Patient engagement (PE) and patient experience data (PED) are increasingly valued but lack consistent integration and visibility in regulatory and HTA decision making.
This CIRS multi-stakeholder workshop explored practical steps for embedding meaningful PE and PED in regulatory and HTA processes.
Background
Patient engagement (PE) and the use of patient experience data (PED) are now recognised as essential elements in the development, regulation, and health technology assessment (HTA) of new medicines, helping ensure decisions reflect patient needs and priorities.
Building on earlier efforts — including the 2015 Centre for Innovation in Regulatory Science (CIRS) workshop and work by the Clinical Trials Transformation Initiative (CTTI), Patients Active in Research and Dialogues for an Improved Generation of Medicines (PARADIGM), and Patient-Focused Medicines Development (PFMD) — CIRS formed a Topic Group in 2022 of various stakeholders to suggest research activities in this area for the CIRS 2024-2026 Research Agenda. This culminated in a multi-stakeholder workshop held in the UK on 1–2 October 2025, which examined how patient input is being integrated and communicated in regulatory and HTA decision making.
The workshop brought together representatives from patient organisations, industry, regulators, HTA agencies and payers to discuss challenges, opportunities, share case studies, and develop recommendations for improving the measurement and articulation of patient input in agency assessments.
Key findings from the workshop
Conclusions
The workshop demonstrated clear consensus that patient engagement (PE) and robust patient experience data (PED) are increasingly valued by regulators and HTA agencies, yet their use remains inconsistent and not always visible. Despite progress, stakeholders noted persistent challenges facing PE and PED, including difficulty quantifying their impact, fragmented approaches across jurisdictions, lack of methodological clarity, inconsistent quality and representativeness of data, and limited transparency about how patient input influences final decisions. Nevertheless, the workshop reinforced a shared commitment to strengthening patient voice throughout the medicine lifecycle.
The discussions emphasised the importance of early, purpose-driven PE in ensuring that patient perspectives shape research questions, evidence generated (including PED), and the outcomes assessed. Embedding PE and PED at the disease level rather than solely per asset was suggested to reduce duplication and improve efficiency. Agency and company case studies showed that patient input can be highly impactful when linked directly to decision‑making frameworks—whether through PRO and endpoint selection, preference studies, qualitative insights, or structured committee contributions. However, clearer expectations from regulators and HTA agencies are needed to incentivise investment in PED generation and reduce uncertainty about its evidentiary weight.
A recurring theme was the need to improve the visibility and transparency of how PE and PED are used in regulatory and HTA assessments and decisions. Current feedback to patients is often limited, risking perceptions of tokenism. Harmonised terminology, structured reporting tools such as standardised PED tables, clearer rationales for accepting or excluding patient evidence, and more accessible public‑facing summaries were seen as key steps toward building trust and demonstrating meaningful use of patient insights by agencies. Enhancing transparency is also essential to support internal decision making within companies, empower patient organisations, and provide assurance to communities that their input matters and is valued.
Looking ahead, participants agreed that progress requires cultural change, stronger infrastructures, and sustained cross‑stakeholder collaboration. Increased alignment between regulatory and HTA agencies was viewed as important for reducing duplication, supporting consistent expectations, and easing the burden on patient organisations. Support for patient groups, including capacity‑building, sustainable funding, and more flexible conflict‑of‑interest policies, will be necessary to ensure diverse, representative patient voices can contribute effectively. Ultimately, the workshop concluded that the field must now move from why PE and PED matter to how to embed them systematically and transparently in evidence generation and decision making.
