Objectives: Some countries make considerable effort to involve patients and patient groups in their health technology assessment (HTA) processes; others are only just considering or are yet to consider patient involvement in HTA.

Methods: This commentary offers four arguments why patient involvement should be prioritized by those HTA agencies that do not yet involve patients: (1) from a patients’ rights perspective, (2) based on patient and community values, (3) centering on evidentiary contributions, and (4) from a methodological perspective.

Results: The first argument builds on the Alma-Ata Declaration, which holds that patients have a right and duty to have a say in the planning and delivery of their health care, individually and collectively. Where HTA is used to determine access to technologies and services, we argue that patients have a right to be heard. The second argues that decisions about treatments and services need to be aligned with the core values and morals of the patients whom the health system serves. The third argues that patients have unique knowledge and insights about living with a health condition and their needs for services and treatments regarding that condition, which can add to the knowledge base and value of the HTA process. The fourth argues that involvement of patients can facilitate methodological advancement of HTA, in areas such as early scientific advice and managed entry with evidence development.

Conclusions: An HTA process that includes patient perspectives can, therefore, provide added value to patients, policy makers and healthcare professionals alike.

Objectives: The headroom method was introduced for the very early evaluation of the potential value of new technologies. It allows for establishing a ceiling price for technologies to still be cost-effective by combining the maximum effect a technology might yield, the maximum willingness-to-pay (WTP) for this effect, and potential downstream expenses and savings. Although the headroom method is QALY-based, not all innovations are expected to result in QALY gain.

Methods: This study explores the feasibility and usefulness of the headroom method in the evaluation of technologies that are unlikely to result in QALY gain. This will be illustrated with the diagnostic trajectory of complex pediatric neurology (CPN).

Results: Our headroom analysis showed a large room for improvement in the current diagnostic trajectory of CPN in terms of diagnostic yield. Combining this with a maximum WTP value for an additional diagnosis and the potential downstream expenses and savings, resulted in a total headroom of €15,028. This indicates that a new technology in this particular diagnostic trajectory, might be cost-effective as long as its costs do not exceed €15,028.

Conclusions: The headroom method seems a useful tool in the very early evaluation of medical technologies, also in cases when immediate QALY gain is unlikely. It allows for allocating healthcare resources to those technologies that are most promising. It should be kept in mind, however, that the headroom assumes an optimistic scenario, and for that reason cannot guarantee future cost-effectiveness. It might be most useful for ruling out those technologies that are unlikely to be cost-effective.

Objectives: Zika virus (ZikaV) is currently one of the most important emerging viruses in the world which has caused outbreaks and epidemics and has also been associated with severe clinical manifestations and congenital malformations. Traditional approaches to combat the ZikaV outbreak are not effective for detection and control. The aim of this study is to propose a cloud-based system to prevent and control the spread of Zika virus disease using integration of mobile phones and Internet of Things (IoT).

Methods: A Naive Bayesian Network (NBN) is used to diagnose the possibly infected users, and Google Maps Web service is used to provide the geographic positioning system (GPS)-based risk assessment to prevent the outbreak. It is used to represent each ZikaV infected user, mosquito-dense sites, and breeding sites on the Google map that helps the government healthcare authorities to control such risk-prone areas effectively and efficiently.

Results: The performance and accuracy of the proposed system are evaluated using dataset for 2 million users. Our system provides high accuracy for initial diagnosis of different users according to their symptoms and appropriate GPS-based risk assessment.

Conclusions: The cloud-based proposed system contributed to the accurate NBN-based classification of infected users and accurate identification of risk-prone areas using Google Maps.

Objectives: The aim of this study was to review 5 years of activity from a new system devised by the National Institute for Health and Care Excellence (NICE), for assessing medical devices and diagnostics aimed at identifying and speeding adoption of technologies with clinical and cost advantages, compared with current practice in the United Kingdom healthcare system.

Methods: All eligible notified technologies were classified using the Food and Drug Administration and Global Medical Device Nomenclature nomenclatures. Decisions about selecting technologies for full assessment to produce NICE recommendations were reviewed, along with the reasons given to companies for not selecting products.

