The primary objectives of this umbrella review were to (a) quantify the relative importance, of “severity” and “rarity” criteria in health resource allocation; and (b) analyze the contextual factors influencing the relative importance. The secondary objective was to examine how “severity” and “rarity” criteria are defined.

Searches were carried out in PubMed and Embase to identify eligible systematic reviews. Quality appraisal of systematic reviews was undertaken. From identified systematic reviews, primary studies were extracted and further screened for eligibility. The inclusion of severity and rarity criteria and their respective weights in primary studies were examined. Descriptive and regression analyses were performed.

Twenty-nine systematic reviews were screened, of which nine met the inclusion criteria. Primary studies included in these systematic reviews were retrieved and screened, resulting in forty articles included in the final analysis. Disease severity was more frequently considered (n = 29/40) than disease rarity (n = 23/40) as an evaluation criterion. Out of all cases where both were included as evaluation criteria, disease severity was assigned higher weights 84 percent of the time (n = 21/25).

Our review found consistent evidence that disease severity is more relevant and preferred to rarity as a priority-setting criterion albeit constraints in statistical analysis imposed by limited sample size and data availability. Where funding for rare diseases is concerned, we advocate that decision-makers be explicit in clarifying the significance of disease severity and/or rarity as a value driver behind decisions. Our findings also reinforce the relevance of disease severity as a criterion in priority setting.

The decision-making (DM) process in public administration is the subject of research from different perspectives and disciplines. Evidence-based policies, such as health technology assessment (HTA), are not the only support on which public policies are designed. During the COVID-19 pandemic WHO, national and subnational institutions developed HTA reports to guide DM. Despite this, inadequate variability was observed in the health technologies recommended and reimbursed by different provincial Health Ministries in a federally organized developing country like Argentina. The processes and results of DM on health technologies for COVID-19 in Health Ministries of Argentina were inquired.

A retrospective research design was developed, with triangulation of quantitative and qualitative methods. We retrieved information for the years 2020–2021 through document review of official webpages, surveys, and interviews with decision-makers of the 25 Argentinian Ministries of Health. We analyzed the recommendations and reimbursement policies of seven health technologies.

In contradiction with WHO’s policies, ivermectine, inhaled ibuprofen, convalescent plasma and equine serum were widely recommended by most of Argentina’s health ministries outside a clinical trial context, with risks for patients and a huge opportunity cost.

Despite an important HTA institutional capacity, the impact of HTA organizations and their technical reports was limited. Health Ministries with institutionalized HTA units had more adherence to WHO recommendations, but the influence of different technical and political criteria was identified. Power relations within and outside the administration, the pharmaceutical industry and academics, the media, social pressure, the judicial and legislative powers, and the political context strongly influenced DM.

Evidence development for medical devices is often focused on satisfying regulatory requirements with the result that health professional and payer expectations may not be met, despite considerable investment in clinical trials. Early engagement with payers and health professionals could allow companies to understand these expectations and reflect them in clinical study design, increasing chances of positive coverage determination and adoption into clinical practice.

An example of early engagement through the EXCITE International model using an early technology review (ETR) is described which includes engagement with payers and health professionals to better inform companies to develop data that meet their expectations. ETR is based on an early evidence review, a framework of expectations that guides the process and identified gaps in evidence. The first fourteen ETRs were reviewed for examples of advice to companies that provided additional information from payers and health professionals that was thought likely to impact on downstream outcomes or strategic direction. Given that limitations were imposed by confidentiality, examples were genericized.

Advice through early engagement can inform evidence development that coincides with expectations of payers and health professionals through a structured, objective, evidence-based approach. This could reduce the risk of business-related adverse outcomes such as failure to secure a positive coverage determination and/or acceptance by expert health professionals.

Early engagement with key stakeholders exemplified by the ETR approach offers an alternative to the current approach of focusing on regulatory expectations. This could reduce the time to reimbursement and clinical adoption and benefit patient outcomes and/or health system efficiencies.

In response to the Omicron surge in early 2022, the HTA Philippines evaluated the acceptability of Filipinos in using self-administered antigen tests (SAAgTs) as part of COVID-19 HTAs in the Philippines.

Scoping review from literature databases was initially conducted to identify preset codes in the use of SAAgT. Preset codes were used to establish the questions for focus group discussions (FGDs). Semi-structured questionnaires were created through Delphi technique. FGDs with four stakeholder groups (i.e., nine healthcare workers [HCWs], seven representatives of at-risk groups, six economic frontliners, and seven representatives of micro–small–medium-sized enterprises) were conducted.

