Exploration of the visibility of patient input in final recommendation documentation for three health technology assessment bodies.

Health technology assessment (HTA) recommendations informed by patient concerns are seen to ensure democracy and legitimacy. We explored how written and oral patient involvement in two HTAs was reported on in publicly available final recommendations and discussion summaries of appraisal committees from three HTA bodies. We aimed to gain insights into how patient input was utilized by appraisal committees to better understand the goals of patient involvement and how these are being achieved. In each of the three HTA bodies, templated submission questionnaires provide a formal process for seeking written patient group input. Additionally, the National Institute for Health and Care Excellence (NICE) selects patient experts to provide a templated submission and attend appraisal committee meetings. For Scottish Medicines Consortium (SMC), a patient advocate and clinician combined meeting (PACE) discussed the cancer drug, referred to in the final recommendation. The discussion summaries of all appraisal committees contained references to patient involvement. Where two mechanisms for patient involvement were provided, oral input from the expert patients and PACE were more clearly documented than information from written patient group submissions. NICE reports focused on the perspective of the patient expert. The SMC report highlighted feedback from the PACE throughout. We suggest that the lack of clear reporting on the use of patient group input in deliberations and therefore accountability to patient groups limits progress in patient involvement in HTA. Patient groups may therefore not have a clear understanding of what information they can best provide to inform deliberations, and in reporting back to members.

The Cochrane Library review titles that are important to users of health care, a Cochrane Consumer Network project.

BACKGROUND: The Cochrane Consumer Network is an internet-based community of international users of health care contributing to the work of The Cochrane Collaboration, whose mission is to inform healthcare decision making through development of systematic reviews of best evidence on healthcare interventions. OBJECTIVE: To prioritize existing review titles listed on The Cochrane Library from a healthcare user perspective, with particular emphasis on patients, carers and health consumers. DESIGN: An online survey was developed and after piloting was made available internationally. The broad dissemination strategy targeted Consumer Network members and Cochrane Review Group editorial staff to identify champions who notified patient support groups and participated in snowballing. The first part of the survey defined criteria that could be applied to review titles and asked survey respondents to rank them. The second part asked respondents to select a health area and prioritize review titles that were of importance to them. Each health area corresponded to a Cochrane Review Group. RESULTS AND DISCUSSION: Sufficient responses were obtained from 522 valid responses to prioritize review topics in 19 health areas. A total of 321 respondents completed the titles assessment. The types of prioritized interventions were determined by the health area. An important observation was the emphasis on lifestyle and non-medication therapies in many of the included health areas. The clearest exception to this broad observation was where acute care is required such as antibiotics for acute respiratory tract and HIV-associated infections and for cardiac conditions. For some cancers, advanced cancer interventions were prioritized. The most important criteria were for the title to convey a clear meaning and the title conveyed that the review would have an impact on health and well-being. The least important criteria were that the topic was newsworthy or prioritized in the healthcare system. CONCLUSION: This project was able to identify priority Cochrane review topics for users of health care in 19 of the 50 areas of health care covered by The Cochrane Collaboration. Reviews addressing lifestyle and non-medical interventions were strongly represented in the prioritized review titles. These findings highlight the importance of developing readable, informative lay summaries to support evidence-based decision making by healthcare users.

Civil society and medical product access in Africa: Lessons from COVID-19.

Understanding health as a human right creates a legal obligation on countries to ensure access to timely, acceptable, and affordable health care. We highlight the importance of a meaningful role for civil society in improving access to well-regulated quality medical products in Africa; to support and be part of a regional social contract approach following the access issues that have been particularly evident during the COVID-19 pandemic. We argue that African communities have a clear participatory role as important stakeholders in the regulatory lifecycle. Solidarity is important for a cohesive approach as formal government healthcare infrastructure may be minimal for some countries, with little training of communities available for disease management and insufficient money to fund people to organise and deliver health care. Some of the issues for civil society engagement with multi-stakeholders, and possible mitigating strategies, are tabulated to initiate discussion on facilitators and concerns of governments and other stakeholders for meaningful participation by patients, communities and civil society within a regional regulatory lifecycle approach. Solidarity is called for to address issues of equity, ethics and morality, stigmatisation and mutual empowerment - to sustainably support the region and national governments to develop greater self-sufficiency throughout the regulatory lifecycle. By creating a participatory space, patients, communities and civil society can be invited in with clear missions and supported by well-defined guidance to create a true sense of solidarity and social cohesion. Strong leadership coupled with the political will to share responsibilities in all aspects of this work is key.

Where National Medicines Policies Have Taken Us With Patient Involvement and Health Technology Assessment in Africa.

