Composite measures and indices are used in medical research to represent certain concepts that cannot be measured with one variable. They can be used to predict outcomes or serve as outcomes in trials. The creation of innovative indices is important to increase publications and secure research funding. However, some assumptions and problems are prevalent among indices. We aim to develop a reporting guide and an appraisal tool for indices based on the issues we identified.

We reproduced the three frailty indices from a previous publication and 134,689 principal component-based indices. We reviewed the index assumptions, bias introduced by data processing, relationships between input variables. We interpreted the indices with input variables.

We identified four major issues to be addressed in a reporting guide: constraints imposed by index creation on the input variables; data processing without evidence base; indices poorly linked to input variables; and, relatively inferior predictive power. We demonstrated a flow diagram and a checklist to report and review these four issues related to innovative indices.

A reporting and critical appraisal tool for innovative indices is lacking and needed. These four issues that need to be explicitly considered are previously neglected. This guide is the first attempt to improve the quality and generalizability of innovative indices. This guide can be used to lead further discussion with other experts and review committees.

In the Philippines, medicines are procured at higher rates in government hospitals. The prices of essential medicines have high variability, and a significant portion of out-of-pocket expenditures by Filipinos is for medicines. This study's objective is to determine the factors associated with the variation in drug pricing among public hospitals.

This was a mixed-methods, case-control study of 57 hospitals. Two tools were developed based on: (i) Management Sciences for Health (MSH)’s Rapid Pharmaceutical Management Assessment and (ii) World Health Organization (WHO)’s Good Pharmaceutical Practices. The dependent variable is a drug price reference ratio of a preselected drug basket. Examples of factors studied are: (i) preference for generics, (ii) procurement type, and (iii) time out of stock.

Hospitals with proper procurement planning and performance monitoring are expected to decrease the price ratio (R = -0.030). However, interview data showed that forecasting is still not robust enough. Past consumption (91 percent) remained the most frequently used input to procurement planning. Few hospitals took into consideration other factors such as morbidity, mortality, and patient demographics. The expertise of hospital procurement staff increases the hospital's price mark-up. Interview results suggest this is because members and hospital units do not meet eye-to-eye to ensure accountability and coordination across units in planning and implementing the procurement procedures.

By having a forward-looking procurement plan, forecasting can be more efficient. Potential improvement lies in finding mechanisms where nearby hospitals could participate in pooled procurement. Pooled procurement could have an impact on reducing prices by capturing economies of scale, provided this is operated efficiently and transparently.

One important objective at the Institut d'excellence en santé et en services sociaux (INESSS) is to guide the implementation of promising new technologies into Québec's healthcare system. A comprehensive evaluation framework was recently developed that takes into account the dynamic and iterative nature of the life cycle of such technologies. This framework is presently being used to inform the decision-making process concerning use of thrombectomy for ischemic stroke.

A field evaluation has been undertaken since April 2016 in all four of Québec's specialized tertiary stroke centers. This real-world evidence is communicated regularly to the clinical teams as well as decision-makers. A systematic literature surveillance is also ongoing, with results being shared amongst clinical experts on our interdisciplinary advisory committee. On the basis of the generated evidence from these sources, recommendations to optimize structures, processes of care and clinical outcomes will be developed, in collaboration with the interdisciplinary committee.

Thrombectomy has been shown to be safe and effective for treating ischemic stroke in the randomized trial setting in high-volume, expert centers. Real-world evidence from Québec indicates increasing use of this new technology but with wide variation across health regions. Observed times to treatment appear favorable for patients admitted directly to tertiary centers, but inter-hospital transfer is associated with important increases in delays from first door to thrombectomy. The documentation of 90-day outcomes is problematic, especially for patients transferred out of tertiary stroke centers prior to discharge. Uncertainties raised in the literature include patient selection criteria and optimal processes of care during prehospital and inter-hospital phases of the patient's trajectory.

The ongoing comprehensive evaluation of thrombectomy for ischemic stroke in Québec is a concrete example of how the use of an innovative, disruptive technology can be optimized. We acknowledge the contribution of the members of the clinical expert committee.

