Demonstrating the influence of HTA: INAHTA member stories of HTA impact.

Introduction:Health technology assessment (HTA) agencies wish to ensure the impact of their HTAs. HTA impact assessment measures the influence of a HTA on decision-making and downstream to patient outcomes. Despite their potential to provide insights, the use of impact assessment frameworks by HTA agencies is limited. Understanding the underlying mechanisms of adopting HTA impact assessment frameworks is therefore important. Using a social cognitions lens, this study aims to provide insights into the enabling and hindering factors associated with the assessment of HTA impact by INAHTA members.Methods:Using an interpretive description design, this cross-sectional study used semi-structured interviews of INAHTA members to gain insight into attitudes, social support, self-efficacy, barriers, and intentions towards HTA impact assessment. Transcriptions were analyzed using a social cognitions lens by two researchers using a constant comparative method to identify themes.Results:Twenty-six of forty-seven INAHTA members participated. Preliminary results showed that interviewees most often perceived support for assessing impact from their ministry of health or from agency staff. Most interviewees noted challenges to measuring impact at the right time and a lack of human resources, methods, and tools as internal barriers. A lack of transparency and a limited impact assessment culture were perceived as the main external barriers. Interviewees reported feeling fairly confident in overcoming internal barriers, but were less confident in overcoming external barriers. Providing feedback for improvement to HTA processes and making achievements visible were the most frequently reported advantages of assessing impact, whereas its time consuming nature was the biggest disadvantage.Conclusions:This is the first study to use a social cognitions model to understand HTA impact assessment. Although the results of this convenience sample need to be interpreted with caution, they contribute knowledge on factors that facilitate and hinder agencies in the assessment of impact and illuminate opportunities for developing effective strategies to support HTA agencies in this area.

Plain English summaryA 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.BackgroundPatient 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.MethodsA 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.ResultsOf 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.ConclusionsAlthough 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.

Health technology assessment (HTA) agencies assess evidence to support decision making about which technologies to provide and pay for in the health system. HTA impact is understood as the influence that HTA report findings can have in the health system, including impacts on reimbursement decisions, changes to health outcomes, or broader system or societal impacts. The International Network of Agencies for Health Technology Assessment (INAHTA) is a global network of publicly funded HTA agencies. INAHTA's mission, in part, is to advance the impact of HTA to support reimbursement decisions and the optimal use of health system resources. Each year, INAHTA awards the David Hailey Award for Best Impact Story to the member agency that shares the best story, as voted by fellow members, about HTA impact. The impact story sharing program in INAHTA contributes to a deeper understanding of what works well (or not so well) in achieving HTA impact. This paper provides six impact stories from agencies that were finalists for the 2021 and 2022 David Hailey Impact Award for Best Impact Story: the Institut national d'excellence en santé et en services sociaux, the Malaysian Health Technology Assessment Section, Ontario Health, the Center for Drug Evaluation, the National Institute for Health and Care Excellence, and Health Technology Wales. These stories demonstrate that HTA agencies can, in differing ways, effectively support governments in their efforts to place evidence at the centre of decision making.

The objectives of the study were to establish a benchmarking tool to collect metrics to enable increased clarity regarding the differences and similarities across health technology assessment (HTA) agencies, to assess performance within and across HTA agencies, identify areas in the HTA processes in which time is spent and to enable ongoing performance improvement. Common steps and milestones in the HTA process were identified for meaningful benchmarking among agencies. A benchmarking tool consisting of eighty-six questions providing information on HTA agency organizational aspects and information on individual new medicine review timelines and outcomes was developed with the input of HTA agencies and validated in a pilot study. Data on 109 HTA reviews from five HTA agencies were analyzed to demonstrate the utility of this tool. This study developed an HTA benchmarking methodology, comparative metrics showed considerable differences among the median timelines from assessment and appraisal to final HTA recommendation for the five agencies included in this analysis; these results were interpreted in conjunction with agency characteristics. It is feasible to find consensus among HTA agencies regarding the common milestones of the review process to map jurisdiction-specific processes against agreed metrics. Data on characteristics of agencies such as their scope and remit enabled results to be interpreted in the appropriate local context. This benchmarking tool has promising potential utility to improve the transparency of the review process and to facilitate both quality assurance and performance improvement in HTA agencies.