The Covid-19 pandemic has highlighted global knowledge about, but lack of equitable access to, life-changing medicines, and other innovative medical products by populations in African low and middle income countries. The World Health Organization (WHO) and other international non-profit foundations and organizations are constantly striving to address inequity. In the 1970s, WHO initiated a regularly updated essential medicines list, together with the concept of national medicines policies (NMPs) to ensure access and availability, affordability, rational, and effective use of medicines which are considered essential in addressing predominant population health issues and disease burden. We studied the NMPs of Ghana, South Africa, Uganda and Zimbabwe to highlight some of the important issues that these countries experience in the safe and effective use of medical products. Thailand is an example of how health technology assessment (HTA) can provide a country with an internationally supported, clearly defined and transparent process to broaden access to medicines and services. These medical services can add considerable value in accordance with local values and priorities. Involvement of civil society adds democratic legitimacy to such processes. Community health workers and patient advocacy groups are important in raising awareness and knowledge of safety issues and the effective use of quality medicines. They can apply pressure for increased funding to improve access to healthcare. Medicines and services that contribute to supported self-care are of benefit in any setting. Joint efforts across African countries such as with the African Medicines Agency are important in addressing some of the major health issues.

Patients and public are important stakeholders in health technology assessment but the level of involvement is low - a call to action.

BACKGROUND: Health technology assessment (HTA) agencies have an important role in the evaluation and approval of new technologies. They determine their value within a health system so to promote equitable, quality care with available healthcare resources. Many HTA agencies have some mechanism for involving patients in their processes, but there is great variability and an absence of comprehensive, robust practices for involvement. The accelerating pace of medical innovation creates a need to improve the depth and breadth of patient involvement in the HTA process. MAIN BODY: In this 'Call to action', we present ideas from three HTA expert commentaries calling for collaborative learning and to share innovative ideas for changes in HTA. We also draw on examples of HTA agencies creatively pursuing this goal. We propose a 'Call to action' for HTA stakeholders to undertake serious dialogue with patient advocates aimed at creating shared goals. HTA agencies can use these goals to ensure meaningful patient involvement at every step of the HTA process. Five elements are explored. In 'Recognizing the value of shared purpose', we highlight examples of HTA agencies that have patients working in partnership with medical practitioners and HTA staff. Results include improved processes that instil confidence. 'Committing to patient involvement as part of HTA culture' highlights several initiatives aimed at changes in HTA organisational culture to be more inclusive of patients. In 'Aligning patient and HTA goals' we cite work in Belgium and New Zealand which places a greater emphasis on quality of life rather than life expectancy and cost-effectiveness. By 'Integrating patient involvement at every step of the HTA process' patients can make vital contributions at every stage of the HTA process. We provide two examples of where HTA agencies have successfully involved patients early in the process in order to broaden the scope of evaluations. 'Developing a common language and working together' can support transformative dialogue through 'unified language'. CONCLUSION: The authors of this commentary ask that agencies and stakeholders involved in HTA take up this call to work together for visionary and transformative elevation of the voice of patients in HTA worldwide.

Can We Afford to Exclude Patients Throughout Health Technology Assessment?

Health technology assessment (HTA) is intended to determine the value of health technologies and, once a technology is recommended for funding, bridge clinical research and practice. Understanding the values and beliefs expressed by patients and health professionals can help guide this knowledge transfer and work toward managing the expectations of end users. We gathered patient and patient group leader experiences to gain insights into the roles that patients and patient advocacy groups are playing. We argue that through partnerships and co-creation between HTA professionals, researchers and patient advocates we can strengthen the HTA process and better align with service delivery where person-centered care and shared decision making are key elements. Patient experiences and knowledge are important to the democratization of evidence and the legitimacy of HTAs. Patient preference studies are used to balance benefits with potential harms of technologies, and patient-reported outcomes (PROs) can measure what matters to patients over time. A change in culture in HTA bodies is occurring and with further transformative thinking patients can be involved in every step of the HTA process. Patients have a right to be involved in HTAs, with patients' values central to HTA deliberations on a technology and where patients can provide valuable insights to inform HTA decision-making; and in ensuring that HTA methodologies evolve. By evaluating the implementation of HTA recommendations we can determine how HTA benefits patients and their communities. Our shared commitment can positively effect the common good and provide benefits to individual patients and their communities.

Making community voices heard in a research-health service alliance, the evolving role of the Community Advisory Group: a case study from the members' perspective.

