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 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

doi:10.1007/s10198-020-01212-w

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 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

^*Corresponding author for this work

Centre for Health Economics

Research output: Contribution to journal › Article › peer-review

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.

Original language	English
Pages (from-to)	1421-1437
Number of pages	17
Journal	European Journal of Health Economics
Volume	21
Issue number	9
Early online date	13 Aug 2020
DOIs	https://doi.org/10.1007/s10198-020-01212-w
Publication status	Published - 1 Dec 2020

Bibliographical note

Publisher Copyright:
© 2020, Springer-Verlag GmbH Germany, part of Springer Nature.

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

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10.1007/s10198-020-01212-w

Cite this

Coyle, D., Durand-Zaleski, I., Farrington, J., Garrison, L., Graf von der Schulenburg, J. M., Greiner, W., Longworth, L., Meunier, A., Moutié, A. S., Palmer, S., Pemberton-Whiteley, Z., Ratcliffe, M., Shen, J., Sproule, D., Zhao, K., & Shah, K. (2020). HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies. European Journal of Health Economics, 21(9), 1421-1437. https://doi.org/10.1007/s10198-020-01212-w

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title = "HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies",

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{\textquoteright}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.",

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note = "Publisher Copyright: {\textcopyright} 2020, Springer-Verlag GmbH Germany, part of Springer Nature.",

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doi = "10.1007/s10198-020-01212-w",

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Coyle, D, Durand-Zaleski, I, Farrington, J, Garrison, L, Graf von der Schulenburg, JM, Greiner, W, Longworth, L, Meunier, A, Moutié, AS, Palmer, S, Pemberton-Whiteley, Z, Ratcliffe, M, Shen, J, Sproule, D, Zhao, K & Shah, K 2020, 'HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies', European Journal of Health Economics, vol. 21, no. 9, pp. 1421-1437. https://doi.org/10.1007/s10198-020-01212-w

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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

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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.

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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

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JO - European Journal of Health Economics

JF - European Journal of Health Economics

IS - 9

ER -

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

Abstract

Bibliographical note

Keywords

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