Shaping a research agenda to ensure a successful European health technology assessment: insights generated during the inaugural convention of the European Access Academy

Abstract

Objectives Key challenges for a joint European Health Technology Assessment (HTA) include consolidated approaches towards the choice of adequate comparator(s), selection of endpoints that are relevant to patients with a given disease, dealing with remaining uncertainties as well as transparent and consistent management of related processes. We aimed to further crystallize related core domains within these four areas that warrant further research and scrutiny. Methods Building on the outcomes of a previously conducted questionnaire survey, four key areas, processes, uncertainty, comparator choice and endpoint selection, were identified. At the inaugural convention of the European Access Academy dedicated working groups were established defining and prioritizing core domains for each of the four areas. The working groups consisted of ~ 10 participants each, representing all relevant stakeholder groups (patients/ clinicians/ regulators/ HTA & payers/ academia/ industry). Story books identifying the work assignments were shared in advance. Two leads and one note taker per working group facilitated the process. All rankings were conducted on an ordinal Likert Response Scale scoring from 1 (low priority) to 7 (high priority). Results Identified key domains include for processes: i) address (resource-) challenge of multiple PICOs (Patient/ Intervention/ Comparator/ Outcomes), ii) time and capacity challenges, iii) integrating all involved stakeholders, iv) conflicts and aligning between different multi-national stakeholders, v) interaction with health technology developer; for uncertainty: i) early and inclusive collaboration, ii) agreement on feasibility of RCT and acceptance of uncertainty, iii) alignment on closing evidence gaps, iv) capacity gaps; for comparator choice: i) criteria for the choice of comparator in an increasingly fragmented treatment landscape, ii) reasonable number of comparators in PICOs, iii) shape Early Advice so that comparator fulfils both regulatory and HTA needs, iv) acceptability of Indirect Treatment Comparisons (ITC), v) ensure broad stakeholder involvement in comparator selection; for endpoint selection: i) approaching new endpoints; ii) patient preferences on endpoints; iii) position of HTA and other stakeholders; iv) long-term generation and secondary use of data; v) endpoint challenges in RCTs. Conclusions The implementation of a joint European HTA assessment is a unique opportunity for a stronger European Health Union. We identified 19 domains related to the four key areas, processes, uncertainty, comparator choice and endpoint selection that urgently need to be addressed for this regulation to become a success.

The role of stakeholder involvement in the evolving EU HTA process: Insights generated through the European Access Academy’s multi-stakeholder pre-convention questionnaire

“…The regulation aims to harmonize methodological standards for HTA and foster collaboration among the European health technology assessment (HTA) bodies [ 1–3 ]. It provides a unique opportunity to consolidate the various national HTA approaches and shape processes and methods to strengthen the European Health Union [ 3 , 4 ]. The harmonized procedure covers the clinical aspects of the assessment of a new technology while responsibility for conclusions on the added value (appraisal) and for decisions on pricing and reimbursement remains within the member states’ remit.…”

“…The harmonized procedure covers the clinical aspects of the assessment of a new technology while responsibility for conclusions on the added value (appraisal) and for decisions on pricing and reimbursement remains within the member states’ remit. Since January 2022, preparatory work has been initiated, including establishing a member states’ coordination group and respective subgroups, establishing a stakeholder network, and drafting guidance documents [ 1 , 3 , 4 ]. The European HTA regulation will be adopted in a stepwise approach.…”

“…A service contract was entered with the European network for Health Technology Assessment (EUnetHTA) 21 joint consortium to support the stepwise implementation of the EU HTA regulation. This joint consortium, led by the ‘Zorginstituut’ (ZIN, The Netherlands), includes 13 European HTA bodies [ 4–6 ]. The EUnetHTA 21 work agenda covers various deliverables, including developing methodological and process guidance and conducting joint clinical assessments (JCA) and joint scientific consultations (JSC) [ 4 , 7 ].…”

“…This joint consortium, led by the ‘Zorginstituut’ (ZIN, The Netherlands), includes 13 European HTA bodies [ 4–6 ]. The EUnetHTA 21 work agenda covers various deliverables, including developing methodological and process guidance and conducting joint clinical assessments (JCA) and joint scientific consultations (JSC) [ 4 , 7 ].…”

“…Many challenges remain for the implementation of the EU HTA regulation and might represent a hurdle to the Commission’s medium-term goal of improving patient access to innovative medicines [ 2 , 4 ]. For the joint European HTA to provide an ‘additional benefit’ over the multitude of currently existing national assessments and to achieve a balanced, improved outcome for all stakeholders, key methodological and process issues need to be addressed [ 4 , 9 ]. During the inaugural convention of the EAA in May 2022, experts from all relevant stakeholder groups developed a research agenda focusing on how to approach these challenges [ 4 , 10 ].…”