Patient Engagement and Patient Experience Data in Regulatory Review and Health Technology Assessment: A Global Landscape Review.

BACKGROUND: Working with patients through meaningful patient engagement (PE) and incorporating patient experience data (PXD) is increasingly important in medicines and medical device development. However, PE in the planning, organization, generation, and interpretation of PXD within regulatory and health technology assessment (HTA) decision-making processes remains challenging. We conducted a global review of the PE and PXD landscape to identify evolving resources by geography to support and highlight the potential of integration of PE and PXD in regulatory assessment and HTA. METHODS: A review of literature/public information was conducted (August 2021-January 2023), led by a multistakeholder group comprising those with lived or professional experience of PE and PXD, to identify relevant regulatory and HTA initiatives and resources reviewed and categorized by geography and focus area. RESULTS: Overall, 53 relevant initiatives/resources were identified (global, 14; North America, 11; Europe, 11; Asia, nine; UK, six; Latin America, one; Africa, one). Most focused either on PE (49%) or PXD (28%); few (11%) mentioned both PE and PXD (as largely separate activities) or demonstrated an integration of PE and PXD (11%). CONCLUSIONS: Our analysis demonstrates increasing interest in PE, PXD, and guidance on their use individually in decision-making. However, more work is needed to offer guidance on maximizing the value of patient input into decisions by combining both PE and PXD into regulatory and HTA processes; the necessity of integrating PE in the design and interpretation of PXD programs should be highlighted. A co-created framework to achieve this integration is part of a future project.

Building from Patient Experiences to Deliver Patient-Focused Healthcare Systems in Collaboration with Patients: A Call to Action.

Patients' experiences of their diagnosis, condition, and treatment (including the impact on their lives), and their experiences surrounding expectations of care, are becoming increasingly important in shaping healthcare systems that meet the evolving needs and priorities of different patient communities over time; this is an ongoing goal of all healthcare stakeholders. Current approaches that capture patient experiences with data are fragmented, resulting in duplication of effort, numerous requests for information, and increased patient burden. Application of patient experience data to inform healthcare decisions is still emerging and there remains an opportunity to align diverse stakeholders on the value of these data to strengthen healthcare systems. Given the collective value of understanding patient experiences across multiple stakeholder groups, we propose a more aligned approach to the collection of patient experience data. This approach is built on the principle that the patients' experiences are the starting point, and not just something to be considered at the end of the process. It must also be based on meaningful patient engagement, where patients are collaborators and decision makers at each step, thereby ensuring their needs and priorities are accurately reflected. The resulting data and evidence should be made available for all stakeholders, to inform their decision making and healthcare strategies in ways that meet patient priorities. We call for multi-stakeholder collaboration that will deliver healthcare systems and interventions that are better centered around and tailored to patient experiences, and that will help address patients' unmet needs.

Valuing patient engagement: Reflexive learning in evidence generation practices for health technology assessment.

Much attention in health technology assessment (HTA), a health system governance mechanism used for determining the value of health technologies, is being paid to improving the quality and patient-relevance of the evidence used in assessment pratices. Whilst the direct involvement of patient actors throughout HTA processes has become a more routine element of institutional practice, the 'impacts' of patient engagement (PE) initiatives have proven difficult to determine and enhance. In reflexive governance theories, reflexive learning is a critical mechanism of multi-stakeholder arrangements that better handles the complexities of technologies and how they are understood through governance practices. This paper explores how reflexive learning can be used to build a richer conceptualisation of PE in HTA, in order to generate suggestions for enhancing PE practices and their impact. We critically apply reflexive learning insights on qualitative data derived from the co-creation process of a PE evaluation framework, organised through an EU project focused on strengthening PE practices across medicines development (2018-2020), including 24 interactive case studies, 3 multi-stakeholder workshops, and our observations throughout the project. The findings characterise two dimensions of reflexive learning in PE: First, reflexive learning refers to the adaptive reorganisation of evidence generating practices, including the revision of medicines' evaluation criteria and the conditions under which evidence 'relevant' to HTA is constructed. Second, reflexive learning spotlights the sociopolitics which shape technology evaluation. Four themes affecting meaningful and sustained PE in medicines development were analysed: institutional boundaries due to established evaluation criteria; timing of engagements; network relations between institutional actors; and the politics of patient representation. Extending beyond discrete PE activities and their reported impacts, reflexive forms of learning are crucial to yielding more 'meaningful' PE for HTA and medicines development, facilitating a HTA practice that more meaningfully deals with the complexities of medicines evidence generation.

Sustaining Meaningful Patient Engagement Across the Lifecycle of Medicines: A Roadmap for Action.

