Table 2.

Examples of patient engagement (PE) and patient experience data (PXD) initiatives in different regions

Initiative	Purpose/summary
Global
International Coalition of Medicines Regulatory Authorities (ICMRA) Statement	Identified opportunities for regulatory authorities to collaborate in considering how real-world evidence (RWE) can inform regulatory decision-making Areas recognized were (1) harmonization of terminology to clearly define real-world data and RWE; (2) convergence on guidance and best practice, including common principles on data quality, identification of situations where RWE can appropriately contribute to regulatory decisions, and templates for study protocols and reports that can be used by multiple authorities; (3) rapid creation of international expert groups and collaboration on processes; and (4) definition of common principles for study registration and publication of results in registries and open-source, peer-reviewed journals [13]
Proposed International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Guideline	Proposed ICH Guideline Work to Advance Patient Focused Drug Development identifies areas where including the patient perspective would improve “the quality, relevance, safety and efficiency of drug development and inform regulatory decision-making” Presents opportunities for global harmonization of how the patient perspective is included using a robust methodology that is appropriate for both regulated industry and regulatory authorities [14]
Global Patient Experience Data Navigator	Co-created by Patient Focused Medicines Development (PFMD) as a tool to help navigate the PXD landscape and to add clarity and structure to understanding the generation, use, and interpretation of PXD Includes tools to identify PXD use by different stakeholders throughout the product development cycle and healthcare process, determine what impacts matter most to patients and families, and select appropriate measurement methods of outcomes that matter most to patients and families [15]
Europe
European Medicines Agency (EMA) Engagement Framework	Supporting access to individual patient’s experiences of living with a condition, its management and use of medicines Promoting the generation, collection, and use of evidence-based PXD for benefit-risk decision-making Developed by the Patients and Consumers Working Party (PCWP)—an organization that also informs global guidance provided by ICH and the Council for International Organizations of Medical Sciences (CIOMS) Patients' and Consumers' Working Party (PCWP) members come from patient organizations, industry, regulators, academia, and the World Medical Association and work collectively to highlight pragmatic points around patient involvement strategies [16]
Health Technology Assessment International (HTAi)	360° HTA Patient Involvement Project (supported by HTAi, the European Patient Academy on Therapeutic Innovation [EUPATI] and the European Patients’ Forum [EPF]) Aims to understand how methods and processes for patient involvement in health technology assessment (HTA) processes are perceived, if all stakeholders feel that patients are sufficiently involved, and to provide advice for future directions [17]
Innovative Medicines Initiative-PREFER (IMI-PREFER)	Framework that provides suggestions on how patients’ perspectives could be measured through patient preference studies and then incorporated into regulatory decision processes Framework has three main sections: (1) defining preference study aims and objectives; (2) study planning, design, and conduct; and (3) interpretation and application of study results [18]
Europe Network for Health Technology Assessment (EUnetHTA)	Joint work plan to help build a European network of experts on patient-reported outcomes (PROs), contribute to guideline development and a workshop on PXD for multistakeholders, as well as following up on areas of action identified in the ICH reflection paper on patient-focused drug development [14] The plan will further describe best practice for issues such as compensation for expert participation and, through educating experts on the difference between HTA and regulatory processes, will provide direction on how to incorporate expert input into the regulatory and HTA outputs [19]
Medicines and Healthcare products Regulatory Agency (MHRA) Patient Involvement Strategy 2021–2025	Sets out objectives to engage the public and patients during all stages of the regulatory process, and to change the internal culture of the agency such that “every member of staff considers the patient and public perspective in their decisions” Sets out objectives for multistakeholder partnerships in acknowledgement that benefits exist from sharing data and avoiding duplication of time and effort [20]
Centre for Research in Public Health and Community Care (CRIPACC)	Provides practical advice to health researchers on giving feedback to patient and public contributors, including thanking contributors and providing detailed feedback (such as impact of patient contribution and study progress) to increase motivation, confidence, learning and development, accountability, and transparency [21]
National Institute for Health and Care Excellence (NICE) RWE Framework	RWE framework is primarily aimed at pharmaceutical and health technology companies developing evidence to inform NICE guidance but is also relevant to patients and organizations that gather data and review evidence During development of the framework, NICE sought feedback through workshops and public consultation from numerous bodies, including patients and patient organizations, and revised the framework accordingly A key recommendation is that data should be collected in a patient-centered way that also minimizes the burden on patients and healthcare professionals [22]
United States
Patient Focused Drug Development (PFDD) Guidance	Guidance published in 2020, addressing collection of patient input including sampling methods and target population definition Guidance issued in February 2022, focused on methods used to identify what is important to patients in relation to the burden of their disease and its treatment. It described best practice in conducting qualitative, quantitative, and mixed methods research, as well as considerations for the use of social media Draft guidance issued in June 2022 to help clinical trial sponsors use high quality measures of trial outcomes that are important to patients. Described how to choose, modify, or develop and validate clinical outcome assessments, including PROs, observer-reported outcomes, clinician-reported outcomes, and performance-based outcomes [23–26] The FDA's Center for Biologics Evaluation and Research (CBER) supports the PFDD mission and has a patient engagement program to incorporate patient input in their work. Initiatives include CBER’s Science of Patient Input (SPI) initiative and their Rare Disease program [27]
Medical Device Innovation Consortium (MDIC) Patient and Public Involvement (PPI) Clinical Trial Framework	Describes considerations for regulators wanting to include PPI in clinical trial design Includes leveraging existing opportunities to incorporate PPI into regulatory decision-making; identifying novel endpoints for patient preference studies and aligning these with traditional endpoints; ensuring that the PPI used is relevant to the intended patient population; and using relevant statistical methods for the trial population [28]
Patient Insights Database	Aims to support early inclusion of patients in clinical trial design Generated through patient interviews and testimonials, RWE studies, and health-related quality of life (HRQoL) research Used to further understanding of symptoms and diagnosis, treatment, HRQoL, economic burden, and patients’ hopes for new treatments [29]
US Food and Drug Administration’s (FDA) Center for Devices and Radiological Health	Patient Engagement in the Design and Conduct of Medical Device Clinical Studies—provides guidance on PE in medical device studies and is intended to help sponsors understand how they can use PXD to improve clinical studies for medical devices, to highlight the benefits of early patient engagement, to clarify what are considered relevant engagement activities, and to address questions/misconceptions about PE data collection for medical device design and clinical studies [30] Principles for Selecting, Developing, Modifying, and Adapting Patient-Reported Outcome Instruments for Use in Medical Device Evaluation—presents concepts to consider when using PRO instruments, provides recommendations around ensuring the chosen PRO instruments are sufficient for the task, and describes best practices to develop, modify, or adapt PRO instruments for an optimized outcome [31]
Japan, China, and Singapore
Pharmaceuticals and Medical Device Agency (PMDA) in Japan	Published a report on patient participation in 2021 with the dual aim of gathering patient input and increasing awareness amongst patients of the agency’s work Made a commitment to include PROs in all medicine and device evaluations [32]
Center for Drug Evaluation (CDE) in China	Published Guiding Principles for the Application of Patient Reported Outcomes in Drug Clinical Research and Development (Trial) [33]
Consumer Engagement and Education (CEE) Agency in Singapore	Provided a forum for patient and volunteer organizations to publicize their work on patient and public involvement Insights included the value of patient involvement in HTA through the provision of unique first-hand experience; the importance of mutual trust, cooperation, and maintaining a two-way conversation with patients and HTAs; the need to improve patient health literacy and clarity of communication using plain language; and the importance of patient insights for rare diseases to fill data gaps [34]