Sector financing reached $19.9B in 2020, doubling from the previous year; 2021 is likely to be a record year for the number of regulatory decisions on new regenerative medicine products
The regenerative medicine and advanced therapies sector raised a record $19.9B in funding in 2020, fueling the broader biotech sector and driving a rapidly advancing pipeline of potentially transformative therapies, according to the Alliance for Regenerative Medicine’s (ARM) 2020 Annual Report released today.
Two new therapies received approval in 2020: Orchard Therapeutics’ gene therapy Libmeldy, by the European Medicines Agency (EMA), and Kite’s Tecartus chimeric antigen receptor T-cell (CAR-T) therapy, by the Food & Drug Administration (FDA). By the end of 2020, there were 152 ongoing Phase 3 trials worldwide in cell, gene, and tissue-based therapies. Regulatory decisions are expected on a record eight new regenerative medicine products – and on a total of ten products across at least four geographies – in 2021, including Breyanzi, a Bristol Myers Squibb CAR-T therapy for relapsed or refractory large B-cell lymphoma that already received FDA approval in February.
“When COVID-19 struck, we joined the rest of the world in not knowing what 2020 would bring,” said Janet Lambert, CEO of ARM. “Instead, not even a global pandemic could derail the scientific advancements, investor appetite, and maturing pipeline that made headlines for the regenerative medicine sector in 2020. For a once aspirational sector, the future is now.”
It was a significant year of growth for ARM as well. The global voice of the sector grew by 80 members, reaching a total global membership of more than 380 organizations. To serve this growing membership, ARM invested in five new full-time hires in 2020 and for the first time integrated its policy, advocacy, and communications activities under the umbrella of public affairs. The organization plans to expand its focus on public affairs in 2021 with new advocacy hires in Europe, at the U.S. federal level, and for U.S. states.
ARM and the sector also achieved notable policy milestones. Sector advocacy efforts led the Centers for Medicare & Medicaid Services (CMS) to create a diagnosis related group (DRG) for CAR-T therapies that will ensure proper reimbursement for providers. In December, CMS also finalized a rule removing barriers to innovative payment models in state Medicaid programs, a significant area of focus for ARM and its members. And the EU Pharmaceutical Strategy, a blueprint for a years-long legislative push, recognized cell and gene therapies as “milestones of major progress” in healthcare.
ARM and its members also made a commitment to improving racial equality in the sector. ARM’s Action for Equality Task Force established the GROW RegenMed Internship Program, which will provide paid opportunities for Black students at ARM member organizations. At least 16 ARM member organizations – as well as ARM itself – will host GROW interns in Summer 2021.
Highlights from ARM’s annual report and full-year 2020 data include:
Entering 2021 with significant momentum, ARM and its members will focus on policy and regulatory initiatives to ensure that transformational therapies continue to advance through the pipeline and that patients can access these treatments post-approval. In particular, ARM will advocate for the adoption of innovative payment models in the U.S. and Europe, and for Chemistry, Manufacturing and Controls (CMC) regulatory flexibility to address manufacturing hurdles in the rapidly evolving cell and gene therapy sector.
For more information or for media requests, please contact Stephen Majors, Director of Public Affairs for ARM, at email@example.com.
About the Alliance for Regenerative Medicine
The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory and reimbursement initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative medicines and advanced therapies. In its 11-year history, ARM has become the global voice of the sector, representing the interests of 380+ members worldwide, including small and large companies, academic research institutions, major medical centers and patient groups. To learn more about ARM or to become a member, visit http://www.alliancerm.org.