BACKGROUND: There is increased recognition that incorporating patients' perspectives and insights into the medicines development process results in better health outcomes and benefits for all involved stakeholders. Despite the increased interest and the existence of frameworks and practical recommendations, patient engagement (PE) is not yet considered standard practice. The objective of this work was to provide a roadmap to support systematic change in all stakeholder organisations involved in medicines development across Europe, patients and patient organisations, medicines developers, academia, regulatory authorities, Health Technology Assessment bodies, payers, policy-makers and public research funders, to sustain PE practices. METHODS: A mixed-methods approach was used by the EU-funded Innovative Medicines Initiative PARADIGM Consortium to co-develop the sustainability roadmap including background work to identify success factors and scenarios for sustainable PE. The roadmap development was based on the Theory of Change concept and populated with findings from (1) interviews with national/ and international institutions with the potential to increase PE uptake by other stakeholders; (2) multi-stakeholder workshops and webinars; and (3) consultations with specific stakeholder groups, Consortium members and a consultative body formed by international PE initiatives. RESULTS: This roadmap sets strategic goals for the PE community to achieve meaningful and systematic PE through changes in the culture, processes and resources of stakeholder organisations. It brings in key PARADIGM outputs to work in a coordinated fashion with existing frameworks and mechanisms to achieve system-wide sustained PE. CONCLUSIONS: The roadmap provides a framework for all stakeholders to take collective action within their organisations and across Europe to implement PE in a sustainable manner.

Understanding multi-stakeholder needs, preferences and expectations to define effective practices and processes of patient engagement in medicine development: A mixed-methods study.

BACKGROUND: The holistic evolution of patient engagement in medicines development requires a more detailed understanding of the needs of all involved stakeholders, and one that better accounts for the specific needs of some potentially vulnerable patient populations and key stages in medicines development. OBJECTIVE: The purpose of this convergent mixed-methods study was to better understand the needs of different stakeholders concerning patient engagement at three key stages in medicines development: research priority setting, clinical trial design and early dialogues with Health Technology Assessment bodies and regulators. DESIGN: This study brought together findings from three sources: i) an online questionnaire, ii) face-to-face consultations with two potentially vulnerable patient populations, a workshop with Health Technology Assessment bodies, and iii) three-step modified Delphi methodology. RESULTS: Overall stakeholders still need additional varied support mechanisms to undertake, sustain or measure value of patient engagement. Health Technology Assessment bodies need better rationale for patient engagement in early dialogue and tools to support its implementation. Improved awareness and understanding of the need and value that involving patients, who are often considered as potentially vulnerable, can bring is needed, as is better accommodation of their specific needs. Similarly, weighted Delphi categories were as follows: aims and objectives, and sustainability. Several additional themes were common across the three key stages in medicines development. CONCLUSION: This broad-reaching study provides the blocks needed to build a framework for patient engagement in medicines development. PATIENT OR PUBLIC CONTRIBUTION: Patients were involved in review and interpretation of data.

Putting Patients at the Centre of Healthcare: Progress and Challenges for Health Technology Assessments.

Health technology assessments (HTAs) are meant to inform health policy by taking account of all the potential impacts of using a health technology. In the 1990s, HTAs included rigorous research to produce patient-based evidence, and some supported participation of patient representatives to help focus HTA research and determine value. In the 2000s, HTAs became more closely linked to reimbursement decisions, focusing on clinical and cost effectiveness. Patient involvement should be tailored to the specific needs of each HTA. As the timeframe for HTAs has reduced, research to produce patient-based evidence has been replaced by input from patient groups. This places a burden on individuals and organizations that needs to be critically reviewed. Therefore, it is imperative that we clarify when patient involvement is likely to add value and support patients to provide their unique knowledge in the most optimal way to influence HTA decision making. To reduce the burden on patient groups, more must be done to encourage research to produce patient-based evidence early in technology development. Like clinical research, a programme of research should be carefully planned, with appropriate methodological rigor for each study, and all research should be published. For this, the development of quality standards for research to produce patient-based evidence may be needed. Patient involvement has inherent value. It should be focused, systematic and transparent, and evolve according to the experiences of all stakeholders. All countries or collaboratives that undertake HTA should consider how they can elicit the needs, preferences and experiences of patients to support creation of patient-centered healthcare policy.

What do stakeholders expect from patient engagement: Are these expectations being met?

BACKGROUND: Meaningful patient engagement (PE) in medicines development and during the life cycle of a product requires all stakeholders have a clear understanding of respective expectations. OBJECTIVE: A qualitative survey was undertaken to understand stakeholder expectations. DESIGN: The survey explored 4 themes from the perspective of each stakeholder group: meaning, views, expectations and priorities for PE. Participants were grouped into 7 categories: policymakers/regulators; health-care professionals (HCPs); research funders; payers/purchasers/HTA; patients/patient representatives; pharmaceutical/life sciences industry; and academic researchers. RESULTS: Fifty-nine interviews were conducted across a range of geographies, PE experience and job seniority/role. There was consensus across stakeholders on meaning of PE; importance of promoting PE to a higher level than currently; need for a more structured process and guidance. There was little consensus on stakeholder expectations and roles. Policymakers/regulators were expected by others to drive PE, create a framework and facilitate PE, provide guidelines of good practice and connect stakeholders, but this expectation was not shared by the policymakers/regulators group. HCPs were seen as the link between patients and other stakeholders, but HCPs did not necessarily share this view. DISCUSSION AND CONCLUSIONS: Despite broad stakeholder categories, clear themes emerged: there is no "leader"; no stakeholder has a clear view on how to meaningfully engage with patients; there are educational gaps; and a structure and guidance for PE is urgently required. Given the diversity of stakeholders, there needs to be multistakeholder collaborative leadership. Effective collaboration requires consensus on roles, responsibilities and expectations to synergize efforts to deliver meaningful PE in medicines life cycle.