Discomfort in being a target of stigma and being prescribed an “illness identity” when suspected or confirmed COVID-19-positive, along with lack of confidence to perform self-test, caused hesitancy in self-testing among participants. The need for subsidies for test kits from the government or employers was emphasized to increase its accessibility. Having a designated access point and reporting system for SAAgT was highlighted to avoid nepotism (padrino system attributed to debt of gratitude), inequitable distribution, and lapses in reporting. A participatory approach to education was perceived as crucial to reduce any misconceptions associated with the use of SAAgT.

All FGD groups expressed favorable reviews on the implementation of SAAgT because it can potentially reduce the burden of health facility-administered tests. These findings were considered by the HTA Council in the recommendation of SAAgT as part of the overarching national strategies for the diagnosis and screening of COVID-19.

Though there have been longstanding discussions on the value of ethics in health technology assessment (HTA), less awareness exists on ethics information retrieval methods. This study aimed to scope available evidence and determine current practices for ethics information retrieval in HTA.

Literature searches were conducted in Ovid MEDLINE, LISTA, Scopus, and Google Scholar. Once a list of relevant articles was determined, citation tracking was conducted via Scopus. HTA agency websites were searched for published guidance on ethics searching, and for reports which included ethical analyses. Methods sections of each report were analyzed to determine the databases, subject headings, and keywords used in search strategies. The team also reached out to information specialists for insight into current search practices.

Findings from this study indicate that there is still little published guidance from HTA agencies, few HTAs that contain substantial ethical analysis, and even less information on the methodology for ethics information retrieval. The researchers identified twenty-five relevant HTAs. Ten of these reports did not utilize subject-specific databases outside health sciences. Eight reports published ethics searches, with significant overlap in subject headings and text words.

This scoping study of current practice in HTA ethics information retrieval highlights findings of previous studies—while ethics analysis plays a crucial role in HTA, methods for literature searching remain relatively unclear. These findings provide insight into the current state of ethics searching, and will inform continued work on filter development, database selection, and grey literature searching.

Early access schemes (EASs) are approaches used by payers to balance and facilitate earlier patient access to innovative health technologies while evidence generation is ongoing. Schemes require investment from payers and are associated with significant risk since not all technologies will be routinely reimbursed. The purpose of this study was to gain the perspectives of policy experts about the key challenges for EASs and potential solutions for their optimal design and implementation.

Two virtual workshops were convened including (i) UK-based policy experts (England, Wales, and Scotland) and (ii) representatives from multiple healthcare systems (England, France, Sweden, Canada, Poland, and Norway). Participants were encouraged to share their experiences with EASs in their healthcare system and highlight key challenges for policy makers. Discussions were transcribed and analyzed using framework analysis.

Participants agreed that EASs have value when targeted toward innovative technologies with the potential for significant clinical benefit in an area of high unmet need. Participants discussed potential solutions to the challenges faced by payers implementing EASs, including defining eligibility criteria, supporting evidence generation, and approaches to reimbursement.

Participants agreed that EASs are one possible solution for their healthcare systems and have the potential to deliver significant clinical value to patients. However, widespread adoption of EASs is limited due to concerns about the risks for patients and healthcare budgets, further solutions are needed to deliver EASs for targeted therapies.

To summarize the key methodological challenges identified by health technology assessment (HTA) agencies assessing gene therapy (GT) and consideration of broad elements of value.

Economic evaluations (EEs) of voretigene neparvovec (VN) in RPE65-mediated inherited retinal disease (IRD) published in English were selected. HTA evaluations from Australia, Canada, Ireland, Scotland, England, and the United States were reviewed. An existing methodological framework was used to identify the challenges and considerations.

Eight unique EEs were identified of which six were evaluated by HTA agencies. Incremental cost-effectiveness ratios ranged from $68,951 to $643,813 per quality-adjusted life-years (QALY) gained (healthcare perspective) and dominant to $480,130 per QALY gained (societal perspective). The key challenges were the lack of validated surrogate outcome, utility values and indirect costs from IRD patients, and limited evidence of the long-term treatment effect. Two HTA agencies reviewed a range of novel broader elements of value and whether they were associated with VN while other agencies discussed some elements of broader value. Caregiver disutility was included in some, but not all, evaluations.

The methodological challenges were consistent with innovative interventions for rare diseases and managed using standard methods. Broader value was important to decision-makers but inconsistently applied across agencies. Possible reasons are limitations in the evidence available of the broader benefits that VN offers and how to incorporate these within an EE. A need exists for greater guidance and consistency across jurisdictions regarding the consideration of broader value that considers latest best practice.

To develop best-practice guidance for health technology assessment (HTA) agencies when appraising diagnostic tests for SARS-CoV-2 and treatments for COVID-19.

