The challenges in reimbursement for cell and gene therapies

Summary

In an insightful discussion, Kristen Wolfe, VP of Global Policy Strategy at Bluebird Bio, addresses the challenges and innovations in the cell and gene therapy landscape. She emphasizes the need for adaptable reimbursement models that reflect the unique nature of one-time, potentially curative therapies. Highlighting recent advancements in U.S. policy, particularly the CMS's value-based payment framework, Wolfe expresses optimism about progress in payer strategies. She underscores the importance of operational ease in implementing these models to enhance patient access. Kristen will further explore these critical issues at the upcoming Cell and Gene Therapy Bioprocessing and Commercialization Meeting.

Takeaways

• 😀 Kristen Wolfe is the VP of Global Policy Strategy and U.S. Government Payer at Bluebird Bio, focusing on the intersection of policy and reimbursement for cell and gene therapies.
• 🤔 The primary challenge in the gene therapy landscape is the shift in paradigms, as these therapies often offer one-time administration with potential curative effects, differing from traditional chronic disease therapies.
• 💡 Wolfe emphasizes the need for flexibility in healthcare systems to accommodate the unique characteristics of gene therapies during the reimbursement process.
• 🔄 There is a significant tension between macro-level policy decisions and local budget constraints, impacting how gene therapies are valued and reimbursed.
• 🛠️ A critical aspect of Wolfe's role includes coordinating global policy strategies while leading negotiations with U.S. government payers like Medicare and Medicaid.
• 📈 Recent changes in the U.S. reimbursement landscape, such as the CMS final rule for value-based payment for drugs, mark important progress towards improving access to gene therapies.
• 🔍 The Medicaid best price rules have historically posed barriers to innovative payment models, but recent developments indicate a willingness to explore creative solutions.
• 🗣️ Wolfe stresses the importance of operational ease in implementing new reimbursement models, as complex processes can hinder patient access to therapies.
• 🚀 Despite challenges, Wolfe notes that significant progress is being made in how payers across Medicare, Medicaid, and commercial sectors approach gene therapies.
• 🤝 The upcoming meeting will feature discussions on these topics, reflecting a growing interest in finding sustainable solutions for gene therapy access.

Q & A

What is Kristen Wolfe's current role at Bluebird Bio?

Kristen Wolfe is the VP of Global Policy Strategy and U.S. Government Payer at Bluebird Bio, where she focuses on bridging policy and reimbursement for cell and gene therapies.

What is the main challenge in the reimbursement landscape for cell and gene therapies?

The main challenge is the newness of these therapies and their one-time administration model, which contrasts with traditional therapies designed for chronic diseases.

How does Wolfe describe the historical context of therapy evaluation?

Wolfe notes that the evaluation standards for therapies have been built around chronic disease treatments over the past several decades, which complicates the assessment of new, potentially curative therapies.

What does Wolfe mean by the 'block and tackle' of negotiations?

The 'block and tackle' refers to the fundamental aspects of negotiating for approved therapies with U.S. government payers like Medicare and Medicaid.

What key changes are happening in the U.S. regarding gene therapy reimbursement?

There have been significant evolutions in how various payers approach reimbursement for gene therapies, including the introduction of more creative payment models.

What recent development did Wolfe highlight regarding CMS and gene therapies?

Wolfe highlighted the CMS final rule for value-based payment for drugs, which aims to remove barriers to creative payment models for gene therapies.

How does Wolfe see the role of operational ease in the reimbursement process?

Operational ease is crucial, as even if regulatory and legal challenges are resolved, complex operational processes can still pose significant barriers to access for patients.

What perspective does Wolfe suggest is necessary for evaluating new therapies?

Wolfe suggests a reorientation of thinking from policymakers and payers, emphasizing the need to recognize the long-term benefits of curative therapies rather than just immediate costs.

What is the significance of the upcoming panel that Wolfe will be part of?

The panel will discuss critical topics around value, regulatory considerations, and access for novel therapies, providing insights into the current challenges and developments in the field.

What overall sentiment does Wolfe express about the future of gene therapy access in the U.S.?

Wolfe expresses cautious optimism, noting that while progress is being made, there is still a need to accelerate efforts to ensure timely access to these therapies for patients.