To develop a health technology assessment (HTA) adaptation toolkit and glossary of adaptation terms for use by HTA agencies within EU member states to support them in adapting HTA reports written for other contexts. The toolkit and glossary were developed by a partnership of 28 HTA agencies and networks across Europe (EUnetHTA work package 5), led by the UK National Coordinating Centre for Health Technology Assessment (NCCHTA). Methods employed for the two resources were literature searching, a survey of adaptation experience, two rounds of a Delphi survey, meetings of the partnership and drawing on the expertise and experience of the partnership, two rounds of review, and two rounds of quality assurance testing. All partners were requested to provide input into each stage of development. The resulting toolkit is a collection of resources, in the form of checklists of questions on relevance, reliability and transferability of data and information, and links to useful websites, that help the user assess whether data and information in existing HTA reports can be adapted for a different setting. The toolkit is designed for the adaptation of evidence synthesis rather than primary research. The accompanying glossary provides descriptions of meanings for HTA adaptation terms from HTA agencies across Europe. It seeks to highlight differences in the use and understanding of each word by HTA agencies. The toolkit and glossary are available for use by all HTA agencies and can be accessed via www.eunethta.net/. These resources have been developed to help HTA agencies make better use of HTA reports produced elsewhere. They can be used by policy-makers and clinicians to aid in understanding HTA reports written for other contexts. The main implication of this work is that there is the potential for the adaptation of HTA reports and, if utilised, this should release resources to enable the development of further HTA reports. Recommendations for the further development of the toolkit include the potential to develop an interactive web-based version and to extend the toolkit to facilitate the adaptation of HTA reports on diagnostic testing and screening.

Background: Evidence-informed deliberative processes (EDPs) were recently introduced to guide health technology assessment (HTA) agencies to improve their processes towards more legitimate decision-making. The EDP framework provides guidance that covers the HTA process, ie, contextual factors, installation of an appraisal committee, selecting health technologies and criteria, assessment, appraisal, and communication and appeal. The aims of this study were to identify the level of use of EDPs by HTA agencies, identify their needs for guidance, and to learn about best practices.Methods: A questionnaire for an online survey was developed based on the EDP framework, consisting of elements that reflect each part of the framework. The survey was sent to members of the International Network of Agencies for Health Technology Assessment (INAHTA). Two weeks following the invitation, a reminder was sent. The data collection took place between September-December 2018. Results: Contact persons from 27 member agencies filled out the survey (response rate: 54%), of which 25 completed all questions. We found that contextual factors to support HTA development and the critical elements regarding conducting and reporting on HTA are overall in place. Respondents indicated that guidance was needed for specific elements related to selecting technologies and criteria, appraisal, and communication and appeal. With regard to best practices, the Canadian Agency for Drugs and Technologies and the National Institute for Health and Care Excellence (NICE, UK) were most often mentioned. Conclusion: This is the first survey among HTA agencies regarding the use of EDPs and provides useful information for further developing a practical guide for HTA agencies around the globe. The results could support HTA agencies in improving their processes towards more legitimate decision-making, as they could serve as a baseline measurement for future monitoring and evaluation.

IntroductionFindable, structured, and understandable data from health technology assessment (HTA) reports is the core of HTA policy research. Available databases with this information, such as the International Network of Agencies for Health Technology Assessment (INAHTA) database, may be incomplete and their common manual data collection is time-consuming. Automated data extraction may offer a solution by creating a standardized, real-time-updating, comprehensive, open-access HTA database.MethodsIn this research, we explore the possibilities of automated data extraction in the context of creating a standardized and comprehensive HTA policy research database. Data points were extracted from publicly available guidance reports of the National Institute for Health and Care Excellence (NICE) using different text extraction techniques such as natural language processing (NLP) and generative pre-trained transformers (GPTs). Future efforts are aiming to expand the database to other HTA bodies and link it to the European Medicines Regulatory Database (EMRD) that is also being developed.ResultsPreliminary results of our research show that it is possible to use existing text extraction techniques to extract relevant information from publicly available HTA recommendations. Scaling the system to include more HTA bodies and data points is challenging as extraction based on document structure is complicated by heterogeneity in document structure within HTA bodies and between HTA bodies. Future results will focus on finding the best data extraction approach for each data point and on validating the system.ConclusionsUsing automated data extraction to extract data from HTA reports can be a viable option for creating a comprehensive database that can be used to enhance comparative HTA policy research. Challenges remain in scaling the system to include more HTA bodies and data points. Results regarding best-performing extraction techniques and data validation of the system are expected soon.