We used a policy sandbox approach to develop best-practice guidance for HTA agencies to approach known challenges associated with assessing tests and treatments for COVID-19. The guidance was developed by a multi-stakeholder workshop of twenty-one participants representing HTA agencies, clinical and patient experts, academia, industry, and a payer, from across Europe and North America. The workshop was supported by extensive background work to identify the key challenges, including: targeted reviews of existing COVID-related methods guidance for assessing interventions and clinical guidelines, engagement with clinical experts, a survey and workshop of HTA agencies, a systematic review of published economic evaluations, and a workshop of health economic modelers.

We suggest HTA agencies should consider using other types of evidence (e.g., real world) where high-quality randomized controlled trials may be lacking and healthcare systems would value timely HTA outputs. A “living” HTA approach may be useful, given the context of an evolving disease, scientific understanding and evidence base, allowing for decisions to be efficiently revisited in response to new information; particularly, if supported by a common “disease model” for COVID-19. Innovative ways of engaging with the public and clinicians, and early engagement with regulators and payers, are recommended.

HTA agencies should consider the elements of this guidance that are most suited to their existing processes to enable them to assess the effectiveness and value of interventions for COVID-19.

The objective of the present policy analysis was to understand how a disinvestment approach to the process of health technology assessment (HTA), applied to the field of medical devices, might help Italian policymakers to properly spend the resources in healthcare.

Previous international and national experiences in disinvestment for medical devices were reviewed. Precious insights for the rational expenditure of the resources were derived by assessing the evidence available.

The disinvestment of ineffective or inappropriate technologies or interventions with an inadequate value-for-money ratio has become a growing priority for National Health Systems. Different international disinvestment experiences of medical devices were identified and described through a rapid review. Although most of them have a strong theoretical framework, their practical application remains difficult. In Italy, there are no examples of large and complex HTA-based disinvestment practices, but their importance is becoming increasingly acknowledged, especially given the need to prioritize the funds provided by Recovery and Resilience Plan.

Anchoring decisions on health technologies without reassessing the current technological landscape through a robust HTA model might expose to the risk of not ensuring the best employment of the resources available. Thus, it is necessary to develop a strong HTA ecosystem in Italy through adequate consultation with stakeholders to enable a data-driven and evidence-based prioritization of resources toward choices characterized by high value for both patients and society as a whole.

The French health technology assessment (HTA) agency initiated a research between 2018 and 2019 with the aim of determining whether other HTA organizations (agencies, bodies, institutes, and expert networks) and researchers had developed an evaluation framework of organizational impacts (OIs).

Three types of investigation were carried out: (i) an analysis of documents published by selected HTA organizations, (ii) a rapid review on the OI issues, (iii) a questionnaire survey to experts of the International Network of Agencies for Health Technology Assessment.

The analyses highlight six key points: (i) there is no explicit conceptual definition of OIs; (ii) OIs are often not included in a specific dimension of the evaluation or in the same dimensions; (iii) three recurring categories emerge from the assessment of OIs: processes, structure, and culture; (iv) despite its limitations, the European Network for Health Technology Assessment framework (Core Model) is the most mature assessment model to date; (v) the question of the scope of OIs to be considered is unresolved (micro-meso-macro); and (vi) the delineation between OI assessment and economic assessment must be addressed.

Although the issue of considering OI in HTA has been raised for many years, it remains largely unresolved. Defining the concept of OI is a prerequisite for taking the next step toward an evaluation framework. As the question of the impact of innovation goes beyond the health sector, extensive research on how to define and take into account these OIs may be relevant.

To clarify the concept of disruptive technologies in health care, provide examples and consider implications of potentially disruptive technologies for health technology assessment (HTA).

We conducted a systematic review of conceptual and empirical papers on healthcare technologies that are described as “disruptive.” We searched MEDLINE and Embase from 2013 to April 2019 (updated in December 2021). Data extraction was done in duplicate by pairs of reviewers utilizing a data extraction form. A qualitative data analysis was undertaken based on an analytic framework for analysis of the concept and examples. Key arguments and a number of potential predictors of disruptive technologies were derived and implications for HTA organizations were discussed.

Of 4,107 records, 28 were included in the review. Most of the papers included conceptual discussions and business models for disruptive technologies; only few papers presented empirical evidence. The majority of the evidence is related to the US healthcare system. Key arguments for describing a technology as disruptive include improvement of outcomes for patients, improved access to health care, reduction of costs and better affordability, shift in responsibilities between providers, and change in the organization of health care. A number of possible predictors for disruption were identified to distinguish these from “sustaining” innovations.

Since truly disruptive technologies could radically change technology uptake and may modify provision of care patterns or treatment paths, they require a thorough evaluation of the consequences of using these technologies, including economic and organizational impact assessment and careful monitoring.

Health technology assessment (HTA) plays a central role in the coverage and reimbursement decision-making process for public health expenditure in many countries, including Thailand. However, there have been few attempts to quantitatively understand the benefits of using HTA to inform resource allocation decisions. The objective of this research was to simulate the expected net monetary benefit (NMB) from using HTA-based decision criteria compared to a first-come, first-served (FCFS) approach using data from Thailand.

