American Society of Hematology

Congress Finalizes FDA Bill on User Fee Programs & Drug Shortages

Published on: June 20, 2012

On June 18, the United State Senate and House of Representatives filed the final Prescription Drug User Fee Act (PDUFA) legislation (The Food and Drug Administration (FDA) Safety and Innovation Act, S. 3187) including important provisions to mitigate drug shortages. In addition to addressing drug shortages, the bill would create user fee programs for generic drugs and generic biologic drugs, or biosimilars, and include provisions that focus on the safety of the drug supply chain, among other issues. The House passed the bill on Wednesday, June 20, and the Senate is expected to take up the bill the week of June 25.

In recent weeks ASH had been leading advocacy efforts to ensure that biological products were included in the bill's requirement that manufacturers provide advanced notification of stoppages or interruptions in production that could result in a drug shortage. In addition, ASH sought to remove the exemption of products derived from human plasma proteins and recombinant products replacing human tissue – products used for the treatment of hemophilia and other bleeding disorders – from the early reporting mandate in the House and Senate bills.

The advanced notification requirements in the final legislation apply to drugs which are "life-supporting, life-sustaining, or intended for use in the prevention of or treatment of a debilitating disease or condition including drugs used in emergency medical care or during surgery that are not radio pharmaceutical drugs." While the legislation does not include biological products in the advanced notification requirements, it gives the Secretary of the Department of Health and Human Services (HHS) the authority to apply these requirements to biological products including plasma protein therapies and their recombinant analogs by regulation if it would benefit public health.

The treatment of biological products in the final legislation reflects the significant difference between the House bill, which included biologics in this section, and the Senate bill, which limited the advanced notification requirements to sterile injectible products. The House and Senate resolved their differences by leaving the decision to the HHS Secretary and the regulatory process. Based on discussions with FDA on the legislation, agency officials have supported the inclusion of biological products in the advanced notification requirements. ASH's advocacy was influential in removing the exemption of plasma protein therapies and their recombinant analogs from the advanced notification requirements in the final bill.

Over the past 18 months, ASH has worked closely with Congressional leaders as they have developed drug shortage legislation. ASH has made several recommendations on ways to prevent and mitigate shortages, including to: increase FDA authority, require that the Secretary disseminate information about drug shortages to patient and provider organizations, enhance inter- and intra-agency coordination on drug shortages, require that the Agency Strategic Plan consider the impact of drug shortages on research and clinical trials, examine the impact of current FDA requirements on shortages, and provide economic incentives to manufacturers of critical drugs.

The Society's advocacy has been successful and the final bill includes several of ASH's key recommendations, including:

  • Requiring that the Secretary disseminate information about drug shortages to patient and provider organizations;
  • Requiring enhanced inter-and intra-agency coordination and requiring increased and effective communication with outside stakeholders; and
  • Requiring that the Strategic Plan consider the impact of drug shortages on research and clinical trials.

More information about drug shortages and ASH advocacy is available on the ASH website.

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