The roles and potential value of patient preference (PP) data in health technology assessment (HTA) remain to be fully realized despite an expanding literature and various efforts to establish their utility. This article reports lessons learned through a series of collaborative workshops with HTA representatives, organized by the Health Technology Assessment International’s Patient Preferences Project Subcommittee.

Five online workshops were conducted between June 2022 and June 2023, seeking to facilitate collaborative learning and reflection on ways that PP data can be integrated into HTA. Participants included nine HTA representatives from the United States, Canada, Australia, England, and the Netherlands. Workshops were recorded, transcribed, and thematically analyzed.

Despite appreciating the value of PP data, participants were ambivalent about their use in HTA. Some felt that they were already getting the information they needed from the cost-effectiveness analysis or existing patient involvement processes. Others thought that PP data would be very helpful at the initial and final stage of the decision-making process and, particularly, in the following cases: (a) when technology has important non-health benefits; (b) when the clinical and/or cost-effectiveness evidence is marginal; and (c) when treatment is indicated for a large and heterogeneous population. Issues related to the validity and reliability of PP studies were frequently raised, with preference heterogeneity at the core of these concerns.

Collaborating with HTA representatives in the “co-creation” of PP research can help address their concerns and facilitate mutual learning about how PP data can be used in HTA.

Healthcare disinvestment requires multi-level decision-making, and early stakeholder engagement is essential to facilitate implementation and acceptance. This study aimed to explore the perceptions of Malaysian healthcare stakeholders to disinvestment initiatives as well as identify disinvestment activities in the country.

A cross-sectional online survey was conducted from February to March 2023 among Malaysian healthcare stakeholders involved in resource allocation and decision-making at various levels of governance. Response frequencies were analyzed descriptively and cross-tabulation was performed for specific questions to compare the responses of different groups of stakeholders. For free-text replies, content analysis was used with each verbatim response examined and assigned a theme.

A total of 153 complete responses were analyzed and approximately 37 percent of participants had prior involvement in disinvestment initiatives. Clinical effectiveness and cost-effectiveness ranked as the most important criteria in assessment for disinvestment. Surprisingly, equity was rated the lowest priority despite its crucial role in healthcare decision-making. Almost 90 percent of the respondents concurred that a formal disinvestment framework is necessary and the importance of training for the program’s successful implementation. Key obstacles to the adoption of disinvestment include insufficient stakeholder support and political will as well as a lack of expertise in executing the process.

While disinvestment is perceived as a priority for efficient resource allocation in Malaysian healthcare, there is a lack of a systematic framework for its implementation. Future research should prioritize methodological analysis in healthcare disinvestment and strategies for integrating equity considerations in evaluating disinvestment candidates.

Multi-level dissemination strategies are needed to increase equitable access to effective treatment for high-risk outpatients with COVID-19, particularly among patients from disproportionately affected communities. Yet assessing population-level impact of such strategies can be challenging.

In collaboration with key contributors in Colorado, we conducted a retrospective cohort study to evaluate a multi-level dissemination strategy for neutralizing monoclonal antibody (mAb) treatment. Real-world data included county-level, de-identified output from a statewide mAb referral registry linked with publicly available epidemiological data. Outcomes included weekly number of mAb referrals, unique referring clinicians, and COVID-19 hospitalization rates. We assessed weekly changes in outcomes after dissemination strategies launched in July 2021.

Overall, mAb referrals increased from a weekly average of 3.0 to 15.5, with an increase of 1.3 to 42.1 additional referrals per county in each post-period week (p < .05). Number of referring clinicians increased from a weekly average of 2.2 to 9.7, with an additional 1.5 to 22.2 unique referring clinicians observed per county per week beginning 5 weeks post-launch (p < .001). Larger effects were observed in communities specifically prioritized by the dissemination strategies. There were no observed differences in COVID-19 hospitalization rates between counties with and without mAb treatment sites.

Real-world data can be used to estimate population impact of multi-level dissemination strategies. The launch of these strategies corresponded with increases in mAb referrals, but no apparent population-level effects on hospitalization outcomes. Strengths of this analytic approach include pragmatism and efficiency, whereas limitations include inability to control for other contemporaneous trends.

Large, transdisciplinary research consortia have increasingly been called upon to address complex and challenging health problems. The National Institutes of Health’s (NIH) Environmental influences on Child Health Outcomes (ECHO) Program developed multisite collaboration strategies to promote impactful collaborative observational research on child health. Team science and implementation science offer theoretical and methodological structure to answer questions about the strategies that facilitate successful consortia. We sought to characterize the elements and conditions that influence the implementation of a complex, interdisciplinary longitudinal research program, ECHO.

Informed by the Practical, Robust, Implementation and Sustainability Model, our ethnographic research included semi-structured interviews with internal stakeholders and program evaluation metrics. We conducted template and matrix analysis and triangulated the qualitative and quantitative data to understand the implementation of ECHO.