A previously published simulation model was adapted to the Thai context which aimed to simulate the impact of using different decision-making criteria to adopt or reject health technologies for public reimbursement. Specifically, the simulation model provides a quantitative comparison between an HTA-based funding rule and a counterfactual (FCFS) funding rule to make decisions on which health technologies should be funded. The primary output of the model was the NMB of using HTA-based decision criteria compared to the counterfactual approach. The HTA-based decision rule in the model involved measuring incremental cost-effectiveness ratios against a cost-effectiveness threshold. The counterfactual decision rule was a FCFS (random) selection of health technologies.

The HTA-based decision rule was associated with a greater NMB compared to the counterfactual. In the investigated analyses, the NMB ranged from THB24,238 million (USD725 million) to THB759,328 million (USD22,719 million). HTA-based decisions led to fewer costs, superior health outcomes (more quality-adjusted life-years).

The results support the hypothesis that HTA can provide health and economic benefits by improving the efficiency of resource allocation decision making.

The extent to which value assessments are uniquely deployed in any given geographic setting is variable. Increasingly, markets are seeking insights from external health technology assessments (HTAs) to assist with decisions surrounding the adoption of new technologies. We reviewed the environment, infrastructure, and practice of value assessment in six countries, with a focus on how these elements influence the transferability of value assessments between settings.

We reviewed the diverse settings in which six organizations conducting HTA operate, and explored how differences might affect the transferability of value assessment. We focused attention on Australia’s Pharmaceutical Benefits Advisory Committee, China’s National Center for Medicine and HTA, Germany’s Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, Japan’s Center for Outcomes Research and Economic Evaluation for Health (Core 2 Health), the National Institute for Health and Care Excellence in England and Wales, and the Institute for Clinical and Economic Review in the United States.

HTA is adopted to address unique objectives for a given health system and is tailored to support local standards and preferences. Some elements of a value assessment, such as evidence on clinical effectiveness, may be more transferable than others. It is challenging to appropriately adjust external assessments to the local context.

Contextual differences influence both the role and application of HTA. These differences limit the transferability of value assessments from one setting to another. De novo appraisals, customized to the local decision context, are the ideal approach to determinations about value.

The National Institute for Health and Care Excellence’s (NICE) method guide for technology appraisals (TAs) encourages medicine manufacturers to use the EuroQol 5 Dimensions (EQ-5D) in relevant clinical trials to obtain utility values; however, the EQ-5D may have low sensitivity when compared to disease-specific measures. This study investigated whether the NICE TA committee’s acceptance of manufacturer-proposed utility values is dependent on the manufacturers’ sources of the utility values.

Using publicly available data for 2011–2020, we identified 136 single TAs of cancer medicines, the health-related quality-of-life-measures used in relevant clinical trials, manufacturers’ sources of utility values, and the NICE TA committee’s acceptance of these values. Fisher’s exact tests were performed to compare the acceptability of different value sources and reasons for non-acceptance.

The number of appraisals for which the EQ-5D in the relevant clinical trials was the source of the manufacturer-proposed utility values increased continuously over time. The TA committee’s acceptance of values was not dependent on the information source. In cases where a submission for which the information source was the EQ-5D was rejected, the reason was generally related to inappropriate values for the UK population or inappropriate data adjustment, not data reliability.

Our results demonstrated that according with the NICE’s method guide regarding utility values does not guarantee acceptance by the TA committee. Manufacturers must consider in advance possible differences between their clinical trials and clinical practice in the UK and refine plans for EQ-5D measurement in order to obtain convincing evidence.

The European Network for Health Technology Assessment (EUnetHTA) was established in 2006. During its final project phase (joint action 3 [JA3]), it undertook an activity to define the scientific and technical principles of a model of health technology assessment (HTA) cooperation in Europe. This policy article presents the key learnings from JA3 partners about developing a model of HTA cooperation.

There were two phases to the activity: (i) A descriptive phase to describe the elements of HTA cooperation that were already in place in EUnetHTA JA3 and to identify which elements could be improved or were missing. (ii) An analytic phase synthesizing the data collected to identify learnings from the JA3 and to define the scientific and technical principles for a future model of HTA cooperation.

Learnings for developing HTA cooperation were identified in regard to the framework used to support the cooperation, the HTA activities undertaken, the involvement of internal and external actors, managing decision making and the required human resources and support services needed to undertake HTA activities and to coordinate collaboration.

These learnings coming from the experiences of the EUnetHTA JA3 are useful to inform discussions on a European Union regulation for HTA cooperation as well as subsequent work to set up the structures that will be defined in the regulation. The findings also have broader applicability and are relevant to individuals, groups, and organizations setting up HTA programs or establishing their own international collaborations.