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Developing a Framework for the Health Technology Assessment of Histology-independent Precision Oncology Therapies

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Abstract

The arrival of precision oncology is challenging the evidence standards under which technologies are evaluated for regulatory approval as well as for health technology assessment (HTA) purposes. Several key concepts are discussed to highlight the source of the challenges in evaluating these products, particularly those impacting the HTA of histology-independent therapies. These include the basket trial design, high uncertainty in (potentially substantial) benefits for histology-independent therapies, and the inability to identify and quantify benefits of standard of care in daily practice when the biomarker is not currently used in practice. There is little precedent for a technology with the unique mixture of challenges for HTA of histology-independent therapies and they will be evaluated using standard HTA, as there currently is no evidence suggesting the standard HTA framework is not appropriate. A number of questions proposed to help guide HTA bodies when assessing the appropriateness of local processes to optimally evaluate histology-independent therapies. Pragmatic solutions are further proposed to decrease uncertainty in the benefits of histology independent therapies as well as fill gaps in comparative evidence. The proposed solutions ensure a consistent and streamlined approach to evaluation across histology-independent products, although with varying strengths and limitations. Alongside these solutions, sponsors should engage early with HTA bodies/payers and regulatory agencies through parallel/joint scientific advice to facilitate the integration of both regulatory and HTA perspectives into one clinical development programme, potentially reconciling evidence requirements.

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Acknowledgements

The authors would like to thank Adam Lloyd, Beth Wehler, Qianyi Li and Peter Rouse for their contribution to this manuscript.

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Authors and Affiliations

  1. IQVIA Ltd., 210 Pentonville Rd, London, N1 9JY, UK

    Jennifer G. Gaultney

  2. National Institute for Health and Care Excellence, London, UK

    Jacoline C. Bouvy

  3. Institute for Clinical and Economic Review, Boston, MA, USA

    Richard H. Chapman

  4. Bayer Healthcare Pharmaceuticals Inc, Whippany, NJ, USA

    Alexander J. Upton

  5. IQVIA, Durham, NC, USA

    Stacey Kowal

  6. University Medical Center, Hamburg-Eppendorf, Germany

    Carsten Bokemeyer

  7. Health Innovation Technology Transfer Foundation, Barcelona, Spain

    Oriol Solà-Morales

  8. Center for Integrated Oncology, University Hospital Cologne, Cologne, Germany

    Jürgen Wolf

  9. London School of Hygiene and Tropical Medicine, London, UK

    Andrew H. Briggs

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  1. Jennifer G. Gaultney
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  2. Jacoline C. Bouvy
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  3. Richard H. Chapman
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Corresponding author

Correspondence to Jennifer G. Gaultney.

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Funding and conflict of interest

JG and SK received funding for medical writing assistance from Bayer. JC has no funding sources or conflicts of interest to declare. RC has received research grants from Blue Shield of California Foundation, California Healthcare Foundation, Laura and John Arnold Foundation, and NE States Consortium Systems Organization, outside the submitted work; other support received from Aetna, AHIP, Allergan, Alnylam, Anthem, AstraZeneca, Biogen, Blue Shield of California, Boehringer-Ingelheim, Cambia Health Services, CVS, Editas, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim Health Care, Health Care Service Corporation, Health Partners, Johnson & Johnson (Janssen), Kaiser Permanente, LEO, Mallinckrodt, Merck, National Pharmaceutical Council, Novartis, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, and United Healthcare, as dues for annual Policy Summit meeting. AU is an employee of Bayer. CB has received speaking honoraria from Merck KGaA, Sanofi, Roche, Bayer, BMS, AstraZeneca and MSD. CB has received consulting fees from, Lilly, Merck Serono, Sanofi, Bayer, MSD, GSO and AOK Health Insurance. CB has further received research funding from Abbvie, ADC Therapeutics, Agile Therapeutics, Alexion Pharmaceuticals, Amgen, Apellis Pharmaceuticals, AstraZeneca, Bayer, BarGen Bio, Blueprint Medicines, BMS, Boehringer Ingelheim, Celgene, Daiichi Sankyo, Eisai, Gilead, Glycotope, GSK, Incyte, IO Biotech, Isofol Medical, Janssen, Karyopharm Therapeutics, Lilly, Millennium, MSD, Nektar, Novartis, Rafael Pharmaceuticals, Roche, Springworks Therapeutics, and Taiho Pharmaceutical. CB has received travel grants from Merck Serono, Sanofi, Pfizer, and BMS. JW has received advisory board and lecture fees from Amgen, AstraZeneca, Bayer, Blueprint, BMS, Boehringer-Ingelheim, Chugai, Daiichi Sankyo, Ignyta, Janssen, Lilly, Loxo, MSD, Novartis, Pfizer, Roche, Seattle Genetics, and Takeda. JW has received research grants from BMS, Janssen, Novartis, and Pfizer. OSM and AB have received consulting fees, travel grants and speaking honoraria from Bayer.

Author contributions

All authors were involved in the manuscript conception. JG and SK drafted the article. JB, RC, AU, CB, OSM, JW and AB provided critical revision of the article. All authors provided final approval of the version to be published.

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Gaultney, J.G., Bouvy, J.C., Chapman, R.H. et al. Developing a Framework for the Health Technology Assessment of Histology-independent Precision Oncology Therapies. Appl Health Econ Health Policy 19, 625–634 (2021). https://doi.org/10.1007/s40258-021-00654-4

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  • DOI: https://doi.org/10.1007/s40258-021-00654-4

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