Helping to shape drug shortage provisions in FDA user fee legislation

Oct. 19, 2012

On July 9, 2012, President Barack Obama signed the U.S. Food and Drug Administration (FDA) Safety and Innovation Act of 2012 (Public Law No: 112-144) into law, reauthorizing the FDA’s user fee programs for five years and creating user fee programs for generic and biologic drugs. In addition to reauthorizing and creating user fees, perhaps the most important provisions in this legislation to members of the American Society of Hematology (ASH) were those intended to mitigate the country’s increasing drug shortage problem. Shortages have had particularly devastating effects on patients managed by hematologists because many of the drugs and biologic products most vulnerable to shortages are used to treat blood disorders. Not only have shortages interrupted care, putting patients’ lives in danger, but they have also delayed clinical research critical to the development of new life-saving drugs.

The drug shortage provisions of this comprehensive and complex piece of legislation establish a process by which drug manufacturers are required to provide the FDA with a six-month advance notification of anticipated stoppages or interruptions in the manufacturing of drugs that are “life-supporting, life-sustaining, or intended for use in the prevention of, or treatment for, a debilitating disease or condition, which could lead to a shortage of that drug in the United States.” The intent is to establish a formal process by which the FDA can collaborate with manufacturers in order to take actions which could prevent or mitigate drug shortages.

Prior to the development of this legislation, ASH had become a vocal advocate for solutions to address this growing crisis. Leadership and staff from ASH, the first medical society to call on Congress to conduct hearings on drug shortages, had already met with senior administration officials and the FDA, were tracking hematologic drug shortages on a special page on its website,, and had begun collaborating with drug companies to obtain products in shortage for its members.

ASH continued its advocacy as Congressional leaders from both sides of the aisle developed the drug shortage provisions in the FDA Safety and Innovation Act of 2012 and, in a rare showing of bipartisanship, brought this legislation to life. Since the bill’s main task was to reauthorize drug user fees that were critical to keeping the FDA operational, the bill was labeled “must pass,” and special interest groups were warned that their lobbying efforts for their cause would likely not be considered in the interest of swift passage. Nevertheless, recognizing the crisis at hand and the enormous threat drug shortages would pose to its members and the patients they treat, ASH redoubled its efforts.

Collaborating with ranking members of the House Energy and Commerce Committee and the Senate Committee on Health, Labor, and Pensions, ASH worked to ensure that biologic products, which were originally exempted from the early notification mandate in previous versions of the legislation, were not specifically excluded. These drugs, which range from chemotherapeutic agents used to treat patients with blood cancers (biologics) to plasma protein therapy products and their recombinant analogs used for the treatment of hemophilia and other bleeding disorders, are critical to the delivery of care for these patients.

Ultimately, the advocacy was successful on several fronts. While the final FDA Safety and Innovation Act does not include biological products in the advanced notification requirements in its drug shortage provisions, it authorizes the Secretary of the Department of Health and Human Services (HHS) to apply these requirements to biological products including plasma protein therapies and their recombinant analogs by regulation if it would benefit public health. The final bill also includes several additional key ASH recommendations. First, the bill requires that the HHS Secretary disseminate information about drug shortages to patient and provider organizations, requires enhanced inter- and intra-agency coordination between the FDA and other organizations, and requires the FDA to increase its communication with outside stakeholders. The bill also mandates that the HHS establish a task force and develop and implement a strategic plan to enhance the Department’s response to prevent and mitigate drug shortages. The strategic plan required by the bill also includes an evaluation of the impact of drug shortages on research and clinical trials.

While these provisions included in the FDA Safety and Innovation Act mark an important first step to address the current drug shortage crisis, ongoing advocacy to prevent future shortages is critical. ASH will continue to monitor all hematologic drug shortages closely and will continue to work with policymakers to prevent and mitigate drug shortages and protect the health of Americans.

Armand Keating, MD, is president of the American Society of Hematology, the world’s largest professional society concerned with the causes and treatment of blood disorders.