Patient Involvement in the Lifecycle of Medicines According to Belgian Stakeholders: The Gap Between Theory and Practice

Abstract

Background: Patient involvement is often acknowledged as an important aspect of the lifecycle of medicines. Although different typologies exist, patient involvement has been described as the involvement of patients in decision-making regarding medicines. In view of the diversity of stakeholders and types of decisions in which patients might be involved, an in-depth understanding of these stakeholders' views toward involving patients in the lifecycle of medicines is essential.Methods: Interviews and surveys were used to gain insights into the perspectives and experiences of Belgian healthcare stakeholders. Interviews (n = 22) were conducted with academics, hospital pharmacists and representatives from health insurance funds, the Belgian reimbursement agency, pharmaceutical industry and patient organizations. Interviews underwent a framework analysis. Surveys (n = 108) were completed by hospital visitors and analyzed descriptively.Results: Despite an increasing amount of efforts to involve patients, interviewees labeled the level of actively involving patients as rather low and scattered across the different phases of the lifecycle of medicines. The main opportunities for patient involvement highlighted by interviewees were for: (i) informing early development decisions on which treatments to develop, (ii) clinical trial endpoint selection and (iii) clinical trial protocol design. However, remaining questions surrounding patient knowledge, and particularly how and which patients to involve represent important barriers toward implementing patient involvement in the lifecycle of medicines. Of survey participants, 77% indicated to be willing to participate in patient preference studies. Reasons for participating mentioned most frequently were “to improve development of treatments,” because “it is important to explore and listen to patient preferences” and “to have a voice as patients”.Conclusions: The barriers identified in this study hamper transitioning patient involvement from theory to practice. Bridging this gap requires addressing the identified barriers and unresolved questions surrounding the right methodology for involving patients, the “right patients” to involve and means to increase patient knowledge. In order to do so, further research should focus on assessing the value of methods that allow to indirectly capture patients' perspective both in the context of development as well as in the context of evaluation.

Treatment innovation for patients: a collaborative network in the Benelux and an inside view of 20 years of Galapagos

“…EU regulation further requires clinical trial sponsors to provide summary results of their trials in a format understandable to laypersons, which may increase the opportunities for collaboration with patient representatives [13]. Patient involvement in the drug life cycle remains challenging, and gaps remain between theory and practice, as shown by a recent Belgian survey [34]. Involving patients and patient perspectives in the design of studies that include patient-reported outcomes is an important step [35].…”

Use of Patient Preference Information in Benefit–Risk Assessment, Health Technology Assessment, and Pricing and Reimbursement Decisions: A Systematic Literature Review of Attempts and Initiatives

“…They are best positioned to provide a real-world understanding of their experiences and define their treatment preferences based on benefits and harms of treatment outcomes (1)(2)(3)(4)(5)(6)(7)(8)(9). As a result, an extensive trend toward making more patient-centric healthcare decisions has emerged, and experts have considered three possible levels of patient involvement referred to as micro, meso, and macro levels reliant on whether the involvement impacts patient-physician day-to-day interactions, a specific disease area, or resource allocation and healthcare policy decisions, respectively (10). While the role of patient with regard to microlevel decision, i.e., shared decision making, has significantly evolved in the last decades, when it comes to health authorities' decision making, the relative importance of patient voice remained unclear.…”

“…In response to the growing interest of patient involvement, two trends, not mutually exclusive, have emerged: the first trend is "direct involvement" of patients in decision making, which is in favor of patients participating in discussions, such as through committees, advisory groups, or just testimonies, while the second trend is "indirect patient involvement" through studies allowing for the assessment of patient preferences (PPs) in a more systematic way (7,10,13). Moreover, researchers and policymakers have developed tools to assess PPs and initiated a variety of efforts and attempts to better include these PPs in benefit-risk assessment (BRA) and HTA decisions.…”

Improving Patient Involvement in the Lifecycle of Medicines: Insights From the EUPATI BE Survey

“…Patient involvement is increasingly recognized as an integral part of healthcare and a critical component of safe and effective people-centered services. Engaged patients are more able to make informed decisions about their care options (1). Furthermore, resources may be better used if they are aligned with patients' priorities and this is critical for the sustainability of healthcare systems worldwide.…”

“…Patient-centered initiatives, such as the Innovative Medicines Initiative (IMI) have greatly added to the awareness that patients need to be at the center of a healthcare system. In order to participate in this innovation process, the European Patients' Academy on Therapeutic Innovation (EUPATI), a patient-led public-private partnership was created in 2012 (1). EUPATI aims to increase patient involvement by developing educational toolboxes and trainings.…”