New Publication: Advanced Therapy Medicinal Products and Health Technology Assessment

Advanced therapy medicinal products (ATMPs) are beginning to reach European markets, and questions are being asked about their value for patients and how healthcare systems should pay for them.

A new publication in the European Journal of Health Economics, co-authored by OHE’s Grace Hampson and Adrian Towse, alongside Bengt Jönsson (Stockholm School of Economics), Jonathan Michaels (University of Sheffield), J.-Matthias von der Schulenburg (Leibniz University, Hannover), and Olivier Wong (Medi-Qualite Omega), identifies potential challenges of ATMPs in view of current health technology assessment (HTA) methodology and explores potential solutions to these challenges.

Three key topics are discussed:

  1. Uncertainty: Although uncertainty in the evidence is an aspect of all HTA processes, the specific nature of the evidence that is available for newly approved ATMPs is considered likely to require special consideration.
  2. Discounting: The nature of the distribution of costs and benefits was felt likely to make the estimation of cost-effectiveness particularly sensitive to decisions about appropriate discount rates.
  3. Health outcomes and value: The way in which potentially curative treatments may be considered differently to treatments that create smaller incremental benefits for larger populations may raise questions regarding the existing methods of assigning value in HTA.

The authors discuss how evidence challenges linked to increased uncertainty may be mitigated by the collection of follow-on data, the use of value of information analysis, and/or outcomes-based contracts. For discount rates, an international, multi-disciplinary forum should be established to consider the economic, social and ethical implications of the choice of rate. Finally, consideration of the feasibility of assessing the value of ATMPs beyond health gain may also be key to decision-making.

The paper concludes with a list of recommendations put forward to initiate and continue the dialogue around HTA for ATMPs in the context of other published reports.

By following these recommendations, the opportunity exists to improve the HTA methods used for the assessment of ATMPs, which would enable healthcare systems to manage some of the uncertainties presented by early data from these products.

Access the full paper (open access) here.


Jönsson, B., Hampson, G., Michaels, J., Towse, A., von der Schulenburg, J.-M.G. and Wong, O., 2018. Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare. The European Journal of Health Economics. DOI

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Marsden, G. and Towse, A., 2017. Exploring the Assessment and Appraisal of Regenerative Medicines and Cell Therapy Products: Is the NICE Approach Fit for Purpose? OHE Consulting Report. RePEc

Hampson, G., Towse, A., Pearson, S.D., Dreitlein, W.B. and Henshall, C., 2018. Gene therapy: evidence, value and affordability in the US health care system. Journal of Comparative Effectiveness Research, 7(1), pp.15–28. DOI | RePEc

Schaffer, S.K., Messner, D., Mestre-Ferrandiz, J., Tambor, E. and Towse, A., 2018. Paying for Cures: Perspectives on Solutions to the “Affordability Issue”. Value in Health, 21(3), pp.276–279. DOI

Posted in Health Technology Assessment, Pricing and Reimbursement, Innovation | Tagged External publications