HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies

Doug Coyle (), Isabelle Durand-Zaleski (), Jasmine Farrington (), Louis Garrison (), Johann Graf von der Schulenburg, Wolfgang Greiner (), Louise Longworth (), Aurélie Meunier (), Anne-Sophie Moutié (), Stephen Palmer (), Zack Pemberton-Whiteley (), Mark Ratcliffe (), Jie Shen (), Doug Sproule (), Kun Zhao () and Koonal Shah ()
Additional contact information
Doug Coyle: University of Ottawa
Isabelle Durand-Zaleski: University of Paris Est
Jasmine Farrington: PHMR Ltd
Louis Garrison: University of Washington
Wolfgang Greiner: Bielefeld University
Louise Longworth: Bielefeld University
Aurélie Meunier: PHMR Ltd
Anne-Sophie Moutié: PHMR Ltd
Stephen Palmer: Center for Health Economics, University of York
Zack Pemberton-Whiteley: Acute Leukemia Advocates Network (ALAN)-Chair
Mark Ratcliffe: PHMR Ltd
Jie Shen: Novartis
Doug Sproule: AveXis
Kun Zhao: China National Health Development Research Center

The European Journal of Health Economics, 2020, vol. 21, issue 9, No 12, 1437 pages

Abstract: Abstract This last decade has been marked by significant advances in the development of cell and gene (C&G) therapies, such as gene targeting or stem cell-based therapies. C&G therapies offer transformative benefits to patients but present a challenge to current health technology decision-making systems because they are typically reviewed when clinical efficacy data are very limited and when there is uncertainty about the long-term durability of outcomes. These challenges are not unique to C&G therapies, but they face more of these barriers, reflecting the need for adapting existing value assessment frameworks. Still, C&G therapies have the potential to be cost-effective even at very high price points. The impact on healthcare budgets will depend on the success rate of pipeline assets and on the extent to which C&G therapies will expand to wider pathologies beyond rare or ultra-rare diseases. Getting pricing and reimbursement models right is important for incentivising research and development investment while not jeopardising the sustainability of healthcare systems. Payers and manufacturers therefore need to acknowledge each other’s constraints—limitations in the evidence generation on the manufacturer side, budget considerations on the payer side—and embrace innovative thinking and approaches to ensure timely delivery of therapies to patients. Several experts in health technology assessment and clinical experts have worked together to produce this publication and identify methodological and policy options to improve the assessment of C&G therapies, and make it happen better, faster and sustainably in the coming years.

Keywords: Cell therapy; Gene therapy; Advanced therapy medicinal products; Health Technology Assessment; Orphan drugs; Pricing and reimbursement; Cost-effectiveness; Value-based pricing; Innovation; Health policy; Budget impact; Patient access; Risk sharing (search for similar items in EconPapers)
Date: 2020
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Citations: View citations in EconPapers (2)

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DOI: 10.1007/s10198-020-01212-w

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