Index mining is a new discipline that aims to search for the composite measures or indices most relevant to the contexts or outcomes. After reviewing three frailty indices and principal component (PC)-based indices, we hereby show certain occasions that can lead to ineffective indices, which consist of bias or fail to represent the theories.

We reproduced and reviewed the three frailty indices and the 134,689 PC (principal component) -based indices from previous publications. The impact of aggregating the input variables on the final indices was analyzed using forward stepwise regression.

Several methods to combine the input variables were related to ineffective projection of information onto the indices. The most common causes leading to ineffective summation of input variables were shown in three examples involving different types of input variables, which were positively or negatively correlated or uncorrelated to the outcome. Ineffective indices were created often because of the summation of redundant information or uncorrelated variables.

The creation of ineffective indices can be avoided if the relationships between input variables and outcomes are properly scrutinized. The creation of composite measures and indices is still a discipline under active development. The three examples we identified are the mistakes that may be repeated unintentionally and need to be addressed with explicit rules. A reporting guide for the creation of composite measures has been proposed. A proper review of index objectives, data characteristics, and data limitations before creating composite measures or indices is recommended.

Dry eye disease (DED) is a common condition that significantly impacts patients’ quality of life. Previous studies have explored the impact of DED on patients’ lives qualitatively; however, patients’ preference structures have not been thoroughly explored quantitatively.

A targeted literature review and social media listening project guided design of a discussion guide for in-depth patient interviews (n=12). These, in turn, guided construction of a quantitative questionnaire administered to moderate to severe DED patients, 40 per country in Australia, Germany, United States and United Kingdom (total n=160). Patients’ preference structures were explored through an online survey using a self-explicated conjoint methodology, because of its high respondent-friendliness. Additionally, we administered the EQ5D-5L instrument to determine the health states/utilities of patients. Reaction to a hypothetical novel treatment was further obtained to check for convergent validity with the self-explicated conjoint. Finally, we asked respondents to rate the ease and relevance of the questionnaire to them.

Qualitative research uncovered important patient perspectives that were built into the quantitative survey. For example, patients seek medical advice when their symptoms are not improving. Patients’ lives are most affected by sensitivity to light, itchy and tired eyes and an inability to perform computer/screen work; however, of most concern/worrying to them is that their DED will get worse and they go blind. Results from the quantitative preference research will also be shared and its implications for future clinical trials in DED outlined. The results of the patient research and preference study are to be shared with health technology assessment (HTA) bodies and regulators through the early dialogue scientific advice process.

A process of using qualitative research to determine what matters to patients and then quantification through respondent-friendly preference research can identify outcomes that are most patient-relevant, to inform future drug development strategies.

The objective of this qualitative analysis was to explore caregiver perceptions and experiences of receiving or not receiving a genetic diagnosis following genome-wide sequencing (GWS) in children with suspected genetic disorders.

One caregiver for each child completed an online survey two weeks after enrolling in the Clinical Assessment of the Utility of Sequencing and Evaluation as a Service (CAUSES) study, beginning in January 2016, and again about six months after receiving the GWS results. The survey covered the caregivers' experiences and quality of life and children's healthcare resource utilization, and provided open-ended questions for comments. The follow-up survey was completed by twenty families who had received a diagnosis with their GWS results and by twenty-two families who had not received a diagnosis. A thematic analysis of the free-text comments from both groups was performed using NVivo 11.4.2.

Caregivers from both groups expressed similar experiences of negative socioeconomic effects of caregiving, particularly related to employment and time burden. Caregivers who did not receive a diagnosis with the GWS results were generally hopeful of receiving a diagnosis in the future and reported expectations of a positive benefit from receiving a diagnosis, both in terms of access to additional resources and of positive psychological effects. The absence of a diagnosis was a source of anxiety for many caregivers. By contrast, caregivers who had received a diagnosis reported positive, neutral, and negative psychological effects from the knowledge gained; no participants commented on the consequences for access to additional services or other socioeconomic effects.

