An Inclusive Civil Society Dialogue for Successful Implementation of the EU HTA Regulation: Call to Action to Ensure Appropriate Involvement of Stakeholders and Collaborators

Abstract

Objectives: Stakeholder involvement has long been considered a success factor for a joint European health technology assessment (HTA) process, and its relevance is now anchored in the EU HTA Regulation’s (EU HTAR) legislative wording. Therefore, we aimed to explore the roles, challenges, and most important activities to increase the level of involvement per stakeholder group. Methods: At the 2022 Fall Convention of the European Access Academy (EAA), working groups addressed the involvement of patients, clinicians, regulators, health technology developers (HTD), and national HTA bodies and payers within the EU HTA process. Each working group revisited the pre-convention survey results, determined key role characteristics for each stakeholder, and agreed on the most important activities to fulfill the role profile. Finally, the activities suggested per group were prioritized by plenary group. Results: The prioritized actions for patients included training and capacity building, the establishment of a patient involvement committee, and the establishment of a patient unit at the EC secretariat. For clinicians, it included alignment on evidence assessment from a clinical vs. HTA point of view, capacity building, and standardization of processes. The most important actions for regulators are to develop joint regulatory-HTA guidance documents, align processes and interfaces under the regulation, and share discussions on post-licensing evidence generation. HTDs prioritized scientific advice capacity and the review of the scoping process, and further development of the scope of the assessment report fact checks. The top three actions for national HTA bodies and payers included clarification on the early HTD dialogue process, political support and commitment, and clarification on financial support. Conclusions: Addressing the activities identified as the most important for stakeholders/collaborators in the EU HTA process (e.g., in the implementation of the EU HTA Stakeholder Network and of the guidance documents developed by the EUnetHTA 21 consortium) will be key to starting an “inclusive civil society dialogue”, as suggested by the European Commission’s Pharmaceutical Strategy.

Navigating the path towards successful implementation of the EU HTA Regulation: key takeaways from the 2023 Spring Convention of the European Access Academy

“…Indeed, the European Medicines Agency and EUnetHTA 21 have been engaged in collaborative efforts since 2010, involving the exchange of information and discussions on matters of mutual interest and aiming to align regulatory evaluations and HTA assessments, working towards harmonization in these domains [ 16 – 18 ]. It is, however, important to note that Regulators are not included in Preamble §44 of the EU HTA Regulation and would therefore technically be considered collaborators/contributors rather than stakeholders [ 2 , 5 , 7 ]. Nonetheless, fostering strong collaboration between Regulators and HTA bodies is considered a key success factor for the Regulation, as continuous involvement in the regulatory process plays a crucial role in facilitating an efficient and predictable JCA process for all stakeholders concerned [ 4 – 6 , 19 – 25 ].…”

“…One of the approaches to address the diverging needs of MS is through the PICO survey, aiming to identify each MS PICO requirements and subsequently consolidate them into the minimum number of PICOs necessary to meet the needs of as many MS as possible [ 34 , 35 ]. Numerous concerns have emerged regarding the possibility of MS requesting multiple divergent PICO schemes, which is considered one of the major hurdles for successful implementation of the Regulation [ 4 , 7 , 36 ]. Indeed, a multiplicity of PICOs significantly increases the time and resources required for HTDs to collect, analyse and present the requested data when available, as well as the time and resources for HTA agencies to assess the data for each PICO, leading to a substantial workload which may result in increased complexity rather than a simplification of the HTA process [ 4 , 7 , 36 ].…”

“…Numerous concerns have emerged regarding the possibility of MS requesting multiple divergent PICO schemes, which is considered one of the major hurdles for successful implementation of the Regulation [ 4 , 7 , 36 ]. Indeed, a multiplicity of PICOs significantly increases the time and resources required for HTDs to collect, analyse and present the requested data when available, as well as the time and resources for HTA agencies to assess the data for each PICO, leading to a substantial workload which may result in increased complexity rather than a simplification of the HTA process [ 4 , 7 , 36 ]. The difficulties of aligning PICO elements have been the subject of extensive research and of discussions at previous EAA Conventions in 2022 [ 4 , 5 , 7 , 20 , 21 , 36 ], which primarily focused on the level of agreement of PICO parameters between jurisdictions and the feasibility of managing multiple PICOs.…”

“…Four dedicated WGs with approximately 15–20 participants each were formed in advance, with the following focus topics: Health Policy Challenges ( WG 1, Health Policy ), Stakeholder Readiness ( WG 2, Stakeholders ), Approach to Uncertainty ( WG 3, Uncertainty ), and Challenges regarding Methodology ( WG 4, Methodology ). These themes were chosen on the basis of outcomes of previous EAA conventions and input from the EAA Faculty [ 4 – 7 ]. The goal of each break-out session was to identify and prioritize key challenges and corresponding action points relating to the EU HTA Regulation within its respective area.…”

“…The EAA held its inaugural convention in May 2022 followed by a second convention in October 2022. These events brought together experts from a range of stakeholder groups to accomplish two respective objectives: first, to develop a research agenda aimed at addressing key challenges in implementing the Regulation and the evolving HTA value framework, and second, to gather insights and develop a call to action for optimal stakeholder involvement [ 5 – 7 , 13 ].…”