Bridging the gap: Can International Consortium of Health Outcomes Measurement standard sets align outcomes accepted for regulatory and health technology assessment decision-making of oncology medicines.

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.

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.

A central function of health technology assessment (HTA) agencies is the production of HTA reports to support evidence-informed policy and decision making. HTA agencies are interested in understanding the mechanisms of HTA impact, which can be understood as the influence or impact of HTA report findings on decision making at various levels of the health system. The members of the International Network of Agencies for HTA (INAHTA) meet at their annual Congress where impact story sharing is one important activity. This paper summarizes four stories of HTA impact that were finalists for the David Hailey Award for Best Impact Story.The methods to measure impact include: document review; claims analysis and review of reimbursement status; citation analysis; qualitative evaluation of stakeholders' views; and review of media response. HTA agency staff also observed changes in government activities and priorities based on the HTA. Impact assessment can provide information to improve the HTA process, for example, the value of patient and clinician engagement in the HTA process to better define the assessment question and literature reviews in a more holistic and balanced way.HTA reports produced by publicly funded HTA agencies are valued by health systems around the globe as they support decision making regarding the appropriate use, pricing, reimbursement, and disinvestment of health technologies. HTAs can also have a positive impact on information sharing between different levels of government and across stakeholder groups. These stories show how HTA can have a significant impact, irrespective of the health system and health technology being assessed.


 Several regulatory agencies have implemented processes to facilitate timely access to innovative therapies for patients with rare or life-threatening diseases with high unmet medical needs, typically with additional data-generation requirements to address important uncertainties in the existing evidence base at the time of regulatory approval. Many health technology assessment (HTA) agencies have adapted their processes to include time-limited recommendations (TLRs) for such therapies, which allows patients access to these medications while additional data are collected to verify the clinical efficacy, real-world effectiveness, or economic value of the new therapeutic. The objective of this Environmental Scan is to identify what HTA agencies have processes in place for TLRs and to describe these processes, if applicable.
 HTA agencies in North America, Europe, Australia, and New Zealand were considered for this report based on commonalities to the Canadian context in terms of geography and relevant regulatory or HTA and reimbursement processes. Seven HTA agencies (the National Institute for Health and Care Excellence, the Scottish Medicines Consortium, France’s Haute Autorité de Santé, the Dutch National Health Care Institute, the Italian Medicines Agency, the Belgium National Institute for Health and Disability Insurance, and the Australian Pharmaceutical Benefits Advisory Committee) that have implemented formal TLR processes, through managed entry agreements, special access funds, or other related programs were identified. Most TLR programs are referred to as managed access or entry agreements, but other types include interim acceptance, conditional inclusion, assessment contingent on additional evidence development, and additional specialized programs or pathways.
 TLRs are primarily used for promising (and sometimes high-cost) medicines intended to treat a condition with an important unmet medical need; some specify that the disease must be serious and life threatening (regardless of prevalence), others indicate that the process is used for orphan drugs intended to treat rare conditions, and others consider multiple criteria or do not have prerequisite criteria for eligible indications.
 Evidence generation requirements for TLRs vary across HTA agencies and are often dependent on the nature of the clinical and economic uncertainties identified in the initial assessment; sources include later follow-up from ongoing clinical trials, new clinical trials, real-world data, or a combination of both clinical and real-world studies. Additionally, most TLR processes have explicit maximum durations for the time-limited period, which range from 2 to 7 years across the included HTA agencies (although some allow for time adjustments or renewals); however, the French and Scottish HTA agencies determine the duration of the time-limited period on a case-by-case basis.
 Overall, this report provides an overview of HTA processes for TLRs across HTA agencies in Europe and Australia, many of which may be useful to inform the implementation of future TLR processes in Canada, as well as to refine existing ones.

