Alliance for Cell Therapy Now and Bipartisan Policy Center Host Roundtable Discussion on Advancing Real-World Data and Evidence to Support Regulatory and Payment Decision-making for Regenerative Cell Therapies

Washington, D.C. (January 27, 2020) – Today, the Alliance for Cell Therapy Now collaborated with the Bipartisan Policy Center to host a roundtable discussion on advancing the use of real-world data and evidence to support regulatory, payment, and patient decision-making for regenerative cell therapies. The event was co-chaired by former Food and Drug Administration (FDA) Commissioner and former Centers for Medicare and Medicaid Services (CMS) Mark McClellan and former FDA Commissioner and former National Cancer Institute (NCI) Director Andrew von Eschenbach.

Public and private sector leaders engaged in a robust discussion on the real-world data and evidence needs associated with regulatory evaluation of and payment for regenerative cell therapies, along with action steps needed to drive patient access to such therapies.

FDA Principal Deputy Commissioner Amy Abernethy kicked off the discussion, describing her vision for a modernized FDA supported by innovative technology and the use of data from real-world sources. FDA CBER Director Peter Marks engaged in a highly interactive discussion with roundtable participants on the actions needed to bring regenerative cell therapies to market, focusing in on the need for greater evidence.

Leaders from government agencies, such as CMS, the Health Resources and Services Administration, and the National Institute of Science and Technology, engaged in the discussion, along with private sector leaders, representing academic and research institutions, cell therapy manufacturers, the pharmaceutical industry, payers, philanthropy, investors, and other non-profit organizations.

Several actions emerged from the discussion, including the need for a collaborative, multi-center trial that combines traditional evidence development with data from real-world sources, as well as robust cell characterization to take into account inherent variation in the manufacturing of human cells.

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About the Alliance for Cell Therapy Now

Alliance for Cell Therapy Now (ACT Now) is an independent, non-profit organization devoted to advancing the availability of and access to safe and effective cell therapies for patients in need. ACT Now convenes experts and stakeholders to develop and advance sound policies that will improve the development, manufacturing, delivery, and improvement of regenerative cell therapies.  See http://allianceforcelltherapynow.org/