Results: Between 2009 and 2014, 186 technologies were notified (46 percent therapeutic and 54 percent diagnostic). Thirty-nine were judged ineligible (no regulatory approval), and 147 were considered by an independent committee. Of these, eighty (54 percent) were not selected for full assessment, most commonly because of insufficient evidence (86 percent): there were uncertainties specifically about benefits to the health service (54 percent), to patients (39 percent), and about cost (24 percent). The remaining 67 were selected and assessed for Medical Technology guidance (52 percent) (noninferior and/or lower cost consequences than current practice), for Diagnostics guidance (43 percent) or other NICE recommendations about adoption and use. Classifying technologies by two different systems showed no selection bias for any technology type or disease area.

Conclusions: Identifying new or under-used devices and diagnostics with potential benefits and promoting their adoption is important to health services in the United Kingdom and worldwide. This new system offers a means of fostering both uptake and further research. Lack of research data on new products is a major obstacle to evaluation.

Objectives: Economic evaluation (EE) is an accepted element of decision making and priority setting in healthcare. As the number of published EEs grows, so does the number of systematic reviews (SRs) of EEs. Although search methodology makes an important contribution to SR quality, search methods in reviews of EEs have not been evaluated in detail. We investigated the resources used to identify studies in recent, published SRs of EEs, and assessed whether the resources reflected recommendations.

Methods: We searched MEDLINE for SRs of EEs published since January 2013 and extracted the following from eligible reviews: databases searched, health technology assessment (HTA) sources searched, supplementary search techniques used. Results were compared against the minimum search resources recommended by National Institute for Health and Care Excellence (NICE) (MEDLINE, Embase, NHS EED, EconLit) for economic evidence for single technology appraisals, and resource types suggested in the summary of current best evidence from SuRe Info (economic databases, general databases, HTA databases, HTA agency Web pages, gray literature).

Results: Sixty-five SRs met the inclusion criteria; data were extracted from forty-two. Five reviews (12 percent) met or exceeded the NICE recommended resources. Nine reviews (21 percent) searched at least four of the five types of resource recommended by SuRe Info. Five reviews (12 percent) searched all five. Twenty-three reviews (55 percent) did not meet the NICE recommendations or four of five of the SuRe Info recommended resource types. Search reporting was frequently unclear or incorrect.

Conclusions: Searches conducted for the majority of recently published SRs of EEs do not meet two published approaches.

Objectives: The aim of this report was to assess the clinical effectiveness of two Gene expression profiling (GEP) and two expanded immunohistochemistry (IHC) tests compared with current prognostic tools in guiding the use of adjuvant chemotherapy in patients with early breast cancer.

Methods: A systematic review of the evidence on clinical effectiveness of OncotypeDX, IHC4, MammaPrint, and Mammostrat, compared with current clinical practice using clinicopathological parameters, in women with early breast cancer was conducted. Ten databases were searched to include citations to May 2016.

Results: Searches identified 7,064 citations, of which forty-one citations satisfied the criteria for the review. A narrative synthesis was performed. Evidence for OncotypeDX demonstrated the impact of the test on decision making and there was some support for OncotypeDX predicting chemotherapy benefit. There were relatively lower levels of evidence for the other three tests included in the analysis. MammaPrint, Mammostrat, and IHC4 tests were limited to a small number of studies. Limitations in relation to study design were identified for all tests.

Conclusions: The evidence base for OncotypeDX is considered to be the most robust. Methodological weaknesses relating to heterogeneity of patient cohorts and issues arising from the retrospective nature of the evidence were identified. Further evidence is required for all of the tests using prospective randomized controlled trial data.

Objectives: The aim of this study was to assess viewpoints of end-users concerning the purchasing process of high-risk medical devices and to discuss the relevance of health technology assessments (HTAs) at the hospital level and other potential areas for improvement of purchasing processes.

Methods: We used a cross-sectional study and assessed the attitudes and thoughts of orthopedic specialists. The study took place between June and October 2015 in Mexico.

Results: We collected data from 187 orthopedic surgeons. Involvement of orthopedic specialists in purchasing was reported by 86 percent. However, clinical practice was perceived as negatively influenced by purchasing outcomes by 92 percent. The problems were described as: material failure; effectiveness of medical devices; obsolete medical device technology; incomplete provision of implant / instrument sets; delayed provision of implants and instruments.

Conclusions: To prevent sub-standard outcomes of purchasing decisions, this study and the current literature suggest that technologies should be assessed during the purchasing process, end-users should be adequately involved, and decisions should be based on multiple criteria including clinical impact in the short-term (e.g., primary stability of implant) and long-term (e.g., survival of implant). The focus on Mexico is particularly novel and provides insights into a health system where HTA is mainly present at the macro level and can be used for the listing of medical device technologies in the standard list. This study concludes that Mexican stakeholders of the purchasing process underestimate the contribution of HTAs at the level of purchasing decisions. HTA in Mexico has improved over the past years but still requires more advancement.

