In this Issue
Regulatory and Drug Development »
Featured Story
FasterCures Roundtable: Manufacturing Challenges of Cell and Gene Therapies
FasterCures hosted a roundtable on March 4, 2021, to discuss the challenges of manufacturing cell and gene therapies. The discussion featured Peter Marks, director of the Center for Biologics Evaluation and Research at the Food and Drug Administration (FDA), who discussed some of the activities being undertaken by the agency to address current manufacturing challenges, such as the creation of the Bespoke Gene Therapy Consortium. Some of the other solutions discussed during the roundtable include increasing interactions between FDA and product sponsors on specific Chemistry, Manufacturing, and Controls (CMC) issues; defining the specific CMC issues where clarity from FDA is most needed; applying regulatory flexibilities for CMC activities; defining critical quality attributes for different product types; piloting new mechanisms; and creating safe, neutral venues for biopharmaceutical companies to solve for common pain points.
Regulatory and Drug Development
FDA Approves Two CAR-T Therapies
The first quarter of 2021 saw two CAR-T therapy approvals:
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Abecma® (Bristol Myers Squibb and bluebird bio) is a CAR-T cell therapy for adults with multiple myeloma approved by the FDA. Abecma was granted Orphan Drug and Breakthrough Therapy designations by the FDA.
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Breyanzi® (Bristol Myers Squibb) is a CAR-T cell therapy to treat adults with relapsed or refractory Large-B Cell Lymphoma. This product was approved with a cross-agency approach from the Center for Biologics Evaluation and Research and the Oncology Center of Excellence. Breyanzi was granted Orphan Drug and Breakthrough Therapy designations by the FDA and is the first therapy to be approved under FDA’s Regenerative Medicine Advanced Therapy designation.
Bill & Melinda Gates Foundation Partners with Novartis to Discover and Develop In Vivo Therapy Gene Therapy for Sickle Cell Disease
The Bill & Melinda Gates Foundation has entered into an agreement with Novartis to support the discovery and development of a single-administration, in vivo gene therapy to cure sickle cell disease, a hereditary blood disorder that disproportionately affects those of African descent. While gene therapies continue to advance and bring new hope to patients, this project in particular is aimed at making them more affordable and practical for low-resource settings, such as sub-Saharan Africa, which carries about 80 percent of the global disease burden for sickle cell disease. The key advantage of this potential new therapy is its ability to be administered once, directly into the patient, eliminating the need to modify cells in a lab and, in turn, prolonged hospital stays. This agreement follows a commitment made by the Gates Foundation, along with the National Institutes of Health, to invest a combined $200 million over the next four years to develop gene therapies for sickle cell disease and HIV that would be made available globally.
Coverage and Reimbursement
ICER Publishes Evidence Report on CAR-T Cell Therapies for Multiple Myeloma
On April 5, the Institute for Clinical and Economic Review (ICER) released an evidence report assessing the comparative clinical effectiveness and value of three new treatments targeting the B-cell maturation antigen for Triple Refractory Multiple Myeloma (TCRMM) patients. TCRMM is a condition caused when multiple myeloma patients no longer respond to traditional treatments, including immunomodulatory agents, proteasome inhibitors, and anti-CD38 monoclonal antibodies. The treatments reviewed included two CAR-T cell therapies idecabtagene vicleucel (”ide-cel,” Abecma®) and ciltacabtagene autoleucel (”cilta-cel”). The third treatment reviewed was belantamab mafodotin-blmf (Blenrep®, GSK). On April 16, this evidence report was reviewed by ICER’s Midwest Comparative Effectiveness Public Advisory Council at a public meeting. Discussions and input collected during this meeting will inform a final evidence report, which is expected to be released on May 11, 2021. Payers may use the findings of this final report in their coverage decision-making about these therapies.
Policy
OIG Issues Advisory Opinion That Paves Way for CAR-T Patient Assistance Program
On March 23, the Department of Health and Human Services Office of Inspector General (OIG) released an advisory opinion in response to a request from a CAR-T therapy manufacturer regarding a potential free drug program. Under the program, the manufacturer would offer the cell therapy free of charge to patients who lack or have been denied coverage of the drug and fall below an annual income threshold. The OIG found that such a program would not violate the US federal anti-kickback statute (AKS), which prohibits the exchange (or offer to exchange) of anything of value in an effort to induce (or reward) the referral of business reimbursable by federal health-care programs. Because of the one-time nature of CAR-T therapy, the OIG found that such a program presents a “sufficiently low risk of fraud and abuse” under AKS, as providing the drug for free would not be contingent on future orders. This opinion paves the way for other CAR-T therapy companies to explore similar programs.