There are wide variations in the practices of patient involvement in health technology assessment (HTA) in Europe. The field is lacking a consensus on good practices, leading to divergent processes, methods, and evaluation of patient involvement. To identify potential good practice approaches and current gaps, a structured online survey was conducted among HTA stakeholders, including HTA practitioners, patient stakeholders, industry representatives, and others who had experienced patient involvement in HTA.

The questionnaire was co-created by HTA experts, patient stakeholders, and industry representatives and disseminated between 29 April and 14 September 2022.

Responses (n = 168) were submitted from thirty-two European countries by HTA practitioners (n = 33), patient stakeholders (n = 75), industry stakeholders (n = 42), providers (n = 5), academics (n = 7), and others (n = 6). The responses indicated that “allowing access to treatments that have demonstrated value”is the principle rationale for conducting HTA. In terms of the importance of patient involvement, there was consensus across stakeholder groups that “patients have insights and information [that] no other stakeholder has” and that patient involvement is important “to inform HTA which evidence is most patient-relevant”. Shortcomings were identified in the lack of systematic and transparent processes, an unsatisfactory level of information and guidance, and minimal communication and collaboration.

The diverse stakeholders who responded highlighted the need for improving specific aspects of patient involvement practices, including better guidance and information, a more consistent flow of communication between the HTA body and participating patient stakeholders, and the need to develop and implement a consensus on good practices.

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.

The main objective was to pilot the culturally adapted “Educate, Nurture, Advise, Before Life Ends” for Singapore (ENABLE-SG) model to evaluate its feasibility and potential effectiveness.

A single-arm pilot trial of ENABLE-SG among patients with advanced solid tumors and caregivers of these patients was conducted in the outpatient oncology clinic setting. Enrolled participants participated in individual ENABLE-SG psychoeducational sessions weekly. Patients had 6 sessions on the topics of maintaining positivity, self-care, coping with stress, managing symptoms, exploring what matters most and life review. Caregivers had 4 sessions on the topics of maintaining positivity, self-care, coping with stress and managing symptoms. At baseline, 4, 8, and 12 months after enrolment, patient’s quality of life was measured using the Functional Assessment of Chronic Illness Therapy – Palliative Care, patient’s mood was measured using the Center for Epidemiologic Studies – Depression scale, and caregiver quality of life was measured using the Singapore Caregiver Quality of Life Scale.

We enrolled 43 patients and 15 caregivers over a 10-month period from August 2021 to June 2022. Although there was a low approach-to-participation rate, most of those who enrolled completed all ENABLE-SG sessions – 72% for patients and 94% for caregivers. Caregivers had better quality of life over time, specifically in the subscales of mental well-being and experience-meaning.

Based on findings from this study, we are planning a randomized waitlist-controlled trial of ENABLE-SG for patients with advanced cancer and their caregivers.

Engaging patients, caregivers, and other stakeholders to help guide the research process is a cornerstone of patient-centered research. Lived expertise may help ensure the relevance of research questions, promote practices that are satisfactory to research participants, improve transparency, and assist with disseminating findings.

Traditionally engagement has been conducted face-to-face in the local communities in which research operates. Decentralized platform trials pose new challenges for the practice of engagement. We used a remote model for stakeholder engagement, relying on Zoom meetings and blog communications.

Here we describe the approach used for research partnership with patients, caregivers, and clinicians in the planning and oversight of the ACTIV-6 trial and the impact of this work. We also present suggestions for future remote engagement.

The ACTIV-6 experience may inform proposed strategies for future engagement in decentralized trials.

To explore how primary healthcare professionals (HCPs) tasked with facilitating primary healthcare service development with patient participation perceived their role.

Patient participation in health service development is a recognized means of ensuring that health services fit the public’s needs. However, HCPs are often uncertain about how to involve patient representatives (PRs), and patient participation is poorly implemented. Inspired by the Promoting Action on Research Implementation in Health Services framework, we address the innovation (patient participation), its recipients (PRs, HCPs, supervisors, and senior managers), and its context (primary healthcare at a local and organizational level).

We conducted semi-structured individual interviews with six HCPs working as internal facilitators in primary healthcare in four Norwegian municipalities. The data were analyzed by applying Braun and Clarke’s reflexive thematic analysis.

The themes show that to develop primary healthcare services with patient participation, facilitators must establish a network of PRs with relevant skills, promote involvement within their organization, engage HCPs favorable toward patient participation, and demonstrate to supervisors and senior managers its usefulness to win their support. Implementing patient participation must be a shared, collective responsibility of facilitators, supervisors, and senior management. However, supervisors and senior management appear not to fully understand the potential of involvement or how to support the facilitators. The facilitator role requires continuous and systematic work on multiple organizational levels to enable the development of health services with patient participation. It entails maintaining a network of persons with experiential knowledge, engaging HCPs, and having senior management’s understanding and support.

