Evaluating the impact of health technology assessment (HTA) is vital to measure its contribution to health and social care decision-making and improving citizen outcomes. Health Technology Wales (HTW) is a HTA body committed to evaluating the impact of our work. Here we present HTW’s impact evaluation approach with a case study for autologous hematopoietic stem cell transplantation (AHSCT) for highly active relapsing remitting multiple sclerosis (RRMS).

Using an outcomes-focused approach based on contribution analysis, HTW has worked with an external evaluation organization to develop a framework to measure the impact of our work. Data on impact was collected from both qualitative and quantitative sources, including social media metrics, surveys, and informal feedback from stakeholders. We engaged with various stakeholders, including clinicians, academics, patient organizations and other HTA bodies.

The technology appraisal and guidance were published in July 2020, recommending AHSCT for routine adoption to treat highly active RRMS. Patient groups welcomed the appraisal findings as an important step forward in recognising the needs of people with RRMS and felt that “people living with MS were listened to throughout the process”. Following publication online, the guidance has had approximately 500 views, and featured on the MS Trust website and in several news articles. The Welsh Health Specialist Services Committee, a commissioning body in Wales, recommended AHSCT for RRMS as a ‘high priority’ for funding in the WHSSC Integrated Commissioning Plan 2021-22.

Since its publication, we have been able to prospectively capture the impact of this guidance through various stakeholders groups and sources. Overall, responses have been positive and the guidance has supported decision makers in Wales. Ongoing evidence capture, including through HTW’s adoption audit processes, will add further understanding to the potential impact of our work.

Telemedicine strategies have been broadly introducing in health services during the COVID-19 pandemic, including in care of neurological diseases.

A rapid realist review was conducted using EUnetHTAs Core Model 3.0 and GRADE evidence to decision frameworks were used as frameworks to describe the ethical, legal, organizational, social and patient aspects (ELSI+) related to the use of teleneurology (TN) A scoping multistakeholder meeting helped defined the scope and research questions of the assessment. Patient representatives, clinicians, scientific society representatives with relevant experience in TN were invited and participated. Industry representatives were also present. Systematic searches for ethical, legal, organizational, social and patients related aspects were conducted. Additional manual searches contributed to contextualize these dimensions in the Spanish context. A narrative synthesis was undertaken.

Main results of the assessment of the ELSI+ aspects of TN were described. TN applications are diverse depending on the condition, objective of care and technology used. The implementation of TN lacks specific legal frameworks which implies legal uncertainty. TN may increase geographical accessibility to neurological care in remote areas and by reducing difficult commuting to specialized care centers. Nevertheless, accessibility is challenged by reduced access to technology, the digital divide, lack of health literacy or technologies not adapted to functional diversity. Therefore, equity is not guaranteed if it is offered as a non-voluntary basis or with no support. TN tends to be accepted by patients and carers if it has enough quality, saves travelling time and costs and does not dehumanize care as it is perceived as more flexible and convenient. Quality of TN needs an interdisciplinary team with skills to coordinate organizational aspects of the implementation which include among others, the planification of the support to patients and carers before, during and after the consultation. Health professionals may also need to learn adapted communicational and technological skills.

The implementation of TN poses many ethical, legal, organizational, social or patient-centered challenges.

In UK males, prostate cancer is the most common cancer, with over 47,500 diagnosed annually. Radiotherapy is a highly effective curative treatment but can be limited by dose to surrounding normal-tissues such as the rectum. Radiation to the rectum can be reduced by increasing the distance between prostate and rectum with a hydrogel spacer. Despite National Institute of Health and Care Excellence guidance, spacers are not widely funded in the UK. Limited funding has necessitated patient prioritization, without any existing consensus on method.

Studies have shown generally homogenous results in reduction of rectal toxicity across assessed subgroups, but the requirement to prioritize remains. One way of addressing the appropriate use of beneficial health technologies is the inclusion of end-user experts in decision-making. The study aim was to identify consensus among radiation oncologists on patient prioritization for rectal hydrogel spacers.

We conducted a Delphi study where six leading clinical oncologists and one urologist from across the UK experienced in using rectal hydrogel spacers participated in two rounds of online questionnaires and two virtual advisory board meetings.