Our findings suggest that caregivers may have high expectations for what a diagnosis can provide to them and their families, which may not be fully met once a diagnosis is obtained. The study underpins the importance of patient-centered communication of genomic testing results so that families can set realistic expectations of what having a diagnosis will achieve.

Globally, countries are investing substantially in e-health. Failures of programs to achieve valuable economic, clinical and societal outcome are increasingly reported. Unsuitable governance models may be one explanation. Research on governance models’ usefulness for realization of valuable outcomes is incomplete and scattered. Our goal is to fill this gap by producing knowledge on e-governance in Norway. Our hypotheses are: i) Co-governance and Relational Coordination will positively impact the realization of valuable outcome; and, ii) Multilateral stakeholder dialogue and collaboration, including health service delivery perspective, have been proposed to innovate health technology assessment (HTA). This will improve the relevance of HTA e-governance research.

We undertook the following: i) Systematic Review of e-governance in healthcare ii) Participatory observations, in depth interviews/focus groups iii) Document retrieval and analyses iv) Creation and support of arenas for dialogue between stakeholders on values and governance v) Analyses of co-produced value adjustments vi) Analyses of the usefulness of the Scientific Dialogue Approach for changing HTA paradigms. The study populations were: i) Governmental bodies responsible for innovation of the electronic health record (EHR) in Norway; ii) Regional and municipal authorities and management responsible for implementation of her; and, iii) The leaders of different levels at a municipal “Health House” established as a hybrid between primary and specialist health services

The project runs between January 2018–2022. Expected findings are: i) Diverging and common values; ii) Diverging governance models; iii) Diverging attitudes towards “best governance practices”; iv) Diverging levels of trust; v) Different world views, belief-systems and individual values; vi) Attitudes towards consensus building or conflict; and, vii) Experiences to feed into the discussion of stakeholder dialogue as an HTA approach.

We expect: i) To present results from the systematic review and preliminary findings from the first phases of participatory observations; ii) That results from the overall project will have high impact on the Norwegian governance models of e-health; and, iii) Publications in high impact scientific journals.

Extracorporeal circulatory membrane oxygenation (ECMO) is a technology that allows recovery of adults in cardiorespiratory failure with encouraging results, but is not available in the Brazilian universal public health system (SUS) due to high implementation costs. Time-driven activity based costing (TDABC) is applied to measure processes in an economic perspective by identifying opportunities to make processes more efficient through the reduction of resources used in each activity. The literature has explored the use of TDABC to measure costs related with clinical procedures and technologies in microcosting studies, identifying opportunities to improve the process by making it more efficient. This research measures the real costs to implement ECMO in Brazil to compare with the current public reimbursement system.

This study applied TDABC using data from 6 patients to measure costs of ECMO intervention considering the public perspective in Brazil. In sequence, standard price payed by SUS was used to estimate the current reimbursement amount received by the hospital for ECMO procedure. Cost variable analysis was conducted to understand when and how patients receiving ECMO are using hospital resources. Cost data were collected from an academic public hospital using an average of 18 months (2016–2017) for the department costs.

The real average cost was USD 128,923. Most significant resource costs was medical staff, particularly for the three survivor patients, and the ECMO equipment presented the second highest cost. ECMO activities were separated into: before implantation of ECMO, period using ECMO, intensive care post-ECMO and rehabilitation, being the period where ECMO is the most expensive, particularly in nurse and physician costs. The SUS average was USD 31,437, which shows a difference of USD 97,485 between the real ECMO cost and the public reimbursement in Brazil.

A critical element of the propagation of ECMO in Brazil and its reimbursement by public health system is the high cost and out-of-date standard payments by the Ministry of Health. Effort to implement a trustworthy method to guide decisions of SUS for the adoption and financing new technologies is essential to contribute to the optimization of public health policies in a country with a universal health system and limited resources dedicated to health sectors.