There is no health system that has the resources to evaluate all technologies. The presence of a clear process to prioritize health technologies for assessment by health technology assessment (HTA) agencies is a good practice principle recognized at the international level. The objective of Health Technology Assessment International's 2020 Latin American Policy Forum (LatamPF) was to explore how to improve the way HTA agencies in Latin America identify and prioritize technologies for assessment. This paper is based on a background document, a survey, and the deliberations of the members of the LatamPF (forty-six participants from eleven countries) using a design thinking methodology. Participants agreed that a lack of clear prioritization mechanisms results in HTA processes and decisions that are perceived to be of low transparency and overly exposed to political or interest group pressures. The LatamPF identified barriers and recommended actions to improve HTA prioritization mechanisms in Latin America. The criteria identified as the most important to be taken into consideration by HTA agencies in the region when prioritizing a technology for assessment were: the burden of illness, the potential clinical benefit, the alignment with national health priorities, the potential impact on equity, a lack of treatment alternatives for patients, and the potential economic impact. Forum participants agreed that the establishment of transparent prioritization processes is a key element for all health systems. Improvements in these processes will strengthen HTA and provide greater legitimacy to decision making.

INTRODUCTION:Priority setting in health care has been long recognized as an intrinsically complex and value-laden process. Yet, Health Technology Assessment (HTA) agencies presently employ value assessment frameworks that are ill-fitted to capture the range and diversity of stakeholder values, and thereby risk to compromise the legitimacy of their recommendations. We propose ‘evidence-informed deliberative processes’ as an alternative framework with the aim to enhance this legitimacy.METHODS:The framework is based on an integration of two increasingly popular and complementary frameworks for priority setting: multi-criteria decision analysis (MCDA) and accountability for reasonableness (A4R), Evidence-informed deliberative processes are, on the one hand, based on early, continued stakeholder deliberation to learn about the importance of relevant social values. On the other hand, they are based on rational decision-making – through evidence-informed evaluation of the identified values.RESULTS:The framework has important implications for how HTA agencies should ideally organize their processes. Firstly, HTA agencies should take the responsibility to organize stakeholder involvement. Second, agencies are advised to integrate their assessment and appraisal phase, allowing for the timely collection of evidence on values that are considered relevant. Third, HTA agencies should subject their specification of decision-making criteria to public scrutiny. Fourth, agencies are advised to use a checklist of potentially relevant criteria, and to provide argumentation how each criterion affected the recommendation. Fifth, HTA agencies must publish their argumentation and install options for appeal.CONCLUSIONS:Adopting ‘evidence-informed deliberative processes’ as a value assessment framework could be an important step forward for HTA agencies to optimize the legitimacy of their priority setting decisions. Agencies can incorporate elements according to their needs and affordances.

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.

IntroductionOmics technologies enable the measurements of genes (genomics), mRNA (transcriptomics), proteins (proteomics) and metabolites (metabolomics) and thus proved to be valuable tools for personalized decision-making in clinical practice. For their evaluation, a health technology assessment (HTA) framework is not standardized and accepted, yet. Therefore, we aim at designing an omics-technologies HTA evaluation framework to facilitate their assessment, through a mixed-method approach. This work is part of the ExACT project, which aims to produce a range of tools to facilitate the implementation of precision health in clinical practice.MethodsA systematic review was conducted to identify the existing HTA frameworks used for the evaluation of omics-technologies. Desk research on the HTA agencies’ websites was performed to identify the reports on omics-technologies HTA evaluation frameworks used by these agencies. A questionnaire evaluating HTA agencies’ experience on evaluation of omics-technologies was designed. The new framework will be elaborated based on the findings from the three methodological steps, and will be validated through a Delphi process.ResultsTwenty-three articles were included in the systematic review. The main identified HTA frameworks were ACCE and “Evaluation of Genomic Applications in Practice and Prevention” (EGAPP). The desk research showed that these frameworks were seldom used by HTA agencies, which for the evaluation of omics-technologies mostly refer to the HTA Core Model®, mainly assessing the following domains: clinical effectiveness and economic evaluation. Data collection process of the questionnaire HTA agencies’ experience is in progress.ConclusionsAlthough two main HTA frameworks for the evaluation of omics-technologies have been identified, these frameworks are sporadically used by HTA agencies in their practice. The particular interest of HTA agencies on clinical effectiveness and economic evaluation, might potentially reflect the uncertainty and difficulties when evaluating omics-technologies. This could indicate that these HTA frameworks are not feasible and practical to be used in routine HTA agency processes for omics technologies, emphasizing the need for a new framework. Our methodological approach might contribute to the development of a new HTA framework, feasible and practical to use not only for HTA agencies.