The experts estimated that 83 percent of patients who could potentially benefit from a spacer were denied access. Overall, ten points of consensus were reached. Key ones concerning patient-access were:

• • Spacer use in eligible patients significantly reduces radiation dose to the rectum and toxicity-related adverse events.

• • Increased benefit is expected in patients on anticoagulation, with diabetes and with inflammatory bowel disease.

• • Increased benefit can be expected with ultra-hypofractionated radiotherapy, but radiotherapy modality is not a key consideration for patient selection.

• • Patients should have the opportunity to actively participate in the discussion regarding the use of a spacer.

Currently, not all patients who would benefit can access funding for hydrogel spacers. Consensus in this study indicates that appropriate health policy and funding mechanisms are warranted for patients, to provide equitable access to technologies improving quality of life.

In the past decade, health technology assessment (HTA) has narrowed its scope to analyses of mainly clinical and economic benefits. Technology challenges in the 21st century emphasize the need for holistic assessments to obtain accurate recommendations for decision-making, as in HTA’s foundations. Using the VALues In Doing Assessments of health TEchnologies (VALIDATE) methodology for complex technologies provides a deeper understanding of problems through analysis of stakeholders’ views, allowing for more comprehensive HTAs. This study aimed to assess a pharmaceutical clinical decision support system (CDSS) using VALIDATE.

Semi-structured interviews with different stakeholders were conducted in the following domains: problem definition (medication error [ME] occurrence and prevention); judgement of solution (existing preventive methods and previous experiences of the CDSS); background theories (future impact and personal beliefs); and barriers to and facilitators of implementation. The following individuals were interviewed: medical informatic specialists (n=3), pharmacists (n=2), nurses (n=2), physicians (n=2), CDSS company representatives (n=1), electronic health record developer (n=1), and health consultancy firm representatives (n=1). Content analysis was used to integrate and analyze the data.

The multistakeholder interviews identified various barriers to the acceptance and implementation of a pharmaceutical CDSS that were different from those reported in the literature. These included: (i) occurrence of ME (no traceability of medication taken or poor patient medication empowerment); (ii) perception of current level of MEs (huge improvement from ten years ago); (iii) perception of technology as a tool to prevent ME (not enough if only implemented at one point of care); (iv) previous experiences with a CDSS (low rates of development of CDSSs are due to medication prescriptions being digitalized last in hospitals); (v) CDSS metrics (input data should be measured to control CDSS performance); and (vi) other barriers.

Including facts and stakeholders’ values in problem definition and the scoping of health technologies is essential for the proper conduct of HTAs. Incorporating views from multiple stakeholders when scoping the assessment of health technologies brings additional values to literature findings, resulting in a more holistic evaluation. The lack of multistakeholder scoping can lead to inaccurate information and result in wrong decisions about if, when, and how to adopt a CDSS.

Before admission to the insured package, the price of a medicine is usually assessed on the basis of the value of the medicine for the patient: the effect size on health and survival must be in line with the costs. That seems like a fair starting point, but the use of such ‘value-driven’ models sometimes results in unrealistic prices. These prices in turn lead to discussions about limitations within the healthcare budget and may result in delays in the accessibility of medicines. The aim of this study was to review several alternative pricing models and propose possible applications of the models.

Six pricing models were selected that encompassed cost-based or cost- and value-based aspects. The models were reviewed within the context of the published group of medicines, followed by a discussion on their potential to aid in creating benchmarks for pricing negotiations.

Five cost-based pricing models and one value-based model with a cost-based aspect were found with potential applications. (i) The AIM-model for innovative medicines. (ii) The adjusted AIM-model for repurposed medicines. (iii) The Cancer drug pricing model for innovative oncolytics with information about health benefit. (iv) The discounted cash flow model for orphan medicines with information about health benefit. (v) The value-based rate of return pricing for innovative orphan medicines with information about health benefit. (vi) The P-quad model for drugs with a high budget impact for which there is no generic competition after intellectual property an regulatory exclusivity end.

We argue that it would be more logical for different categories of medicines to base prices on average costs, possibly combined with a bonus for innovation: the cost-based pricing method. The next step is to discuss the possible application of cost-plus methods with stakeholders including patients, industry, payers, and healthcare professionals.

