In December 2015, the Bipartisan Policy Center –a Washington, D.C. nonprofit think-tank– published a seminal paper entitled Advancing Regenerative Cellular Therapy -Medical Innovation Healthier Americans. This Report provided information about the nature of cell therapies, international competitive landscape and US regulatory analysis. Guiding the document was a blue-ribbon panel including experts and leaders from Duke University School of Medicine, Wake Forest Institute for Regenerative Medicine, California Institute for Regenerative Medicine, Mayo Clinic, Sanford Burnham Prebys Discovery Institute, and Harvard Stem Cell Institute.
The report served as the framework for a groundbreaking bipartisan legislative initiative in 2016, the REGROW Act, that sought to elevate regenerative medicine as a national priority and called for an accelerated approval pathway for regenerative cell therapies.
After much public input, the proponents of REGROW, working with a supportive coalition of stakeholders including our team at the Regenerative Medicine Foundation, helped craft innovative proposals that evolved into the Regenerative Advanced Therapies section of the 21st Century Cures Act, setting forth a priority review and accelerated approval pathway for regenerative medicine advanced therapy products and mandates that FDA to consider real world evidence.
Passage was a celebrated event, and even the loudest critics from industry who fought the regenerative medicine legislation down to the wire, finally embraced it wholeheartedly at the end. To paraphrase Tacitus and JFK, victory has 100 fathers, and failure is an orphan!
“Seriously now, who can oppose bringing regenerative medicine clinical practice out of the anecdotal fogginess and into the sunlight of transparency?”
The Bipartisan Policy Center has now published a April 2017 report titled Accelerating the Development and Delivery of Safe and Effective Cures for Patients in the United States recommending that Congress supports a federally funded registry for regenerative cell therapies.
At the moment, the general public, clinicians and researchers are frustrated. How can we embrace these potential curative therapies if the outcomes are not shared? Where’s the data? A national registry will go a long way to solving that problem.
Registries are real world evidence. A national registry It’s the next logical step and one that all proponents of regenerative medicine should embrace.
The Bipartisan Policy Center report underscores that there is a critical need to establish a national registry for such therapies to support research, rapidly improve practice and provide additional data to inform decision-making.
The Bipartisan Policy Center specifically recommends that Congress first clarify existing law to include regenerative cell therapies in the existing Stem Cell Therapeutic Outcomes Database, beyond bone marrow and cord blood. The Outcomes Database presently includes compiling information in a standardized electronic format with patients diagnosis, procedures, results, long-term follow up and such other information that HHS determines, in order to conduct an ongoing evaluation of the scientific and clinical status involving recipients.
“How can we embrace these potential curative therapies if the outcomes are not shared? Where’s the data? A national registry will go a long way to solving that problem”
Seriously now, who can oppose bringing regenerative medicine clinical practice out of the anecdotal fogginess and into the sunlight of transparency? Patients would have information necessary for decision making and clinicians would have information to improve outcomes. It’s all upside.
The Bipartisan Policy Center further recommends that Congress authorize additional funding to support an outcomes database. Without sufficient national funding, there is a risk that parochial interests will rise developing confusing, underfunded and competitive databases.
In the divided world of Washington D.C, we should congratulate the good work of the Bipartisan Policy Center that consistently seeks common ground and progress.
It’s time to advance a registry for all regenerative cell therapy outcomes.
Bernard Siegel is the Founder and Executive director of the Regenerative Medicine Foundation and the World Stem Cell Summit. As a recognized policy expert on stem cell research, regenerative medicine and cloning, Mr. Siegel is a frequent panelist and keynote speaker on the subject of stem cells, public policy, patient advocacy and the societal implications of longevity. He has spoken at the United Nations, the Salk Institute, the German National Academy of Science (The Leopoldina), and others. He has lectured at more than 40 colleges and universities around the globe.