In this Issue

Featured Story»
Regulatory»
Coverage and Reimbursement»
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Featured Story

The Alliance for Regenerative Medicine kicked off the year with its annual “Cell and Gene State of the Industry Briefing,” which provides an overview of the industry’s progress in 2020 and key milestones for the year ahead. Looking back, 2020 was a year of progress and challenges. The Food and Drug Administration (FDA) approved regenerative medicine advanced therapy (RMAT) designations for 12 investigational drugs, a designation that bestows advantages on the sponsor, including early and frequent interactions with FDA and expedited review timelines. 2020 also saw six programs delayed due to manufacturing or data-related issues. In 2021, regulatory decisions are expected on six cell and gene therapy products. They include StrataGraft® (Mallinckrodt plc), idecabtagene vicleucel (ide-cel) (BMS/bluebird bio), lisocabtagene maraleucel (liso-cel) (BMS), PTC-AADC (PTC Bio), Lumevoq® (GenSight Biologics), and elivaldogene autotemcel (eli-cel, Lenti-D™) (bluebird bio). 

Regulatory

NCATS and FDA Workshop Addresses Immunogenicity of Gene Therapies 

The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) and FDA’s Center for Biologics Evaluation and Research (CBER) held a virtual workshop on November 30–December 1, 2020, “Systemic Immunogenicity Considerations for Adeno-Associated Virus (AAV)-Mediated Gene Therapy.” This workshop featured scientists, product developers, and patient representatives to discuss immunogenicity issues related to the administration of AAV gene therapies. The objectives of this meeting included exchanging lessons learned from AAV gene therapy clinical trials, exploring new strategies for modulating the immune response to AAV gene therapies, identifying knowledge gaps, and exploring possible solutions. The discussion will inform a research agenda that builds on the current science to address these issues. A key opportunity discussed was the role of an entity like NCATS to facilitate pre-competitive data sharing activities. 

FDA Publishes Guidance on Gene Therapies for Neurodegenerative Diseases

In January 2021, FDA CBER released a Draft Guidance for Industry titled “Human Gene Therapy for Neurodegenerative Diseases,” which provides recommendations to sponsors developing new gene therapies for the adult and pediatric neurodegenerative diseases populations. The guidance discusses considerations for chemistry, manufacturing, and controls (CMC), preclinical studies, and clinical trials. It also highlights the FDA’s expedited programs that can be used for gene therapies, particularly the regenerative medicine advanced therapy designation and breakthrough therapy designations. The guidance encourages product sponsors to engage with FDA early (before the submission of an investigational new drug application) and discusses the meeting types available to sponsors to receive early regulatory advice, including the INTERACT meeting, which can be used to discuss a product’s preclinical program. 

FDA Issues Guidance on COVID-19 Considerations for Cell and Gene Therapy

FDA CBER published a final guidance in January 2021 titled, “Manufacturing Considerations for Licensed and Investigational Cellular and Gene Therapy Products During COVID-19 Public Health Emergency,” which provides recommendations to minimize the risk of transmitting coronavirus in the manufacture of cell and gene therapies. The guidance’s recommendations address the assessment of donors, source cells and tissues, manufacturing processes, manufacturing facility control, and materials testing. 

 Coverage and Reimbursement

“Cures 2.0” Expected to Include Proposals to Modernize CMS Coverage and Reimbursement

At the Milken Institute Future of Health Summit, Representatives Diana DeGette (D-CO) and Fred Upton (R-MI) updated the audience on the status of “Cures 2.0,” the widely anticipated follow-on legislation to the 21st Century Cures Act that was enacted in 2016. Representative DeGette indicated that a goal of the new legislation would be to modernize the Centers for Medicare & Medicaid Services’ coverage and payments approach to better accommodate new innovative technologies. In a concept paper released in April 2020, cell and gene therapies were among the new technologies that garnered interest. The lawmakers indicate they are continuing to work with experts in the field to develop the legislation and expressed optimism it will have broad, bipartisan support in the new Congress. 

Policy

CMS Finalizes New Medicaid Rules on Value-Based Pricing Agreements 

On December 21, 2020, CMS finalized a proposed rule issued in June 2020 to give states and manufacturers more flexibility to enter into value-based agreements. Specifically, CMS addresses barriers created by Medicaid’s “best price” rules, which manufacturers and payers have argued discourage the use of value-based contracts. In the December rule, CMS provides new methods for manufacturers to calculate the “best price” for their drug. These changes will take effect in January 2022. 

Value-based contracting is just one of the approaches that states will pursue to provide Medicaid beneficiaries with access to cell and gene therapies and other novel but costly medical advances. With the budget strains created by the ongoing COVID-19 pandemic, state Medicaid programs will continue to seek to experiment with new approaches to finance new therapies. 

Cures for Life Quarterly compiles new policy, regulatory, and reimbursement-related developments that have implications for access to cell and gene therapies. For questions regarding this Cures for Life issue, please contact Allyson Conlin at [email protected].