BACKGROUND: The Melbourne Genomics Health Alliance (the Alliance) is a collaboration of leading hospitals, research and academic organisations, supported by its member organisations and the Victorian Government. The Alliance was set up by its members in 2013 to steer the translation of genomics, making it an integral part of health care in Victoria, Australia. The Community Advisory Group (CAG) was formed soon after, to give input and advice across the program. This was to ensure consideration of community values, perspectives and priorities, and knowledge translation for patient care. The CAG was charged with providing a strong community voice for the duration of the program. Appointed members were experienced consumer advocates with developed connections to the community. MAIN BODY: The Alliance progressed from an initial Demonstration Project (2013-2015) to a multifaceted program (2016-2020). The CAG worked strategically to help address complex issues, for example, communication, privacy, informed consent, ethics, patient experience, measurement and evaluation standards and policies, data storage and re-use of genomic data. Many aspects of translating genomics into routine care have been tackled, such as communicating with patients invited to have genomic testing, or their caregivers, and obtaining informed consent, clinical questions across 16 areas of health care, training and education of health and laboratory professionals, genomic data management and data-sharing. Evidence generated around clinical utility and cost-effectiveness led to government funding of testing for complex genetic conditions in children. CONCLUSION: The CAG activities, recorded in a CAG-inspired Activity register, span the full spectrum of information sharing and consultation to co-design and partnership. The CAG were involved at multiple levels of participation and in all tiers of activity including governance, development of policies and procedures, program planning and evaluation. Working relationships were built up and a level of trust instilled to advance the Alliance work program in ensuring an effective patient-care model of delivery of genomics. CAG input into project deliverables has been tangible. Less tangible contributions included presentations at external meetings and conferences, direct interactions at meetings with Alliance members, interactions with visitors and external experts, taking part in consultations with experts, state and federal government.

Melbourne Genomics Health Alliance was established in 2013 to steer genomics into health care in Victoria, Australia. The Community Advisory Group (CAG) was formed soon after to provide advice and insights from the patient perspective. The CAG has added value to the Alliance's complex research-to-clinical service program of work over eight years to date. Following an explanation of the program, the CAG members identified priority areas and mechanisms for their involvement. Areas that members were involved in included: communication, visual identity and website, patient portal and its evaluation, information management, consent processes, laboratory requirements, tools for patient experience and quality of life measures, predictive health issues study, storage and sharing of data, databases, CAG Communication Plan, the Patient Guide, role with Victorian Government Department of Health and Human Services, implementation plan, workshop to upskill patient advocates, financial and strategic planning. Members also presented on the role of the CAG at conferences and symposia. The balanced, trusting relationship that developed between the CAG, the Program Team and its governance structure was of great value to and an achievement for the Alliance. CAG input into project deliverables and impact was recorded in a CAG inspired Activity Register and has been very tangible. Their less tangible contribution to the project is also important. Contributions included presentations at external meetings, direct interactions at annual meetings with Alliance members, interactions with visitors and external experts, taking part in consultations with experts, state and federal government. These provided opportunities to influence mindsets.

Patient advocate perspectives on involvement in HTA: an international snapshot.

PLAIN ENGLISH SUMMARY: A number of health technology assessment (HTA) organisations have developed processes to engage patients in the assessment of new health technologies such as pharmaceuticals, diagnostic tests, devices or medical procedures. Typically, this involves the HTA agency providing an opportunity for patient advocates and their patient organisations (support groups for patients with a specific disease or condition) to provide submissions detailing experiences with the disease and the health technology that is being assessed. While some literature exists about how HTA agencies view the engagement of patients in the HTA process, it is not yet clear how the patient advocates and patient organisations themselves view this engagement. To answer this question, we surveyed the views of patient advocates who were members of patient organisations known to be engaged in the process of HTA or evidence-based practice. Snowballing - that is, passing on the survey invitation from individuals invited to take part in the survey to other individuals - occurred in one of the countries. The responses in this country provided a very useful comparison between the views of people who were appointed as the 'patient representatives' on an HTA committee with those who contributed input as part of the general patient organisation engagement process. Our findings identify gaps in understanding of the purpose of patient involvement and whether patient organisations felt their input made a difference, the information and support provided, and if and how feedback is given to the patient organisations. Our work can help inform further research as well as continuing improvements in HTA patient engagement processes. ABSTRACT: Background Patient involvement in health technology assessment (HTA) processes is becoming more frequent. However, it is not clear how patient advocates and their disease-based patient organisations that are involved in HTA view their involvement. We report on the results of an international survey of patient advocates and members of patient organisations about their experiences and perceptions of that involvement. Methods A 16-question survey was sent out to patient advocates and members of patient groups known to be involved in HTA processes or evidence-based practice. The survey consisted of open-ended questions focusing on respondent characteristics, stage and nature of involvement, support from HTA agencies for involvement, purpose of involvement, feedback on involvement, and whether the respondents felt that their input made a difference. Results Of 16 individuals who received the survey, 15 responded. Three, from Italy, Israel and Japan, were not involved in HTA in their country. Respondents from the following countries reported involvement in HTA processes: Canada, England, Scotland, and Wales, The Netherlands, Australia, Taiwan. The respondents indicated that HTA agencies reach out to them either actively or passively, and that their involvement is often at the appraisal stage of HTA. Typically, they reported involvement as either participants in committees or providers of submissions to HTA agencies. A wide range of approaches to supporting patient involvement by the HTA agencies was identified by respondents - including personal and telephone support, online resources, training and provision of information - but the level and type of support reported was uneven across jurisdictions. Not all respondents were clear on the purpose of their involvement in HTA, although some were able to cite specific examples of how their input made a difference; members of an HTA decision-making committee appeared to have a better understanding and were able to give examples. Feedback from HTA agencies to the patient groups on their submissions is often not provided. Conclusions Although considerable progress has been made in terms of engaging patients and patient groups in HTA, gaps remain in how involvement is supported, including facilitating involvement, clarity on roles, two-way flow of information, and methods for enhancing communication between patient organisations and HTA agencies.

