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As science progresses, we need parallel progress in making breakthrough treatments widely available, BIO President & CEO John F. Crowley said at a Jan. 8 White House Cell and Gene Therapy Forum.
“Innovation without access for all isn’t enough,” Crowley told a panel that he moderated at the event. “The science is hard enough, so let’s work to increase access.”
Several other attendees said access is becoming the biggest challenge to delivering the remarkable advances enabled by cell and gene therapies (CGT). Solutions for improving access presented by the forum included:
- Supporting the Centers for Medicare & Medicaid Services Cell and Gene Therapy Access Model and learning from the process of expanding access at the state level.
- Considering reform of the Employee Retirement Income Security Act (ERISA).
- Passing the Rare Pediatric Priority Review Voucher (PPRV) Act and other supportive legislation.
The issue is something Crowley’s Biotechnology Innovation Organization (BIO) has been seeking to address for a while.
Earlier discussions hosted by BIO have emphasized the need for innovations in paying for CGTs. One of the unique challenges of these therapies is that they may have high upfront costs but reduce health costs overall, which is why BIO has advocated value-based payment models.
CGTs have been enabled by advances in genomic research and offer previously unachievable results for patients. Cell therapies include CAR T cell therapy, in which cells taken from a patient are genetically altered to attack cancer cells and then reintroduced into the patient. Gene therapies, which alter defective genes, can be especially effective against inherited genetic conditions, such as sickle cell disease and other rare diseases.
Rare Disease Congressional Caucus Co-Chair Sen. Amy Klobuchar (D-MN) was one of the speakers at the White House Forum. She heard from experts who echoed Crowley’s calls for policy to encourage CGT innovation and access.
“The science of CGTs has progressed unbelievably and can change the course of disease,” Crowley said. “But we still have much work to do to get a cure or therapy to patients.”
2024 National Medal of Technology and Innovation
Biotech was also in the spotlight at the White House on Jan. 3 when President Biden awarded the 2024 National Medal of Technology and Innovation. Among the 11 recipients were three biotech leaders, according to an announcement from the U.S. Patent and Trademark Office:
- Jennifer A. Doudna, Innovative Genomics Institute: “For development of the revolutionary CRISPR-Cas9 gene editing technology, with widespread applications in agriculture and health research. Jennifer Doudna’s innovations are fundamentally transforming our collective health and well-being and have contributed to the development of treatments for sickle cell disease, cancer, type 1 diabetes, and more.”
- Moderna, Inc.: “For saving millions of lives around the world by harnessing mRNA vaccine technology to combat a global pandemic. In 2020, Moderna rapidly developed and deployed a COVID-19 mRNA vaccine that was essential to ending the COVID-19 pandemic, opening new frontiers in immunology and advancing America’s leadership in research innovation.”
- Pfizer Inc.: “For saving millions of lives around the world by harnessing mRNA vaccine technology to combat a global pandemic. In 2020, Pfizer rapidly developed and deployed a COVID-19 mRNA vaccine that was essential to ending the COVID-19 pandemic, opening new frontiers in immunology and advancing America’s leadership in research innovation.”
Featured photo: White House CGT Forum panel moderated by BIO President & CEO John F. Crowley, from left: Rebecca Gardner, St. Jude / American Society for Transplantation and Cellular Therapy; Matthew Durdy, Catapult UK; Crowley; Karen Skinner, LifeArc; and Priti Hegde, Kite Pharma.
