Unlike randomized controlled trials, lack of methodological rigor is a concern about real-world evidence (RWE) studies. The objective of this study was to characterize methodological practices of studies collecting pharmacoeconomic data in a real-world setting for the management of type 2 diabetes mellitus (T2DM).

A systematic literature review was performed using the PICO framework: population consisted of T2DM patients, interventions and comparators were any intervention for T2DM care or absence of intervention, and outcomes were resource utilization, productivity loss or utility. Only RWE studies were included, defined as studies that were not clinical trials and that collected de novo data (no retrospective analysis).

The literature search identified 1,158 potentially relevant studies, among which sixty were included in the literature review. Many studies showed a lack of transparency by not mentioning the source for outcome and exposure measurement, source for patient selection, number of study sites, recruitment duration, sample size calculation, sampling method, missing data, approbation by an ethics committee, obtaining patient's consent, conflicts of interest, and funding. A significant proportion of studies had poor quality scores and was at high risk of bias.

RWE from T2DM studies lacks transparency and credibility. There is a need for good procedural practices that can increase confidence in RWE studies. Standardized methodologies specifically adapted for RWE studies collecting pharmacoeconomic data for the management of T2DM could help future reimbursement decision making in this major public health problem.

According to international experience in the field of hospital-based health technology assessment (HB-HTA), most of the implemented new health technologies must undergo a clinical and economic assessment (CEA) of their viability by creating a mini-health technology assessment report. However, HB-HTA should not be limited only to the initial CEA; further monitoring of the effectiveness of implemented new health technologies is necessary.

We developed a special reporting form for creating a CEA of implemented new health technologies and integrated it into the hospital information system. Indicators of clinical effectiveness are determined individually for each implemented technology. The main indicators of economic effectiveness are financial results (or net profit) and profitability—high-cost and high-tech health technologies have priority for monitoring.

In order to ensure a more detailed and complete CEA of implemented health technologies, the following measures were proposed: (i) before implementing the technology, determine the key clinical effectiveness criteria for further monitoring for each implemented health technology; (ii) if possible, determine comparative technologies (alternatives or analogs) for conducting comparative CEA of the implemented health technologies; and (iii) carry out a prospective CEA of the implemented health technologies with a view to publishing the results.

The organization of a continuous monitoring process that analyzes the effectiveness and usage of new health technologies in hospital practice will allow assessment of the following: the clinical effectiveness and safety of the implemented technologies in comparison with world data; the economic effectiveness of the technology, including an accurate calculation of the payback period for investments; and the “real” data on the effectiveness of implemented health technologies in comparison with the initial request for implementation.

The 2019 Health Technology Assessment International (HTAi) Asia Policy Forum (HAPF) discussed the role of horizon scanning (HS) and health technology reassessment (HTR) in supporting sustainable healthcare in Asia.

Discussions and presentations at the 2019 HAPF, informed by a literature review, results of a premeeting survey, and case studies of HTA agencies from the region form the basis of this paper.

Five of the fourteen HTA agencies surveyed have established or are developing HS systems (HSSs), and six reported some experience with HTR. Although there were many differences in the respective HSSs, all were proactive and identified technologies within a 1–3-year time horizon. Commonalities included the criteria used to prioritize the technologies including burden and rarity of the disease, and cost to the health system and patients. Experience in HTR was not as extensive with most HTR activity being conducted on an ad hoc basis. Challenges for both HS and HTR include a lack of HTA capacity and good quality data, as well as a need for transparency and collaboration with industry.

With developing universal health care and HTA capacity in many countries in Asia, HS and HTR are in their infancy. Whilst several countries in the region are experienced in all facets of HTA, other countries are developing their HTA skill base. As such, there was a clear appetite for setting up a collaborative network in the Asia region to facilitate information sharing around HTA activities, and more specifically, HS and HTR methodologies, experiences, and assessments.

Advanced Therapy Medicinal Products (ATMPs) are innovative biologics (gene, cells and tissue-based products) with the potential to treat diseases with significant unmet clinical need. ATMPs pose distinct regulatory, health technology assessment (HTA) and patient access challenges, hence early identification and prioritization of ATMPs is now recognized as a key concern in England. The National Institute for Health Research Innovation Observatory (NIHRIO) uses a robust methodology to identify and monitor health technologies, including ATMPs that meet the remit of key HTA stakeholders in England. This analysis provides a global overview of the current ATMPs pipeline to administer useful insights for policymakers, funders and innovators.