Objectives: The aim of this study was to present the experience of a Canadian hospital-based health technology assessment (HTA) unit that performed the traditional functions of the HTA process along with many other activities to facilitate the choice of smart pumps.

Methods: A rapid literature review was initiated, but little evidence was found. Moreover, the evidence provided was too far from our hospital context. To help our decision makers, we offered them a list of various services based on the skills of our HTA unit staff.

Results: To involve our HTA unit in the choice of the new smart pumps led to a strong collaboration between hospital services. After a rapid review on smart pumps, we proceeded to establish the clinical needs, followed by an evaluation of technical features. To ascertain clinical needs, we participated in the establishment of a conformity list for the tender, a failure and mode-effect analysis, an audit on the use of actual smart pumps, and simulation exercises with nurses and doctors to evaluate the ease of use and ergonomics. With regard to technical tests, these were mainly conducted to identify potential dysfunction and to assess the efficiency of the pump. This experience with smart pumps was useful for evidence-based procurement and led to the formulation of a nine-step process to guide future work.

Conclusions: HTA units and agencies are faced with rapid development of new technologies that may not be supported by sufficient amount of pertinent published evidence. Under these circumstances, approaches other than evidence-based selection might provide useful information. Because these activities may be different from those related to classic HTA, this widens the scope of what can be done in HTA to support decision making.

Objectives: Patient Web portals (PWPs) have been gaining traction as a means to collect patient-reported outcomes and maintain quality patient care between office visits. PWPs have the potential to impact patient–provider relationships by rendering additional channels for communication outside of clinic visits and could help in the management of common chronic medical conditions. Studies documenting their effect in primary care settings are limited. This perspective aims to summarize the benefits and drawbacks of using PWPs in the management of chronic conditions, such as diabetes mellitus, hypertension, and asthma, focusing on communication, disease management, compliance, potential barriers, and the impact on patient–provider dynamic. After a review of these topics, we present potential future directions.

Methods: We conducted an exploratory PubMed search of the literature published from inception through December 2015, and focused our subsequent searches specifically to assess benefits and drawbacks of using PWPs in the management of diabetes mellitus, hypertension, and asthma.

Results: Our search revealed several potential benefits of PWP implementation in the management of chronic conditions with regards to patient–provider relationships, such as improved communication, disease management, and compliance. We also noted drawbacks such as potentially unreliable reporting, barriers to use, and increased workload.

Conclusions: PWPs offer opportunities for patients to report symptoms and outcomes in a timely manner and allow for secure online communication with providers. Despite the drawbacks noted, the overall benefits from successful PWP implementation could improve patient–provider relationships and help in the management of chronic conditions, such as diabetes mellitus, hypertension, and asthma.

Objectives: We conducted a meta-analysis of recently published randomized controlled trials (RCTs) to identify the most effective and safe etanercept dosing regimen and duration of therapy for the treatment of patients with ankylosing spondylitis (AS).

Methods: We systematically reviewed PubMed, Embase, Cochrane Library, and Web of Science databases for RCTs. The proportion of patients attaining 20 percent improvement (according to the Spondyloarthritis International Society response criteria [ASAS 20]) was evaluated as a primary outcome. Secondary outcomes included 50 percent increase in the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI 50) used for evaluating efficacy, as well as the BASDAI/Bath Ankylosing Spondylitis Functional Index (BASFI) scores and adverse events.

Results: ASAS 20 indicated that the efficacy of etanercept did not differ amongst dosing regimens (25 mg twice-weekly versus 50 mg once-weekly: relative risk [RR], 2.18, 95 percent confidence interval [CI], 1.78–2.67 versus RR, 2.00, 95 percent CI, 1.70–2.37). The ASAS 20 reported subgroup differences among treatment durations of less than 12 weeks (RR, 2.70; 95 percent CI, 2.09–3.49); 12 weeks (RR, 1.74; 95 percent CI, 1.37–2.22); and more than 12 weeks (RR, 2.56; 95 percent CI, 1.88–3.48). Other outcomes included BASDAI, BASDAI 50, and BASFI. Drug safety differed according to the treatment regimen and duration.

Conclusion: Our meta-analysis found that there was no significant efficacy difference between 50 mg once-weekly and 25 mg twice-weekly dosing for the treatment of AS, and a dosing duration of less than 12 weeks was more effective for treating AS patients.

