Stakeholders in the drug life cycle recognize the importance of integrating patient experience data (PED) into healthcare decision-making. PED includes patient input, patient reported outcomes (PROs), patient reported experiences, and patient preference information. However, it remains unclear if, how, and to what extent PED are used in reimbursement decisions in Belgium.

A document analysis was performed to evaluate and compare the reporting of PED in Belgian reimbursement documents for COVID-19, oncology, and cardiology. Documents of medicinal products that received a European marketing authorization between 1 January 2015 and 30 September 2023 and were reimbursed in Belgium were included. Data were analyzed descriptively and qualitatively.

Preliminary results showed that PED was generally either not reported or not explicitly reported in Belgian reimbursement documents. In some documents, patient reported outcome measures (PROMs) were mentioned. Additionally, few documents stated PROs such as quality of life. PROMs, PROs, and quality of life outcomes appeared in the reimbursement dossier section describing the therapeutic value of the product. From the preliminary findings it seems that PED were more often present in oncology documents. The reimbursement dossiers did not report whether or how PED were used to inform a particular reimbursement decision.

Preliminary findings suggest that PED are not systematically included in Belgian reimbursement documents. To improve transparent submission and reporting of PED in reimbursement dossiers, guidance from reimbursement agencies as well as checklists and evaluation templates are needed.

Patient experience data (PED), encompassing patient preferences (PP), patient-reported outcomes (PROs), and patient input, play a pivotal role in understanding patient needs and informing healthcare decision-making, including reimbursement decisions. This study aimed to assess the current barriers hindering the integration of PED into practice and its particular challenges, opportunities, and concrete policy actions for the systematic implementation of PED.

Semistructured interviews (n=38) were conducted with industry (n=12), non-profit organizations and academia (n=4), regulatory authorities (n=6), health technology assessment (HTA) bodies and reimbursement agencies (n=6), and patient organizations (n=10) in Europe. A thematic analysis was conducted to explore stakeholders’ perspectives and to gain a comprehensive understanding of challenges and opportunities related to the systematic implementation of PED. Interview transcripts were analyzed using the thematic framework analysis to extract and elucidate the insights provided by the diverse stakeholders.

HTA and reimbursement interviewees agreed on the value of including quality-of-life data, particularly when assessed using validated PRO measures. Despite acknowledging the potential of PP, there remained reluctance to integrate PP into reimbursement decision-making. Participants expressed divergent opinions regarding who should collect PED, with some regulators favoring industry, while HTA and reimbursement agencies emphasized transparency and independent PED collection. Limited experience in assessing PED also contributed to hesitancy, underscoring the need for more guidelines, especially at the national reimbursement level. Stakeholders endorsed collaboration through joint scientific consultations, expressing optimism about the impact of the Regulation (EU) 2021/2282 on health technology assessment (HTAR).

This study emphasizes the high potential of PED in informing reimbursement decision-making, fostering a more patient-centered approach. Stakeholder disparities highlight the complexity, necessitating more guidance, scientific robustness, transparency, and collaboration. In light of these stakeholder considerations, the upcoming HTAR holds promise to enhance the systematic implementation of PED, aligning healthcare decision-making with patients’ needs and preferences.

Uncertainty is a fundamental component of decision making regarding access to and pricing and reimbursement of drugs. The context-specific interpretation and mitigation of uncertainty remain major challenges for decision makers. Following the 2021 HTAi Global Policy Forum, a cross-sectoral, interdisciplinary HTAi-DIA Working Group (WG) was initiated to develop guidance to support stakeholder deliberation on the systematic identification and mitigation of uncertainties in the regulatory-HTA interface.

Six online discussions among WG members (Dec 2021–Sep 2022) who examined the output of a scoping review, two literature-based case studies and a survey; application of the initial guidance to a real-world case study; and two international conference panel discussions.

The WG identified key concepts, clustered into twelve building blocks that were collectively perceived to define uncertainty: “unavailable,” “inaccurate,” “conflicting,” “not understandable,” “random variation,” “information,” “prediction,” “impact,” “risk,” “relevance,” “context,” and “judgment.” These were converted into a checklist to explain and define whether any issue constitutes a decision-relevant uncertainty. A taxonomy of domains in which uncertainty may exist within the regulatory-HTA interface was developed to facilitate categorization. The real-world case study was used to demonstrate how the guidance may facilitate deliberation between stakeholders and where additional guidance development may be needed.

The systematic approach taken for the identification of uncertainties in this guidance has the potential to facilitate understanding of uncertainty and its management across different stakeholders involved in drug development and evaluation. This can improve consistency and transparency throughout decision processes. To further support uncertainty management, linkage to suitable mitigation strategies is necessary.