Technology Assessment Reports Research Articles

Metformin and second- or third-generation sulphonylurea combination therapy for adults with type 2 diabetes mellitus.

Metformin and second- or third-generation sulphonylurea combination therapy for adults with type 2 diabetes mellitus.

Innovation as a value in healthcare priority-setting: the UK experience

All healthcare systems operate with limited resources and therefore need to set priorities for allocating resources across a population. Trade-offs between maximising health and promoting health equity are inevitable in this process. In this paper, we use the UK’s National Institute for Health and Care Excellence (NICE) as an example to examine how efforts to promote healthcare innovation in the priority-setting process can complicate these trade-offs. Drawing on NICE guidance, health technology assessment reports and relevant policy documents, we analyse under what conditions NICE recommends the National Health Service fund technologies of an “innovative nature”, even when these technologies do not satisfy NICE’s cost-effectiveness criteria. Our findings fail to assuage pre-existing concerns that NICE’s approach to appraising innovative technologies curtails its goals to promote health and health equity. They also reveal a lack of transparency and accountability regarding NICE’s treatment of innovative technologies, as well as raising additional concerns about equity. We conclude that further research needs to evaluate how NICE can promote health and health equity alongside healthcare innovation and draw some general lessons for healthcare priority-setting bodies like NICE.

Interactive Summary of Findings tables: the way to present and understand results of systematic reviews.

The JBI Database of Systematic Reviews and Implementation Reports continues to enhance its systematic reviews by including GRADE (Grading of Recommendations Assessment, Development and Evaluation) Summary of Findings (SoF) tables. Summary of Findings tables are concise, tabular summaries of the evidence that address a specific health-related question.1 They include information about the main outcomes, the type and number of studies, the relative and absolute estimates of the effect or association, important comments and a plain language summary to aid interpretation and a rating of the certainty of evidence (also known as quality of the evidence).1 Several randomized trials have shown that well designed SoF tables improve understanding and retrieval of information from a systematic review2-4 and they are a standard feature of Cochrane and are often used in other reviews.5-7 However, in the GRADE Working Group's DECIDE project (www.decide-collaboration.eu)8 and during guideline development work, it became apparent that a “flexible standardization” of presenting information in SoF tables is required to further enhance understanding and uptake of information. These observations led to the development of electronic interactive versions of SoF tables (iSoFs) that allow presenting the same underlying information in several formats that vary in content and graphical layout. These electronic versions are available on dedicated websites and, from this issue of the journal, are linked directly from the standard SoF and text of a Joanna Briggs Institute (JBI) systematic review. An example is available in Vincze et al.9 evaluating the use of nutrition for gestational and postpartum weight management in this issue of the journal. Low certainty evidence from 23 studies in 5230 patients compiled in the review suggests that nutritional interventions may reduce gestational weight gain, a continuous outcome described in this iSoF: https://bit.ly/2Ofg9Av, by 1.25 (95% confidence interval: 0.4 to 2.1) kg compared with other interventions. Authors of JBI systematic reviews will use the GRADE Working Group's official tool, GRADEpro (McMaster University/EvidencePrime, Inc., Hamilton, ON, Canada), to create these iSoF tables. The integrated GRADE Handbook provides guidance for how to create iSoFs, how to embed them in other documents, such as systematic reviews, Health Technology Assessment reports and healthcare guidelines where they can function as decision support tools and aids. Detailed guidance for how to produce SoF tables is also available in GRADE research articles that describe good practices and the process for creating accurate SoF tables for interventions and diagnostic test accuracy reviews.4,10-14 Examples of the use of iSoFs include recent European Commission Breast Cancer guidelines and American Society of Hematology guidelines where recommendations are supported by an iSoF.15-18 We are delighted that the JBI has taken this innovative step to include iSoF tables in their reviews. These steps will make for better evidence integration in decision tools and sharing globally.