While patient participation in individual health technology assessments (HTAs) has been frequently described in the literature, patient and citizen participation at the organizational level is less described and may be less understood and practiced in HTA bodies. We aimed to better understand its use by describing current practice.

To elicit descriptive case studies and insights we conducted semi-structured interviews and open-ended questionnaires with HTA body staff and patients and citizens participating at the organizational level in Belgium, France, Quebec, Scotland, and Wales.

We identified examples of organizational participation in managerial aspects: governance, defining patient involvement processes, evaluation processes and methods, and capacity building. Mechanisms included consultation, collaboration, and membership of standing (permanent) groups. These were sometimes combined. Participants were usually from umbrella patient organizations and patient associations, as well as individual patients and citizens.

Although the concept, participation at the organizational level, is not well-established, we observed a trend toward growth in each jurisdiction. Some goals were shared for this participation, but HTA bodies focused more on instrumental goals, especially improving participation in HTAs, while patients and citizens were more likely to offer democratic and developmental goals beyond improving participation processes.

Our findings provide rationales for organizational-level participation from the perspectives of HTA bodies and patients. The case studies provide insights into how to involve participants and who may be seen as legitimate participants. These findings may be useful to HTA bodies, the patient sector, and communities when devising an organizational-level participation framework.

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.

The Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines on behalf of NHSScotland. Assessment of end-of-life (EoL), orphan, and ultra-orphan medicines includes a process to gather evidence from patients and carers during Patient and Clinician Engagement (PACE) meetings. The output of PACE meetings is a consensus statement describing the medicine’s added value from the perspective of patients/carers and clinicians. The PACE statement is used by SMC committee members in decision making. This study compared how PACE participants and SMC committee members rate the importance of information in PACE statements for these medicines.

A survey was undertaken of patient group (PG) representatives and clinicians who participated in PACE meetings, and SMC committee members.

PACE participants who responded (26 PG representatives and 14 clinicians) rated health benefits and ability to take part in normal life as important/very important. Convenience of administration and treatment choice received the lowest rating. Hope for the future received the most diverse response. PACE participants generally rated the importance of quality of life themes higher than committee members (n = 20) but the rank order was similar. Differences between the proportion of PACE participants and committee members who rated themes as important/very important were greatest for treatment choice and hope for the future.

In general, PACE themes and subthemes that were rated highly by PACE participants were also considered important by SMC committee members, indicating that information captured during PACE meetings is relevant when making decisions on EoL, orphan, and ultra-orphan medicines.

The objective of Health Technology Assessment International’s 6th Latin America Policy Form, held in 2021, was to explore the implementation of deliberative processes in the framework of health technology assessment (HTA) and how agencies in the region could involve stakeholders in this process.

This paper is based on a preparatory survey, a background document, and the deliberative work of participants at the virtual Forum conducted in 2021. There were ninety-one participants in the open session and fifty-two in the closed sessions, representing twelve countries and diverse areas of the health sector.

While there are mechanisms in most countries in Latin America to consider stakeholder involvement to some degree, it remains reduced or limited to a consultative role, making true participative involvement rare. There are significant barriers and structural and contextual limitations that have impeded or slowed progress toward deliberative processes. Relatively low levels of institutionalization and knowledge about HTA, as well as the lack of trust among stakeholders are important challenges. This situation has impacted health systems by diminishing the legitimacy of decisions and the very structures and processes of HTA.

The Forum’s broad group of participants identified barriers, facilitators, and recommendations to improve the use of deliberative processes in Latin America to foster improved fairness and reasonableness in HTA and decision making.

The Pharmaceutical Benefits Advisory Committee (PBAC) is an independent expert body that recommends new technologies for listing on the Pharmaceutical Benefits Scheme. Its decision-making process is evidence-based and considers a technology's clinical effectiveness, safety, and cost-effectiveness compared with other technologies. Since 2014, the PBAC has formally taken into account input from those impacted by the technology via an online consumer comments portal and has also reported on received comments in the Public Summary Documents (PSDs). Comments are welcomed from those whose health the technology is trying to improve, as well as carers, clinicians, and organizations. Our objective was to analyze and review consumer comments in the PBAC's decision-making process.

We extracted information about consumer comments from the PBAC PSDs from 2014–9. We conducted simple descriptive analyses.

Our findings reveal that two thirds of all submissions did not receive a single consumer comment. Of the remaining third, eight submissions (less than 1 percent) had a substantial number of consumer comments (>500). For these technologies, multiple submissions were required before a recommendation was issued. Submissions spanned multiple therapeutic areas, the therapeutic areas with the most consumer comments were genetic disease, pediatrics, and oncology.

In the light of our review, we have identified limitations to the current consumer comments process, and after an examination of the processes of other comparable health technology assessment agencies, we have identified a number of improvements that could be made to the PBAC's process to increase consumer engagement.

