the FDA Office of Hematology and Oncology Drug Products, “[T]he drugs are better,” and it is now a question of “how fast we’re going to approve them” (2). Clinical translation depends on an optimally functioning FDA, and sequestration may slow the approval process.

The pace of recent and ongoing advances is impressive by any historical comparison (particularly given the uncertain economy of the past few years). In 2012, 16 of the 39 new drugs approved were given Priority Review designation because of their potential to “provide a significant advance in medical care” (3). The priority designation requires that FDA complete its review of an investigational new drug (IND) application in 6 months rather than 10. In addition, FDA has classified 25 drugs with the new Breakthrough Therapy designation (signed into law on 9 July 2012) (4), because the drugs treat serious or life-threatening illnesses and demonstrate substantial improvement over existing therapies (5). This designation demands more interaction between the inventing company and FDA with a goal of shortening the review process. Both of these designations—along with FDA’s Fast Track status and Accelerated Approval (6)—reflect the agency’s public-health mission to speed access to safe and effective treatments, especially for life-threatening illnesses. This efficiency of review is reflected in the fact that 30 of the 39 new drugs (77%) that came onto the market in 2013 were approved first in the United States. To continue this effort, FDA requires dedicated staff and sufficient resources.

ALL WORK AND NO PAY

Even in tough fiscal times, FDA has broad and important responsibilities beyond the review and approval of new drugs. The agency oversees ~25% of all consumer spending in the United States, including 100% of drugs, vaccines, medical supplies, and personal care products and 80% of our nation’s food supply (7). Yet, Congress continues to ask the agency to do more with less. Since 2009, Congress has assigned FDA with many additional responsibilities through the Family Smoking Prevention and Tobacco Control Act (2009), Biologics Price Competition and Innovation Act (2010), Secure and Responsible Drug Disposal Act (2010), Combat Methamphetamine Enhancement Act (2010), Food Safety Modernization Act (2011), and FDA Safety and Innovation Act (FDASIA; 2012).

FDA’s role in maintaining the safety and availability of health-related and food products could not be more important to the American people. Just to name a few, FDA’s responsibilities include identifying problems in a production facility or compounding pharmacy across state lines when a cluster of infections is noted, linking a nationwide food-borne illness with a specific contamination within the entire country’s food supply, and managing the crisis of shortages of life-saving and palliative cancer therapeutics that has arisen in recent years. In part because of the determined efforts of the FDA Drug Shortages Program, we have seen a reduction in new drug shortages from the highs reached in 2011. But despite the improvement, existing shortages remain unresolved. All of these disparate functions require a robust infrastructure and a diversely experienced staff.

Despite FDA’s central importance to so many aspects of American life, sequestration acutely threatens the agency’s ability to fulfill its current mission, let alone expand it. Under sequestration, the agency is losing 5.1% of its current-year budget. Worse still, the Office of Management and Budget has testified that the actual impact is closer to 9%. This is a cut of ~$209 million, which will have an impact on all of the agency’s critical functions. Policy-makers and the public should be concerned and outraged that sequestration may cause slower approval of new and potentially life-saving drugs, less rigorous monitoring of food and drug safety, and an inability to keep up with advancing science and technology. This last aspect is especially in jeopardy because of limits these cuts place on the FDA staff’s participation in scientific meetings and training.

The biomedical community has played an important role in voicing concerns about the impact that sequestration cuts are having on federally funded research at the U.S. National Institutes of Health. We should also be involved in advocating for sufficient resources for FDA. Ultimately, biomedical research offers no value to the public if our work does not result in the availability of safe and effective interventions to cure, treat, palliate, diagnose, and prevent disease. Adequate resources for FDA are a vital link in the translational chain, and sequestration is a threat to the health and welfare of everyone.

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