Technological innovations in the health sector have economic implications that go beyond their effects on health expenditure, expanding into other areas of the state budget (e.g., the social security system). Furthermore, innovation can affect the production of wealth by workers and companies, which in turn affects tax revenues. In addition, the presence of chronic diseases tends to reduce the propensity to consume and changes the allocation of consumption between the different sectors. Allocative decisions in the health system are rarely supported by an analysis that combines the health effects of innovations and their consequences in the economic system.

The objective of this study was to estimate the value of management programs for patients suffering from type 2 diabetes mellitus that involved different levels of use of innovative technologies and drugs. A tax impact assessment methodology was adopted in the context of chronic conditions to analyze the effect of adopting alternative management models for patients with diabetes on the broader economic system.

Assuming a policy that reduces annual complications by 0.42 percent, there was an increase in tax revenue (cumulative value) of approximately EUR 28,175 and a reduction in productivity losses (cumulative value) of EUR 4,049,890. Projecting the impact on the age trend of the population up to 65 years of age with these estimates, it is possible to have an increase in tax revenue (cumulative value) equal to approximately EUR 7,050,598 and a reduction in productivity losses (cumulative value) equal to EUR 140,235,923.

In light of this work, providing remote patient support (telemedicine) and expanding the provision of innovative oral antidiabetic drugs to family physicians could improve care for patients with type 2 diabetes mellitus. This study provides decision makers with an immediately usable model to broaden the information base for planning and regulatory choices. In addition, it supports the use of economic evaluations that calculate the entire value of a technological innovation or health program.

Patients with severe rheumatoid arthritis (RA) disease may be treated with tumor necrosis factor (TNF)-α inhibitors. However, their efficacy is reduced by the presence of anti-drug antibodies. The objective of this HTA was to conduct a systematic review to investigate the effectiveness of using enzyme-linked immunosorbent assay (ELISA) tests (Promonitor, IDKmonitor, LISA-TRACKER, RIDASCREEN, MabTrack, and Sanquin Diagnostic Services) to measure levels of the drug and anti-drug antibodies for monitoring response to TNF-α inhibitors in patients with RA.

A range of bibliographic databases including MEDLINE, EMBASE and CENTRAL were searched from inception to November 2018. Studies were eligible for inclusion if they investigated ELISA tests in RA patients receiving treatment with a TNF-α inhibitor who had achieved the treatment target (remission or low disease activity [LDA]) or experienced a primary or a secondary non-response. The tests must compare with standard care where treatment decisions are based on clinical judgements and monitoring using a composite score such as the disease activity score 28 joints (DAS28). Risk of bias was assessed using the Cochrane (ROBINS-1) tool for non-randomized studies.

Two studies were included. One non-randomized controlled trial (non-RCT) compared standard care with therapeutic drug monitoring using Promonitor test kits in RA patients in remission/LDA receiving adalimumab, and a historically controlled study investigated Sanquin ELISA kits. The non-RCT study showed that there was a non-significant reduction in the risk of flare in the intervention group compared with the control group. Patients’ health-related quality of life outcomes were higher in the intervention group at all visits compared with the control group. However, this study had serious limitations because analyses were not performed using an intention-to-treat approach. The historically controlled study did not provide valid evidence on whether ELISA-based monitoring is clinically effective.

There is only limited, poor evidence available. There are considerable uncertainties on the effectiveness of using ELISA tests for monitoring response to TNF-α inhibitors in RA. Further controlled trials are required.

The incidence of beta thalassemia varies greatly in different regions of China. Blood transfusion combined with iron chelation and hematopoietic stem cell transplantation (HSCT) is the main treatment for beta thalassemia in China. This study aimed to reveal the specific treatment schemes used for patients with beta thalassemia and to evaluate their effects.

A search strategy was developed to identify articles published between 1 January 2010 and 30 August 2021 in the following literature databases: PubMed, Embase, the China National Knowledge Infrastructure, Wanfang Data, and the Chinese BioMedical Literature Database.