Objectives: Some experts have promoted preparticipative cardiovascular screening programs for young athletes and have claimed that such programs were cost-effective without performing a critical analysis of studies supporting this statement. In this systematic review, a critical assessment of economic evaluations on these programs is performed to determine if they really provide value for money.

Methods: A systematic review of economic evaluations was performed on December 24, 2014. Web sites of health technology assessment agencies, the Cochrane database of systematic review, the National Health Service Economic Evaluation Database of the Cochrane Library, EMBASE, Medline, Psychinfo, and EconLit were searched to retrieve (reviews of) economic evaluations. No language or time restrictions were imposed and predefined selection criteria were used. Selected studies were critically assessed applying a structured data extraction sheet.

Results: Five relevant economic evaluations were critically assessed. Results of these studies were mixed. However, those in favor of screening made (methodological) incorrect choices, of which the most important one was not taking into account a no-screening alternative as comparator. Compared with no screening, other strategies (history and physical examination or history and physical examination plus electrocardiogram) were not considered cost-effective.

Conclusions: Results of primary economic evaluations should not be blindly copied without critical assessment. Economic evaluations in this field lack the support of robust evidence. Negative consequences of screening (false positive findings, overtreatment) should also be taken into account and may cause more harm than good. A mass screening of young athletes for cardiovascular diseases does not provide value for money and should be discouraged.

Background: Defining relevant outcome measures for clinical trials on medical devices (MD) is complex, as there is a large variety of potentially relevant outcomes. The chosen outcomes vary widely across clinical trials making the assessment in evidence syntheses very challenging. The objective is to provide an overview on the current common procedures of health technology assessment (HTA) institutions in defining outcome measures in MD trials.

Methods: In 2012–14, the Web pages of 126 institutions involved in HTA were searched for methodological manuals written in English or German that describe methods for the predefinition process of outcome measures. Additionally, the institutions were contacted by email. Relevant information was extracted. All process steps were performed independently by two reviewers.

Results: Twenty-four manuals and ten responses from the email request were included in the analysis. Overall, 88.5 percent of the institutions describe the type of outcomes that should be considered in detail and 84.6 percent agree that the main focus should be on patient relevant outcomes. Specifically related to MD, information could be obtained in 26 percent of the included manuals and email responses. Eleven percent of the institutions report a particular consideration of MD related outcomes.

Conclusions: This detailed analysis on common procedures of HTA institutions in the context of defining relevant outcome measures for the assessment of MD shows that standardized procedures for MD from the perspective of HTA institutions are not widespread. This leads to the question if a homogenous approach should be implemented in the field of HTA on MD.

Objectives: The aim of this study was to evaluate available knowledge and identify knowledge gaps within the field of oral and maxillofacial surgery, by systematically collecting and evaluating systematic reviews. Twelve specific domains were selected: surgical removal of teeth, antibiotic and corticosteroid prophylaxis, orofacial infections, dental and facial trauma, orthognathic surgery, reconstructive surgery, benign tumors, cysts, premalignant lesions, oral complications of treatment of malignant tumors, hyperbaric oxygen therapy, temporomandibular joint surgery, cost effectiveness of different surgical treatments, and ethics.

Methods: The literature search, covering four databases, was conducted during September 2014: PubMed, The Cochrane library, Centre for Reviews and Dissemination and EBSCO dentistry and oral science source. Retrieved systematic reviews were quality assessed by AMSTAR.

Results: In all, 1,778 abstracts were identified, of which 200 met the inclusion criteria. Forty-five systematic reviews were assessed as of high to moderate quality. The results disclosed some existing evidence in a few domains, such as surgical removal of teeth and implant survival after sinus lifts. However, in all domains, the search revealed a large number of knowledge gaps. Also of concern was the lack of data regarding health economics and ethics.

Conclusions: In conclusion, there is a need for well-conducted clinical research in the fields of oral and maxillofacial surgery.

Background: The aim of this study was to assess the quality of reporting sample size calculation and underlying design assumptions in pivotal trials of high-risk medical devices (MDs) for neurological conditions.

Methods: Systematic review of research protocols for publicly registered randomized controlled trials (RCTs). In the absence of a published protocol, principal investigators were contacted for additional data. To be included, trials had to investigate a high-risk MD, registered between 2005 and 2015, with indications stroke, headache disorders, and epilepsy as case samples within central nervous system diseases. Extraction of key methodological parameters for sample size calculation was performed independently and peer-reviewed.