Healthcare-associated infections (HAIs) are an important, potentially preventable reason to maintain a clean healthcare environment. However, guidelines from Europe and North America do not concur—European guidelines recommend using neutral detergent (followed by chlorine-based disinfection (CBD) if required), whilst North American guidelines recommend using detergent or hospital-grade disinfectant-detergents for routine cleaning or decontamination of noncritical healthcare environmental surfaces. The objective of this study was to compare the effectiveness on rates of HAIs of: (i) disinfectant-detergents versus detergents; and (ii) the active ingredient of many disinfectant-detergents—quaternary ammonium compounds (QAC)—versus CBD.

A rapid review of systematic reviews was conducted using the following search terms: keywords and controlled vocabulary terms for the concepts of “healthcare environmental surfaces” AND (“QAC-based disinfectants” OR “disinfectant-detergents” OR “decontamination”) AND (“environmental contamination” OR “colonization” OR “HAIs”). The search filters included systematic reviews, guidelines, and technology reports. The following databases were searched: The Cochrane Library; PubMed; and health technology assessment and guideline websites for gray literature. Systematic reviews of studies comparing the effects of disinfectant-detergents with detergent, or comparing QAC with CBD, on rates of HAIs in the healthcare environment were included. Reviews on the cleaning or disinfection of body surfaces or disinfection of invasive medical devices were excluded. Quality assessment was not conducted. Data extraction was performed using a pro forma.

The literature search resulted in 356 titles. From ninety-four potentially relevant abstracts, fifty-seven full-texts were evaluated: fifty-one were excluded (eight non-English) and six were included. All review authors cautioned that the evidence was low level, methodologically poor, subject to confounding, and didn't address adverse outcomes. The reviews identified eight relevant primary studies, three of which compared disinfectant-detergents with detergent and found no difference in rates of HAI. Five studies compared QAC with CBD. All five demonstrated that CBD was superior to QAC and reduced Clostridium difficile infection rates in outbreak contexts. Furthermore, QAC may induce sporulation and microbial resistance.

Low-level evidence suggested that: there is no advantage in using disinfectant-detergents for routine cleaning of noncritical surfaces; CBD is superior to QAC-based disinfection in reducing clostridial infections; and QAC agents may induce sporulation or microbial resistance.

The Patient Experiences and Satisfaction with Medications (PESaM) questionnaire was recently developed. It consists of two disease-specific modules for evaluating drug treatment of idiopathic pulmonary fibrosis (IPF) and atypical hemolytic uremic syndrome (aHUS): (i) a generic module applicable to any medication, and (ii) a patient expectations module. This study assessed the validity and reliability of the generic module in a large sample of patients in the Netherlands.

In 2017, the PESaM-questionnaire was sent out to IPF patients on pirfenidone or nintedanib, aHUS patients receiving eculizumab, and patients using advagraf after kidney transplantation. The generic module consists of 16 items related to the domains effectiveness, side-effects and ease of use, and assesses patient experiences regarding the impact of the medication on daily life and health, and satisfaction. Mean scores for each domain were calculated using a scoring algorithm. Content validity, construct validity, and reliability were assessed using recommended methods.

Patients (n=188) completed the generic module of whom 48 percent used pirfenidone, 36 percent nintedanib, 11 percent advagraf, and 5 percent eculizumab. Content validity was established. Expected associations between patient experiences, satisfaction, and quality of life (QoL) were generally confirmed, demonstrating construct validity. For example, a moderate to strong positive association was found between patient experiences and satisfaction with side-effects (correlation coefficient 0.625, p < 0.05), and low (positive) associations were found between patient experiences and QoL. Importantly, the PESaM-questionnaire was able to discriminate between patients using different medications. Intraclass correlation coefficients, for test-retest reliability, ranged between good and excellent for most domains.