The most used iron chelation schemes in China for patients with beta thalassemia included deferoxamine (DFO) monotherapy, deferiprone (DFP) monotherapy, deferasirox (DFX) monotherapy, and combinations of DFP and DFO. Most studies reported that combinations of DFP, DFO, and DFX monotherapy performed better than DFO or DFP monotherapy alone in reducing the blood, heart, and liver iron load. However, the adverse effects of iron chelation affected patient compliance with treatment to a certain extent. Stem cells for patients receiving HSCT in China were mainly donated by human leukocyte antigen (HLA)-matched siblings or unrelated individuals. The five-year overall survival rate after HSCT ranged from 83 to 90 percent, while the five-year beta thalassemia-free survival rate ranged from 65 to 87 percent. Graft-versus-host disease and infection were the most common serious complications experienced by transplant recipients.

For patients in China with beta thalassemia, the most effective iron chelation treatment schemes were combinations of DFP, DFO, and DFX monotherapy. HSCT from HLA-matched siblings or unrelated donors resulted in a significant improvement in the cure rate for beta thalassemia. However, patients still need safer and more effective innovative treatments, and further evidence on existing treatments needs to be generated from larger scale studies in the Chinese population.

The use of Health Technology Assessment (HTA) in hospitals contributes to decision-making, professional training, greater interaction with technical to scientific knowledge, resource-saving, and partnerships. Hospitals are strategic for the field of clinical management and quality of care and are open to partnerships with national and international agencies and groups. Despite the hospital-based (HB)-HTA movement, the incipient application of HTA to the hospital decision-making process is related to incipient planning, and there is still much room for progress worldwide. The aim of this study was to analyze the uptake level of HB-HTA in diverse contexts.

A scoping review was conducted according to the methodology of the Joanna Briggs Institute, whose data analysis model consisted of the combination of Donabedian’s structure, process, and outcome categories and the dimensions of the project Adopting Hospital Based Health Technology Assessment in European Union (AdHopHTA).

We identified 270 studies, and after removing duplicates and reading full texts, 36 references met the eligibility criteria. Thirty-six hospitals were identified, of which there were 24 largescale hospitals with extra bed capacity. Twenty-three hospitals were affiliated with universities. Canada stood out with five university hospitals, four of which with public funding. Half of the identified hospitals had HB-HTA units (18/36). Hospitals with full uptake level of HTA corresponded to 75 percent of the sample (27/36), and the remainder had partially uptake level of HTA, or 25 percent of the hospitals in the review (9/36). There were no hospitals with incipient uptake level of HTA.

Measuring the uptake level of HTA in hospitals contributed to understanding how their participation has occurred in the field of HB-HTA. This study revealed the importance of identifying factors such as sustainability, growth, and evolution of HB-HTA in countries with and without a tradition in this field.

Pulsed-field ablation (PFA) is a new non-thermal ablative approach for treating paroxysmal and persistent atrial fibrillation (AF). It provides an alternative to the conventional thermal techniques of radiofrequency ablation and cryoablation. PFA is currently performed with the Farapulse PFA system (Boston Scientific, USA), which recently received CE marking in Europe. In this study, we describe the current evidence on the efficacy, safety, and cost-effectiveness of this new technology.

The Early Awareness and Alert System of the Agencia de Evaluación de Tecnologías Sanitarias-at the Instituto de Salud Carlos III- identified this new ablation approach in June 2021. An early technology assessment was conducted by reviewing relevant literature published to November 2021. The literature was identified by searching PubMed, Embase, the International Clinical Trials Registry Platform, ClinicalTrials.gov, and the Cochrane Library.

Seven relevant publications were identified, including three open-label trials for paroxysmal AF and one for persistent AF. Three studies comparing PFA with conventional thermal ablation techniques were also included. The safety results showed a lower incidence of complications (esophageal injury, pulmonary venous stenosis, pericardial effusion, and aortic injury) after PFA relative to conventional techniques. In terms of efficacy, PFA resulted in lower rates of AF recurrence in the medium and long term, compared with conventional techniques, and there were no differences in the lengths or success rates of the procedures. No cost-effectiveness studies were identified. Seven ongoing trials were identified, but no results have been published.

The current evidence for PFA in the treatment of AF showed good safety and promising efficacy. However, there is a lack of information on its effect on quality of life and the risk of death, disabling stroke, or cardiac arrest. Early evaluation of new and emerging technologies makes it possible to gather the minimum information necessary to support decisions on their inclusion in the healthcare system. In this case, based on current evidence, the widespread use of PFA is not recommended, except under a rigorous research protocol.