The Canadian BIA guideline was published by the Patented Medicine Prices Review Board (PMPRB) in 2007. Our initial systematic literature review of national and international BIA guidelines showed that a number of new recommendations relating to BIA model structure, input data and reporting format have been adopted in other jurisdictions such as UK, Australia, Poland, Ireland, Belgium, France and the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). The main objective of the present study was to conduct a comparative review of national, international and Canadian Federal, provincial and territorial BIA guidelines and provide a list of new recommendations related to the BIA key elements which have not been discussed or included in the Canadian PMPRB BIA guidelines.

BIAs guidelines were searched in databases such as MEDLINE, EMBASE, Cochrane, and the gray literature including regulatory agency websites. An Excel-based data abstraction form was designed in order to highlight differences between recommendations related to the BIA key elements provided by PMPRB, provincial, and other national and international BIA guidelines.

Twelve guidelines were reviewed in detail. Sixty percent of the recommendations were new or were different from recommendations in the Canadian PMPRB BIA guidelines. They related to BIA key elements such as perspective, target population, costing, presenting results, data sources and handling the uncertainty.

The present literature review is the initial step towards updating the Canadian BIA guidelines. This study presents a comparative review of key elements in BIA among different guidelines and provides a list of relevant practical recommendations for the improvement of the Canadian BIA guidelines. The new methodologic advancements and recommendations that were identified are being presented to Canadian stakeholders for their opinion and feedback prior to the development of a proposed new set of Canadian guidelines.

Since 2005, new hospital examination and treatment methods (NUB) were reimbursed by hospital individual supplementary fees as long as they were not sufficiently covered by a DRG. In 2016, the NUB procedure was decisively changed by legal norm §137 h SGB.V to evaluate medical devices (MD) of high risk classes, particularly invasive, or new theoretical-scientific concepts versus treatment alternatives by the Federal Joint Committee (G-BA). Hospitals and manufacturers have to submit detailed information on the application of the MD and the scientific evidence to G-BA along with a NUB application. This assessment may lead to exclusion of the method from reimbursement by the statutory health insurance (SHI).

The published MD consultation submissions, assessments and G-BA resolutions (to date) were analyzed regarding evaluation criteria, treatment potential and study obligation.

In 2017, nineteen procedures were reviewed by G-BA with respect to §137 h. Two ultra-controlled high-intensity focused ultrasound (HIFU) indications were regarded as having potential benefit but not sufficient evidence yet, thus respective studies have to be initiated. Three procedures were regarded as eligible according to §137 h but not yet evaluated. Six procedures (ultra-controlled HIFU in five indications, targeted lung denervation in chronic obstructive pulmonary disease) were rated as having no potential benefit, while eight procedures were regarded as not eligible according to §137 h.

Initially put into place for high risk class and primarily invasive devices, consultations and assessments under §137 h show that there is some uncertainty around applicability criteria. The majority of those procedures which fell under the assessment law failed to be granted potential benefit as treatment alternative. Currently consultations are ongoing which could possibly lead to the exclusion of these methods from the performance spectrum of the SHI. Manufacturers should revise their study concepts in order to fulfill the specific demand for robust evidence.

Smartphones have been one of the success stories of the last decade. Recently apps have been used to promote, manage, and provide medical and healthcare education. Since smartphones are used to support healthcare and public health interventions, they can also provide a useful and easy method for collecting data for healthcare research. In addition, smartphone apps have been successfully used to support telemedicine and remote healthcare in developing nations. This study aimed to assess the effectiveness and analyze costs before and after the inclusion of type 2 diabetic patients in a smartphone app healthcare program.

The smartphone app healthcare program is available for Android and IOS systems, and is used to manage behavior changes and to improve patient adherence to pharmacotherapy. Patient follow up is done through a specialized telephone monitoring center made up of a physician, nurses, nutritionists, and psychologists who provide constant monitoring and guidance to patients. A retrospective study was conducted of twenty-nine patients before (year 2016) and 12 months after they were included in the smartphone app healthcare program. Data on physician visits, hospitalizations, and medical and laboratory exams were collected from medical records. The cost analysis was conducted from the private healthcare group perspective and was performed using the micro-costing method.

