Reimbursement and Payment Models for Therapies With Transformative and Curative Intent

Abstract In today’s healthcare environment there is a great need for treatments capable of reversing or significantly impacting the progression of and costs associated with serious illnesses. Regenerative medicines, including cell and gene therapies and other advanced approaches, offer the potential to transform the healthcare landscape through transformative, durable and (in some cases) even potentially curative outcomes targeting many of our highest unmet need scenarios, including life-threatening acute and chronic conditions, injuries, degenerative diseases, genetic disorders, and cancer. These therapies are complex and costly to bring to market, and often require payment commensurate with their transformative value. Given the increasing number of such therapies and eligible patients, global health systems are straining to adjust under designs not intended to accommodate large-scale reimbursement of innovative, high value treatments. Health systems and therapy developers must address the challenges of developing and providing patients access to transformative treatments, including defining what constitutes ‘transformative’ or ‘curative’ in a given scenario in terms of evidence required and assessing value. In this chapter we discuss the challenges faced by transformative therapy developers and global health systems to reimbursement and adoption of these innovative therapies, and emerging solutions to address how to pay for high value treatments.