Enzymatic replacement therapy for lysosomal storage disorders: Drug evaluations review in Spain

IntroductionIn the European Union companies only need to demonstrate that the risk-benefit balance of the new drug is favourable to obtain the authorization to sell new drugs. Hence a comparison with available treatments, a cost-effectiveness analysis and the place in the therapy of the new drug are not required. Therefore, it is necessary to carry out these analyses in an additional study. In Spain there is no national government agency, which conducts a centralized evaluation and makes decisions on funding and are the Pharmacy and Therapeutics committees that take mandatory decisions for the entire region. ObjectivesTo identify all drug assessments and health technology assessment reports of the enzymatic replacement treatments for the four LSD considered in this study, including Mucopolysaccharidosis (MPS), Gaucher, Fabry and Pompe disease at the national, regional and hospital level; and to summarize the efficacy in terms of outcome measures, adverse events, economic impact and final recommendations and indicate potential improvements. Methods19 reviews at the regional and hospital level and one therapeutic positioning report at national level for MPS were considered for this. ResultsIn general, the drugs evaluated in LSD have proved to be effective in the most frequent clinical forms of disease. However, there are clinical phenotypes that still do not have available an effective treatment. ConclusionsIt is expected that new therapies, such as intrathecal therapies or gene therapy could be evaluated in the different types of LSD demonstrating positive effects, especially in the previous phenotypes.

VP11 Use Of Health Technology Assessment Adaptation In Latin America

IntroductionThe development of health technology assessment (HTA) reports is a time-consuming process that requires highly trained human resources. In many Latin American countries this type of personnel is scarce. The adaptation of HTA could be a time-saving process to get inputs for decision. The objective of this study is to determine the frequency of use of HTA adaptation process and to describe type of tools used in this process in Latin American countries.MethodsThe Health Technology Assessment Network of the Americas (REDETSA) is a non-profit network formed by ministries of health, regulatory authorities and health technology assessment agencies (PAHO/WHO). During the last meeting of REDETSA in November 2018, we performed an exploration survey to gather information related to the topic in order to promote the creation of an adaptation working group. The question was whether HTA agencies did adaptation of HTA reports and, if so, what methods and tools were used and what sections of the report were adapted.ResultsThirty-three institutions from fourteen Latin American countries answered to the consultation. Seven countries do adaptation of HTA (50 percent) and one country does adoption. Of those countries that adapt HTA, three do only economic transferability. Methods and tools are usually developed locally or there is not a systematic approach. In two countries, the economic study transferability tool developed by Hutter and Antoñaza is used.ConclusionsAdaptation of HTA is not well developed among Latin American agencies, although it seems to be an efficient strategy when assessing efficacy and safety. Adaptation of economic studies is still controversial; nevertheless, it is used in some of the countries of the region. It is necessary to advance in the development of HTA adaptation tools, developed and adapted to local contexts in the region.

PP72 Using INTEGRATE-HTA On The Example Of Rasterstereography For Scoliosis

IntroductionFull health technology assessment (HTA) reports discuss not only the safety and efficacy of a technology, but also the economic, ethical, legal, socio-cultural, and organizational aspects. INTEGRATE-HTA is a completed European Union project that developed concepts and methods for a patient-centered, integrated assessment of complex technologies. Technologies can be considered complex if they are characterized by a large number of interacting components, a wide variability of outcomes, or a high degree of flexibility. In contrast to the usual linear approach of addressing individual HTA domains separately, the INTEGRATE-HTA methodology is based on the assumption that different aspects of the domains interact. From the very beginning, these interactions are captured systematically using various tools. Continuous reflection and compaction of these relations can lead to an extended perspective on a technology. As a result, complexity and mechanisms of action open up, helping to channel public discussion and implementation. We investigated whether using the INTEGRATE-HTA methodology improves the understanding of individual domains and their interactions.MethodsAccording to the methodology, an initial logic model for rasterstereography in patients with scoliosis was developed and successively expanded. A synoptic table, showing multiple maps of individual aspects to domains, and a complexity checklist were used. In addition, harvest plots were created and the socio-cultural impact of the disease was highlighted as a semantic complex. A final logic model and an interaction figure were established to initiate discussion.ResultsHaving been classified as slightly complex in the beginning, rasterstereography turned out to be highly complex after using a variety of tools and a final graphical representation; the multiple mapping of individual aspects to domains resulted in a high density of interactions.ConclusionsThe INTEGRATE-HTA methodology helped to identify interactions between domains and significantly expanded the perspective on a technology. It improved patient-centered understanding and facilitated the discussion of single aspects.