IntroductionAfter surveying its members on ethical issues (2003), the International Network of Agencies for Health Technology Assessment (INAHTA) mandated its Ethics Working Group (2005) to reflect on the role of health technology assessment (HTA) organizations in meeting social expectations. Some aspects of these have since been clarified by two studies addressing either the official position of INAHTA's members or the publication authors. An international survey was carried out on the perception of HTA professionals’ expectations when producing HTA reports: how to fulfil HTA's social role, which value judgments should be made explicit and what should be the status of ethical analysis.MethodsA twenty-two question, web-based, anonymous survey was devised from our recent systematic review on the integration of ethics into HTA and carried from April to July 2018. The information on 328 HTA agencies/contact persons from seventy-five countries was collected from the website of INAHTA, Health Technology Assessment International (HTAi), the European Network for Health Technology Assessment (EUnetHTA), EuroScan International Network, the HTA Network of the Americas (RedETSA) and the HTA Network of Asia (HTAsiaLink), a 2015 World Health Organization survey, HTAi members, and our local HTA network (Québec, Canada).ResultsEighty-nine participants completed and submitted a finalized survey for a 27 percent participation rate representing thirty-three countries. Regarding how the HTA reports should fulfil their social role, our results showed that over 84 percent of the respondents agreed upon the necessity to address it to decision makers, patients and citizens. At a lower and more variable level, the same result was found about the necessity to make value judgements explicit in different sections of the report, including ethical analysis. This contrasts with the variability of responses obtained on the status of ethical analysis although an agreement on the expertise required was observed. Variability in the usefulness of patient, public or stakeholder participation was observed.ConclusionsAt the dawn of this decade, this study reveals high expectations on context-dependent decisions in HTA: the necessity to integrate the ‘explicitation’ of value judgements and systematic ethical analysis to fulfil HTA's social role.