The PESaM questionnaire is a promising tool to provide scientific evidence regarding the patient's perspective in health technology assessments and reimbursement decision-making regarding (expensive) medications, but can also support shared decision-making and appropriate use of medication at the individual patient level. Further research will assess the questionnaire's responsiveness and generalizability of results to other patient populations.

To stay at home, have social interaction, or work, people experiencing urinary retention or uncontrolled urine or feces leakages need specific medical devices (MDs). In France, the MDs used to be covered by the health insurance system if included on a specific list of products and services qualifying for reimbursement. These MDs for urinary and fecal drainage and collection are included under a generic description corresponding to a class of products with the same indications. This coverage modality offered low resistance to unnecessary or wasteful spending. Furthermore, a periodic update of the list is required whereas it has not been done for more than 10 years.

In 2016, Haute Autorité de santé (HAS) assessed the actual clinical benefit of these MDs using a standard health technology assessment method (systematic literature review, opinions of health professionals and patients’ representatives). Manufacturers were asked to provide technical specifications on their MDs.

The lack of professional guidelines and well-conducted comparative clinical trials has to be pointed out; among 516 identified publications screened, only seven recommendations, one technological review and one randomized controlled study were selected. Despite this, HAS defined specifications for each generic description, based on users’ experience (patients and caregivers). These included specific indications, minimum technical specifications and, when applicable, conditions of prescribing and use. This assessment took into account individual preferences, the role of the natural carers and the conditions, and opportunities for patients to improve and update their self-care and rehabilitation skills.

The HAS assessment of MDs for urinary and fecal drainage and collection provides a cornerstone for the enhancement of the access to the necessary devices for homecare. The expected benefits are an improvement of the quality of life and a reduction of health expenditure due to misuse, complications or hospitalizations.

Prostatic artery embolization (PAE) was introduced in the 1970s to control major bleeding after prostate surgery. The procedure was noticed to improve the lower urinary tract symptoms of benign prostatic hyperplasia (BPH) and in 2010, PAE was first investigated as an alternative treatment for BPH. A rapid health technology assessment (HTA) was carried out to inform our hospital's decision on introducing this procedure.

The rapid HTA investigated the safety and clinical effectiveness of PAE for patients with BPH. The PICO elements were: Population- Patients with symptomatic BPH; Intervention- PAE; Comparator- Conventional management; Outcomes- Adverse effects, clinical outcomes. The NHS Centre for Reviews & Dissemination databases, Cochrane Database of Systematic Reviews, and PubMed (MEDLINE) were searched for systematic reviews and HTA reports.

Eight systematic reviews from the most recent two years were found. The primary evidence base consists of two randomized controlled studies of PAE versus transurethral resection of the prostate (TURP), one matched pair analysis of PAE versus open prostatectomy in patients with large prostates, and several non-comparative studies. The comparative studies showed patients had better International Prostate Symptom score, quality of life and reduced prostate volume with TURP and open prostatectomy from 1 to 24 months. With respect to adverse events, embolized patients had more adverse events than controls, particularly acute urinary retention and post-embolization syndrome. However, controls had more abnormal ejaculation; and adverse effects from surgery naturally only occurred in controls.

PAE appears to be a promising technology lacking long term outcomes. It has potential for patients who are not fit or not keen on surgery, or who may have large prostates, but who are still vascularly suitable for embolization. It would be suitable to carry out under clinical research conditions to clarify the incremental benefits of the technology and which patient groups are best served by the procedure.

Health technology assessment (HTA) must adapt to support the changing health system landscapes and improve access to valuable innovation under budgetary constraints. This is exemplified by the pricing and reimbursement of high-cost combination therapies increasingly used in oncology. Variability exists in current HTA practices across different countries, resulting in discrepancies in reimbursement outcomes and patient access. Using Italy as a case study, the objective was to assess the challenges faced by HTA agencies in the negotiation of pricing and reimbursement of combination therapies.

A targeted literature review of Italian HTA agency websites was undertaken to identify any literature/guidance relating to HTA decision-making for combination oncology therapies.

