Abstract
BackgroundAdvanced 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.ObjectivesTo 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.MethodsAn Expert Panel reviewed current HTA principles and practices in relation to the specific characteristics of ATMPs.ResultsThree 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.ConclusionsATMPs 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 codeI.
Highlights
The European Medicines Agency’s (EMA) definition of advanced therapy medicinal products (ATMPs) is: “medicines for human use that are based on genes or cells ... [and] offer groundbreaking new opportunities for the treatment of disease and injury” [1]
The approach takes a broad view of health technology assessment (HTA) methodology, including the assessment of cost-effectiveness and individual and social value, as well as issues related to implementation and pricing and payment models for Advanced therapy medicinal products (ATMPs) in different healthcare systems
Prioritisation was based on the existence of methodological uncertainties that are likely to be relevant to the evaluation of ATMPs
Summary
The European Medicines Agency’s (EMA) definition of advanced therapy medicinal products (ATMPs) is: “medicines for human use that are based on genes or cells ... [and] offer groundbreaking new opportunities for the treatment of disease and injury” [1]. Health technology assessment (HTA) is a “multidisciplinary activity that systematically examines the technical performance, safety, clinical efficacy and effectiveness, cost, cost-effectiveness, organisational implications, social consequences, legal and ethical considerations” [3] of a health technology It is a form of policy research with specific methodology that examines the short- and long-term consequences of the application of a healthcare technology to inform policy decision-making and enable rational decisions to be made for healthcare resource allocation [4]. Drummond et al proposed a set of 15 principles that can be used in assessing existing or establishing new HTA activities [5] These principles describe and discuss elements of good practice in developing the structure and remit of HTA organizations, the methods of and processes for conducting HTA, and the use of HTA in decision-making [5]. The approach takes a broad view of HTA methodology, including the assessment of cost-effectiveness and individual and social value, as well as issues related to implementation and pricing and payment models for ATMPs in different healthcare systems
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