Patiëntervaringsgegevens in de cardiologie: Een weg naar meer patiëntgerichte besluitvorming in de gezondheidszorg

Healthcare systems are increasingly expected to deliver care that is not only effective and safe, but also responsive to what matters most to patients. Yet, patients' role and influence in drug development, regulatory assessment, and pricing and reimbursement (P&R) evaluations is limited and fragmented. Decision-makers continue to rely predominantly on the perspectives of non-patient stakeholders and prioritise product-related criteria, potentially overlooking patients' distinct needs and preferences. At the same time, patients increasingly advocate for meaningful involvement in decisions that affect them, emphasising the principle: "Nothing about us, without us". As healthcare systems face rising cost pressures, involving patients is no longer a 'nice to have' but is critical for high-quality decision-making. Healthcare is therefore undergoing a cultural shift worldwide: from research on people to research with them. In this regard, patient experience data (PED) is gaining recognition as an important source of patient-based evidence alongside clinical and economic evidence across the medical product life cycle (MPLC). PED can improve research quality, feasibility, and legitimacy, while strengthening public trust, reducing costs, and avoiding research waste. Despite growing international consensus on its value, the availability and systematic use of high-quality PED remain inconsistent, unclear, and poorly integrated into decision-making processes. This persistent gap between recognition and impact raises a fundamental question: how can patients' perspectives be systematically, rigorously, and meaningfully integrated into healthcare decision-making? The PhD thesis addresses this gap by examining PED implementation across the MPLC, with a focus on cardiology. The research was structured around three objectives: (i) mapping current implementation practices and stakeholders' insights, (ii) optimising emerging patient-centred research methods, and (iii) translating findings into actionable recommendations. To achieve these objectives, this thesis adopted a mixed-methods design, combining qualitative and quantitative approaches across different stages of the MPLC and integrating perspectives from multiple stakeholder groups. The PhD thesis begins with Chapter 1, providing a comprehensive overview on patient-centred healthcare decision-making and PED, followed by Chapter 2 setting out the research objectives, design, and structure. Next, the PhD thesis is organised in three parts corresponding to the three objectives. Part I examines how PED and patient involvement practices are currently implemented in drug development and assessment, incorporating stakeholders' perspectives to identify remaining gaps and optimisation avenues. As key decisions about treatments are first made during drug development, Chapter 3 investigates the reporting and evolution of patient involvement in randomised controlled trials published over time in major medical journals and their corresponding protocols. This meta-epidemiological evaluation reveals that fewer than one in five trial articles and protocols reported patient involvement. When reported, descriptions are often vague and provide limited information on specific outcomes or impacts on decision-making. This chapter reveals a gap in transparency and reporting: even when patients are involved, their contributions remain largely invisible, underscoring the need for more consistent, detailed, and transparent reporting practices. To further understand current practices and stakeholders' perspectives, Chapter 4 explores clinical trial design processes, barriers, and opportunities for PED implementation in clinical research. Through a structured literature review and stakeholder interviews, this chapter demonstrates that while incorporating patient perspectives into clinical trials is acknowledged as strategically important, systematic implementation remains inconsistent. Patient-reported outcomes (PROs) are most frequently used, but their application varies across disease areas. Validated patient-informed measures are limited and PROs rarely serve as primary endpoints. Active patient input remains limited, but its integration is gaining institutional support, while approaches to better align research with patients' unmet needs are emerging yet still largely absent in practice. Patient-reported experiences (PREs) and patient preference data are far less frequently used, also highlighting awareness and methodological gaps. Clinical trials mark the beginning of a treatment's journey, but subsequent assessment phases determine whether treatments will come to market and ultimately reach patients. To better understand PED integration in downstream decision-making, Chapter 5 explores multi-stakeholder perspectives on challenges, opportunities, and concrete policy ways forward for systematically implementing PED in healthcare decision-making. Semi-structured interviews with regulators, P&R agencies, industry representatives, academics, and patient organisations across Europe reveal several shared barriers, including the lack of clear policy frameworks, absence of harmonised methodological standards, limited validated tools, and insufficient transparency in PED assessment. Although stakeholders broadly agreed on the value of PED, uncertainty remains regarding which stakeholder group should initiate first actions in advancing systematic implementation. Complementing this broader assessment perspective, Chapter 6 focuses specifically on patient involvement in P&R procedures. Through a scoping literature review, semi-structured interviews, and focus group discussions, this chapter identifies international practices, barriers, and enablers for meaningful patient involvement. While patient involvement in P&R procedures remains uneven and inconsistently implemented across countries, the findings indicate a clear gradual shift towards more structured participation. However, the results also show that patient involvement cannot be realised solely through isolated initiatives. Achieving meaningful and sustainable involvement requires the integration of structural, organisational, procedural, technical, and financial enablers. Having mapped the current landscape and stakeholders' insights on PED implementation in drug development and assessment, Part II focuses on optimising patient-centred research methods and their implementation, particularly patient preference studies (PPSs) and needs assessments. As shown in Part I, these methods remain underutilised due to remaining methodological challenges. Therefore, Part II generates methodological insights and optimises implementation through applied case studies in cardiology and conceptual framework development. In treatment settings such as heart failure, decision-makers still rely primarily on the perspectives of non-patient stakeholders, which may deviate from the unique perspectives of patients. Hence, Chapter 7 explores heart failure patients' unmet medical needs, treatment priorities, risk tolerance, and information needs. Interviews indicate that patients prioritised improvements in quality of life alongside life expectancy. This qualitative research suggests a potential misalignment between traditional clinical endpoints in cardiovascular research, where survival outcomes often dominate clinical evaluation, and symptom-based outcomes that matter most to patients. These findings emphasise the importance of collecting bottom-up patient insights to inform drug development and evaluation. To explore patient preferences in a different clinical context, Chapter 8 investigates how preferences may differ in a medical device setting by conducting a qualitative PPS among patients with cardiac pacemakers. Despite expressing trust in their physicians to decide on specific device types, patients still value specific disease and treatment-related characteristics, mainly as informational needs rather than decision-making drivers. These findings show that patient preferences and their potential role in decision-making differ depending on the clinical context. By using deliberative approaches combining both scoring and ranking exercises, this study also demonstrates that methodological optimisation has helped patients to reflect more deeply on trade-offs, resulting in a more stable set of patient priorities. As development should also be guided by patients' needs, conducting needs assessments is essential for identifying unmet needs and research gaps. Chapter 9 applies and refines a needs assessment framework (developed by Belgian Health Care Knowledge Centre) to assess unmet health-related needs unique to patients with rare diseases. This clinical context complements the treatment and medical device settings explored in previous chapters, as rare diseases are also present in cardiology and often pose distinct challenges due to limited treatment options and smaller patient populations. Through a rapid literature review and stakeholder consultations, this chapter reveals multidimensional unmet needs spanning health, healthcare, and social dimensions for both patients and caregivers. The findings highlight the necessity of holistic approaches to needs identification and policy prioritisation. In response to criticisms of the supply-driven approach to healthcare decision-making, both PPSs and needs assessments have emerged as methods that can support a more patient-centred shift. Building on the previous chapters, Chapter 10 explores the potential synergy between these two methods to maximise their impact and implementation in healthcare decision-making. The chapter proposes a conceptual framework in which needs assessments first identify broader unmet needs, after which PPSs can quantify patients' trade-offs and prioritise these needs. By positioning these methods as sequential and complementary rather than independent approaches, the chapter provides methodological g