In Italy, there is no fixed cost-effectiveness threshold and decisions are based on multiple criteria. Managed market entry agreements are extensively used; price-volume agreements and drug registries are common. While this framework allows flexibility and avoids the rigidity of incremental cost-effectiveness ratio thresholds, it has raised concerns about transparency and budget impact. Combination therapies are not given specific concessions; however, market access for a combination of a new high-cost drug with an existing one is complex, particularly if the drugs are manufactured by different companies. The added value provided by the new drug in the combination should be rewarded while the older product benefits from the increased volume of use. The price of the older drug cannot be lowered unless the pricing and reimbursement contract is expiring or a new indication/formulation is pending, presenting a challenge to both pharmaceutical companies and HTA agencies.

Combination therapies pose a challenge for HTA agencies. In the Italian system this is partially mitigated by the use of multiple criteria for decision-making and managed access agreements. However, these approaches have also led to concerns about a lack of transparency in decision-making.

Interventions and services for people with mental health problems can have broad remits: they are often designed to treat people with a variety of diagnoses. Furthermore, addressing mental health problems can have long-term implications for economic, social, and health outcomes. This represents a challenge for health technology assessment, for which long-term trial data can be lacking. In this review, we sought to identify how analysts have tackled this problem. We reviewed the methods used to extrapolate costs and outcomes for the purpose of economic evaluation where long-term trial data are not available.

We conducted a systematic review of the medical and economic literature evaluating long-term costs and outcomes for mental health interventions and services designed to treat or prevent more than two mental health conditions. We searched key databases including MEDLINE, Embase, PsycINFO, CINAHL, and EconLit. Two authors independently screened citations. Articles were excluded if they reported within-trial analyses or employed a time horizon of less than 5 years.

The search identified 829 unique records. No papers could be included in the review.

This review highlights the lack of research and understanding available to inform the appraisal of broad mental health interventions. In light of our findings, we consider the reasons for this lack of information and review relevant literature on the subject. Potential barriers to research in this context include: (i) challenges in understanding the value of broad mental health services, such as the mental and physical health nexus, intersectoral costs and benefits, and interpersonal impacts, (ii) methodological difficulties, such as data availability, patient heterogeneity, and the challenge of extrapolation, and (iii) parity of esteem. We make recommendations for resolving this problem with regard to funding, data collection, modelling methods, and outcome measurement.

Recently, voluminous research and commentary have touted genetic and molecular testing to improve the management of urologic cancer. The purposes of such testing include screening, risk assessment, diagnosis, prognosis, pharmacogenetics, and monitoring (for example, recurrence, predicting treatment response). An interactive graphical tool (“evidence map”) would help policy makers examine the current state of research, identify prevailing trends, and prioritize research efforts.

A professional information specialist searched MEDLINE/EMBASE for articles published in 2010 or later that primarily focused on genetic/molecular testing and either kidney or bladder/urothelial cancer. Two research analysts classified all relevant abstracts regarding to cancer type, genetic marker(s), clinical purpose(s), assay methods, publication type, and author country/region. We created an interactive map using HTML5 and JavaScript.

We identified 4,731 articles, 828 (18 percent) of which met our inclusion criteria. Our map has interactive filters which allow flexible selection of articles and automatic updating of the counts. For example, one can quickly redraw the map to focus only on U.S./European systematic reviews and meta-analyses. Research on bladder/urothelial cancer focuses on both diagnosis and prognosis, with some interest in monitoring. In kidney cancer, research on prognosis outweighs research on diagnosis. Overall, research on genetic/molecular markers is in an exploratory phase, e.g. for kidney cancer prognosis alone, 173 empirical studies considered hundreds of different markers.

Assessing prognosis is a common purpose of genetic tests for both bladder/urothelial and kidney cancer. Increased research on the monitoring of bladder/urothelial cancer may be due to its high recurrence rates, whereas lower interest in genetic tests to diagnose kidney cancer may be due to effective imaging tests. For policy makers, evidence maps can inform decisions about the scope of commissioned systematic reviews as well as the targets for recommendation statements. Interactive features allow maps to be redrawn to align with users’ specific interests.