Results: In a final sample of seventy-one eligible trials, we collected data from thirty-one trials. Eighteen protocols were obtained from the public domain or principal investigators. Data availability decreased during the extraction process, with almost all data available for stroke-related trials. Of the thirty-one trials with sample size information available, twenty-six reported a predefined calculation and underlying assumptions. Justification was given in twenty and evidence for parameter estimation in sixteen trials. Estimates were most often based on previous research, including RCTs and observational data. Observational data were predominantly represented by retrospective designs. Other references for parameter estimation indicated a lower level of evidence.

Conclusions: Our systematic review of trials on high-risk MDs confirms previous research, which has documented deficiencies regarding data availability and a lack of reporting on sample size calculation. More effort is needed to ensure both relevant sources, that is, original research protocols, to be publicly available and reporting requirements to be standardized.

Objectives: The aim of this study was to adapt and assess the value of a Multi-Criteria Decision Analysis (MCDA) framework (EVIDEM) for the evaluation of Orphan drugs in Catalonia (Catalan Health Service).

Methods: The standard evaluation and decision-making procedures of CatSalut were compared with the EVIDEM methodology and contents. The EVIDEM framework was adapted to the Catalan context, focusing on the evaluation of Orphan drugs (PASFTAC program), during a Workshop with sixteen PASFTAC members. The criteria weighting was done using two different techniques (nonhierarchical and hierarchical). Reliability was assessed by re-test.

Results: The EVIDEM framework and methodology was found useful and feasible for Orphan drugs evaluation and decision making in Catalonia. All the criteria considered for the development of the CatSalut Technical Reports and decision making were considered in the framework. Nevertheless, the framework could improve the reporting of some of these criteria (i.e., “unmet needs” or “nonmedical costs”). Some Contextual criteria were removed (i.e., “Mandate and scope of healthcare system”, “Environmental impact”) or adapted (“population priorities and access”) for CatSalut purposes. Independently of the weighting technique considered, the most important evaluation criteria identified for orphan drugs were: “disease severity”, “unmet needs” and “comparative effectiveness”, while the “size of the population” had the lowest relevance for decision making. Test–retest analysis showed weight consistency among techniques, supporting reliability overtime.

Conclusions: MCDA (EVIDEM framework) could be a useful tool to complement the current evaluation methods of CatSalut, contributing to standardization and pragmatism, providing a method to tackle ethical dilemmas and facilitating discussions related to decision making.

Objectives: Japanese authorities have announced a plan to introduce a health technology assessment (HTA) system in 2016. This study assessed the potential impact of such a policy on the price of the antivirologic drug simeprevir.

Methods: Taking the antivirologic drug simeprevir as an example, we compared the current Japanese price with hypothetical prices that might result if a U.K. (cost-utility) or German (efficiency frontier) style HTA assessment was in place.

Results: The simeprevir unit price under the current Japanese pricing scheme is 13,122 Japanese yen (equivalent to 109.35 U.S. dollars as of April 2015). Depending on the selection of comparators and the pricing method, and assuming that HTA will be used as a basis for price setting, the estimated prices of simeprevir vary up to four times higher than under the current Japanese pricing scheme.

Conclusions: Although the analysis is based on only one drug, it cannot be taken for granted that a new HTA system would reduce public healthcare expenditure in Japan.

Objectives: The aim of this work was to provide a comprehensive overview of the evolution of the health technology assessment (HTA) concept in the scientific literature through a scientometric approach.

Methods: A literature search was conducted, by selecting publications, as well as news from the media, containing “health technology assessment” in their title, abstracts, or keywords. We then undertook a bibliometric and network analysis on the corpus of 2,865 publications thus obtained.

Results: Since a first publication in 1978, interest in HTA remained marginal until a turning point in the late 1980s, when growth of the number of publications took off alongside the creation of the U.K.’s NICE agency. Since then, publications have spread across several journals. The ranking of the organizations that publish such articles does not reflect any hegemonic position. However, HTA-related scientific production is strongly concentrated in Commonwealth and Nordic countries. Despite its transnational aspects, research on HTA has been framed within a small number of scientific networks and by a few opinion leaders.

Conclusions: The “career” of the HTA concept may be seen as a scientific-knowledge based institutionalization of a public policy. To succeed in a country, HTA first needs scientific prerequisites, such as an organized scientific community working on the health sector and health services. Then, it appears that the recognition of this research by decision makers plays a key role in the development of the field.