The emergence of direct oral anticoagulants (DOACs) has revolutionized the prevention of stroke-related non-valvular atrial fibrillation (NVAF). Although several DOACs are available, studies comparing the cost effectiveness of DOACs with vitamin K antagonists for NVAF are scarce. The objective of this study was to assess the cost effectiveness of DOACs and warfarin from the Hong Kong public institutional perspective to inform formulary listing decisions.

A previously developed Markov model was adapted to simulate the lifetime disease progression of a hypothetical cohort of 1,000 patients. Net monetary costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) were computed for the following competing alternatives: warfarin, apixaban (5 mg twice daily), dabigatran 110mg or 150mg (twice daily), and rivaroxaban (20mg once daily). Model inputs were sourced from local real-world evidence, landmark trials, and comprehensive literature reviews. Probabilistic sensitivity analyses and deterministic sensitivity analyses were conducted to address study uncertainties. The willingness-to-pay threshold was set at one times the gross domestic product (GDP) per capita (USD 46,091) per QALY gained.

In base case results, all DOACs provided greater improvements in QALYs at a lower cost than warfarin. Using apixaban as the reference for comparisons among the DOACs, dabigatran 110 mg resulted in greater costs and lower QALY gains and was dominated by apixaban, whereas dabigatran 150 mg provided an incremental QALY of 0.005 at an incremental cost of USD 326, leading to an ICER of USD 67,633 per QALY. The lifetime cost associated with rivaroxaban was lower than for apixaban (-USD 151), but with lower QALY gains (-0.147), resulting in an ICER of USD 1,029 per QALY. In probabilistic sensitivity analysis, the probability of warfarin, rivaroxaban 20 mg, dabigatran 110 mg, dabigatran 150 mg, and apixaban 5 mg being cost effective out of 2,000 iterations was 0 percent, 0 percent, 29.4 percent, 33.2 percent, and 37.4 percent, respectively.

The results indicated that apixaban was the most cost-effective treatment in the management of NVAF, compared with other DOACs and warfarin. This conclusion was consistent under all the uncertainty test scenarios.

Traditional Chinese Medicine (TCM) has become a common kind of health care in several countries, with increasing demands. This review aimed to appraise the reporting quality of economic evaluations of TCM in the National Reimbursement Drug List (NRDL) of China (2020 version), based on the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement.

The reporting quality of included economic evaluations was assessed by two independent reviewers using the CHEERS statement.

A total of 360 articles were retrieved, but only 38 economic evaluations met the inclusion criteria. No articles were compliant with all items of the CHEERS checklist. On average, the included economic evaluations satisfactorily met 10.93 of the CHEERS items (51.31%). The least reported CHEERS checklist items included: “Characterizing heterogeneity”, “Conflicts of interest”, “Discount rate”, and “Study perspective”, with an average score of 0.00, 0.05, 0.08, and 0.16, respectively.

The economic evaluation of TCM is still at an early stage, with an urgent need for improving the reporting quality. To promote the reporting quality of economic evaluations and further development of TCM, multiple measures focusing on reporting formula, policy, training, and new methodology are required.

Cataract surgery is the most frequently performed surgical procedure in the UK and posterior capsule opacification (PCO) is the most common complication post-surgery. Nd:YAG capsulotomy is the standard of care for treating PCO, although it bears a cost and is also associated with complications. The objective of this research was to estimate costs from a budget holders perspective associated with PCO related, post-cataract surgery resource use, comparing different single-piece intraocular lenses (IOLs) and utilizing results from a recently published audit of PCO incidence in the UK (n=601,084).

This research adapts the findings of the aforementioned audit to develop a cost-consequence analysis. The model is underpinned by the Nd:YAG rates of the included single-piece acrylic IOLs at 5 years. Nd:YAG related additional consultations and reported complications of the procedure were also included as variables of efficiency in the model. Estimates are presented from the perspective of a hospital setting in the UK, performing 3,000 cataract surgeries annually and extrapolated out to the broader cataract population (n=472,000). Costs were sourced from NHS Tariff documentation.