Recommendations by countries' health technology assessment (HTA) agencies are used to decide which new therapies warrant the allocation of limited health-care resources to make them available through publicly funded health systems. This process is of public health importance for balancing the dual aims of optimising patient outcomes while ensuring financial sustainability. We evaluated which factors affect HTA outcomes and the time to positive HTA outcome, focussing on the role of clinical benefit evaluated with the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS). In this retrospective analysis, data were extracted from publicly available HTA reports and related sources from six country settings and their respective HTA agencies (Australia, Canada, England, France, the Canadian province of Quebec, and Scotland). We evaluated new cancer medicines for treating solid tumours in a non-curative setting with published ESMO-MCBS scores and that had been assessed by at least three HTA agencies between Jan 1, 2011, and Dec 31, 2020. Using ESMO-MCBS score as an independent variable, we did descriptive and multivariable regression analyses to evaluate: (1) factors associated with the time between marketing authorisation and positive (unrestricted [List] and restricted [List with Constraints]) HTA outcome; and (2) factors associated with HTA outcomes. 67 medicine-indication pairs used in non-curative settings were identified, totalling 360 HTA submissions (medicine-indication-country triplets) reviewed by the six HTA agencies. Factors significantly associated with a reduced interval between marketing authorisation and a positive (unrestricted or restricted) HTA outcome included a high ESMO-MCBS score (ie, 4 or 5, vs a low or average score of 1-3; hazard ratio [HR] per 1 month increment 1·42 [95% CI 1·11-1·81], p=0·0055), parallel review (vs standard marketing authorisation process; HR 1·69 [1·13-2·54], p=0·011), having a risk-sharing agreement or special funding arrangements (vs no funding agreement, HR 4·62 [95% CI 2·51-8·51], p<0·0001, and HR 4·16 [2·03-8·50], p=0·0001, respectively), and assessment by particular HTA agencies (pan-Canadian Oncology Drug Review vs National Institute for Health and Care Excellence [NICE], HR 2·82 [1·68-4·75], p=0·0001; and Haute Autorité de Santé vs NICE, HR 5·70 [2·87-11·33], p<0·0001). Accelerated marketing authorisation was significantly associated with a longer time to positive HTA outcome (vs standard authorisation process; HR 0·70 [95% CI 0·51-0·95], p=0·024). Positive HTA outcomes (both unrestricted and restricted) were significantly associated with a high ESMO-MCBS score (vs low or average ESMO-MCBS score; relative risk ratio [RRR] 14·10 [95% CI 3·54-56·20], p=0·0002, and RRR 4·52 [1·90-10·75], p=0·0006, respectively) and acknowledgment of unmet medical need (vs unmet need not recorded, RRR 22·73 [5·51-93·73], p<0·0001, and RRR 1·87 [1·18-2·97], p=0·0075, respectively). By contrast, positive HTA outcomes (unrestricted and restricted) were inversely associated with uncertainties regarding inputs to economic models informing HTA submissions (vs uncertainties not recorded, RRR 0·28 [0·10-0·78], p=0·014, and RRR 0·45 [0·25-0·82], p=0·010, respectively). Regarding country-relevant effects, inverse associations with positive HTA outcomes (both unrestricted and restricted) were observed for assessment in Quebec (vs England; RRR 1·15×10-6 [1·44×10-7-9·09×10-6], p<0·0001, and RRR 0·33 (0·24-0·46), p<0·0001, respectively) and for assessment in Australia (vs England; RRR 1·78×10-6 [1·04×10-8-3·00×10-4], p<0·0001, and RRR 0·30 [0·15-0·61], p=0·0008, respectively). Several factors informed HTA outcomes for new cancer medicines. A high ESMO-MCBS score, defined as indicating substantial clinical benefit, increased the likelihood of a positive HTA outcome and shortened the interval between marketing authorisation and HTA outcome, and this association was not affected by other variables. Additional factors informing HTA outcomes include evidence uncertainties and unmet medical need. Country-relevant differences exist in the time-to-HTA outcome and the propensity of some countries to achieve positive (restricted or unrestricted) outcomes compared with others. None.

The purpose of this study was to systematically review the process for topic selection by health technology assessment (HTA) agencies around the world to provide the knowledge base for the improvement of topic selection frameworks in HTA agencies. A systematic search was conducted in PubMed and EMBASE to identify papers up to February 2019. Gray literature was identified by screening the Web sites of HTA agencies on the nonprofit member list of the International Network of Agencies for Health Technology Assessment (INAHTA). Data were extracted for each HTA agency and synthesized, with issues including general contextual information about each agency and the process of topic selection. Out of forty-nine nonprofit members of INAHTA, a total of seventeen HTA agencies with a framework for topic selection were identified from twenty-two included papers/documents. Multiple criteria were used for topic selection in all frameworks and agencies undertook multiple steps, which could include the specification of criteria for topic selection, identification of topics, short listing of potential topics, scoping of potential topics, scoring and ranking of potential topics, and deliberation and decision on final topics for HTA. Shortcomings were found in relation to methods of scoring and ranking as well as lack of monitoring and the evaluation of the process. Our study provides insights into the current practice of topic selection in HTA agencies. Multiple criteria decision analysis methodology appears highly relevant to these processes. A consensus approach for the development of methods of topic selection would be valuable for the HTA community.

