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 As the timeframe for HTAs has reduced, research to produce patient-based evidence has been replaced by input from patient groups.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.

BackgroundMeaningful patient engagement (PE) in medicines development and during the life cycle of a product requires all stakeholders have a clear understanding of respective expectations.ObjectiveA qualitative survey was undertaken to understand stakeholder expectations.DesignThe 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.ResultsFifty‐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 conclusionsDespite 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.

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.

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.

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.