NIHRIO's database tracks pharmaceuticals from phase I/II onwards, but this analysis focuses on late-stage development. The database (N > 12,000 records) was filtered to identify potential ATMPs using a predefined criteria based on the European Medicine's Agency's classification. Each record is categorized by stage: ‘Active’, (with an estimated three years to European licence); ‘Monitoring’ (in development with no licence date); and ‘Finished’, (output produced/discontinued and no longer tracked). Subsequently, records in ‘Active’ and ‘Monitoring’ were examined further.

Analysis identified 636 ATMPs: five percent ‘Active’, 40 percent ‘Monitoring’ and 55 percent ‘Finished’. ATMPs in the Active/Monitoring stages included: gene therapies (52%), somatic cells (43%) and tissue-engineered products (5%). Of these, 40 percent were oncological with the majority targeting hematological cancers (lymphomas). Prevalent non-oncology areas included musculoskeletal (10%) and ophthalmology (8%). Over one-third of trials were phase IIs, with almost half of all trials were based in the US.

The overarching findings here indicate increasing development of the ATMP pipeline towards indications with significant unmet clinical need. In oncology, the high prevalence of hematological ATMPs is largely due to recent chimeric antigen receptor T cells (CAR-T) innovation. In non-oncology areas, ATMP development is increasing due to advances in regenerative medicine. With a significant number of ATMPs projected to be licenced within three years, and many more in active late-stage trials, HTA bodies and health systems are challenged to prepare for the entry of these innovative therapies.

Because of a lack of suitable heart donors, alternatives to transplantation are required. These alternatives can have high costs. The aim of this study was to perform a systematic review of cost-effectiveness studies of ventricular assist devices (VADs) and to assess the level of evidence of relevant studies. The purpose was not to present economic findings.

A systematic review was performed using four electronic databases to identify health economic evaluation studies dealing with VADs. The methodological quality and reporting quality of the studies was assessed using three different tools, the Drummond, Cooper, and CHEERS (Consolidated Health Economic Evaluation Reporting Standards) checklists.

Of the 1,258 publications identified, thirteen articles were included in this review. Twelve studies were cost–utility analyses and one was a cost-effectiveness analysis. According to the Cooper hierarchy scale, the quality of the data used was heterogeneous. The level of evidence used for clinical effect sizes, safety data, and baseline clinical data was of poor quality. In contrast, cost data were of high quality in most studies. Quality of reporting varied between studies, with an average score of 17.4 (range 15–19) according to the CHEERS checklist.

The current study shows that the quality of clinical data used in economic evaluations of VADs is rather poor in general. This is a concern that deserves greater attention in the process of health technology assessment of medical devices.

Gather health technology assessment (HTA) experts' insights on the desirability and acceptability of treatment-sequencing models applied to relapsing-remitting multiple sclerosis (RRMS).

Primary data.

In-depth double-blind semi-structured telephone interviews.

General themes were extracted from qualitative interviews.

Although experts confirmed the importance of evaluating the clinical and cost-effectiveness of treatments as part of a sequence, the current HTA decision making framework is not conducive to this. Developing an RRMS treatment-sequencing model that meets HTA requirements is difficult, in particular due to scarcity of effectiveness data in later treatment lines.

At present, a treatment-sequencing model for RRMS may be desirable yet not requested by HTA bodies for their decision making. However, there could be other areas where a treatment-sequencing model for RRMS is of use.

In Japan, a new cost-effectiveness evaluation system for medicine and medical device pricing was employed in April 2019 after a trial implementation. This study describes the discussions held from April 2016 to March 2019 concerning the newly introduced system.

Using published government documents, discussions with stakeholders, and the minutes of the Chuikyo committee meetings, the following issues are addressed: (i) the results of the trial implementation and (ii) an overview of the newly introduced system.