Strengthening international patient advocacy perspectives on patient involvement in HTA within the HTAi Patient and Citizen Involvement Interest Group - Commentary.

PLAIN LANGUAGE SUMMARY: Health Technology Assessment (HTA) is an evidence-based decision-making process, focusing on evaluating health technologies for funding within a healthcare system. 'Health technologies' include medications, medical devices, diagnostics, medical procedures and services. Health Technology Assessment international (HTAi) is a global society for those who produce, use, participate in, or encounter HTA. The HTAi Secretariat supports special interest groups within HTAi. One such special interest group, the Patient and Citizen Involvement in HTA Interest Group (PCIG), focuses on strengthening patient involvement in HTA. PCIG's members are from HTA agencies, research, industry, and patient organisations. We describe the steps PCIG has taken to form an international panel of patient advocates (the Patient Panel) as an autonomous group within its governance structure that reports directly to the PCIG Steering Committee. The Patient Panel was established in order to strengthen meaningful patient involvement in HTA by working co-productively with other PCIG members to develop resources accessible to patient organisations. Patient advocates known to be active within HTA in their own countries were invited to form the inaugural group, with one person appointed to chair the group for the first year. Documentation had been prepared to inform potential members and set out potential roles as seen by the Steering Committee. The appointed Patient Panel is defining its own terms of reference, goals and objectives. The Panel Chair is a member of the PCIG Steering Committee, and is mentored by the retiring PCIG chair. A registry of activities is part of monitoring and evaluation. ABSTRACT: Background Health Technology Assessment (HTA) is an evidence-based decision-making process, focusing on evaluating health technologies for funding within a healthcare system or medical insurance system. 'Health technologies' are understood broadly, and include medications, medical devices, diagnostics, medical procedures and services. Health Technology Assessment international (HTAi) is a global society for those who produce, use, participate in, or encounter HTA. The HTAi Secretariat supports special interest groups within HTAi, including the Patient and Citizen Involvement in HTA Interest Group (PCIG), which focuses on strengthening patient involvement in HTA. PCIG's members come from HTA agencies, research, industry, and patient organisations. Main body We describe the steps the PCIG has taken to form an international panel of patient advocates as an autonomous group within its governance structure, reporting directly to the PCIG Steering Committee. Patient advocates known to be active within HTA in their own countries were invited to form the inaugural group. One person was invited to chair the Panel for the first year of its operation, based on the candidate's experience of working across diseases and countries, and being new to HTAi. Documentation was prepared to inform potential members of the Panel and set out potential roles as seen by the Steering Committee. The Panel came into being in March 2016 and is now setting out its own working principles, goals and objectives. The Panel Chair is a member of the PCIG Steering Committee, and is mentored by the previous PCIG chair. A registry of activities has been set up as part of monitoring and evaluation. The Patient Panel is intended to provide a focus within the PCIG for patient advocates to work among themselves and co-productively through the PCIG Working Groups to develop mutual understanding and strengthen meaningful patient involvement in HTA internationally. Patient advocates can benefit from a clear understanding the evidence requirements within HTA and how information can be effectively presented by patient groups to decision-making bodies. Conclusion The HTAi Patient and Citizen Involvement Interest Group has set up a Patient Panel consisting of patient advocates. The intent is to work co-productively to advance patient involvement in HTA.