HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies
- PDF / 806,317 Bytes
- 17 Pages / 595.276 x 790.866 pts Page_size
- 111 Downloads / 252 Views
ORIGINAL PAPER
HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies Doug Coyle1 · Isabelle Durand‑Zaleski2 · Jasmine Farrington3 · Louis Garrison4 · Johann‑Matthias Graf von der Schulenburg5 · Wolfgang Greiner6 · Louise Longworth6 · Aurélie Meunier3 · Anne‑Sophie Moutié3 · Stephen Palmer7 · Zack Pemberton‑Whiteley8 · Mark Ratcliffe3 · Jie Shen9 · Doug Sproule10 · Kun Zhao11 · Koonal Shah3 Received: 7 November 2019 / Accepted: 18 June 2020 © Springer-Verlag GmbH Germany, part of Springer Nature 2020
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 Abbreviations ACEA Augmented cost-effectiveness analysis ATMP Advanced therapy medicinal product ADA-SCID Adenosine deaminase deficiency-severe combined immunodeficiency CADTH Canadian Agency for Drugs and Technologies in Health C&G Cell and gene CEA Cost-effectiveness analysis EMA European Medicine Agency EU European Union FDA Food & Drug Administration * Anne‑Sophie Moutié Anne‑[email protected] Extended author information available on the last page of the article
HSCT Hematopoietic stem cell transplantation HST Highly specialised technology HTA Health technology assessmen
Data Loading...