Specialized Component of Pharmaceutical Service (SCPS) is a strategy to access high cost medicines in the National Health System (NHS) of Brazil, ensuring the completeness of medical treatment in which lines of care are defined in the Clinical Protocols and Therapeutic Guidelines (CPTG). To access the SCPS, the physician has to give to the patient a filled form, following some requirements and the CPTGs. In order to improve rational prescription and to facilitate patients’ access to medicines, we visited physicians and presented key information regarding the CPTGs of rheumatoid arthritis (RA) and the SCPS medicines request process; then, we sought to know their perceptions.

From August to September 2017, 43 specialists and general practitioners that prescribed medicines of SCPS in Minas Gerais were visited by researchers about the CPTG of RA and how to prescribe to provide easier access to these medicines to their patients. After the visits, a researcher contacted the physicians by phone to evaluate their satisfaction with the visits and about the program through a brief questionnaire.

Twenty-eight physicians answered to our phone call, providing a response to the questionnaire. Sixty-eight percent indicated they were very satisfied with the visit. Fifty percent stated that the content of the visit was relevant to their practice, and 60.7 percent said that the distributed material was going to be useful for their professional practice. Regarding the guidelines, 43 percent affirmed that the visit really helped them to improve their understanding of medicine requests in the NHS and 42.9 percent said that the visit increased their understanding of which patients are eligible for RA treatment in the SCPS; 57.1 percent of those affirmed that the visits increased their knowledge.

The physicians, who were mostly specialists, already had knowledge about CPTG and prescription practices of SCPS’s medicines, nevertheless, they showed interest in the visits to review and improve their knowledge and clinical practice.

We conducted health technology assessments (HTAs) of the interventions used between 2012 and 2014 to improve the treatment of homeless people with pulmonary tuberculosis in the Federal District of Brazil. The HTA, which was not ordered by policymakers, was based on the evidence-based national theoretical model compared with local interventions indicated in focus groups, semi-structured interviews, and secondary data produced by the Health Secretariat. The results demonstrated that the implementation of the interventions was unsatisfactory. Our objective was to present the feedback process for policymakers and the Health Secretariat, particularly its challenges.

The feedback was categorized as: (i) an executive abstract with key messages (i.e. underreporting of cases in the surveillance system, lack of primary care, and underestimation of the health problem) reported to policymakers involved in the surveillance and healthcare systems; and (ii) oral presentations in eight meetings organized by the research group and local policymakers.

Between 2016 and 2017 we conducted eight feedback meetings. All of the professionals (n = 8) involved in the Tuberculosis Surveillance and Control Program were present in at least one of the meetings, but healthcare professionals and the Secretary of Health did not participate. The barriers presented by the professionals were: (i) lack of material resources (i.e. cars and gas, phones, diagnostic tests, medications); (ii) lack of human resources (i.e. suboptimal professional staff); and (iii) feeling insecure when performing extramural activities due to the potentially unsafe work environment.

Gathering feedback on a HTA that was not ordered by policy makers can be a challenge. Mainly we demonstrated a negative result on research done in a vulnerable population with a neglected disease, in this case tuberculosis. However, this provided an opportunity for professionals in the surveillance system to discuss the challenges of implementing tuberculosis control among the homeless population.

In the fast-paced world of health technology innovation, early health technology assessment (HTA) gained recognition as a tool to help prioritize and steer the development of those innovations that potentially add value. Much of early HTA seems technology-driven; a certain novel technology is introduced and the focus is on assessing its expected cost-effectiveness. We argue that a first step in assessing innovation would be to derive proof-of-problem through combining evidence from literature and stakeholder engagement. We applied this approach to a novel surgical instrument aimed to facilitate meniscus surgery.