Ninety-eight percent of patients had reduction or maintenance of glycosylated hemoglobin levels, reaching the therapeutic goal (glycosylated hemoglobin of less than 7%). The cost analysis showed a twenty-five percent total cost saving due to a twenty-three percent reduction in the number of physician visits, a thirty-three percent decrease in hospitalizations, and a thirty-five percent cutback in medical and laboratory exams.

The smartphone app healthcare program can facilitate and improve diabetes care, especially with respect to controlling and managing the use of health resources.

High-cost innovative therapies are considered as high-risk investments in the reality of limited health care budgets. Health technology assessment (HTA) facilitates evidence-based decision making that relies on robust assessment of the clinical cost-effectiveness of the technology that is often not available for expensive cancer therapies (CT).

The objective of the study is to undertake a systematic comparison between HTA agencies worldwide in order to identify similarities and differences in the methods and processes in HTA of CT. A cross-sectional web-based survey was conducted between September 2013 and May 2015. We received responses from 161 HTA organizations based in thirty-nine countries.

HTA of CT is mainly performed by agencies in South America (38.46 percent), Australia (37.05 percent) and Europe (36.07 percent), followed by agencies in North America (20.00 percent) and Asia (16.67 percent). Logically, the agencies in high income countries produce more assessments of CT (40.23 percent), which in 34.43 percent they determine as innovative technologies compared with 10.00 percent of the units based in middle income countries and active in CT assessment (11.11 percent). We prove association (p < 0.05) between (i) the type of HTA and income per capita; the level at which the organization operates; its main activity; and the level of recommendation dissemination; (ii) the main target group and consumers of the final HTA product; the stage of evolution of the technology, on which it is likely to be assessed; and approaches to identify innovative technologies. The most active in the preparation of HTA reports are biomedical companies (50.00 percent), government agencies (42.11 percent) and professional organizations (40.00 percent). HTA bodies that assess CT distribute recommendations (37.50 percent) nationally and they are mainly addressed to private health care providers (66.67 percent).

Making coverage decisions based on HTA recommendations control the technologies introduction into the healthcare system that is why it's very important this tool to be properly adjusted to the specific needs of CT assessment.

In decision-making processes, health technology assessment (HTA) plays an important role ensuring the adoption of effective technologies and translating scientific evidence into decisions. Bambino Gesù Children's Hospital developed a new method which integrates EUnetHTA Core Model with multi-criteria decision analysis (MCDA) enabling decision makers to make a more informed decision between different alternatives. This approach quantifies assessment parameters, which are defined by literature evidence, or by expert opinion when lacking such evidence. MCDA results (i.e. decision tree of assessment elements, weighting systems and numerical values of technology’ performance) are derived from expert judgement. This means that indicators are weighed by the same weight system; either they are supported by strong literature evidence or otherwise based on expert opinion. The objective of this work is to use the GRADE approach to weight the relevance of each indicator starting from its source of information because different level of evidence should result in different weights.

A GRADE level was associated with each judgement value of performance indicators and a normal probability function was built with the standard deviation inversely proportional to GRADE level to describe the possible dispersion of the judgement due to the different levels of evidence that support each indicator. The higher the GRADE value, the lower the associated standard deviation. A Monte Carlo simulation was carried out to evaluate the expected value of technology’ performance modulated by GRADE level.

Four Gaussian distributions were built and associated to four GRADE levels. When an indicator has a low GRADE level, its performance value will vary in a broader way according to the linked Gaussian distribution.

This study showed the importance of applying the GRADE system to indicators’ sources of information because this can modify the overall computation of parameter weights and performance, proportionally to their robustness.

Health technology assessment (HTA) is a resource-intensive decision support tool that is typically structured around a set of defined domains. Sometimes urgent requests for HTAs arise that may be subject to very short timeframes, creating unique challenges. This study aimed to describe some of the options for carrying out a “hasty HTA”, and the impact of those options.

