Obesity, defined as a body mass index (BMI) greater than 30kg/m2, is a multifactorial disease with severe health and economic consequences. As obesity associated events impact long-term survival, health economic (HE) modelling is commonly used. However, the current set of modelling approaches are very diverse and lack external model validation. The aim of our research was to compare the event simulation and the HE outcomes of different structural approaches.

We performed an external validation of three main structural modelling approaches for estimating obesity-associated events: (i) continuous BMI-related risks; (ii) general risk equations; and (iii) categorical BMI-related risks. The Swedish Obese Subjects (SOS) intervention study was used for validation. Outcomes compared were mortality, cardiovascular events (CVE), and type 2 diabetes (T2D), over time using the long-term data from the SOS study, looking at both the surgery arm and the control arm. Concordance between modelling results and the external validation study was measured by visual examination of the best fitting linear regression lines, R2 coefficients, the root mean squared errors, and F-tests. Furthermore, we compared the HE modelling results, comparing surgery versus control, expressed as cost per quality-adjusted life-year (QALY) gained based on 1,000 Monte Carlo simulation samples.

Mortality was overestimated by all approaches irrespective of the study arm. For CVE an overestimation by all structural approaches was observed for the control arm. The CVE surgery arm was overestimated by the categorical BMI approach and slightly underestimated by the others. For T2D an underestimation was observed for the continuous and the categorial BMI approaches, whereas there was an overestimation by the risk equation approach. Considering different confidence interval limits, the cost per QALY gained are comparable between all structural approaches.

None of the structural approaches provided perfect external event validation results although the risk equation approach showed the smallest deviations compared to the external validation study. The cost per QALY gained resulting from the three approaches were fairly comparable.

The objective of the European Network for Health Technology Assessment (EUnetHTA) Joint Action 3 (JA3) was to develop a sustainable European model for future collaboration on HTA, by reducing duplication in HTA production and increasing patient access to health technologies. Compared to the previous JA2, several procedural changes were made aiming to increase usability, transparency, and inclusiveness of relative effectiveness assessments (REAs). This article presents and highlights these changes, explains their rationale as well as their influence on HTA production.

Feedback from REA teams and project managers was collected. At the end of JA3, all lessons learned were mapped, resulting in a set of recommendations for a future REA production process.

In JA3, forty-three EUnetHTA REAs have been produced. Efforts to increase the usability of the REAs were made by focussing on the needs of REA producers and users (HTA agencies) and by increasing stakeholder involvement. Huge steps were taken with regard to transparency, which was achieved through publication of guidances, templates, and up-to-date information on the EUnetHTA website. In an attempt to improve inclusiveness, (stakeholder) interaction and involvement as well as feedback procedures were enhanced and streamlined. The fine-tuned project management brought all aspects together and facilitated a consistent and reliable workflow.

Despite that HTA agencies have different national requirements, the procedural changes made in JA3 proved to counteract some of these challenges. Nevertheless, it is of utmost importance that further perceived methodological differences are being resolved to ensure a strong base for future European collaboration on REA production.

With the disease spectrum changing in China, type 2 diabetes mellitus (T2DM) has become the main chronic disease which affects people’s health severely, bring patients serious economic burden of disease. For T2DM patients, reliable quality of evidence in decision-making are significant, improving the efficiency of the adjustment of the National Reimbursement Drug List (NRDL). Based on the Consolidated Health Economic Evaluation Reporting Standards (CHEERS), we aimed to evaluate the quality of all published pharmacoeconomic evaluations on T2DM drugs in 2020 NRDL.

Because the 2020 NRDL came into effect on 1 March 2021, we searched all published pharmacoeconomic evaluations about T2DM drugs in 2020 NRDL before March 2021 in China National Knowledge Infrastructure (CNKI), Wan fang Data, China Science and Technology Journal Database (VIP), PubMed, and Web of Science. According to the criterion of inclusion and exclusion, all documents were screened and then relevant basic information of targeted documents was extracted. The quality was evaluated by calculating the final scores based on CHEERS. Two reviewers assessed each publication’s quality using the CHEERS instrument and summarized study quality.