First, we identified a broad scope of stakeholders in meniscus surgery (i.e. meniscectomy). Through interviewing them we derived key problems in meniscectomy as-is, and determined which outcomes matter most. We used stakeholder and literature input to quantify the room for improvement in current meniscectomy. Together with stakeholders we interpreted the problem quantification and conducted an early assessment of the proposed surgical innovation. Finally, we made use of this early stakeholder engagement to uncover possible barriers and facilitators to the innovation's implementation.

While all stakeholders were enthusiastic about the innovation, there was a shared perception that there is little room for improvement in meniscectomy at present. Put differently; the innovation poses a great solution to problems that may not exist. In addition, by involving a broad range of stakeholders we were able to identify barriers and facilitators to future implementation early on, such as surgeons’ preferences.

We conclude that the innovation's value may lie with applications outside of meniscus surgery. Regarding methodology, we showed how a shift of focus from solution to problem definition provides a different perspective on an innovation's potential value, borne out of needs not currently met. In doing so, early HTA is in a unique position to help navigate the stream of health technology innovation before actual development of the innovation.

Subsequent to review by Canada's two central health technology assessment (HTA) agencies, confidential drug prices are negotiated by the pan-Canadian Pharmaceutical Alliance (pCPA) on behalf of public drug plans. This analysis is the first to examine characteristics of drugs considered for negotiation, and the duration of negotiations, from inception in 2011 to August 2017. The objectives were to identify how HTA recommendations impacted price negotiations, and in particular the role of health economics in the process.

The dataset contained 208 drug indications from the pCPA archives: those with a decision to negotiate (n=155) or a decision not to negotiate (n=53). Data were abstracted from the publicly-maintained websites of the respective agencies; descriptive statistics were conducted.

There was close but imperfect alignment between the HTA agency listing recommendation and the pCPA's decision to negotiate. The incremental cost-effectiveness ratio (ICER) of negotiated drugs (as estimated by HTA agencies) approached CAD 200,000/QALY (i.e. USD 157,000) for oncology drugs, but was closer to CAD 100,000/QALY (i.e. USD 78,000) for non-oncology drugs, revealing that negotiations would require a substantial discount to achieve conventionally ‘acceptable’ value-for-money. ICERs were influential to non-oncology drug recommendations (and were increasingly used to set pCPA negotiation targets) but did not appear to influence oncology drug HTA recommendations. The time period required to initiate negotiations was dramatically shorter for oncology versus non-oncology drugs (53 versus 263 days), and also differed markedly between therapeutic areas. The time period for pCPA activities was surprisingly similar for drugs recommended without a price condition and for those conditional on a price reduction.

These findings revealed an implicit prioritization pattern at the pCPA, as well as the evolving role of health economics in Canada's two-stage reimbursement process.

Medication delivery is one of the most common interventions in clinical practice. It requires the direct involvement of nurses and high precision infusion pumps in order to increase the infusion accuracy. Any mistake in the medication delivery process can lead to a medication error, resulting in adverse events with considerable increases in hospital length of stay and cost. Research studies should analyze this area more in emerging countries, as their realities differ from the realities of developed countries, where most of the literature of this area has been developed. This research study analyses this area in Brazil, a leading emerging country. The incorporation of these technologies in health services have caused two major problems: uncertainty around its effectiveness in reducing adverse drug event rates related to infusion dose errors, and the high cost of their inputs. The objective of this study was to analyze the cost-effectiveness of intelligent drug library infusion pumps to reduce adverse drug events during intravenous medication delivery in pediatric and neonatal patients.

Cost-effectiveness was evaluated using a decision-tree framework, considering two scenarios as the base case: the reference one, which uses conventional infusion pumps for intravenous medication delivery with a volume greater than 60 mL, and an alternative one, which uses the drug library infusion pumps. The analysis is with the Unified Health System (Brazil's publicly funded health care system) perspective. The Monte Carlo simulations addressed the uncertainties of the framework. The effectiveness measure was avoidance of adverse drug events.

The probabilistic analysis showed the drug library infusion pumps to be more cost-effective than conventional pumps. This ratified what had already been revealed by acceptance curve, which demonstrated that the drug library infusion pumps are more likely to be cost-effective compared to the conventional infusion pumps (with a minimum of the incremental cost-effectiveness of USD 1,501.28).

