Patient and citizen participation at the organizational level in health technology assessment: an exploratory study in five jurisdictions

Abstract

Objective While patient participation in individual health technology assessments (HTAs) has been frequently described in the literature, patient and citizen participation at the organizational level is less described and may be less understood and practiced in HTA bodies. We aimed to better understand its use by describing current practice. Method To elicit descriptive case studies and insights we conducted semi-structured interviews and open-ended questionnaires with HTA body staff and patients and citizens participating at the organizational level in Belgium, France, Quebec, Scotland, and Wales. Results We identified examples of organizational participation in managerial aspects: governance, defining patient involvement processes, evaluation processes and methods, and capacity building. Mechanisms included consultation, collaboration, and membership of standing (permanent) groups. These were sometimes combined. Participants were usually from umbrella patient organizations and patient associations, as well as individual patients and citizens. Discussion Although the concept, participation at the organizational level, is not well-established, we observed a trend toward growth in each jurisdiction. Some goals were shared for this participation, but HTA bodies focused more on instrumental goals, especially improving participation in HTAs, while patients and citizens were more likely to offer democratic and developmental goals beyond improving participation processes. Conclusion Our findings provide rationales for organizational-level participation from the perspectives of HTA bodies and patients. The case studies provide insights into how to involve participants and who may be seen as legitimate participants. These findings may be useful to HTA bodies, the patient sector, and communities when devising an organizational-level participation framework.

The European Voice of the Patient living with pulmonary hypertension associated with interstitial lung disease: Diagnosis, symptoms, impacts, and treatments

“…11 The European Medicines Agency (EMA) supports the collection and use of evidence‐based PED for benefit‐risk decision‐making, 12 and the interest of payers and health technology assessment (HTA) bodies is equally increasing. 13 …”

“…11 The European Medicines Agency (EMA) supports the collection and use of evidence-based PED for benefit-risk decisionmaking, 12 and the interest of payers and health technology assessment (HTA) bodies is equally increasing. 13 In Idiopathic Pulmonary Fibrosis (IPF), patients report that the aspects most important to them are not routinely captured by clinical studies, despite trial protocols including PRO measures such as the St George's Respiratory Questionnaire, Living with Pulmonary Fibrosis, Short Form 36, and the EQ-5D. [14][15][16] A lack of specificity and validity results in studies often not detecting a clinically meaningful PRO-score difference in response to therapy, supporting the need for further research on the health-related quality of life (HRQoL) impacts in PH-ILD.…”

“…In its Patient‐Focused Drug Development (PFDD) initiative, the US Food and Drug Administration (FDA) defines PED as quantitative data generated with clinical outcome assessments (COAs), including patient‐reported outcomes (PRO), as well as qualitative and patient preference research 11 . The European Medicines Agency (EMA) supports the collection and use of evidence‐based PED for benefit‐risk decision‐making, 12 and the interest of payers and health technology assessment (HTA) bodies is equally increasing 13 …”