AcrySof IQ was associated with lower Nd:YAG procedures and additional consultations at 5 years post-cataract surgery compared to all other single-piece monofocal acrylic lenses included in UK Audit Report. Assuming 3,000 cataract surgeries carried out annually, this translated into potential cost savings for the AcrySof IQ lens ranging from GBP 7,993 (EUR 9,379) (versus Eyecee One) to GBP 194,502 (EUR 228,243) (versus Akreos Adapt). Extrapolating to the broader population cataract patients in the UK would provide for a cost-saving estimates in the region of GBP 1.25 to GBP 30.6 million (EUR 1.47 to EUR 35.91 million).

This economic analysis highlights that the appropriate choice of IOL for cataract surgery, as a direct consequence of lower ND:YAG capsulotomy rates may translate into significant savings both for UK hospitals and the national healthcare system.

The study aims to systematically review all articles on the economic evaluation (EE) of coronary stenting, to critically assess the reporting quality, and to summarize the results.

A systematic search was undertaken through seven databases (PubMed, Web of Science, Embase, CNKI, Wanfang data, Vip data and SinoMed.) from inception until March 2021, to identify economic evaluation articles comparing coronary stenting with other therapies, or among different stenting procedures. After screening articles and extracting data independently, we summarized methods, contents, and outcomes of the included articles and appraised their methodological quality using the CHEERS (Consolidated Health Economic Evaluation Reporting Standards) checklists. Then, the literature scores were standardized as a proportion of the total score, and stepwise multiple regression was constructed to verify the factors that might influence the quality of literature.

Of the 3,622 publications identified, 59 articles were included in this review. There were 33 cost-effectiveness studies and 26 were cost-utility studies. The quality of the reports varied between studies, with a standardized mean score of 0.76 (0.40-0.98). According to the Cheers checklist, “Introduction” had the lowest overall score (0.53), with many articles deficient in the description of the study’s perspective; “Discussion” had the highest overall score (0.86), with nearly three-quarters of the articles reporting the full content; “Title and abstract”, “Methods”, “Results”, and “Other” scored 0.71, 0.78, 0.74 and 0.66, respectively. According to the results of the stepwise multiple regression model, “Published year”, “National type”, and “Type of economic analysis” research were significantly associated with the quality of literature.

The quality of current research reports on the economics of coronary stenting is generally satisfactory, but there is potential for improvement and high quality reports can provide evidence to support decision making for policy makers.

With the increasing use of health economic evaluation (HEE) in decision-making and health resource allocation and management policy design has seen an increase in HEE studies on screening programs in China l. In addition to the quantity of HEE, the quality may be of particular concern as it influences the reliability of HEE evidence adopted in policy formulation. This study sought to assess the reporting quality of HEE on screening programs over the last 20 years in China and identify potential predictors and relevant recommendations to improve the quality of study reporting.

A search of HEE studies published in PubMed, Embase, CNKI and WANFANG from 2000 to 2021 was performed. Two reviewers independently extracted data and assessed the quality if reporting using the 24 item Consolidated Health Economic Evaluation Reporting Standards ( CHEERS) checklist. The CHEERS score for each study was converted into standardized 0-1 point scale. General liner regression was used to identify predictors associated with the reporting quality.

One hundred and thirty-three studies met the inclusion criteria. The mean standardized score for the included studies was 0.56 (title), 0.64 (abstract), 0.74 (introduction), 0.58 (methods), 0.40 (results), 0.70 (discussion), and 0.54 (other section). The number and reporting quality of articles published each year showed an overall upward trend. A greater proportion of studies were published in Chinese journal (69.2%), modelling-based (54.9%), conducted by universities/research institutions (45.9%), focused on non-infectious disease (84.2%), using cost-effectiveness analysis method (50.4%), published in non-specialty journal (60.2%), and declaring the funding support (76.7%). Items related to study perspective, discount rate, measurement of effectiveness, currency and price, analytical methods, uncertainty, heterogeneity and conflicts were under-reported. Published year, journal type, first author affiliation and economic evaluation type predicted higher score in regression analyses (p<0.05).

Overall, the quantity and quality of HEE on screening programs in China is improving, although there is a need to improve the use of on specific reporting items in the CHEERS criteria. The use of suitable evaluation guidelines will make the decision-making process more scientific.

