TY - JOUR

T1 - Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare

AU - Jönsson, Bengt

AU - Hampson, Grace

AU - Michaels, Jonathan

AU - Towse, Adrian

AU - von der Schulenburg, J. Matthias Graf

AU - Wong, Olivier

N1 - Funding information: Conflict of interest All authors report receipt of consultancy fees from Kite Pharma in relation to this work. In addition, Grace Hampson is an employee of the Office of Health Economics, a registered charity, which receives funding from a variety of sources, including the Association of the British Pharmaceutical Industry. Jonathan Michaels has have been in receipt of funding from the NIHR in the UK for ongoing research projects that are unrelated to this paper, and participated in a mock appraisal process for regenerative medicines carried out by the National Institute for Health and Care Excellence (NICE). Adrian Towse is an employee of the Office of Health Economics, a registered charity, which receives funding from a variety of sources, including the Association of the British Pharmaceutical Industry. Olivier Wong is Chief Medical Office of Medi-Qualite Omega SARL and the company receives fees from various life science industries.

PY - 2019/4/1

Y1 - 2019/4/1

N2 - 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.

AB - 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.

KW - Advanced therapy medicinal products

KW - Cell therapy

KW - Gene therapy

KW - Health technology assessment

KW - Regenerative medicine

KW - Value

UR - http://www.scopus.com/inward/record.url?scp=85053672374&partnerID=8YFLogxK

U2 - 10.1007/s10198-018-1007-x

DO - 10.1007/s10198-018-1007-x

M3 - Article

C2 - 30229376

AN - SCOPUS:85053672374

VL - 20

SP - 427

EP - 438

JO - European Journal of Health Economics

JF - European Journal of Health Economics

SN - 1618-7598

IS - 3

ER -