The study demonstrated that the use of the drug library infusion pumps in the pediatric and the neonatal intensive care unit can improve the results of the adverse drug event reduction strategy.

Many countries face the challenge of an aging population. Development of suitable technologies to support frail elderly living in care homes, sheltered housing or at home remains a concern. Technology evaluation in real-life conditions is often lacking, and randomized controlled trials of ‘pre-designed’ technologies are expensive and fail to deliver. A novel alternative would be ‘living labs’-real-life test and experimentation environments where users and producers co-create innovations and large-scale data can be collected.

The goal of the living labs and Data Driven Research and Innovation (DDRI) Programme is to use data driven analytics and insights to support technology development for independent living, healthy aging and more cost-effective care. This involves a cluster of long-term residential care facilities providing 24/7 living lab settings, linked to an embedded innovation hub. DDRI also encompasses private vehicles (e.g. sensors in cars) to enable elderly to drive safely for longer. Collaborations have been established with Universities in England, Scotland and Ireland and with international industry partners.

Several projects are underway: (i) develop machine learning algorithm from non-intrusive sensor data to build a well-being representation for individual residents/citizens; (ii) evaluate innovative interventions for good sleep environment and nutritional support; and (iii) establish ethics framework to ensure that needs of residents, families and staff are embedded in design, communication, and evaluation of future DDRI projects. In addition, fifteen interdisciplinary doctoral fellowships are in place, six universities are working closely with individual living lab settings, and an innovation hub has been established in one care home for horizon-scanning and strategic technology selection and implementation.

Over the next five years, a national network of 20 residential living labs with over 1,500 participants will be established. Generation of new user-led technologies, blueprints for capture of individual data at significant scale, and ethical and organizational guidelines will be developed. Intelligent mobility via data capture/feedback in vehicles will be established.

The use of multi-criteria decision analysis (MCDA) in health technology assessment (HTA) studies has become more common due to the fact that MCDA offers a comprehensive technique for decisions that involve multiple criteria and stakeholders. How MCDA contributes to the HTA decision making process is an issue to be investigated. A systematic review was carried out in order to provide an overview of the benefits identified in MCDA applications for the strategic HTA decision making process.

A systematic review developed by Philip Wahlster et al. (2014) was updated. The papers were analyzed in order to determine how MCDA is connected with traditional HTA, and to identify opportunities through the application of MCDA. In total 965 papers were found, and 43 articles were included in the review. The included articles detailed MCDA applications oriented to tactical and strategic decision making processes. The review was conducted by two researchers.

Of the available studies published on MCDA, 76 percent were published between 2014 and 2017, and 24 percent were published prior to 2014. Regarding the MCDA methodology defined in the included studies, 10 used the analytical hierarchy process, four used multi-attribute theory, and others refer the methodology only as “MCDA”. Seventeen studies also included health technology economic analysis, in special cost-effectiveness, safety and technological innovation. The studies suggest MCDA adds value since it allows different stakeholders to be engaged in the decision making process.

The increase in studies on MCDA and healthcare point to the possibility to add different criteria, engage people with different knowledge levels, and make the decision-making process more transparent. In comparison with other technical areas, the use of MCDA in healthcare is more focused on achieving the decision about adding the new technology, and to show how to engage stakeholders than to explain how to develop the algorithms and methodologies.

Hemophilia gene therapy trials demonstrate a “cure” could be achievable, thereby changing disease management. CoreHEM aims to develop multi-stakeholder consensus around a clearly defined, core outcome set (COS) - a minimum set of outcomes that should be measured and reported in all clinical trials of a specific condition - that will demonstrate and allow differentiation of the effectiveness and value of gene therapy relative to the current standard of care. Health technology assessment (HTA) frequently suffers from a lack of relevant, consistently reported outcomes. When uniformly implemented, COS increase the predictability and consistency of appraisals, coverage, and reimbursement decisions by payers and HTA agencies.

