Exploring the Truth of Reimbursement Challenges for Cell and Gene Therapies

Trinity has worked with over a third of companies with cell and gene therapies (CGTs) on market or in Phase 3 clinical trials. Our work has supported business development, launch planning, evidence generation (HEOR, etc.), market access, pricing, and other critical commercial activities to support the development and launch of these therapies. As such, we have seen CGTs morph from a small niche into possibly the most exciting market in pharma. 2019 is poised to be an inflection point for these transformational therapies, changing the way we care for patients and disrupting the pharmaceutical & biotech industry. Despite this potential, however, there remains a lot of misconceptions about the unique challenges facing CGTs in terms of reimbursement, notably:

• Payment requirements (i.e., large lump sum costs, Medicaid best price)
• Coding requirements (i.e., to ensure that providers are adequately reimbursed)
• High price requirements and payer stakeholder pushback
• Patient affordability (e.g., given the potential high out of pocket exposure for patients)
• Portability and outcome tracking requirements (i.e., to address patient turnover across plans)

In our experience, most of these issues, while important, are manageable by US (and to some extent EU and Japan) payers, and payers are welcoming of these new paradigms in patient treatment. However, as the CGT market is poised to dramatically expand in the coming years, both in terms of the number of products and the patient population volume they target, it is not clear how sustainable the current reimbursement model is and what changes may take place or need to take place. To unravel this, we conducted both primary payer research and economic analyses. This paper provides an overview of our findings and their implications.