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

Abstract

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 As the timeframe for HTAs has reduced, research to produce patient-based evidence has been replaced by input from patient groups.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

Cost-Effectiveness of Sacubitril/Valsartan in Germany: An Application of the Efficiency Frontier

“…Additionally, the EF's ability to consider various outcomes may be helpful if alternative outcomes, such as patientreported outcomes, are generated by clinical trials. 54 We think it may be useful to perform similar comparisons in various other fields, where patented drugs reflect important treatment modalities, such as oncology.…”

Use of Patient Preferences in Health Technology Assessment: Perspectives of Canadian, Belgian and German HTA Representatives

“…Patients can provide insights about the impact of conditions and treatments on their lives, outcomes that matter to them, and their needs and fears [3,4]. Currently, patients are sometimes directly involved in discussions [5,6]. However, direct patient involvement is thought to be subjective, potentially biased, and of limited representativeness [7][8][9][10].…”

Patient Preference Studies During Early Drug Development: Aligning Stakeholders to Ensure Development Plans Meet Patient Needs

“…Rather, the process should serve as a framework, whereby the importance of generating new patient-based evidence needs to be considered by the manufacturer together with input from a multi-stakeholder advisory board (including patients) throughout development. This notion parallels the recognition of HTA agencies, that it is “imperative that we clarify when patient involvement is likely to add value” (18). From the HTA perspective, the generation of patient-based evidence in early product development is encouraged to reduce the burden on patients and patient groups later in the product life cycle (18).…”

“…Such research meets the increasing requests that the evidence the pharmaceutical industry brings to the table for its new products not only includes efficacy and safety data but also endpoints that are relevant to the target patients and that the product will deliver “patient value” (5, 18–20). Currently, patient-based evidence is still not reported sufficiently in pharmaceutical company submission dossiers, as reported by the Canadian HTA agency (CADTH) when they reviewed dossiers submitted between December 2012 and June 2014 (21).…”

“…Many agencies involve individual patients or patient representatives to inform the regulatory or HTA process ( patient input, patient involvement ) (25). In this function, patients can contribute their experience of living with the disease and thereby support the interpretation of the clinical evidence with impact on the decision to make new interventions available (18, 25, 26). Likewise, patients can contribute to the design of clinical trials by being collaborative research partners for the research team (27).…”