IntroductionOver the past decade there have been increasing interactions between health technology assessment (HTA) agencies through international networks at the policy level and European joint actions at the product level. A pilot project is underway to explore collaboration beyond Europe between HTA agencies in Australia, Canada, and the UK. This study the compared HTA recommendations of new active substances (NAS) appraised by Australia’s Pharmaceutical Benefits Advisory Committee (PBAC), Canada’s CADTH, and England’s National Institute for Health and Care Excellence (NICE).MethodsUsing publicly available data and established benchmarking methodology, we examined 45 NAS appraised by PBAC, CADTH, and NICE between 2017 and 2021. Analysis was performed to assess rollout time from regulatory to HTA recommendation, and to the first HTA recommendation.ResultsMost products were submitted to the Europe Medicine Agency first (89%). However, 71 percent of NAS in Australia and 69 percent in Canada were submitted to HTA in parallel with regulatory review, which shortened overall rollout time. The median HTA submission gap among the three agencies was 140 days in Australia, 102 days in Canada, and 8 days in England. PBAC had the highest number of negative recommendations (51%), followed by CADTH (18%), and NICE (5%). The congruence of HTA decisions was highest between CADTH and NICE (56%), compared with PBAC and CADTH (11%), and PBAC and NICE (20%)ConclusionsTo achieve a more collaborative HTA process it is necessary to understand the rollout time in these jurisdictions. This study identified submission gaps among the regulatory agencies, but there may be more synergy in the future as regulators in the three jurisdictions work collaboratively through the Access Consortium. Currently, the submission gap for the three HTA agencies was mostly within six months, making collaboration on joint assessment possible. We observed divergences in HTA recommendations due to the methodology and decision criteria applied by each agency. Therefore, collaboration on assessment should build on the clinical aspects, although HTA decisions should be grounded in the local context.

Introduction: In Europe and beyond, the rising costs of healthcare and limited healthcare resources have resulted in the implementation of health technology assessment (HTA) to inform health policy and reimbursement decision-making. European legislation has provided a harmonized route for the regulatory process with the European Medicines Agency, but reimbursement decision-making still remains the responsibility of each country. There is a recognized need to move toward a more objective and collaborative reimbursement environment for new medicines in Europe. Therefore, the aim of this study was to objectively assess and compare the national reimbursement recommendations of 9 European jurisdictions following European Medicines Agency (EMA) recommendation for centralized marketing authorization.Methods: Using publicly available data and newly developed classification tools, this study appraised 9 European reimbursement systems by assessing HTA processes and the relationship between the regulatory, HTA and decision-making organizations. Each national HTA agency was classified according to two novel taxonomies. The System taxonomy, focuses on the position of the HTA agency within the national reimbursement system according to the relationship between the regulator, the HTA-performing agency, and the reimbursement decision-making coverage body. The HTA Process taxonomy distinguishes between the individual HTA agency's approach to economic and therapeutic evaluation and the inclusion of an independent appraisal step. The taxonomic groups were subsequently compared with national HTA recommendations.Results: This study identified European national reimbursement recommendations for 102 new active substances (NASs) approved by the EMA from 2008 to 2012. These reimbursement recommendations were compared using a novel classification tool and identified alignment between the organizational structure of reimbursement systems (System taxonomy) and HTA recommendations. However, there was less alignment between the HTA processes and recommendations.Conclusions: In order to move forward to a more harmonized HTA environment within Europe, it is first necessary to understand the variation in HTA practices within Europe. This study has identified alignment between HTA recommendations and the System taxonomy and one of the major implications of this study is that such alignment could support a more collaborative HTA environment in Europe.

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.