Diagnostic testing and patient monitoring are important to diagnose potential diseases and to evaluate treatment regimens. Since diagnosis and treatment monitoring have no intrinsic effects, an economic evaluation of biomarkers is inevitably linked to the resulting therapeutic interventions, which depend on both physicians’ decisions and diagnostic accuracy of the test (i.e., sensitivity, specificity). In this review we analyzed the methodology of economic studies evaluating the management of the five most relevant non-communicable diseases, that is, obesity, cancer, diabetes, cardiovascular and chronic respiratory diseases.

A systematic search in Medline and the National Health Service Economic Evaluation Database (NHS EED) covering the last ten years served to identify health economic analyses of biomarkers used in diagnosing / monitoring. Findings were reviewed with respect to analytical method, reported outcomes and comparability.

The search yielded 680 abstracts in total out of which 280 full texts were reviewed and 77 sources included following predefined criteria. Most economic analyses (94%) evaluated the clinical outcome and costs of testing / monitoring in correlation to a corresponding intervention, 6 percent of the sources focused on the accuracy of the test or monitoring methods only. There were 61 studies that included an economic model; overall, 15 sources presented the outcome as cost per life year gained (CEA), 37 sources as cost per QALY gained (CUA), and 12 provided the outcome as both a CEA and CUA. In 16 analyses the outcome was presented in other economic terms as, for example, cost per additional case detected.

Determining the value of biomarkers requires consideration of the clinical consequences of a test result (incorrect treatment decisions, impact on morbidity, mortality, or quality of life) as well as the corresponding economic outcomes. Most of the identified studies considered at least one of these aspects. Results are presented in manifold ways but do not necessarily address decision makers’ needs. Thus, clear guidelines on economic evaluations of biomarkers are needed and should include broader health system views like affordability or the number of unnecessary interventions avoided.

The public health areas without commercial value continue to be underserved, while those of high profit for industry will not be sustainable for much longer. We hypothesize that the lack of equity and efficiency in the biomedical research and development system is mainly due to a pharma-led short-term profit orientation that ignores the values of other relevant stakeholders.

This pilot study reached some consensus on the principles of a co-created biomedical research and development process based on the preferred supplier (PS) model, which proposes a public health procurement system prioritizing business with companies fulfilling the “4 Share” criteria of priorities, risks and rewards, results, and outcomes to ensure that health needs are met.

A constructive health technology assessment, which included VALues In Doing Assessments of health TEchnologies (VALIDATE) methodology, was used to analyze the values and dissent of a pilot sample of ten global key informants. The methodology comprised qualitative techniques such as an online preliminary survey, in-depth semi-structured interviews, and a Delphi survey to reach a joint construction by reconstructing the stakeholders’ interpretive frames and applying an adaptation of the Richardson model to contested values.

There was consensus on combining efficiency and social justice norms by incentivizing diseases affected by market failure due to small subpopulations (e.g., rare diseases), low availability to pay, restricted use (e.g., antibiotics), and difficulty demonstrating results (e.g., Alzheimer’s disease). Stakeholders mainly agreed on the PS 4 Share principles, highlighting the need for price to be linked to impact modulated by tracked research and development costs and investments, as proposed by the PS model. More market incentives such as push, and especially pull incentives (market access), should be included. The PS model should be cause-solution oriented, promote open-disruptive innovation, and guarantee fast patient access.

The PS model achieved multistakeholder consensus for desirability (values) and plausibility (shifting incentives). Stakeholders pointed out the need to apply health technology assessment to further develop the model and pilot it in the European Union with the NextGenerationEU.

Horizon scanning (HS) is an early health technology assessment (HTA) method for raising early awareness among policymakers of promising new and emerging health technologies. The Agency for Care Effectiveness (ACE) in Singapore established a HS system in 2019 to complement its HTA process. While the HS system initially focused on cell and gene therapies, this was expanded in 2020 to include medical technologies (MTs). This abstract focuses on the role of HS in nominating MTs for early evaluation to guide subsidy decisions, with the intention of avoiding challenges in altering deeply entrenched practices.

The ACE methodology for HS aligns with the core principles and methods of international best practice. Generally, MTs addressing national healthcare priorities are tracked. To identify topics for subsidy evaluation, the local registration status of an MT was used as the main selection criterion because of its proximity to the technology’s early diffusion into the healthcare system. MTs with regulatory approval were selected for HTA and subsidy consideration. All nominated technologies were checked against eligibility criteria for HTA and then assessed against a standard checklist for prioritizing HTA topics.