IntroductionThe world is facing an urgent environmental emergency that calls for ambitious, coordinated action by governments to improve humankind’s relationship with nature. Mitigation (reducing pollution) and adaptation (adjusting to pollution) are both necessary, and “NextGen” health technology assessment can play a key role in helping to guide health system decision-makers to achieve environmental sustainability.MethodsIn 2022, the International Network of Agencies for Health Technology Assessment (INAHTA) identified environmental impact assessment (EIA) as an important and urgent topic for a white paper. An international author group (n=10), formed of staff from INAHTA member agencies, wrote the paper. The author group met seven times over 2022 to 2024 to develop the paper, which is based on a literature review and expert opinion of INAHTA members. A second group of members formed an international advisory group (n=11), which reviewed the draft twice. The paper was approved by the INAHTA Board before release on the INAHTA website.ResultsEIA can be useful to help achieve the “green” policy goals of health systems by identifying technologies that are comparatively harmful to the environment. However, health technology assessment (HTA) agencies are subject to the institutional structures and regulations of their local health system that can constrain their ability to independently adapt to incorporate EIA. Nevertheless, HTA agencies are starting to do this with some success, but difficult challenges remain, particularly around lack of data and methods. Looking ahead, raising awareness and working together across the HTA ecosystem will be key to achieving the shared goal of environmental sustainability.ConclusionsThe successful inclusion of EIA in priority setting, assessment, and appraisal, as well as knowledge sharing and dissemination activities of HTA, is a challenge since the operating principles, methods, and data in this area are not yet mature. The successful inclusion of environmental impacts in HTA will require some reconsideration of existing value frameworks and methods.

IntroductionProducing new health technology assessments (HTA) can be a time-consuming process. With finite resources in HTA agencies, limited capacities in countries without formalized HTA processes, and growing interest for lifecycles approaches valuing health technologies; innovative and efficient HTA processes are needed. “Adaptive HTA”, referring to the pragmatic use of HTA methods and existing (HTA) evidence, might offer solutions. We will present the results from a scoping review that mapped existing tools, methods, practices to transfer existing HTAs; and reflect on these findings given our own experiences of adaptation processes in LMICs.MethodsWe undertook a scoping review and systematically searched five electronic databases. Inclusion of articles followed strict in- and exclusion criteria. Data extraction focused on information regarding tools, methods, and practices that could aid the transferability of HTA analysis. Here, HTAs referred to full-HTAs and other HTA products, as partial HTAs, economic evaluations, or systematic reviews. Lastly, we mapped the possible overarching factors that can affect transferability.ResultsThe search (November 2020) identified 2030 hits, of which 19 were included. Most HTA transfers followed five steps that closely resemble a de novo HTA process. The identified transferability tools, often checklists, were merely aids or a “catalyst” for the transfer and provided limited guidance for the whole transfer process. Contrastingly, we identified three frameworks that can support the whole process: European Network for HTA (EUnetHTA) Adaptation Toolkit, TRANSFER framework for systematic reviews, and paper series on systematic reviews for economic evaluations. Lastly, our findings pointed to various challenges and knowledge gaps; especially for transfers in low and middle income countries evidence is limited.ConclusionsThe re-use of existing evidence in HTA reports is not new; and readily part of de novo and adaptive processes. The innovative nature of adaptive HTA comes from its ability to unpack the process of adaptation and transferability. Simultaneously, this scoping review highlighted gaps in existing adaptive methods, and could aid future adaptive HTA process for experienced and new HTA-doers.

IntroductionThe Department of Management and Incorporation of Technologies and Innovation in Health (DGITIS) acts as Conitec's Executive Secretariat. Among its attributions, it promotes the public/patient involvement in the health technology assessment (HTA) process. Recently, Conitec has been working on the inclusion of patient's testimonials about their illness experience in the plenary sessions, that is, the monthly meeting where technologies are assessed.MethodsTo support the action of including patient reporting in Conitec's HTA process, DGITIS developed research on HTA agencies websites worldwide. The main criteria was the inclusion of patients’ reports in their Committee meetings. DGITIS contacted some of these agencies and requested a listserv question to the International Network of Agencies for Health Technology Assessment (INAHTA) members. These findings supported the DGITIS for the inclusion of patient participation in Conitec's meetings, from the selection process to the actual participation.ResultsFor the Conitec's HTA process, the patients’ participation should occur in the prior session to the public consultation, guaranteeing the inclusion of their perspective since the recommendation process beginning. Hence, every demand for incorporation to be discussed at Conitec's meeting should be preceded by a public call for patients with the clinical condition. The DGITIS will also hold preparatory meetings, which will serve as moments for shared construction of knowledge and literacy.ConclusionsThe nomination process, so far, has been grounded as a consensus among the patients. Thus, Conitec acts as a mediator, connecting the involved stakeholders, in a way that they can autonomously organize themselves and indicate the main representative and an alternate one. With the inclusion of the patient's perspective in the Conitec's meeting, another form of patient participation was opened in the HTA process. Therefore, the consolidation of this participation space is feasible and contributes to enrich the Brazilian HTA process.