Although patient and carer involvement in research is well-developed in many countries, this area has been largely overlooked in South-East European countries.

To explore experiences of patients participating in newly set up lived experience advisory panels (LEAPs) within a European Commission funded, large-scale, multi-country mental health research project that focused on improving treatment of individuals with psychosis.

Twenty-one mental health patients were individually interviewed across five countries: Bosnia and Herzegovina, Kosovo, Montenegro, North Macedonia and Serbia. Topic guides covered the experience of participating in LEAPs and their sustainability. Data were analysed by framework analysis.

Seven themes were identified about participating in LEAPs: predominantly positive evaluation, high levels of participant motivation, therapeutic benefits for participants, few challenges, various future perspectives, positive appraisal of the research project and mixed reflections on mental healthcare. Overall, patients’ experiences were positive and enabled them to feel empowered. Patients expressed interest in remaining involved in advisory panels. Additionally, they felt that they could potentially contribute to the work of non-government organisations.

This study is among the few studies exploring patient participation in research projects, and the first such study conducted in South-East European countries. Patients are highly motivated for this engagement, which has the potential to empower them to take on new social roles. Significant efforts at the national level are needed in each country, to make patient involvement in research standard practice.

Accounts of patient experiences are increasingly used in health technology assessment (HTA) processes. However, we know little about their impact on the decision-making process. This study aims to assess the level and the type of impact of patient input to highly specialised technologies (HSTs) and interventional procedures (IPs) guidance at the National Institute for Health and Care Excellence (NICE).

A questionnaire was developed to capture quantitative and qualitative data on the amount and type of impact of patient input into NICE HTAs. It was completed by committee members of the guidance-producing programs after a discussion of the considered topics. The data were analyzed by topic and overall, for each program, and compared across programs.

Patient input was assessed on ten pieces of HST guidance published between January 2015 and November 2019, and on twenty-six pieces of IP guidance scoped between February 2016 and October 2018. A total of 96 responses were collected for HST and 440 for IP. The level of impact of patient input was higher for HST than for IP. For HST, no respondents stated that it had no impact, whereas in IP, 35 percent of respondents did. The most common types of impact found for HST and IP were that it helped interpret the other evidence and that it provided new evidence.

The impact of patient input is not necessarily explicit in changing recommendations, but it provides context, reassurance, and new information to the committee for the decision-making process in HTAs.

The Patient-Centered Outcomes Research Institute (PCORI) horizon scanning system is an early warning system for healthcare interventions in development that could disrupt standard care. We report preliminary findings from the patient engagement process.

The system involves broadly scanning many resources to identify and monitor interventions up to 3 years before anticipated entry into U.S. health care. Topic profiles are written on included interventions with late-phase trial data and circulated with a structured review form for stakeholder comment to determine disruption potential. Stakeholders include patients and caregivers recruited from credible community sources. They view an orientation video, comment on topic profiles, and take a survey about their experience.

As of March 2020, 312 monitored topics (some of which were archived) were derived from 3,500 information leads; 121 met the criteria for topic profile development and stakeholder comment. We invited fifty-four patients and caregivers to participate; thirty-nine reviewed at least one report. Their perspectives informed analyst nominations for fourteen topics in two 2019 High Potential Disruption Reports. Thirty-four patient stakeholders completed the user-experience survey. Most agreed (68 percent) or somewhat agreed (26 percent) that they were confident they could provide useful comments. Ninety-four percent would recommend others to participate.

The system has successfully engaged patients and caregivers, who contributed unique and important perspectives that informed the selection of topics deemed to have high potential to disrupt clinical care. Most participants would recommend others to participate in this process. More research is needed to inform optimal patient and caregiver stakeholder recruitment and engagement methods and reduce barriers to participation.

This paper aims to describe the development of a flowchart to guide the decisions of researchers in the Spanish Network for Health Technology Assessment of the National Health System (RedETS) regarding patient involvement (PI) in Health Technology Assessment (HTA). By doing so, it reflects on current methodological challenges in PI in the HTA field: how best to combine PI methods and what is the role of patient-based evidence.

A decisional flowchart for PI in HTA was developed between March and April 2019 following an iterative process, reviewed by the members of the PI Interest Group and other RedETS members and validated during an online deliberative meeting. The development of the flowchart was based on a previous methodological framework assessed in a pilot study.

The guidelines on how to involve patients in HTA in the RedETS were graphically represented in a flowchart. PI must be included in all HTA reports, except those that assess technologies with no relevant impact on patients’ experiences, values, and preferences. Patient organizations or expert patients related to the topic of the HTA report must be identified and invited. These patients can participate in protocol development, outcomes' identification, assessment process, and report review. When the technology assessed affects in a relevant way patient experiences, values, and preferences, patient-based evidence should be included through a systematic literature review or a primary study.

The decisional flowchart for PI in HTA contributes to the current methodological challenges by proposing a combination of direct involvement and patient-based evidence.