During the trial implementation, thirteen products were evaluated and their prices adjusted. The process of the new system—which was to be implemented in FY 2019—takes about 15–18 months to complete after listing of the target products by the National Health Insurance. The target products are selected principally based on sales volume, degree of innovation (premium), and disclosure of rationale for price setting. First, a manufacturer submits the cost-effectiveness data, which is then reviewed by the Center for Outcomes Research and Economic Evaluation for Health (C2H) in collaboration with academics. The results of the cost-effectiveness evaluation are not considered during the decision-making process concerning the product's listing. The price adjustment system is similar to value-based pricing (VBP); hence, the new system can be considered as VBP adjustment.

Cost-effectiveness evaluation can help promote both technological innovation and sustainability of the healthcare system. We need to create a greater capacity for enhancing this academic review system.

Current clinical practice is based on guidelines and local protocols that are informed by clinical evidence. This means that clinical variability is reduced, but can lead to inefficient clinical decision-making and can increase medical errors, decreasing patient's safety. The aim of the EXCON project is to investigate the innovative concept of Intelligent Clinical History (ICH), and to develop functional prototypes of high added-value in healthcare services.

The innovative EXCON project will take advantage of recent advances in technologies for coding, structuring and semantizing medical information. Thanks to this new structuring, the EXCON platform will be developed. The final users will be health professionals and other decision-makers. Doctors, nurses, epidemiologists and information specialists will be involved in the development and subsequent validation of the platform.

The EXCON platform identifies profiles of patients with a high probability of ischemic heart disease. In the sample analyzed (n = 4,700), 17 percent of patients were admitted to a cardiology unit with suspected coronary heart disease. Of the patients admitted, 53.7 percent did not have ischemic heart disease at discharge. If we apply the algorithm developed by the EXCON project, 24.8 percent of patients would not have been admitted and did not have ischemic heart disease.

In coming decades, patient management will be impacted by the application of new advanced data analytics tools. This will allow for safer and more efficient clinical management, decrease variability in clinical practice, and improve equity. That is why the development and assessment of these technologies is necessary.

Stunting increases a child's susceptibility to diseases, increases mortality, and is associated over long term with reduced cognitive abilities, educational achievement, and productivity. We aimed to assess the most effective public health nutritional intervention to reduce stunting in Myanmar.

We searched the literature and developed a conceptual framework for interventions known to reduce stunting. We focused on the highest impact and most feasible interventions to reduce stunting in Myanmar, described policies to implement them, and compared their costs and projected effect on stunting using data-based decision trees. We estimated costs from the government perspective and calculated total projected cases of stunting prevented and cost per case prevented (cost-effectiveness). All interventions were compared to projected cases of stunting resulting from the current situation (e.g., no additional interventions).

Three new policy options were identified. Operational feasibility for all three options ranged from medium to high. Compared to the current situation, two were similarly cost-effective, at an additional USD 598 and USD 667 per case of stunting averted. The third option was much less cost-effective, at an additional USD 27,741 per case averted. However, if donor agencies were to expand their support in option three to the entire country, the prevalence of 22.5 percent would be reached by 2025 at an additional USD 667 per case averted.

A policy option involving immediate expansion of the current implementation of proven nutrition-specific interventions is feasible. It would have the highest impact on stunting and would approach the WHO 2025 target.

The aim of this study was to suggest options for a national and standardized process for the reimbursement of costly drugs provided in Austrian hospitals.

For answering the research questions, reimbursement processes of ten countries were investigated and the strengths and weaknesses of elaborated options of actions were analyzed, resulting in suggestions for solutions in the Austrian reimbursement processes for hospital drugs.

Based on the information derived from the international analysis and the deliberation of the strengths and weaknesses on optional approaches, as well as, on the consideration of the existing reimbursement processes in Austria, three options to reorganize the current decentralized inpatient reimbursement process in Austria were suggested. The first option presents a process following the established processes of the decision making for outpatient drugs. The second option suggests stronger coordination of and cooperation across the existing processes of the nine regional “Pharmaceutical and Therapeutics Committees”. The third option proposes to expand the already established reimbursement process for non-drug interventions.

Evidence-based, transparent, fair and efficient resource allocations are needed for priority setting decisions. However, a decision process can be based on the best available evidence, can be fair and transparent, although it might be substantially more time-consuming. Thus, a pragmatic balance between quality, transparency and timeliness is crucial.

To establish how real-world evidence (RWE) has been used to inform single technology appraisals (STAs) of cancer drugs conducted by the National Institute for Health and Care Excellence (NICE).