A total of 910 papers were searched, and 24 papers were included. These involved six T2DM drugs, specifically IDegAsp, exenatide, liraglutide, lixisenatide, dapagliflozin and empagliflozin. The average score was 18.31, the standard deviation was 3.67, and the average scoring rate was 77.41 percent. Among all items, “characterizing heterogeneity” scored 0.04, least satisfied with requirements. “Setting and location”, “choice of health outcomes” and “assumptions” scored one, most satisfied with requirements. Among the average scores of all parts, “results” scored lowest at 0.55, and “methods” scored highest at 0.85. The Wilcoxon sum-rank tests showed that score rate which represented reporting quality of economic evaluation (EE) was significantly related to “journal type”, “EEs type”, “model choice” and “study perspective”.

The methodological quality of pharmacoeconomic evaluations about T2DM drugs in 2020 NRDL needs to be improved. Improving the quality of literature is the basic guarantee of scientific decision-making in national medical insurance negotiation.

The interactions between regulators, health technology assessment (HTA), and companies play a significant role in the process of getting medicine to patients. These have evolved at a product level as well as at a policy and cross-jurisdictional level; however, it is important these activities are adding value for stakeholders involved. A survey conducted in March 2021 assessed the current interactions from multi-stakeholders, and their perceptions on the added value these interactions bring to better decision-making.

Three separate questionnaires containing nine questions were developed to assess the perceptions from pharmaceutical companies, regulators, and HTA agencies. The three questionnaires contained analogous questions where appropriate. The company questionnaire was sent to senior management at 19 international pharmaceutical companies, the agency survey was sent to 32 agencies (17 regulatory agencies and 15 HTA agencies) in Australia, Canada, Europe, and Asia.

Seven regulators, seven HTA agencies, and nine companies responded to the survey. All regulators and HTAs indicated they have interactions with their peer agencies, as well as between regulators and HTA. The top areas of interactions for regulators were formal work-sharing between regulators during review (86% response) and regulatory strengthening (86%), whilst for HTAs, interactions between HTA on methodology/framework (83%) and HTA capacity building (67%). Regulatory-HTA interactions were seen to have fewer practical benefits, which may suggest areas for improvement. Both companies and agencies believed an effective engagement model should support evidence generation; agencies also viewed an aligned process and improved decision-making as important. Respondents believed that an ideal ecosystem for interactions should facilitate separate remits for stakeholders, converged requirements, aligned process and increased transparency and trust.

This survey provided a snapshot of the current landscape interactions between stakeholders during the life cycle of new medicines, identified the areas where value is added and improvement are needed. Suggested building blocks to improve future interactions included early scientific advice, alignment of evidence requirements, and a collaborative approach among all stakeholders.

There is a range of activities that health technology assessment (HTA) doers have started to improve the process of generation of required evidence for new technologies, and the alignment of regulatory and reimbursement processes that retard the access to patients to them. Different organizations call those processes early advice, early dialogue, or scientific advice to those activities.

We performed a systematic review of the activities named scientific advice (SA), early advice (EA) and early dialogue (ED). Major databases and HTA organizations were explored. The protocol and search strategy were published in PROSPERO. The selection of final articles and documents was done in pairs, and when discrepancies were found a third person resolved with the consensus of the others. A matrix was used to define the commonalities and differences of the described processes.

We initially retrieved 949 documents, after the analysis of duplications and the full text reading of the selected ones, we finally selected 39 documents and described: the type of technologies, the process, the stakeholders, the duration, the costs, and the impact. Big HTA agencies such as the Canadian Agency for Drugs and Technologies in Health (CADTH) or the National Institute for Health and Care Excellence (NICE) included EA or SA among their portfolio of activities as well as networks (European Network for HTA (EUnetHTA) or smaller agencies such as HTA Wales or Basque Office for HTA (Osteba) among others. The type of activity, the process, duration, purpose and costs differ among HTA doers.

There is a need to define what we meant when we are talking about SA, ED, and EA. In fact, regulators used the same processes with different purposes. Our systematic review and the lessons learnt from the European-funded SAFENMEDTECH project will propose a detailed framework that can be useful to better understanding the needs of each of the involved parties and how to make the processes involved more efficient.