VP06 HTA And Health Industry: Key Aspect Of Their Relationships

IntroductionConclusions and recommendations of health technology assessment (HTA) reports have an impact on all relevant actors involved in the health system (health authorities, administrators, health professionals, patients, citizens and industry). The involvement of all those relevant stakeholders in the HTA process facilitates making valid and informed decisions and an efficient allocation of resources. Improving communication, participation and transparency among all agents will lead to more efficient evaluation and decision-making processes.MethodsTo review key aspects of the relations between HTA agencies and health industries, two process were carried out: a narrative review of literature searched in Medline, PubMed, Embase, CINAHL and WOS (2007-2017) and a review of websites of international HTA agencies. References and webs with information on the framework, objectives, methodologies, impact or results of the relationships were included.ResultsA total of 1961 references were located and forty-five were selected. From the synthesis of the selected references the following key aspects of the relationships between HTA and industry were identified: (i) the importance of early dialogues with industry to align HTA objectives with the generation of evidence; (ii) challenges of the bias in the evidence produced by industry; (iii) difficulties in industry engagement in HTA processes; and (iv) industry interest in HTA. The review of six agency websites provided information on industry involvement in strategic activities, early dialogues, provision of documentation, management of industry clarifications, review of the report/allegations and other forms of relationship.ConclusionsBoth the review of the literature and the contents of the web pages of international agencies with experience in relations with industry show that the interest is in the creation of collaborative frameworks between regulatory authorities that decide on authorization and price and reimbursement and HTA agencies, while both try to maintain an early, transparent and systematic interaction with the healthcare industry.

PP159 Is Community Paramedicine A Safe/Effective Alternative To Usual Care?

IntroductionDue to an aging population, shortage of healthcare staff, and escalating healthcare costs, there has been a recent shift in the professional roles and responsibilities in acute care settings to help bridge the care gap. Paramedics, whose primary responsibilities have been in emergency/transportation services, are increasingly involved in the management of chronic diseases in the community setting. However, even with additional training, there are concerns about the safety and effectiveness of this expanded role. The objective of this presentation is to highlight some of the key findings from a health technology assessment report on the safety and effectiveness of community paramedicine in assessing and managing conditions/diseases with low acuity.MethodsA systematic review was conducted to identify studies that evaluated the safety and effectiveness of different community paramedicine programs.ResultsFour systematic reviews and 20 primary studies (one randomized controlled trial (RCT) and 19 observational studies) were identified. Of these, two systematic reviews and 14 primary studies focused on the safety and effectiveness of Emergency Care Practitioner (ECP) programs ̶ widely implemented programs whereby a paramedic or nurse undertakes activities traditionally performed by physicians, such as the initial assessment of patients, provision of simple treatment, or referral of patients to other clinical care. Limited evidence showed that ECP programs are promising in reducing repeated emergency calls, emergency department visits, hospital admissions/readmissions, and emergency transport charges. While the majority of included studies did not report any safety outcomes, no significant safety issues were identified from the cluster RCT. Evidence for other types of community paramedicine is limited.ConclusionsEvaluation of the impact of community paramedicine programs remains methodologically challenging. Additional cluster RCTs may help determine the effectiveness of community paramedicine programs; safety outcomes should be a key element of future observational studies.

Zasady przeprowadzania strategii wyszukiwania na potrzeby raportów HTA

Literature search strategy for Health Technology Assessment Reports Preparation of HTA reports requires access to the latest and most reliable knowledge in the field of health care. Systematic reviews are the basic tools used to obtain it. In order to find the literature necessary for a systematic review, search strategies are performed in databases. With their help it is possible to thoroughly and reliably search the resources of individual databases and find the required information. Properly executed search strategies also reduce the risk of missing valuable publications. During the preparation of the search strategy, one should use the tools and additional functions implemented in individual databases, thanks to which it is possible to strictly control the process and adapt it to your own needs. Performing a search strategy is a basic skill for anyone who prepares HTA reports.

