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US can continue to lead in drug innovation

Bringing life-saving therapies to patients who need them is a critical societal goal. Many of the 51 novel drugs and biologics that the U.S. Food and Drug Administration (FDA) approved in 2014 are intended to treat, prevent and even cure serious or life-threatening diseases such as cancer, bleeding disorders, pulmonary fibrosis, bacterial infections and hepatitis C. However, the escalating costs and time needed to move a new drug from discovery through human testing is threatening future drug development. As Congress explores legislation to modernize drug discovery and development for the 21st century, we look forward to collaborating on innovative and flexible approaches to bring novel medicines forward as quickly as possible.

It is important to remember, however, that innovative therapies only save lives if they work properly. U.S. citizens rely on the FDA to ensure that the drugs they take are effective and that their benefits outweigh their risks. Improving a patient’s life or lifespan must be central to the concept of drug innovation.

{mosads}Congress and the FDA have worked together previously to make the drug review process more efficient. A quarter-century ago, Congress authorized the first Prescription Drug User Fee Act, giving the FDA vital new resources that resulted in drastic reductions in the time it takes the agency to review new drugs.

As a result, the FDA consistently reviews new drugs faster than all other advanced regulatory authorities around the world, including the European Medicines Agency, and American patients are frequently the first in the world to receive new, innovative drugs. In 2014, the FDA approved the largest number of new drugs and biologics in almost 20 years, including unprecedented numbers of drugs for rare diseases and first-in-class drugs, the most innovative type.

The FDA uses tremendous flexibility when evaluating new drugs intended to treat severe illnesses. More than one-third of the novel drug applications approved by the FDA from 2008 to 2013 were approved on the basis of just one human efficacy study, with supporting evidence, and almost two-thirds of those applications utilized either a flexible development program and/or engaged in one of the FDA’s expedited development programs. For example, the breakthrough therapy designation, created by Congress in 2012, contributed to the approval of 10 highly innovative new medicines in 2014. To date, the breakthrough program has shown promising results in reducing development time as well as FDA review time. 

While Congress and the FDA have dramatically shortened FDA review times, challenges in the preclinical and clinical phases of drug development remain. The very high failure rates during development and the rising costs of clinical trials are most problematic. The FDA shares Congress’s interest in reducing the time and investment needed to develop new drugs. Greatly improved translational science and clinical trial infrastructures will be required to accomplish this. Multiple interventions — scientific, legislative, policy and procedural — will likely be needed. The FDA has been actively involved in working on these problems for the last decade and continues to participate in multiple partnerships with the public and private sectors to address these issues. It is imperative that the pathway from the laboratory to the market enable efficient translation of scientific advances into beneficial medicines. The FDA stands ready to partner with Congress, the National Institutes of Health and other stakeholders to keep the United States at the forefront of drug innovation.

For more information about FDA’s 2014 drug approvals, see the FDA’s Novel New Drug Summary for 2014. Lists of the Center for Drug Evaluation and Research’s 2014 novel new drug approvals and the Center for Biologics Evaluation and Research’s 2014 approvals are available on

Woodcock is director of the FDA Center for Drug Evaluation and Research. Midthun is director of the FDA Center for Biologics Evaluation and Research.

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