The Washington Health Technology Assessment (HTA) program has been guiding coverage decisions in the state since 2007. The Center for Evidence-based Policy works with the program to develop evidence-based HTA reports. In 2020, we presented an HTA on cell-free DNA prenatal screening for chromosomal aneuploidies. In the committee’s discussion, questions around access to screening tests and to prenatal care more generally were raised. We present a case study of how health equities were considered in the development of coverage criteria.

We conducted an HTA using standard systematic review methodologies. Outcomes focused on test accuracy and the impact of screening. We did not look for evidence on access to prenatal screening tests or people’s experience of prenatal screening. We reviewed the meeting transcript to identify issues of health equity and how they influenced the final decision.

During the discussion of the evidence, the committee raised concerns around equitable access to cell-free DNA tests and prenatal screening, including: direct costs to the person; access to the full range of prenatal screening, including ultrasound; and, uptake of prenatal screening. Based on the findings from the evidence report, expert testimony, and public comment, the committee voted to cover cell-free DNA prenatal screening for chromosomal aneuploidies unconditionally.

Health equity is increasingly important in healthcare decision-making. Decision makers should consider how a decision may reduce health inequities and how it may inadvertently increase existing health inequities. Decision makers also need to understand the context within which the decision will be implemented. Consideration of health equity can be addressed in a number of ways, for example through systematic review of equity issues or patient experience or through the use of contextual knowledge from expert and public testimony. Regardless of the method, decision makers should remain transparent in how health equity considerations influenced their final determination.

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.

Coverage of childhood cancer treatment under the Ghanaian National Health Insurance Scheme (NHIS) has been a policy discussion recently. To improve priority setting, Ghana introduced and used health technology assessment (HTA) processes to guide the resource allocation. To understand the role of stakeholders throughout the HTA lifecycle and for this decision, a stakeholder mapping and engagement was undertaken. We share our results of this mapping and analysis for improving management and financing of childhood cancers in Ghana.

We used two main approaches; first, we undertook a systematic policy documents and literature review of stakeholders relevant in childhood cancer management and financing in Ghana. This was followed by a stakeholder engagement workshop of key stakeholders from the Ministry of Health, Ghana Health Service (GHS), National Health Insurance Authority (NHIA), academia, non-governmental organizations (NGOs), private sector, teaching hospitals, patient groups, and civil society organizations. Participation was done in-person and virtual. Questions were moderated using a focus group discussion approach where responses were recorded. Data were analysed using synthesis and development of themes. Mapping of stakeholders was done using Mendelow’s power-interest grid.

The mapping identified eight key stakeholders in different roles: policy makers (Ministry of Health), payer (NHIA), healthcare providers (teaching hospitals, GHS, private hospitals), pharmaceutical companies, patient group (Ghana Parents Association for Childhood Cancers), and advocacy group (NGOs). Analysis showed that power and interest are concentrated with Ministry of Health and NHIA primarily because of control over resources. Also, while healthcare providers, patient groups, and advocacy groups have high interest, their power ranges from low to moderate. Further analysis of data from the workshop revealed that inability to pay for high cost of treatment leads to treatment abandonment. Payment for treatment was mainly out-of-pocket and by donation from philanthropist.

There was a strong will from stakeholders to extend coverage of the NHIS to childhood cancers in Ghana. Stakeholder engagement is a powerful tool and should be an integral part of every HTA process.

Blood collection in a pediatric population is a time-consuming activity and an unpleasant experience. Moreover, many laboratory tests require only small amounts of blood while larger quantity of blood is usually drawn, generating excess waste that must be properly disposed of. To solve patient concerns and workflow inefficiencies biomedical companies developed Pain-Free Blood Draw (PFBD) devices. The aim of this health technology assessment (HTA) study is to compare the performances of PFBD devices with the standard venipuncture to evaluate the potential benefits of introducing PFBD devices into clinical practice.

PFBD devices use microneedles that breach the stratum corneum, significantly reducing the pain perception due to the superficial skin penetration. Decision-oriented HTA method, was applied to conduct the HTA process. It is an analytical instrument that integrates the EunetHTA CoreModel with the analytic hierarchy process, to choose the best technology solution by identifying the main evaluation criteria and defining the weightsof system and performance values. Eight professionals have been involved to define the evaluation criteria and to measure the two technologies’ performance. As the method requires, a literature review was conducted to define the evaluation scheme represented by a multilevel decision tree composed of evaluation areas (domains) and key performance indicators (KPI).

