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Regulatory & Drug Development
Coverage & Reimbursement
Featured Story
Lawmakers Release Cures 2.0 Discussion Draft
In June, Representatives Diana DeGette (D-CO) and Fred Upton (R-MI) released a discussion draft of Cures 2.0 following the landmark 21st Century Cures Act biomedical innovation law. The draft bill includes provisions related to the authorization of medical research, the Food and Drug Administration’s (FDA) regulation of medical products, and Medicare’s payments for new, cutting-edge treatments. It would also establish a new medical research agency, the Advanced Research Projects Agency for Health (ARPA-H), for which the Biden administration has requested a $6.5 billion initial budget.
Several sections of the discussion draft have implications for cell and gene therapies. They include a requirement that the Health and Human Services (HHS) secretary produce a report to Congress on the current state of cell and gene therapy regulation. This report to Congress identifies current obstacles to the development of and access to these therapies. The draft bill also calls on FDA to develop guidance on Chemistry, Manufacturing, and Controls (CMC) programs in the context of expedited reviews.
Lawmakers will continue to collect input on this bill from various stakeholders across the health-care ecosystem before releasing a final bill following Congress’ August recess. FasterCures’ comment letter on the discussion draft can be viewed here.
Regulatory and Drug Development
FDA’S CBER Holds Workshop on Patient Engagement and Regenerative Medicine
The FDA’s Center for Biologics Evaluation and Research (CBER) held a workshop in May called Patient Engagement & Regenerative Medicine: An FDA CBER Workshop for Patient Advocates. The convening included a presentation highlighting the FDA’s efforts to advance regenerative medicine and two panels that discussed the value of the patient perspective in regenerative medicine and shared strategies for how patient organizations might more closely engage with the FDA during the drug development and regulatory approval process.
The role of the patient perspective was emphasized through two case studies examining patient organizations’ interactions with the FDA’s Office of Tissues and Advanced Therapies (OTAT). The first highlighted a Critical Path Innovation Meeting (CPIM) held between the Lou Lou Foundation and the FDA, scheduled after the two organizations had initial contact in an externally-led Patient-Focused Drug Development (PFDD) meeting. The Lou Lou Foundation sought this CPIM to review their clinical outcome assessments and natural history study designs to inform their observational study design.
The second case study featured a private listening session organized by the National Organization for Rare Disorders (NORD) and Reagan-Udall Foundation for the FDA, patients with Glycogen Storage Disorder 1A (GSD1A), and their caregivers. The meeting helped stakeholders better understand GSD1A and understand the caretaker perspective on the burden of the disease and its symptoms. During this listening session, the FDA inquired about the burden associated with current therapies and patients’ risk tolerance related to participation in clinical trials—especially gene therapy trials.
This workshop builds on ongoing efforts by the FDA to engage key patient stakeholders in the drug development process. Toward that end, FDA also used the workshop to highlight the various forums that are currently in place for patients to engage with FDA, including advisory committee meetings, special government employee consultants, public meetings and workshops, patient-focused drug development meetings, FDA/NORD rare diseases listening sessions, and meetings with patient organizations.
The workshop recording and materials can be found here.
FDA CBER Director Speaks on the Cell and Gene Therapy Landscape Amid COVID-19
Peter Marks, director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA), spoke at the Drug Information Association (DIA) Global Annual Meeting about the effects of COVID-19 on the agency’s review of cell and gene therapies. While he noted that no aspect of biomedical research and development was left untouched by the fallout from the pandemic, he observed that some parts of the cell and gene therapy R&D pipeline were not completely sidelined. Notably, the agency received 237 Investigational New Drug Applications (INDs) in 2020, only six fewer than in 2019. Dr. Marks also shared that although work on guidance documents slowed down due to the pandemic, the FDA also became more efficient at issuing guidance and that these learnings and efficiencies can be applied toward producing guidance documents more rapidly in the future.
ARM and NIIMBL Release Project A-Gene
Among the many challenges of cell and gene therapy development is scaling up manufacturing. In June, Alliance for Regenerative Medicine (ARM) and National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) released Project A-Gene to bring standardized methodology to Chemistry, Manufacturing, and Controls (CMC) programs. A-Gene builds on the work of past efforts to incorporate Quality by Design (QbD) principles in the manufacture of monoclonal antibodies (A-Mab) and vaccines (A-Vax), which were instrumental in advancing manufacturing in these areas. A-Gene focuses on adeno-associated virus (AAV) as the case study. ARM will host a webinar series highlighting specific A-Gene chapters and a half-day virtual workshop in November focusing on the best practices for the gene therapy sector.
Coverage and Reimbursement
MIT NEWDIGS Identifies Emerging Market Solutions for Cell and Gene Therapies
In June, MIT NEWDIGS’ Financing and Reimbursement of Cures in the U.S. (FoCUS) Project published a white paper, “Emerging Market Solutions for Financing and Reimbursement of Durable Cell and Gene Therapies.” The paper discusses findings from a request for information (RFI) survey of companies that offer solutions to address cell and gene therapies' financing and reimbursement challenges. The survey found that there are a diverse array of products and services on the market to help payers, providers, and product developers manage the financial impact of cell and gene therapies. FoCUS grouped these products and services into four categories: payer reinsurance/stop-loss/risk carve out solutions; contract negotiation and data management services for payers and pharmaceutical companies; provider contract negotiation services; and financial and pharmaceutical company warranty services. The paper notes that some of these solutions are still in their infancy as only a few cell and gene therapies have been approved to date. Looking ahead, FoCUS will continue to track the evolution of these financing solutions as viable cell and gene therapies continue to emerge and anticipates developing a toolkit aimed at helping stakeholders to evaluate products and services in this area.