Strengthening international patient advocacy perspectives on patient involvement in HTA within the HTAi Patient and Citizen Involvement Interest Group - Commentary.

PLAIN LANGUAGE SUMMARY: Health Technology Assessment (HTA) is an evidence-based decision-making process, focusing on evaluating health technologies for funding within a healthcare system. 'Health technologies' include medications, medical devices, diagnostics, medical procedures and services. Health Technology Assessment international (HTAi) is a global society for those who produce, use, participate in, or encounter HTA. The HTAi Secretariat supports special interest groups within HTAi. One such special interest group, the Patient and Citizen Involvement in HTA Interest Group (PCIG), focuses on strengthening patient involvement in HTA. PCIG's members are from HTA agencies, research, industry, and patient organisations. We describe the steps PCIG has taken to form an international panel of patient advocates (the Patient Panel) as an autonomous group within its governance structure that reports directly to the PCIG Steering Committee. The Patient Panel was established in order to strengthen meaningful patient involvement in HTA by working co-productively with other PCIG members to develop resources accessible to patient organisations. Patient advocates known to be active within HTA in their own countries were invited to form the inaugural group, with one person appointed to chair the group for the first year. Documentation had been prepared to inform potential members and set out potential roles as seen by the Steering Committee. The appointed Patient Panel is defining its own terms of reference, goals and objectives. The Panel Chair is a member of the PCIG Steering Committee, and is mentored by the retiring PCIG chair. A registry of activities is part of monitoring and evaluation. ABSTRACT: Background Health Technology Assessment (HTA) is an evidence-based decision-making process, focusing on evaluating health technologies for funding within a healthcare system or medical insurance system. 'Health technologies' are understood broadly, and include medications, medical devices, diagnostics, medical procedures and services. Health Technology Assessment international (HTAi) is a global society for those who produce, use, participate in, or encounter HTA. The HTAi Secretariat supports special interest groups within HTAi, including the Patient and Citizen Involvement in HTA Interest Group (PCIG), which focuses on strengthening patient involvement in HTA. PCIG's members come from HTA agencies, research, industry, and patient organisations. Main body We describe the steps the PCIG has taken to form an international panel of patient advocates as an autonomous group within its governance structure, reporting directly to the PCIG Steering Committee. Patient advocates known to be active within HTA in their own countries were invited to form the inaugural group. One person was invited to chair the Panel for the first year of its operation, based on the candidate's experience of working across diseases and countries, and being new to HTAi. Documentation was prepared to inform potential members of the Panel and set out potential roles as seen by the Steering Committee. The Panel came into being in March 2016 and is now setting out its own working principles, goals and objectives. The Panel Chair is a member of the PCIG Steering Committee, and is mentored by the previous PCIG chair. A registry of activities has been set up as part of monitoring and evaluation. The Patient Panel is intended to provide a focus within the PCIG for patient advocates to work among themselves and co-productively through the PCIG Working Groups to develop mutual understanding and strengthen meaningful patient involvement in HTA internationally. Patient advocates can benefit from a clear understanding the evidence requirements within HTA and how information can be effectively presented by patient groups to decision-making bodies. Conclusion The HTAi Patient and Citizen Involvement Interest Group has set up a Patient Panel consisting of patient advocates. The intent is to work co-productively to advance patient involvement in HTA.

Patient Centricity and Pharmaceutical Companies: Is It Feasible?

Various health care bodies (regulatory, health technology assessment, academia, health care providers, scientific journals) request patient input in their decision-making processes. This represents a shift from disease-centered to patient-centered approaches to health care. What does this "patient centricity" mean for the pharmaceutical industry? A panel of senior pharmaceutical industry representatives discussed the following key issues: why the pharmaceutical industry needs to be part of the patient-centric movement; how the industry can become patient-centric; and what a patient-centric company actually does. We summarize the panel's point of view on these key questions. The industry's role has been to develop the science and medicines for prevention or treatment of disease. In response to changes in the current health care environment, the industry should focus its efforts on initiatives that will improve impact and value for patients and carers. True patient centricity requires a change in the industry's cultural mindset, an increase in public trust, clearer roles and responsibilities within pharmaceutical organizations, openness to learn from others, and a framework to measure success. There are examples of industry engagement with patients throughout the drug discovery and development process. Patient-reported outcomes are becoming increasingly important endpoints in trials; they capture information of relevance to patients, identify preferences, and better inform treatment decision making. Understanding the patient experience can improve disease management at critical points in the disease course. The future of patient centricity lies in coordinated efforts by and alignment of multiple health care stakeholders, which can only be achieved through collaborations and consortia, with the industry playing a key role.