IntroductionThe Health Technology Assessment (HTA) Network of the Americas (RedETSA) is composed of health ministries, HTA agencies, regulatory authorities, and collaborating centers that exchange valuable cooperation and information to promote the HTA processes in the Americas region. Since its creation, the network has grown significantly, and several milestones have been reached.MethodsA retrospective review of HTA milestones in the region and RedETSA network achievements was conducted, covering its inception at the HTAi annual meeting in Rio de Janeiro, Brazil, in 2011 to the present. The review included: the number of member countries and institutions; milestones in HTA development, implementation, and institutionalization; key Pan American Health Organization (PAHO) resolutions supporting HTA; advances in the Regional Database of Health Technology Assessment Reports of the Americas (BRISA); the number of annual meetings; regional and international cooperation; and capacity building support in the region.ResultsFollowing the approval of the CSP28.R9 resolution on HTAs and Health Decision-Making by the PAHO in 2012 and the WHA67.23 resolution (World Health Organization) in 2014, HTA was implemented or institutionalized in several countries. Since its inception, RedETSA has grown to include 21 member countries in the Americas, represented by 40 institutions. HTA capacity building was supported by 482 scholarships and courses since 2015, as well as 43 webinars by RedETSA. The BRISA database now includes over 3,800 HTA reports. Moreover, RedETSA collaborates with various HTA institutions and initiatives, including HTAi, the International Network of Agencies for HTA (INAHTA), the Spanish Network of Agencies for Assessing National Health System Technologies and Performance (RedETS), RedCritieria (a network initiative of the Inter-American Development Bank), HTAsiaLink, and EuroScan.ConclusionsSignificant progress in HTA has been made in the Americas, with RedETSA playing a key role in these achievements. Challenges remain, including establishing explicit links between HTA and decision-making, consolidating institutional frameworks, increasing local data availability, continuing capacity building efforts, and standardizing HTA reports. Continued cooperation between countries and institutions through network collaboration is essential to address these needs.

Standard outcome sets developed by the International Consortium for Health Outcomes Measurement (ICHOM) facilitate value‐based health care in healthcare practice and have gained traction from regulators and Health Technology Assessment (HTA) agencies that regularly assess the value of new medicines. We aimed to assess the extent to which the outcomes used by regulators and HTA agencies are patient‐relevant, by comparing these to ICHOM standard sets. We conducted a cross‐sectional comparative analysis of ICHOM standard sets, and publicly available regulatory and HTA assessment guidelines. We focused on oncology due to many new medicines being developed, which are accompanied by substantial uncertainty regarding the relevance of these treatments for patients. A comparison of regulatory and HTA assessment guidelines, and ICHOM standard sets showed that both ICHOM and regulators stress the importance of disease‐specific outcomes. On the other hand, HTA agencies have a stronger focus on generic outcomes in order to allow comparisons across disease areas. Overall, similar outcomes are relevant for market access, reimbursement, and in ICHOM standard sets. However, some differences are apparent, such as the acceptability of intermediate outcomes. These are recommended in ICHOM standard sets, but regulators are more likely to accept intermediate outcomes than HTA agencies. A greater level of alignment in outcomes accepted may enhance the efficiency of regulatory and HTA processes, and increase timely access to new medicines. ICHOM standard sets may help align these outcomes. However, some differences in outcomes used may remain due to the different purposes of regulatory and HTA decision‐making.