Alpha-glucosidase inhibitors for prevention or delay of type 2 diabetes mellitus and its associated complications in people at increased risk of developing type 2 diabetes mellitus.

Alpha-glucosidase inhibitors for prevention or delay of type 2 diabetes mellitus and its associated complications in people at increased risk of developing type 2 diabetes mellitus.

Use of Decision Modelling in Economic Evaluations of Diagnostic Tests: An Appraisal and Review of Health Technology Assessments in the UK.

Diagnostic tests play an important role in the clinical decision-making process by providing information that enables patients to be identified and stratified to the most appropriate treatment and management strategies. Decision analytic modelling facilitates the synthesis of evidence from multiple sources to evaluate the cost effectiveness of diagnostic tests. This study critically reviews the methods used to model the cost effectiveness of diagnostic tests in UK National Institute for Health Research (NIHR) Health Technology Assessment (HTA) reports. UK NIHR HTA reports published between 2009 and 2018 were screened to identify those reporting an economic evaluation of a diagnostic test using decision analytic modelling. Existing decision modelling checklists were identified in the literature and a modified checklist tailored to diagnostic economic evaluations was developed, piloted and used to assess the diagnostic models in HTA reports. Of 728 HTA reports published during the study period, 55 met the inclusion criteria. The majority of models performed well with a clearly defined decision problem and analytical perspective (89% of HTAs met the criterion). The model structure usually reflected the care pathway and progression of the health condition. However, there are areas requiring improvement. These are predominantly systematic identification of treatment effects (20% met), poor selection of comparators (50% met) and assumed independence of tests used in sequence (32% took correlation between sequential tests into consideration). The complexity and constraints of performing decision analysis of diagnostic tests on costs and health outcomes makes it particularly challenging and, as a result, quality issues remain. This review provides a comprehensive assessment of modelling in HTA reports, highlights problems and gives recommendations for future diagnostic modelling practice.Electronic supplementary materialThe online version of this article (10.1007/s41669-018-0109-9) contains supplementary material, which is available to authorized users.

Economic Burden of Renal Cell Carcinoma-Part I: An Updated Review.

The economic burden of renal cell carcinoma (RCC) had been reported to be significant in a previous review published in 2011. The objective of this study was to perform an updated review by synthesizing economic studies related to the treatment of RCC that have been published since the previous review. We performed a literature search in PubMed, EMBASE, and the Cochrane Library, covering English-language studies published between June 2010 and August 2018. We categorized these articles by type of analyses [cost-effectiveness analysis (CEA), cost analysis, and cost of illness (COI)] and treatment setting (cancer status and treatment), discussed findings from these articles, and synthesized information from each article in summary tables. We identified 52 studies from 2317 abstracts/titles deemed relevant from the initial search, including 21 CEA, 23 cost analysis, and 8 COI studies. For localized RCC, costs were found to be positively associated with the aggressiveness of the local treatment. For metastatic RCC (mRCC), pazopanib was reported to be cost effective in the first-line setting. We also found that the economic burden of RCC has increased over time. RCC continues to impose a substantial economic burden to the healthcare system. Despite the large number of treatment alternatives now available for advanced RCC, the cost effectiveness and budgetary impact of many new agents remain unknown and warrant greater attention in future research.

Robotic Surgery: Transforming Patient Care in the 21st Century?

Inspired by a notion that machines can emulate human touch, the paper first examines the adoption of surgical robots in operating rooms at the global and local levels. The adoption of the technology has occurred almost concurrently in many countries without much time lag, and this near-simultaneous acceptance of the technology invites us to rethink technological supremacy in relation to patient care in the field of surgery. With focus on the da Vinci surgical system, this paper presents social and ethical implications of how (not) to regulate robotic surgery with potential and invisible risks with respect to patient care. Based on documentary analysis of news media in combination with literature review of medical journals, Korean news media, and Korean technology assessment reports on surgical robots, the paper explores how prevalently the idea of social progress is embedded in the promotion of advanced technology at the societal and governmental levels. I contend that the ideology of social enhancement has served to shape regulatory practices (or their absence) at the expense of patient care in emergent technologies such as robotic surgery in South Korea. Thus, social scientists and bioethicists should be involved in articulating ethical dimensions of the technology even from the stage of development in order to remedy the gap between technical advancement in surgery and patient care.

