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The European Journal of Health Economics
Published in: The European Journal of Health Economics 3/2019

Open Access 01-04-2019 | Original Paper

Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare

Authors: Bengt Jönsson, Grace Hampson, Jonathan Michaels, Adrian Towse, J.-Matthias Graf von der Schulenburg, Olivier Wong

Published in: The European Journal of Health Economics | Issue 3/2019

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Abstract

Background

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.

Objectives

To identify and discuss potential challenges of ATMPs in view of current health technology assessment (HTA) methodology—specifically economic evaluation methods—in Europe as it relates to ATMPs, and to suggest potential solutions to these challenges.

Methods

An Expert Panel reviewed current HTA principles and practices in relation to the specific characteristics of ATMPs.

Results

Three key topics were identified and prioritised for discussion—uncertainty, discounting, and health outcomes and value. The panel discussed that evidence challenges linked to increased uncertainty may be mitigated by collection of follow-on data, 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 for decision-making.

Conclusions

ATMPs face a challenge in demonstrating their value within current HTA frameworks. Consideration of current HTA principles and practices with regards to the specific characteristics of ATMPs and continued dialogue will be key to ensuring appropriate market access.

Classification code

I.
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Metadata
Title
Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare
Authors
Bengt Jönsson
Grace Hampson
Jonathan Michaels
Adrian Towse
J.-Matthias Graf von der Schulenburg
Olivier Wong
Publication date
01-04-2019
Publisher
Springer Berlin Heidelberg
Published in
The European Journal of Health Economics / Issue 3/2019
Print ISSN: 1618-7598
Electronic ISSN: 1618-7601
DOI
https://doi.org/10.1007/s10198-018-1007-x

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