STAs published by NICE from April 2011 to October 2018 that evaluated cancer treatments were reviewed. Information regarding the use of RWE to directly inform the company-submitted cost-effectiveness analysis was extracted and categorized by topic. Summary statistics were used to describe emergent themes, and a narrative summary was provided for key case studies.

Materials for a total of 113 relevant STAs were identified and analyzed, of which nearly all (96 percent) included some form of RWE within the company-submitted cost-effectiveness analysis. The most common categories of RWE use concerned the health-related quality of life of patients (71 percent), costs (46 percent), and medical resource utilization (40 percent). While sources of RWE were routinely criticized as part of the appraisal process, we identified only two cases where the use of RWE was overtly rejected; hence, in the majority of cases, RWE was accepted in cancer drug submissions to NICE.

RWE has been used extensively in cancer submissions to NICE. Key criticisms of RWE in submissions to NICE are seldom regarding the use of RWE in general; instead, these are typically concerned with specific data sources and the applicability of these to the decision problem. Within an appropriate context, RWE constitutes an extremely valuable source of information to inform decision making; yet the development of best practice guidelines may improve current reporting standards.

Mixed home care, in which informal and professional actors work closely together, contributes significantly to ensuring home care up to old age. In this context, collaboration applications can considerably enhance the interactions among caregivers. However, although much research is conducted on need and requirement analyses of such applications, little is known about their introduction and use in care models. The purpose of this contribution is to identify studies that evaluate collaboration applications for mixed home care and compare their outcomes.

To identify literature on mixed home care collaboration applications (mHCA) and their evaluation, a systematic literature review was conducted in five bibliographic databases covering the years 2008 through 2019. The results were supplemented by a search in the meta-database Google Scholar. The evaluation approaches of the studies were analyzed and results compared by using the NASSS framework. Finally, a context concretized model was derived which summarizes interrelations.

Twelve qualitative studies evaluating eleven applications could be identified. They report on increased competency in self-management, psychological relatedness, involvement, and understanding. However, most studies conclude that large scale platform tests are still needed to prove significant changes in care processes, communication, or organization.

Among other things, their implementation is rather difficult due to the specifics of the target group. To enable a more targeted and successful implementation, it might be helpful to classify care networks beforehand and assess their communication behavior and needs. To prove the added value of mHCAs standardized assessment tools should be used.

Clinical atherosclerotic cardiovascular disease (ASCVD) patients are judged to be very-high-risk if they had a history of multiple major ASCVD events, or one major ASCVD event with multiple high-risk conditions. Very-high-risk ASCVD patients are under high risk of adverse clinical events and need more attention in the management of secondary prevention. This real-world study aimed at estimating the prevalence of very-high-risk ASCVD and investigating the occurrence of adverse clinical events and associated risk factors among patients with very-high-risk ASCVD in China.

Data were obtained from the Urban Employee Basic Medical Insurance database in Tianjin, China. Very-high-risk ASCVD patients were identified from 2014 to 2015 through the history of ASCVD events and evidence of high-risk conditions, and followed for 24 months. Adverse clinical events were measured by major adverse cardiovascular events (MACE), a composite endpoint of stroke, myocardial infarction (MI) and death. A Cox regression model was used to identify risk factors of MACE, adjusting for potential confounders.

The percentage of clinical ASCVD patients identified as very-high-risk was 35.2 (N = 41,181), while 34,740 patients with continuous enrollment were included (mean age: 67.1 years; 42.5% female). The percentage of patients who had MACE in the 24-month follow-up period was 27.7, with stroke (22.3%) as the most prevalent event followed by death (6.9%) and MI (1.3%). Male gender, older age, and having MI or ischemic stroke (versus unstable angina) as the index major ASCVD event were risk predictors of MACE.

More than one-third of patients with clinical ASCVD are under very-high-risk in China, and among them 27.7 percent experience MACE during a 24-month follow-up period. Male patients, older patients, and patients who had MI or ischemic stroke are under higher risk of experiencing MACE. Future studies are warranted for comparing the differences in characteristics, pattern of drug use, occurrence of adverse clinical events and medical burden between very-high-risk ASCVD patients and ASCVD patients not at very-high-risk.