HT3 - Using Real-World Evidence for Health Technology Assessment Submissions: Lessons and Insights from Review Of Nice’s Technology Assessment Reports (2016-2017)

HT3 - Using Real-World Evidence for Health Technology Assessment Submissions: Lessons and Insights from Review Of Nice’s Technology Assessment Reports (2016-2017)

PSY126 - Trends in HTA Submissions for Rare Diseases: Insights from Review of Nice’s Highly Specialized Technology (HST) Assessment Reports

PSY126 - Trends in HTA Submissions for Rare Diseases: Insights from Review of Nice’s Highly Specialized Technology (HST) Assessment Reports

Bariatric Surgery Coverage: a Comprehensive Budget Impact Analysis from a Payer Perspective.

The objective of this study was to estimate a payer's budget impact of bariatric surgery coverage under (1) unrestricted, (2) budget-restricted ($500,000/year), and (3) quantity-restricted (100/year) medical benefit plan scenarios versus non-coverage in general and type 2 diabetes mellitus (T2DM) populations over a 10-year period. Using recently published literature and health technology assessment reports, the model evaluated a hypothetical payer population of 100,000 members under current real-world trends: BMI-defined obesity groups (31.3% normal/underweight, 33% overweight, 20.4% obese, 9% severely obese and 6.3% morbidly obese), T2DM prevalence (6.7-27.5%; 100% for the T2DM model), surgery type (LAGB, BPD/DS, VSG, and RYGB), and differential outcomes (T2DM resolution, costs, and reoperation and complications rates). Assuming a surgery election rate of 1.42% among eligible candidates with a 3% discount rate and 10% annual surgery turnover rate, the model calculated the incremental cost per-member-per-month (PMPM) by estimating the difference in total non-T2DM and T2DM-related expected costs and savings. One-way (± 25%) sensitivity analysis was performed. The impact of covering bariatric surgery under multiple scenarios for a general (or T2DM) population ranged from an additional $0.3 to $3.6 (T2DM: $0.3 to $10.5) PMPM in year 1. Incremental costs diminished over time, breaking even between years 5 and 9 (T2DM: 5-6), and by year 10, cost savings were estimated to be between $1.5 and $4.8 (T2DM: $1.2 and $31.8). Providing bariatric surgery coverage may have a modest short-term budget impact increase but would lead to long-term net cost savings in a general population model. The cost savings were much more pronounced in the T2DM model.

Why laparoscopists may opt for three-dimensional view: a summary of the full HTA report on 3D versus 2D laparoscopy by S.I.C.E. (Societ\xe0 Italiana di Chirurgia Endoscopica e Nuove Tecnologie)

BackgroundThree-dimensional view in laparoscopic general, gynaecologic and urologic surgery is an efficient, safe and sustainable innovation. The present paper is an extract taken from a full health technology assessment report on three-dimensional vision technology compared with standard two-dimensional laparoscopic systems.MethodsA health technology assessment approach was implemented in order to investigate all the economic, social, ethical and organisational implications related to the adoption of the innovative three-dimensional view. With the support of a multi-disciplinary team, composed of eight experts working in Italian hospitals and Universities, qualitative and quantitative data were collected, by means of literature evidence, validated questionnaire and self-reported interviews, applying a final MCDA quantitative approach, and considering the dimensions resulting from the EUnetHTA Core Model.ResultsFrom systematic search of literature, we retrieved the following studies: 9 on general surgery, 35 on gynaecology and urology, both concerning clinical setting. Considering simulated setting we included: 8 studies regarding pitfalls and drawbacks, 44 on teaching, 12 on surgeons’ confidence and comfort and 34 on surgeons’ performances. Three-dimensional laparoscopy was shown to have advantages for both the patients and the surgeons, and is confirmed to be a safe, efficacious and sustainable vision technology.ConclusionsThe objective of the present paper, under the patronage of Italian Society of Endoscopic Surgery, was achieved in that there has now been produced a scientific report, based on a HTA approach, that may be placed in the hands of surgeons and used to support the decision-making process of the health providers.

