Details
| Original language | English |
|---|---|
| Pages (from-to) | 1421-1437 |
| Number of pages | 17 |
| Journal | European Journal of Health Economics |
| Volume | 21 |
| Issue number | 9 |
| Publication status | Published - 13 Aug 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
- Advanced therapy medicinal products, Budget impact, Cell therapy, Cost-effectiveness, Gene therapy, Health policy, Health Technology Assessment, Innovation, Orphan drugs, Patient access, Pricing and reimbursement, Risk sharing, Value-based pricing
ASJC Scopus subject areas
- Economics, Econometrics and Finance(all)
- Economics, Econometrics and Finance (miscellaneous)
- Medicine(all)
- Health Policy
Sustainable Development Goals
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In: European Journal of Health Economics, Vol. 21, No. 9, 13.08.2020, p. 1421-1437.
Research output: Contribution to journal › Article › Research › peer review
}
TY - JOUR
T1 - HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies
AU - Coyle, Doug
AU - Durand-Zaleski, Isabelle
AU - Farrington, Jasmine
AU - Garrison, Louis
AU - Graf von der Schulenburg, Johann Matthias
AU - Greiner, Wolfgang
AU - Longworth, Louise
AU - Meunier, Aurélie
AU - Moutié, Anne Sophie
AU - Palmer, Stephen
AU - Pemberton-Whiteley, Zack
AU - Ratcliffe, Mark
AU - Shen, Jie
AU - Sproule, Doug
AU - Zhao, Kun
AU - Shah, Koonal
PY - 2020/8/13
Y1 - 2020/8/13
N2 - 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.
AB - 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.
KW - Advanced therapy medicinal products
KW - Budget impact
KW - Cell therapy
KW - Cost-effectiveness
KW - Gene therapy
KW - Health policy
KW - Health Technology Assessment
KW - Innovation
KW - Orphan drugs
KW - Patient access
KW - Pricing and reimbursement
KW - Risk sharing
KW - Value-based pricing
UR - http://www.scopus.com/inward/record.url?scp=85089378574&partnerID=8YFLogxK
U2 - 10.1007/s10198-020-01212-w
DO - 10.1007/s10198-020-01212-w
M3 - Article
C2 - 32794011
AN - SCOPUS:85089378574
VL - 21
SP - 1421
EP - 1437
JO - European Journal of Health Economics
JF - European Journal of Health Economics
SN - 1618-7598
IS - 9
ER -