In this Cures for Life Issue

Featured Story
Regulatory
Coverage and Reimbursement
Policy
FasterCures’ Resources

By 2025, the Food and Drug Administration (FDA) anticipates approving 10 to 20 cell and gene therapy products a year. FasterCures’ Cures for Life project aims to ensure patients will have access to these life-changing therapies as they are approved by shining a light on potential roadblocks to access and bringing diverse stakeholders together to help solve for these hurdles.

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

Cures for Life Featured Story

FasterCures Webinar Brings Together Experts to Discuss Long-Term Data Collection for Cell and Gene Therapies

In a webinar on September 23, FasterCures brought together Peter Marks, director of FDA’s Center for Biologics Evaluation and Research (CBER), Gwen Nichols, chief medical officer of the Leukemia & Lymphoma Society, and John Doyle, vice president and global healthcare innovation lead of Pfizer to discuss the opportunities and challenges of long-term follow-up data collection for cell and gene therapies. Over the hour-long discussion, the panelists talked about the importance of helping patients understand the value of their data and the need to ensure harmonization in data collection efforts. The panelists agreed on the potential of custom long-term data platforms to help answer outstanding questions about cell and gene therapies. The development of these platforms would be facilitated by a neutral party, like FDA or a patient organization, and created with the input of a coalition of interested stakeholders.

Regulatory

COVID-19 Demands Raise Concerns about FDA’s Capacity for Timely Cell and Gene Therapy Reviews

The demands being placed on FDA’s Center for Biologics Evaluation and Research by COVID-19 has raised concerns about the center’s ability to keep pace with its other responsibilities, including in the rapidly growing field of cell and gene therapy. Peter Marks, director of CBER, has acknowledged this strain in remarks, most recently at FasterCures’ webinar on September 23, where he noted that the “extra workload has slowed things down. Our ability to respond to meeting requests is nowhere near where I would like it to be.” In an investor call, the CEO of Sarepta Therapeutics also cited a slowdown in communications with FDA as a contributing factor to a recently announced setback to its gene therapy candidate for Duchenne muscular dystrophy. Even before the COVID-19 pandemic, FDA’s CBER had signaled that it would need to ramp up resources to support an acceleration in investigational new drug applications for cell and gene therapies.

WHO Drafting a Regulatory Framework for Cell and Gene Therapies

The World Health Organization (WHO) is expected to issue a white paper to propose a harmonized regulatory framework for cell and gene therapies. The framework is intended to increase alignment among high-income countries while providing a framework for low- and middle-income countries. According to a May 2020 technical report released by the WHO Expert Committee on Biological Standardization, the white paper will include definitions, identify the key elements of a conceptual framework, and set out the considerations for quality, safety, efficacy, and post-market surveillance.

FDA Delays Decision on Gene Therapy for Hemophilia A

In what was widely seen as an unexpected move, the FDA has asked Biomarin Pharmaceuticals for more data on its gene therapy candidate for hemophilia A due to concerns about the durability of its effects. The therapy, which had received a breakthrough designation and a priority review status from the FDA, would have been the first gene therapy to treat hemophilia A. According to Biomarin, FDA requires two additional years’ data from an ongoing trial to understand the duration of the therapy’s effect. The European Medicines Agency, the European Union regulatory agency, has also requested additional data from the ongoing Phase III trial for the therapy.

Coverage and Reimbursement

CMS Changes Medicare Reimbursement of CAR-T Therapies

The Centers for Medicare & Medicaid Services (CMS) finalized changes to how hospital inpatient treatments using chimeric antigen receptor T-cell (CAR-T) therapies will be reimbursed under Medicare. CMS will create a new Medicare Severity Diagnosis-Related Group (MS-DRG 018) for CAR-T treatment stays. This change is expected to improve the predictability of payments for CAR-T therapies, but some hospitals may still fall short of fully offsetting provider costs. In the final rule, CMS also announced its intent to engage stakeholders on payment mechanisms for solid tumor cell therapies, such as Tumor-Infiltrating Lymphocyte Therapy and Engineered T Cell Receptor Therapy.

Large Employers Worry about High-Cost Drug Therapies

According to the 2021 survey of large employers by Business Group on Health, 67 percent of respondents cited new million-dollar treatments as their top pharmacy concern. Strategies used or being considered by large employers to manage high-cost drugs include delaying placing these therapies on formularies, purchasing stop-loss insurance to mitigate catastrophic losses, using outcomes-based pricing, participating in a pharmacy benefit manager or health plan risk pool, or implementing a drug financing model.

Policy

Medicaid Paving the Way for More Value-Based Pricing Agreements

Value-based agreements may become more common between states and manufacturers. Under a proposed rule released in June, CMS proposes to allow manufacturers to report multiple “best prices” for drugs in value-based agreements. Under Medicaid’s “best price” rule, manufacturers must offer the lowest negotiated price with insurers to Medicaid plans. This policy has frequently been cited as a barrier to implementing value-based arrangements. This policy change could pave the way for greater value-based agreements for cell and gene therapies, which are considered ideal candidates for testing new payment approaches, given their high upfront costs and potential to offer substantial long-term value.

FasterCures’ Resources

Read FasterCures’ report on Long-Term Follow-Up Data Collection for Cell and Gene Therapies, which explores the data needs of different stakeholder groups, the strengths and limitations of current models for data collection, and future considerations relevant to each stakeholder group.