PD30 Cost-Effectiveness Of Stereo-Electroencephalography For Refractory Epilepsy

Introduction:Stereo-electroencephalography (SEEG) has been shown to be a valuable tool for the anatomo-electroclinic definition of the epileptogenic zone (EZ) in some patients with medically refractory epilepsy considered for surgery. In Spain, many of those patients are not offered this diagnostic procedure. The objective of our health technology assessment (HTA) report was to evaluate the effectiveness, safety and cost-effectiveness of SEEG to define the EZ in patients with refractory epilepsy considered for surgery compared to no SEEG intervention (i.e. remaining with further antiepileptic drugs).Methods:We undertook a systematic review with meta-analyses on the effectiveness and safety of SEEG. A cost-effectiveness analysis was conducted using a Markov model which simulates the costs and health outcomes of individuals for a lifetime horizon from the perspective of the Spanish National Health Service (NHS). The effectiveness measure was quality-adjusted life years (QALYs). We ran extensive sensitivity analyses, including a probabilistic sensitivity analysis.Results:The EZ was found in 92 percent of patients who underwent SEEG, 72 percent were eligible for epilepsy surgery and 33 percent were free of seizures after surgery (47 percent of those who received surgery). Any complications related to insertion and monitoring of SEEG and the subsequent intervention occurred in 1.3 percent of patients. In the base case analysis, SEEG led to higher QALYs and healthcare costs with an estimated incremental cost-effectiveness ratio of EUR 10,368 (USD 12,217) per QALY. The sensitivity analyses showed that the results of the study were robust.Conclusions:SEEG is a cost-effective technology in patients with refractory epilepsy considered for surgery when compared to no SEEG intervention.

OP102 Multiple Criteria Decision Analysis In The Field Of Hospital-Based Health Technology Assessment

Introduction:One of the main tools for Hospital-Based Health Technology Assessment (HB HTA) is the preparation of a mini-health technology assessment (HTA) report. Despite the high value of the results of mini-HTA reports for hospital decision-makers, the classical mini-HTA report does not allow a direct comparison of several health technologies among themselves.Methods:Based on the analysis of international experience of using the principles of multiple-criteria decision analysis (MCDA) in the field of HB HTA, we created and approved our own managerial decision-making model which includes five standardized multiple criteria. The value (weight) of each criterion was defined as the arithmetic mean obtained as a result of interviewing hospital decision-makers and an HTA expert group.Results:Five standardized multiple criteria were included in the structure of our mini-HTA report. These criteria presented the main results of assessment of the viability of implementing new health technologies (HTs) in hospital practice and contain the following: i) Novelty/innovation; ii) Comparative clinical effectiveness and safety; iii) Relevance (demand); iv) Economic effectiveness; and, v) Payback period. We conducted the modeling of various options of HTA results by using multiple criteria, which allowed us to determine the threshold values of the evaluated HTs corresponding to their priority for implementation: i) High priority - HTs are recommended for implementation; ii) Medium priority - HTs can be recommended only if there are sufficient financial resources in hospital; and, iii) Low priority - HTs may be recommended only if there are strong reasons for their need.Conclusions:Integration of the principles of MCDA in the structure of mini-HTA reports gives the opportunity to i) make comparative assessments of implementing new health technologies based on standardized criteria; ii) determine the priority for implementation of newly evaluated health technologies; iii) avoid the influence of subjective factors on the managerial decision-making in hospitals.

Diet, physical activity or both for prevention or delay of type 2 diabetes mellitus and its associated complications in people at increased risk of developing type 2 diabetes mellitus.

Diet, physical activity or both for prevention or delay of type 2 diabetes mellitus and its associated complications in people at increased risk of developing type 2 diabetes mellitus.