PLENARY 1 OF ISPOR 21ST ANNUAL INTERNATIONAL MEETING FOCUSES ON ISSUES SURROUNDING ACCELERATED DRUG APPROVALS

Washington, DC —May 23, 2016—The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) opened its 21st Annual International Meeting this morning with the first plenary session, Accelerating Cures: Addressing Unmet Patient Need or Putting Patients at Risk?, in Washington, DC, USA.

The plenary was moderated by Eleanor M. Perfetto, PhD, MS, Senior Vice President, Strategic Initiatives, National Health Council, Washington, DC and Professor, Pharmaceutical Health Services Research, School of Pharmacy, University of Maryland. The session featured alternative viewpoints on accelerated development efforts. Policy efforts have focused on reducing the time needed to bring medical products to market and to patients in need. Legislative proposals intended to accelerate development of biopharmaceutical and other medical products have had bipartisan support; however, these efforts have also received criticism from opponents who have expressed concern that accelerated development endeavors may compromise patient safety.

Patricia Furlong, Founding President and Chief Executive Officer for Parent Project Muscular Dystrophy, opened the session and provided a poignant perspective by sharing her personal experience as the mother of two boys diagnosed with Duchenne muscular dystrophy. Furlong stressed that while rigorous data is key for new drug approvals, the needs of the patient must be considered. She remarked that when assessing risks and benefits, it is important to note that, “doing nothing, is also doing harm.”

Jerry Avorn, MD, Professor of Medicine, Harvard Medical School and Chief of the Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital provided an academic researcher’s perspective on the issue of accelerating drug approvals, posing the question, “Are we trying to solve a problem that barely exists?” While acknowledging the significance of Furlong’s experience, Avorn cautioned that attempts to loosen drug approval standards may actually end up causing harm to patients, citing historical cases with drugs like laetrile, thalidomide, and Avastin. He noted that while some improvements are needed, the US Food and Drug Administration (FDA) approves drugs as fast as any other regulatory body, with an average time for priority review for a new drug of 6 months.

Scott Gottlieb, MD, Resident Fellow at the American Enterprise Institute, provided a perspective that offered a unique glimpse into the regulatory process and reforms that have been implemented to accelerate drug approvals. Gottlieb reviewed the history of the Breakthrough Therapy Program, noting that oncology products have benefited most from this legislation, but that the Breakthrough Therapy Program is available across all therapeutic areas. He noted that the FDA review process has become increasingly siloed and that this impedes prompt approvals. Gottlieb also spoke about the issue of ultra-rare diseases in which conventional clinical development programs are often not easily obtainable and noted a number of opportunities for additional reform.

When asked what one thing could be done to improve the overall process, Furlong and Gottlieb pointed to the need for greater transparency and cross-departmental communication in FDA decision making, respectively; Avorn encouraged Congress to appropriately fund the FDA and National Institutes of Health (NIH), which could ultimately help address and resolve these important issues.

Presentations and slide decks from the ISPOR 21st Annual International meeting can be found here. Interested parties can follow news and developments from the meeting on social media using the hashtag #ISPORDC.

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