Five evaluation domains were included in the analysis (clinical effectiveness, safety, costs, organizational aspects, and technical characteristics), described by 35 KPIs. Preliminary clinical effectiveness results showed diagnostic concordance between blood samples obtained with PFBD and venipuncture. Even if the additional costs of PFBD, these devices seem to improve the safety by reducing the biological risks for operators. Moreover, considering pediatric patients, organizational aspects would benefit by the use of PFBD in terms of ease of use, compliance of patients, and time reduction for blood collection.

Results showed that PFBD not only have great repercussions in terms of clinical benefits, especially for pediatric patients, but also a significant impact in terms of organizational aspects.

SINTESIS-new technologies is the early warning system for new and emerging technologies of the Agencia de Evaluación de Tecnologías Sanitarias, Instituto de Salud Carlos III. SINTESIS is part of the Action Plan for the Early Detection of New and Emerging Technologies of the Spanish Network of Health Technology Assessment Agencies (RedETS). In RedETS, four agencies are responsible for the identification of innovative technologies. These agencies have been collaborating since 2016 according to the early awareness methods contained in the EuroScan Methods Toolkit. SINTESIS focuses on secondary information sources (i.e., experts and literature). This study describes the experience of SINTESIS in identifying and filtering new technologies in recent years.

Retrospective analysis of all new and emerging technologies notified by SINTESIS to RedETS since 2018. Technologies were analyzed on a year-by-year basis for their source of information, the clinical specialties involved, and whether technologies found in the identification phase were selected for further assessment.

Between 2018–2020, SINTESIS identified 69 emerging and new technologies. Most of the information came from medical press news (35%), and medical web news (22%); other sources included experts (15%), licensing news search (12%), general press (12%), and scientific websites (6%). Almost 37 technologies (54%) were selected for further analysis. Reasons for exclusion included too early identification of technologies/prototypes without enough evidence (52%), technologies already implemented (28%), overlapping technologies between agencies (17%), and not being medical technologies (3%).

Experience suggests that news sections of general and medical journals, websites, and expert consultation are useful sources to identify new and emerging health technologies. The main limitation is that the technologies identified are often at too early a stage of development for further assessment. SINTESIS contributes, within a national horizon scanning system with other agencies, to broaden the information sources and provide useful data on early awareness of innovative technologies. Further studies are needed to assess the impact of emerging technologies detection on healthcare delivery.

The reimbursement of medicines by the Spanish National Health System (NHS) is based on a set of criteria included in the Royal Legislative Decree 1/2015 (RDL 1/2015). The Interministerial Committee on Pricing of Medicines and Healthcare Products (CIPM) is responsible for the final price and reimbursement (P&R) decision, including on its resolutions the criteria listed in the law by which the reimbursement of a drug is approved or denied. Nevertheless, the information behind its reasoning is not provided. The present study aims to characterize the P&R criteria of the RDL 1/2015 through criteria definitions from other countries to improve the P&R evaluation in Spain.

A multidisciplinary experts panel with relevant experience in drug evaluation and decision making at national, regional, and local level in Spain was selected for this study. A literature review to characterize the criteria listed in the RDL 1/2015 was performed based on the most relevant and recognized Health Technology Assessment (HTA) agencies in Europe, UK, and Canada. Eventually, a feasibility study was performed to evaluate the novel drug cefiderocol using the characterized criteria, including a reflective discussion of the results.

Consensus was reached among the multidisciplinary experts on the characterization of the criteria set by the law. The feasibility of their application to a new drug was exploratory, notwithstanding it showed the potential to improve the transparency as well as to offer a more structured rationale for the CIPM to evaluate the inclusion of new drugs in the Spanish NHS.

The aim of this study was to find ways of bridging the gap in opinions concerning health technology assessment (HTA) in reimbursement submission between manufacturers and payers to avoid access delays for patients of vital medicines such as oncology drugs. This was done by investigating differences and similarities of opinion among key stakeholders in Australia.

The survey comprised of nine sections: background demographics, general statements on HTA, clinical claim, extrapolations, quality of life, costs and health resource utilization, agreements, decision making, and capability/capacity. Responses to each question were summarized using descriptive statistics and comparisons were made using chi-square statistics.