BackgroundFew antibiotics have entered the market in recent years despite the need for new treatment options. Some of the challenges of bringing new antibiotics to market are linked to the marketing authorization and health technology assessment (HTA) processes. Research shows great variation in geographic availability of new antibiotics, suggesting that market introduction of new antibiotics is unpredictable. We aimed to investigate regulatory authorities’ and HTA agencies’ role in developing non-financial incentives to stimulate antibiotic research and development (R&D).MethodsWe conducted individual, semi-structured, stakeholder interviews. Participants were recruited from regulatory authorities (EMA and FDA) and HTA agencies in Europe. Participants had to be experienced with assessment of antibiotics. The data were analyzed using a deductive and inductive approach to develop codes and identify key themes. Data were analyzed using thematic analysis including the constant comparison method to define concepts, and rival thinking to identify alternative explanations.ResultsWe found that (1) interpretation of key concepts guiding the understanding of what type of antibiotics are needed vary (2) lack of a shared approach on how to deal with limited clinical data in the marketing authorization and HTA processes is causing barriers to getting new antibiotics to market (3) necessary adaptations to the marketing authorization process causes uncertainties that transmit to other key stakeholders involved in delivering antibiotics to patients.ConclusionsA shared understanding of limited clinical data and how to deal with this issue is needed amongst stakeholders involved in antibiotic R&D, marketing authorization, and market introduction to ensure antibiotics reach the market before resistance levels are out of control. Regulatory authorities and HTA agencies could play an active role in aligning the view of what constitutes an unmet medical need, and direct new economic models towards stimulating greater diversity in the antibiotic armamentarium.

In the drive towards faster patient access to treatments, health technology assessment (HTA) agencies are increasingly faced with reliance on evidence from surrogate endpoints, leading to increased decision uncertainty. This study undertook an updated survey of methodological guidance for using surrogate endpoints across international HTA agencies. We reviewed HTA and economic evaluation methods guidance from European, Australian and Canadian HTA agencies. We considered how guidelines addressed the methods for handling surrogate endpoints, including (1) level of evidence, (2) methods of validation, and (3) thresholds of acceptability. Across the 73 HTA agencies surveyed, 29 (40%) had methodological guidelines that made specific reference to consideration of surrogate outcomes. Of the 45 methods documents analysed, the majority [27 (60%)] were non-technology specific, 15 (33%) focused on pharmaceuticals and three (7%) on medical devices. The principles of the European network for Health Technology Assessment (EUnetHTA) guidelines published in 2015 on the handling of surrogate endpoints appear to have been adopted by many European HTA agencies, i.e. preference for final patient-relevant outcomes and reliance on surrogate endpoints with biological plausibility and epidemiological evidence of the association between the surrogate and final endpoint. Only a small number of HTA agencies (UK National Institute for Care and Excellence; the German Institute for Medical Documentation and Information and Institute for Quality and Efficiency in Health Care; the Australian Pharmaceutical Benefits Advisory Committee; and the Canadian Agency for Drugs and Technologies in Health) have developed more detailed prescriptive criteria for the acceptance of surrogate endpoints, e.g. meta-analyses of randomised controlled trials showing strong association between the treatment effect on the surrogate and final outcomes. As the decision uncertainty associated with reliance on surrogate endpoints carries a risk to patients and society, there is a need for HTA agencies to develop more detailed methodological guidance for consistent selection and evaluation of health technologies that lack definitive final patient-relevant outcome evidence at the time of the assessment.

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.

ObjectivesTo identify and describe patient experience data (PED)-related guidance issued by regulatory and health technology assessment (HTA) agencies in North America, Europe, and Asia Pacific.MethodsNational agency websites were manually searched (October 2021, updated October–December 2023) to identify standalone PED guidance, and general submission/clinical trial or disease-specific guidance in four therapy areas, including PED-related recommendations. Identified documents were reviewed for requirements/expectations on types of PED; concepts of interest and instruments/tools; validation, analysis, and interpretation; study design, and endpoint definitions.ResultsA total of 34 regulatory and 21 HTA documents were reviewed across 7 and 11 agencies, respectively. Most regulatory agencies expected data collected via clinical outcome assessments; concepts of interest were similar; specific tools were not usually recommended, although the use of validated instruments was expected; and meaningful within-patient change was generally considered key to interpretation. HTA guidance on PED varied primarily based on country requirement for utility estimates to inform economic evaluation (little/no guidance beyond the concept of interest) versus interest in PED on signs, symptoms, or disease impact on functioning (generally more comprehensive details on PED expectations).ConclusionThrough PED, patient voice is an increasingly important factor in regulatory, access, and reimbursement decision-making. Regulatory and HTA agencies in North America, Europe, and Asia Pacific have varying expectations regarding PED. This misalignment can necessitate the collection of multiple types of PED to meet all requirements (resulting in complex clinical trial protocols and significant patient burden), and lead to differences in data interpretation or gaps in the expected data. Therefore, PED measurement standards should be harmonized globally, and the collection of required PED should be ensured early during drug development to ensure decisions can be made based on valid and reliable data.