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

authored by

Doug Coyle, Isabelle Durand-Zaleski, Jasmine Farrington, Louis Garrison, Johann Matthias 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, Koonal Shah

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.

Organisation(s)

Center for Health Economics Research Hannover (CHERH)

External Organisation(s)

University of Ottawa
Paris-Est Sup
PHMR Ltd
University of Washington
Bielefeld University
Univ. York, Dep. Comput. Sci., Non-Stand. Comput. Group
Acute Leukemia Advocates Network (ALAN)
Novartis AG
AveXis
China National Health Development Research Center (NHDRC)

Type

Article

Journal

European Journal of Health Economics

Volume

21

Pages

1421-1437

No. of pages

17

ISSN

1618-7598

Publication date

13.08.2020

Publication status

Published

Peer reviewed

Yes

ASJC Scopus subject areas

Economics, Econometrics and Finance (miscellaneous), Health Policy

Sustainable Development Goals

SDG 3 - Good Health and Well-being

Electronic version(s)

https://doi.org/10.1007/s10198-020-01212-w (Access: Closed)