Between February and May 2022, we conducted 24 virtual interviews with representatives from ECHO components. The main cross-cutting topics that emerged from thematic analysis were collaboration and team science; communication and decision-making; data processes and harmonization; and diversity, equity, and inclusion. Both the qualitative and secondary quantitative evaluation data provided insights into the reach, adoption, implementation, and effectiveness of the program.

A large, multidisciplinary research consortium such as ECHO has produced conceptual, instrumental, capacity building, and connectivity impact for internal and external stakeholders. Facilitators included infrastructure that supported collaboration and learning, alignment of data processes, and harmonization. Opportunities for enhanced impact include multidisciplinary, multimethod communication strategies, and alignment of research priorities.

Spain incorporated in 2020 changes in its health technology assessment (HTA), pricing, and reimbursement system for medicines including publishing reports, development of networks of experts, or consultation with stakeholders. Despite these changes, it is unclear how deliberative frameworks are applied and the process has been criticized for not being sufficiently transparent. This study analyses the level of implementation of deliberative processes in HTA for medicines in Spain.

We review the grey literature and summarize the Spanish HTA, pricing, and reimbursement process of medicines. We apply the deliberative processes for HTA checklist, developed to assess the overall context of the deliberative process, and identify the stakeholders involved and type of involvement following the framework for evidence-informed deliberative processes, a framework for benefit package design that aims to optimize the legitimacy of decision making.

In the Spanish HTA, pricing, and reimbursement process deliberation takes place in order to exchange viewpoints and reach common ground, mainly during the prioritization, assessment, and appraisal steps. It is closed to the public, not clearly summarized in published documents and limited to the Ministry of Health, the regulatory agency, other Ministries, and experts with mostly clinical and/or pharmaceutical background. The views of stakeholders are only represented through consultation. Communication is the most commonly used form of stakeholder engagement.

Despite improvements in transparency of the Spanish HTA process for evaluating medicines, aspects related to stakeholder involvement and implementation of deliberative frameworks need further attention in order to achieve further legitimacy of the process.

Despite increasing emphasis on the inclusion of patient input in health technology assessment (HTA) in Europe in particular, questions remain as to the integration of patient insight alongside other HTA inputs. This paper aims to explore how HTA processes, while ensuring the scientific quality of assessments, “make do” with patient knowledge elicited through patients’ involvement mechanisms.

The qualitative study analyzed institutional HTA and patient involvement in four European country contexts. We combined documentary analysis with interviews with HTA professionals, patient organizations, and health technology industry representatives, complemented with observational findings made during a research stay at an HTA agency.

We present three vignettes which showcase how different parameters of assessment become reframed upon the positioning of patient knowledge alongside other forms of evidence and expertise. Each vignette explores patients’ involvement during an assessment of a different type of technology and at a different stage of the HTA process. First, cost-effectiveness considerations were reframed during an appraisal of a rare disease medicine based on patient and clinician input regarding its treatment pathway; in the second vignette reframing amounted to what counts as a meaningful outcome measure for a glucose monitoring device; in the third, evaluating pediatric transplantation services involved reframing an option’s appropriateness from a question of moral to one of legal acceptability.

Making do with patient knowledge in HTA involves reframing of what is being assessed. Conceptualizing patients’ involvement in this way helps us to consider the inclusion of patient knowledge not as complementary to, but as something that can transform the assessment process.

We created a COVID-19 Research Patient and Community Advisory Board (PCAB) to provide patient and community input into clinical and translational research studies. The purpose of this article is to describe the PCAB creation, implementation, and evaluation.

We identified PCAB members who had participated in previous stakeholder engaged activities at our institution and invited their participation. We created a systematic consultation process where researchers could submit plain language research summaries and questions for the PCAB. A facilitated 1-hour virtual consultation was then held where PCAB members provided feedback. We assessed satisfaction of PCAB members and researchers who received consultations using surveys. We also reviewed video recordings of PCAB consultations and reflections from team meetings to identify key lessons learned.

Twenty-seven PCAB members took part in 23 consultation sessions. Twenty-two completed an evaluation survey (81% response rate). Most members agreed or strongly agreed their opinions were valued (86%), it was a productive use of time (86%) and were satisfied (86%). Nineteen researchers completed an evaluation survey (83% response rate). Researchers reported positive experiences of working with the PCAB. Additional insights include limited funding in COVID-19 research for equitable community engagement, deficiencies in researcher communication skills, and a lack of cultural humility incorporated into study activities.

PCAB members provided recommendations that maximized the patient-centeredness and health equity focus of COVID-19 research. The detailed description of the process of developing, implementing, and evaluating our PCAB can be used as a template for others wishing to replicate this engagement model.