Among the 1,025 MTs tracked by the HS system, 89 were locally registered and nominated for HTA. Following eligibility assessment, 26 topics remained. After the prioritization exercise six topics were shortlisted. To date, two evaluations have been completed to guide subsidy decisions and four topics are undergoing evaluation. Notably, 16 of the 26 eligible topics were excluded due to a lack of sufficient evidence, in terms of both quantity and quality, for evaluation.

HS can be a useful tool for identifying new MTs for evaluation and possible funding prior to further diffusion, but careful selection of the technologies is required to ensure a sufficient evidence base for evaluation. Moving forward, HS can also play a more active role in disinvestment of obsolete or low value health technologies.

The number of anti-diabetics listed in the Ministry of Health Medicine Formulary (MoHMF) and the proposal to change prescribing category for usage in primary care facilities has increased each year causing difficulty in monitoring effectiveness, safety and rational use of the treatment and budget management. This study aimed to describe the reassessment and selection strategy of Dipeptidyl Peptidase-4 Inhibitors (DPP4-i), a class of anti-diabetic medicines in the MoHMF.

A literature review on the comparative effectiveness and safety of all available DPP4-i in MoHMF were conducted. Comparative treatment cost and utilization of DPP4-i were analyzed. Approved MoHMF indications were listed and compared against approved Drug Control Authority (Malaysia) indications. Information on clinical guidelines recommendations and listing or reimbursement status in non-Ministry of Health Malaysia (MoHM) public institutions and other countries were obtained. All findings were presented to MoHM drug expert committee for DPP4-i selection strategy. The final recommendation based on consensus among drug expert committee and Pharmaceutical Services Program were presented to the MoHMF Panel.

The MoHMF Panel acknowledged that the efficacy and safety profile of all DPP4-i were equivalent across therapeutic group as supported by strong evidence, hence, their selection can be made via cost-minimization strategy. The call for contract tender for single tablet was conducted based on therapeutic group (drug class) to encourage price competition and contracts were awarded to two DPP4-i which offered the lowest treatment costs. Saxagliptin and vildagliptin were awarded as contract items while sitagliptin remained as local purchase item. Prescribing category for all DPP4-i in MoHMF were streamlined accordingly. Linagliptin was disinvested due to sufficient availability of alternatives.

Selection strategy and disinvestment has successfully reduced the number of DPP4-i listed in MoHMF thus allowing more efficient clinical and cost monitoring. Cost minimization through tender by therapeutic group was the first to be done and has efficiently provided an avenue for price competition which results in saving to MoHM.

The National Institute for Health and Care Excellence (NICE) intends to automate the way it monitors the uptake, impact, and value of its guidance. Traditionally this has been done by developing impact reports, long documents that, while well received, are time consuming to develop and can quickly become outdated.

We focused on a novel topic that would benefit from new data sources to examine its impact: a rapid guideline for managing the long-term effects of coronavirus disease 2019 (COVID-19). We shortlisted “measurable” recommendations within the guideline that were likely to be captured in data collections. We then reviewed available data sources that included relevant up-to-date data. Finally, we explored what existing methods were available to NICE for automating impact reporting.

For long COVID-19 we accessed OpenSAFELY, a secure, transparent software platform for primary care COVID-19 data that was developed in response to the pandemic. This captured data on the management of long COVID-19 in primary care as well as onward referral to specialist clinics. In addition, we accessed data from the CVD-COVID-UK/COVID-IMPACT Consortium, which links general practice records with primary care dispensing data. This enabled us to analyze the impact of the pandemic on the prescribing and dispensing of cardiovascular disease medications. Working with our digital team we developed an automated impact reporting dashboard using Google’s data studio. This enabled different views of the data, for example by region or socioeconomic status, to be presented in an automated way.

Automating the impact reporting of NICE guidance provides up-to-date information on its value to the health system. While we were able to collect new sources of data and automate some aspects of how these were viewed, full automation requires several enablers. These include an application programming interface between the data sources and NICE, and ensuring that NICE guidance is computer readable so that its measurement is practical in healthcare systems.