A COS was developed using a modified Delphi process, including online surveys and an in-person consensus meeting. A literature review and key informant interviews were used to create an initial list of outcomes for voting. Participants (patients, including representatives from the National Hemophilia Foundation and the World Federation of Hemophilia, healthcare providers, payers, HTA agencies, regulators and industry representatives) condensed and prioritized the list by rating each outcome on a scale of 1 (not important to include) to 9 (essential). Participants could also suggest outcomes for voting. Outcomes were eliminated from consideration if <70 percent rated the outcome from 7–9, unless the patient stakeholder group average score was ≥7.

After two Delphi rounds, there was consensus on three outcomes: frequency of bleeds, factor activity level, and duration of expression. Additional outcomes included after an in-person consensus meeting were chronic pain, mental health status, and utilization of the healthcare system (direct costs). Adverse events of interest were evaluated and separately reported.

Including the coreHEM COS in clinical development programs will ensure that relevant, consistent outcomes are available for decisions by HTA agencies, clinicians, and patients. This should result in faster access to novel, high-value therapies for appropriate patients.

São Paulo city in Brazil has implemented social and health care for homeless people with pulmonary tuberculosis since 2007. We conducted a health technology assessment (HTA) of the interventions provided based on a national theoretical model using 2015 data and an overview of systematic reviews. The HTA was requested by national policymakers. The results demonstrated that the interventions for pulmonary tuberculosis were satisfactory. The municipal secretariat implemented actions to improve the national treatment recommendations and adopted incentives to increase adherence to treatments. Our objective was to describe the feedback process for the Health Secretariat.

The feedback was categorized as: (i) an executive abstract with key messages (i.e. ninety-seven percent of notified cases underwent sputum smears, nineteen percent were hospitalized, and fifty-nine percent were cured) reported to policymakers involved in the surveillance program; and (ii) three meetings were organized jointly by the research group and local policymakers.

In 2016 we conducted a meeting to present the results. Thirty-nine professionals involved in the primary care team working on the streets (thirty-five percent) and the Tuberculosis Surveillance and Control Program (five percent) were present. The main barriers presented by the professionals were issues of human resources (i.e. suboptimal professional staff and having two different social organizations responsible for health care). The main facilitators presented by professionals were: (i) using homeless-peers as healthcare workers; (ii) having a network linking the primary care and surveillance programs; and (iii) periodic training.

In addition to the positive results, the HTA presented an opportunity to discuss the sustainability of incentives for adhering to treatments adopted by the policymakers, such as meal allowances and housing support, to improve social conditions among the homeless.

The dual mobility concept was invented to prevent postoperative hip dislocation. It has been used for 40 years, but demonstrative data are limited and many designs are available. In France, many implants for hip arthroplasty are reimbursed by the national health insurance system through a generic description that corresponds to a class of products having the same indications and technical features (designs, coatings, types and systems of fixation). Dual mobility cups were one of them until 2017. The ministry decided to set up the assessment of each design of dual mobility cup, marketed in France for their reimbursement.

Manufacturers of these devices submitted medico-technical application dossiers, with technical characteristics and clinical data, to the French National Authority for Health (HAS). HAS has assessed prospectively the actual benefit of these implants.

Sixteen companies marketing 42 kinds of dual mobility cups associated with 22, 28 or 32 mm femoral heads and cementless or cemented fixations have submitted dossiers. Their demands were argued on non-specific and specific clinical data. For few implants with non-specific clinical data, arguments to demonstrate the equivalence towards other implants were not accepted for reimbursement. For other implants, the specific data available were only low quality studies. No randomized controlled trials were identified. When the risk/benefit balance was acceptable, the implants were approved for reimbursement and HAS required post-launch data to assess the real-world safety of these implants (i.e. dislocation and long-term survival).

All the dual mobility cups marketed in France have been assessed by the French health technology assessment authority. Most of them have been approved for reimbursement, despite of low level of evidence. The brand name inscription enables a specific follow up and the analysis of a post-approval study results in five years.