How can a joint European health technology assessment provide an ‘additional benefit’ over the current standard of national assessments?

Abstract

Objectives We conducted a multi-stakeholder survey to determine key areas where a joint European health technology assessment (HTA) could provide ‘additional benefit’ compared to the status quo of many parallel independent national and subnational assessments. Methods Leveraging three iterative Delphi cycles, a semiquantitative questionnaire was developed covering evidence challenges and heterogeneity of value drivers within HTAs across Europe with a focus on hematology/oncology. The questionnaire consisted of five sections: i) background information; ii) value drivers in HTA assessments today; iii) evolving evidence challenges; iv) heterogeneity of value drivers across Europe; v) impact of Europe’s Beating Cancer Plan (EBCP). The questionnaire was circulated across n = 189 stakeholder institutions comprising HTA and regulatory bodies, clinical oncology associations, patient representatives, and industry associations. Results N = 30 responses were received (HTA bodies: 9; regulators: 10; patients’ and physicians’ associations: 3 each; industry: 5). Overall, 17 countries and EU level institutions were represented in the responses. Consistency across countries and stakeholder groups was high. Most relevant value drivers in HTAs today (scale 1, low to 5, high) were clinical trial design (mean 4.45), right endpoints (mean 4.40), and size of comparative effect (mean 4.33). Small patient numbers (mean 4.28) and innovative study designs (mean 4.1) were considered the most relevant evolving evidence challenges. Heterogeneity between regulatory and HTA evidence requirements and heterogeneity of the various national treatment standards and national HTA evidence requirements was high. All clinical and patient participants stated to have been with EBCP initiatives. Conclusions For a European HTA to provide an ‘additional benefit’ over the multitude of existing national assessments key methodological and process challenges need to be addressed. These include approaches to address uncertainty in clinical development; comparator choice; consistency in approaching patient-relevant endpoints; and a transparent and consistent management of both HTA and regulatory procedures as well as their interface, including all involved stakeholder groups.

Umgang mit best-verfügbarer Evidenz in der Nutzenbewertung von Arzneimitteln: Eine Analyse von Verfahren ohne Vorliegen einer randomisiert-kontrollierten Studie

“…In all 42 procedures, the pharmaceutical company presented at least one single-arm study (SAT). Among these, ≥ Konsequenterweise wurde der Umgang mit der aus diesen aktuellen Trends resultierenden wissenschaftlich-statistischen Unsicherheit als eine zentrale Herausforderung nicht nur bei der Zulassung von Arzneimitteln, sondern auch bei aktuellen und künftigen HTA-Verfahren identifiziert [12]. Eine wesentliche Rolle kommt hierbei dem Konzept der 'Best Available Evidence' zu.…”

Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments

“…A major aim of the strategy is to enable parallel scientific advice on clinical study design for medicines by HTA bodies and the EMA. This would enhance the harmonization of evidence-based methodology across European countries and government authorities, including translating surrogate endpoints to patient-centric metrics [ 93 , 94 ]. Such an approach can improve consistency in the methodologies, especially when dealing with HTA in the context of limited efficacy-safety data.…”

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

“…In December 2021, the European Regulation on Health Technology Assessment (EU HTA R (EU)2021/2282), a fundamental pillar of the EU Pharmaceutical Strategy, was adopted by the Council and the European Parliament and is effective as of January 2022. 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. 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.…”

“…The EAA’s aim is to facilitate the implementation of the EU HTA R through the development of a joint European value framework for the assessment of innovative health technologies. Thereby, the EAA supports the regulation’s vision of achieving high-quality joint clinical assessments, inclusiveness, and transparency in the HTA process and predictability both for authorities and industry with the ultimate goal of facilitating patient access to innovative medicines and some medical devices at EU level [ 1 , 8 ].…”