American Society of Hematology

Congress Passes FDA Bill on User Fee Programs & Drug Shortages; Bill Sent to President for Signature

Published on: June 27, 2012

On June 26, the United State Senate passed 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 bill, which was passed by the House on Wednesday, June 20, will now go to President Obama for his signature.

ASH believes this legislation represents an important first step in addressing the drug shortage crisis. However, the Society is disappointed that the final legislation does not include biologics in the list of drugs covered under the bill's requirement that manufacturers provide advanced notification of stoppages or interruptions in production that could result in a drug shortage. These drugs, ranging 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. They are critical to the delivery of care for patients with blood disorders, and the non-inclusion of these drugs puts these patients in jeopardy.

In recent weeks ASH had been leading advocacy efforts to ensure that biological products were included in the bill's early warning requirement. In addition, ASH sought to remove the exemption of products derived from human plasma proteins and recombinant products replacing human tissue from the early reporting mandate in the House and Senate bills. 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. ASH will continue its advocacy efforts with the Secretary and FDA to see that biologics are included in the advanced notification requirements through the regulatory process.

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, which were included in the final bill, 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.

ASH will provide members with a comprehensive analysis of the full bill in the coming weeks and will continue to monitor all hematologic drug shortages closely. More information about drug shortages and ASH advocacy is available on the ASH website.

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