We recently completed a HTA for the Department of Health in Ireland with a strict 2-month deadline. We considered the impact of the short timeline using the project management triple constraint framework whereby the quality of a project is constrained by cost, scope, and schedule.

When delivering HTAs within short timeframes the schedule is an inflexible constraint. Providing interim advice pending a full assessment may set a precedent, or may not be possible if capital expenditure is already entailed. Additional staff should enable research to be completed faster, although economies of scale may not fully apply. Frequently such resources are not readily available. The reduction of scope through the omission of domains offers the best prospect of facilitating a short timeframe for a HTA. Scope may also be reduced through a less comprehensive analytical approach, but this creates a risk of reduced accuracy. Curtailing data collection and analysis is likely to increase uncertainty in the findings. Risk management is important when comprehensive quality assurance may not be possible.

Carrying out HTAs in short timeframes has implications for content, approach, and, potentially, quality. Agencies must consider how they can meet the needs of the decision maker without overly compromising accuracy or relevance. Due to resource constraints, the best approach is likely to be judicious changes to the scope to remove assessment elements that are unlikely to have a substantive impact on the decision.

Our research group recently evaluated a minimally invasive surgical procedure in order to inform a reimbursement decision. The application for funding was designed around the study selection criteria from a single pivotal randomized controlled trial (RCT). The aim of this study review was to evaluate the safety and effectiveness of this minimally invasive surgical procedure, and document challenges faced in evaluating a technology based on a highly targeted population.

A systematic literature search of four biomedical databases was conducted (PubMed, Embase, Cochrane library, York CRD) up to 8 August 2017. Specific elements related to the population were patient age, together with level and duration of pain. Primary effectiveness outcomes included pain, patient reported quality of life, mortality and adverse events. The included RCTs were critically appraised against the Cochrane risk of bias tool. Meta-analysis was not possible due to the limited availability of evidence with consistent outcomes.

From 4,718 search results, only one pivotal RCT specifically met the inclusion criteria, which demonstrated favorable safety and effectiveness of the procedure; however, the sample population in the trial had limited external validity to the proposed reimbursement population and follow-up was limited to six months. As a result, the selection criteria were broadened to better reflect the manner in which the service may be provided in clinical practice, and capture longer-term safety concerns. Four additional RCTs were included, which provided contradictory results.

The results of this review identified two important issues in evaluating a health technology where the assessment has been focused to the results of a single trial. In particular, the generalizability of a trial is defined by the demographic distribution of the sample, not the selection criteria. Designing the review selection criteria around the selection criteria for a single trial can have consequences for a funding decision.

With heightened awareness of the value of patient and provider perspectives to decision making, Qualitative Evidence Synthesis (QES) is increasingly used within a health technology assessment (HTA) context. Acceptability, feasibility and implementation can all be addressed by synthesis of qualitative research. Concerns have been raised about the quality of the synthesis product, especially when conducted within a constrained time window. How can we test the validity of qualitative studies and assess confidence in synthesized qualitative findings, particularly when time is tight?

A brief examination of issues relating to production and use of QES identified from within the Canary Islands HTA agency will identify practical and methodological challenges. How can existing approaches address wider patient, social, organizational and ethical considerations that inform HTA? The potential for use of Evidence To Decision frameworks and approaches such as GRADE CERQual (a transparent method for assessing the confidence of evidence from reviews of qualitative research) will be briefly examined.

This presentation will identify potential gaps between the needs of a small HTA agency and the methodological support and tools required to address these gaps, based on experience of conducting QES to date. Issues identified are particularly relevant to other small HTA agencies but are also generalizable to larger agencies and guideline producers worldwide. Pragmatic solutions are suggested. A future research agenda for potential methodological and applied research is outlined and current GRADE-CERQual development initiatives briefly shared.

Despite significant progress in developing methodologies for integrating QES within HTA decision making, substantive challenges remain. Observations derived from this small HTA agency can inform further developments across all HTA organizations. Research is required to examine the impact of potential dissemination bias, application of tools across a wider HTA decision making framework and use of rigorous approaches within a time-limited evaluation window.

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.