One of the good practice principles for health technology assessment (HTA) is having a clear link between the assessment and decision making. The objective of the 2019 Latin American Policy Forum (LatamPF) of Health Technology Assessment International was to explore different models of connection between HTA and decision making and to discuss the potential applicability of such models in Latin America.

This paper is based on a background document and the deliberations of the members of the LatamPF (fifty-four participants from twelve countries) where a design-thinking methodology was used.

The participants agreed that insufficient links between HTA and decision making undermine the legitimacy of decisions, expose the HTA process to excessive political and judicial influence, and promote the exclusion of some stakeholders from participating in the assessment process and decision making. High priority aspects of the HTA process that could feasibly be improved and which hold the greatest potential to generate positive changes in the health systems in the region were identified. The majority of these aspects were associated with the appropriate institutionalization of HTA, a greater degree of participation by different stakeholders, and improved transparency in the HTA process.

The LatamPF identified barriers and recommended actions to strengthen the link between HTA and decision making. Participants emphasized that there is now a window of opportunity in the region as many societal actors see this as a priority. For this reason, health system stakeholders must take this opportunity to increase efforts toward strengthening the link between HTA and decision making.

There are large differences between the prenatal screening strategies for Down Syndrome (DS) in different provinces in China. In Henan province there is a serological triple screening in the second trimester (STS) strategy, while in Shandong province contingent non-invasive prenatal testing (NIPT) screening strategy (NIPT delivered to older pregnant women) is used, and there is a universal NIPT screening strategy in Anhui province. Moreover, many factors varied widely in different regions, such as the proportion of older pregnant woman and the ability of people to pay. This study aimed to determine the cost-effectiveness of current strategy in Shandong compared with strategies in other provinces.

A decision tree model was developed according to the screening strategies in different provinces. Four screening strategies were involved, universal STS strategy, contingent STS strategy, contingent NIPT strategy, and universal NIPT strategy. Cost-effectiveness analysis was conducted from a societal perspective in a simulated cohort of 100,000 pregnant women. The data of costs and epidemiologic parameters were collected from field surveys in Shangdong and a literature review.

The universal STS strategy, contingent STS strategy, contingent NIPT strategy, and universal NIPT strategy could prevent 17.0, 40.0, 46.2, and 53.6 DS births, respectively. There was no strategy dominated by others. The universal NIPT strategy and contingent NIPT strategy would decrease invasive procedures for prenatal diagnosis, resulting in fewer procedure-related miscarriages. The sensitivity analysis showed that the effectiveness of the screening strategy is significantly influenced by the resident's acceptance of NIPT.

From the perspective of maximizing the effect, the universal NIPT strategy is the optimal strategy. But taking into account the resident's and government's ability to pay, contingent NIPT Strategy may be appropriate for the current situation in Shandong. To ensure a better cost-effective advantage in the universal NIPT strategy, the government should provide health education to increase the acceptance of NIPT while controlling the price of NIPT.

The pan-Canadian Oncology Drug Review (pCODR) evaluates new cancer drugs for public funding recommendations. While pCODR's deliberative framework evaluates overall clinical benefit and includes considerations for exceptional circumstances, rarity of indication is not explicitly addressed. Given the high unmet need that typically accompanies these indications, we explored the impact of rarity on oncology HTA recommendations and funding decisions.

We examined pCODR submissions with final recommendations from 2012 to 2017. Incidence rates were calculated using pCODR recommendation reports and statistics from the Canadian Cancer Society. Indications were classified as rare if the incidence rate was lower than 1/100,000 diagnoses, a definition referenced by the Canadian Agency for Drugs and Technologies in Health. Each pCODR final report was examined for the funding recommendation/justification, level of supporting evidence (presence of a randomized control trial [RCT]), and time to funding (if applicable).

Of the ninety-six pCODR reviews examined, 16.6 percent were classified as rare indications per above criteria. While the frequency of positive funding recommendations were similar between rare and nonrare indication (78.6 vs. 75 percent), rare indications were less likely to be presented with evidence from RCT (50 vs. 90 percent). The average time to funding did not differ significantly across provinces.

Rare indications appear to be associated with weaker clinical evidence. There appears to be no association between rarity, positive funding recommendations, and time to funding. Further work will evaluate factors associated with positive recommendations and the real-world utilization of funded treatments for rare indications.