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

Figures & data

Figure 1. A five-step procedure utilizing a modified Delphi approach was applied to develop and execute the survey.

Figure 2. The final questionnaire was developed in two distinct versions: version 1 for self-rating by the ‘key’ stakeholder groups (patients’, clinicians’, regulatory and HTD representatives) and version 2 reflecting the external perception of those 4 ‘key’ stakeholder groups by HTA body representatives, payers, and policy makers. Any other stakeholders that participated in the survey were also provided with version 2.

Figure 3. a-b: Background information on submitted questionnaire responses. (a: Number of respondents per stakeholder/collaborator group; b: Number of respondents per country or region, where provided (9 respondents did not provide a country)).

Figure 4. a-d: Box Plots (Mean [x]; Medium; Max; Min; Upper and Lower Quartile) of stakeholder involvement self-rating versus external perception of respective stakeholder/collaborator involvement as rated by HTA bodies; payers; and health policy makers. Scale ranging from 1 to 5, per stakeholder group (a: patients’ representatives; b: clinicians’ representatives; c: regulatory representatives; d: HTD representatives).

Table 1. Key Insights generated within the qualitative part of the questionnaire regarding the content and key challenges for the involvement of the four ‘key’ stakeholder groups.

Focus of Involvement	Patients	Clinicians	Regulatory	HTDs
Content	Definition of unmet need Relevance of innovation for patients Definition of patient relevant endpoints Assessment of patient reported outcomes Acceptability of adverse events	Definition of PICO Scheme i.e., definition of patient population; comparator choice; clinical trial endpoints etc. Input on applied methodology Application of standards of ‘evidence-based medicine’ Involvement in early multistakeholder dialogues on HTA processes (with patients, regulators, and HTDs)	Approach to unmet medical need Early determination of evidence requirements for both benefit-risk evaluation and comparative benefit Joint scientific consultations including feedback on key elements of PICO scheme and study design that reflect both regulatory and HTA requirements	Design of PICO scheme i.e., definition of patient population; comparator choice; clinical trial endpoints etc. Design of applicable comparative evidence package
Key Challenges	Lack of standardized procedure for involvementLack of capacities & resourcesAlignment of EU umbrella organizations with national organizationsManagement of conflict of interestNo consistent recognition of the value of patient input	Lack of standardized procedure for involvement Requirement of additional resources Alignment of EU umbrella organizations with national organizations e.g., regarding guidelines Management of conflict of interest Heterogeneity in market access and clinical standards across Europe (e.g., eastern vs. Western Europe)	Lack of standardized procedure for involvement Timing of EU HTA Process and EMA regulatory process will overlap and might incur time conflicts Tailoring of EMA documents (e.g., EPAR) for utilization in the subsequent HTA processes	Lack of standardized procedure for involvement Time constraints of EU HTA process Limited opportunities of direct involvement before (e.g., in joint consultations) or after submission of a dossier Concern that EU HTA might result in many diverging PICO schemes

Table 2. RACI (Responsible/Accountable/Consulted/Informed) Chart for the role definitions of the relevant stakeholder/collaborator groups for the PICO (Population/Intervention/Comparator/Outcomes) categories in the EU HTA procedure as defined in the EU HTA Regulation.

	Patients	Clinicians	Regulators^§	HTDs	EU – HTA
Population	C	C (important)	C (optional)	I (evt C)	R*
Intervention	I (optional)	I	n/a	A/R	I
Comparator	C	C (important)	C (optional)	I (evt C)	R*
Outcomes	C (Important)	C (important)	C (optional)	I (evt C)	R*

Note: *: HTA bodies are consulted (C) during the development phase; accountability (A) resides with national HTA bodies. §: For the regulatory approval process, regulators are A/R throughout the process which is creating the frame for EU HTA to operate. evt: eventually.