There were ninety-seven respondents in total, thirty-seven from the public sector (academia/government) and sixty from the private sector (industry/consultancies). Private and public sector respondents had similar views on clinical claims. They were divided when it came to extrapolation of survival data and costs and health resource utilization. However, they generally agreed that rebates are useful, outcomes-based agreements are difficult to implement, managed entry schemes are required when data are limited, and willingness to pay is higher in cancer compared to other therapeutic areas. They also agreed that training mostly takes place through on the job training and that guideline updates were a least favored opportunity for continued training.

Private sector respondents favor methods that reduce the incremental cost-effectiveness ratio when compared to the public sector respondents. There still exist a number of challenges for HTA in oncology and many research opportunities as a result of this study.

While various criteria exist to define or categorize innovative medicines as new or repurposed, to our knowledge there are no standardized systems that sufficiently capture the range of pipeline products. The National Institute for Health and Care Research Innovation Observatory (NIHR IO) undertakes routine horizon scanning to support health technology assessment (HTA) in England and maintains a comprehensive Medicines Innovation Database (MInD). The aim of this project is to develop a ‘technology type’ (new versus repurposed) classification system for application within the MInD and to provide a high-level analysis of the emergent data.

We reviewed gray literature, regulatory websites, and drug repositories to identify existing ‘technology type’ classification criteria. Preliminary definitions and classifications for use on the MInD were discussed, refined, and agreed by consensus. Innovative medicines on the MInD were classified as either new or repurposed based on their regulatory approval status (Marketing Authorization) using data from the electronic medicines compendium. For repurposed medicines, further classification was undertaken using abbreviated new drug application (ANDA) data from the FDA Orange Book to identify generic medicines (patency and exclusivity status). We combined a range of semi-automated and manually derived data during this process.

Six technology types were identified and applied to the MInD: (i) new technology; (ii) repurposed technology (on-patent/branded); (iii) repurposed drug (off-patent/generic); (iv) repurposed technology (never commercialized); (v) new and repurposed technology (combinations); and (vi) repurposed technology (combinations). Preliminary analysis of a subset of MInD records identified in July 2021 (n = 113) found mainly 52 percent new technologies, 27 percent new and repurposed technologies (combinations) and 14 percent repurposed technology (never commercialized). Further analysis of approximately 7000 MInD records are ongoing and will report temporal trends, regulatory status, and key challenges.

Our novel evidence-based approach to developing classifications for technology types of innovative medicines resulted in six mutually exclusive states that can be applied to a larger dataset. We believe this offers HTA stakeholders a mechanism to gain valuable insights into the innovation trends, gaps, and areas of unmet need.

The National Institute for Health and Care Research Innovation Observatory (IO) is a horizon scanning centre based at Newcastle University, United Kingdom. The IO provides horizon scanning intelligence on new and innovative medicinal products to the National Institute for Health and Care Excellence (NICE) as technology briefing notifications (TBNs). We present an analysis of how TBNs produced between April 2017 and October 2021 feed into the NICE HTA process and used to inform their Technology Appraisal (TA) programme.

TBNs were mapped to relevant published NICE TA guidance and time from horizon scanning identification to NICE recommendation was studied. For mapping technologies undergoing appraisal, provisional guidance-in-development (GID) identification numbers (IDs) were used. For technologies that had not reached the NICE scoping stage yet, the NICE Topic Selection decision and ID was used.

Six hundred and ninety-three TBNs were submitted to NICE between April 2017 and October 2021; 653 were prioritised for TA. Of those, eleven percent mapped to a published NICE TA guidance; forty-three percent to a GID, twenty-two percent were undergoing consultation, and three percent were not traced. Further twenty-one percent mapped to a suspended or terminated TA. Reasons for this included HTA timeliness, regulatory issues or companies unwilling to submit evidence to NICE. Time from technology identification to TA guidance publication ranged from twenty-two to 115 months. The average time from TBN submission to NICE recommendation was thirty months.

Timely notification is key in achieving TA recommendation aligned with market authorization but not the only influencing factor. After issuing a TBN, the NICE appraisal process might be terminated, suspended or withdrawn due to unforeseen factors. Horizon scanning plays a key role triggering the NICE TA process; understanding factors that influence the successful TA completion would streamline processes and find efficiencies.