Patient Engagement and Patient Experience Data in Regulatory Review and Health Technology Assessment: Where Are We Today?

Abstract

Background: As healthcare stakeholders aim to support patient-centered care, patients play an increasingly important role in pharmaceutical and medical technology development and healthcare decision-making. Patient engagement (PE), patient experience data (PED), and meaningful integration of PE to enrich PED have been evolving rapidly. This landscape review focuses on emerging PE/PED practices and guidelines in 2023.

Methods: References published between January-December 2023 on the use of PE and PED from health technology assessment (HTA) and regulatory bodies in different countries, three peer-reviewed journals, and referred resources from collaborators were analyzed. These references were compared with those in our previous publication (August 2021-January 2023, 17-month period).

Results: Overall, 28 references from HTA/regulatory bodies, 26 from peer-reviewed articles, and 17 referred resources were identified. Eight references on PE and PED integration (PE + PED) were identified in 2023 from HTA/regulatory bodies, compared with none in the previous 17-month analysis. Emerging trends on the role of PE, PED, and real-world evidence in HTA/regulatory deliberations, transparency and geographic variations in the use of such evidence and practices, and gaps thereof have been highlighted.

Conclusions: The increase in PE, PED, and PE + PED references worldwide in 2023 versus the prior 17-month analysis suggests accelerated adoption of PE + PED practices. However, a need remains for comprehensive, actionable guidance on best practices for use of PE and PED for harmonization and incorporation into HTA/regulatory processes. Patient input-essential for evidence-based decision-making-provides valuable insights that enhance care quality, treatment relevance and effectiveness, and builds trust and sustainability.

Keywords: Health technology assessment; Patient engagement; Patient experience data; Real-world evidence; Regulatory assessment.

Fig. 1

(A) References containing information on PE, PED, and PE + PED published by HTA and regulatory bodies in 2023 (categorical assignments were not mutually exclusive); and (B) comparison with numbers of references from HTA and regulatory bodies in the previous publication [10] HTA, health technology assessment; PE, patient engagement; PED, patient experience data

Fig. 2

Types of PED in new drug or biologic applications approved by FDA’s Center for Drug Evaluation and Research in 2023^a [87]. ^aAmong 55 new drug applications and biologics license applications approved by the US FDA’s Center for Drug Evaluation and Research in 2023. Orange bars represent the types of clinical outcome assessments (COAs) included with FDA submissions. Individual submissions sometimes included more than one COA. ClinRO, clinician-reported outcomes; FDA, Food and Drug Administration; ObsRO, observer-reported outcomes; PED, patient experience data; PerfO, performance outcome; PFDD, patient-focused drug development; PRO, patient-reported outcomes

Fig. 3

Examples of ongoing international efforts to engage patients and leverage PED in HTAs in 2023. ACE, Agency for Care Effectiveness; CDA, Canada’s Drug Agency; CONITEC, National Committee for Health Technology Incorporation; COS, core outcome set; EUnetHTA, European Network for Health Technology Assessment; HTA, health technology assessment; ICER, Institute for Clinical and Economic Review; PED, patient experience data