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

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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.

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Notes

  1. All clinical trials of ATMPs conducted between 1999 and June 2015 were searched using three clinical trials databases: ClinicalTrials.gov, the International Clinical Trials Registry Platform (ICTRP) of the World Health Organization (WHO), and EudraCT.

  2. INN = international non-proprietary name.

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

HSCT:

Hematopoietic stem cell transplantation

HST:

Highly specialised technology

HTA:

Health technology assessment

ICER:

Incremental cost-effectiveness ratio

ICER:

Institute for Clinical and Economic Review

ISPOR:

International Society for Pharmacoeconomics and Outcomes Research

KOL:

Key opinion leader

MCDA:

Multi-criteria decision analysis

MUD:

Matched unrelated donor

NICE:

National Institute for Health and Care Excellence

P4P:

Pay-for-Performance

PRO:

Patient-reported outcome

QALY:

Quality-adjusted life year

RCT:

Randomised controlled trial

RWE:

Real-world evidence

R&D:

Research and development

SMA:

Spinal muscular atrophy

SMN1:

Survival motor neuron 1

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Acknowledgements

We sincerely thank Dr Jonathan Michaels (University of Sheffield, UK) for his input and stimulating discussions during the process of writing this publication.

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Authors and Affiliations

  1. University of Ottawa, Ottawa, Canada

    Doug Coyle

  2. University of Paris Est, Champs-Sur-Marne, France

    Isabelle Durand-Zaleski

  3. PHMR Ltd, London, UK

    Jasmine Farrington, Aurélie Meunier, Anne-Sophie Moutié, Mark Ratcliffe & Koonal Shah

  4. University of Washington, Seattle, USA

    Louis Garrison

  5. Center for Health Economics Research, Hannover, Hannover, Germany

    Johann-Matthias Graf von der Schulenburg

  6. Bielefeld University, Bielefeld, Germany

    Wolfgang Greiner & Louise Longworth

  7. Center for Health Economics, University of York, York, UK

    Stephen Palmer

  8. Acute Leukemia Advocates Network (ALAN)-Chair, Bern, Switzerland

    Zack Pemberton-Whiteley

  9. Novartis, Basel, Switzerland

    Jie Shen

  10. AveXis, Chicago, USA

    Doug Sproule

  11. China National Health Development Research Center, Beijing, China

    Kun Zhao

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  1. Doug Coyle
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Correspondence to Anne-Sophie Moutié.

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Coyle, D., Durand-Zaleski, I., Farrington, J. et al. HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies. Eur J Health Econ 21, 1421–1437 (2020). https://doi.org/10.1007/s10198-020-01212-w

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