Objectives: The objective of this study was to compare evidence requirements for health technology assessment of pharmaceuticals by national agencies across Europe responsible for reimbursement decisions focusing specifically on relative effectiveness assessment.

Methods: Evidence requirements from thirty-three European countries were requested and twenty-nine national agencies provided documents to review. Data were extracted from national documents (manufacturer's submission templates and associated guidance) into a purpose-made framework with categories covering information about the health condition, the technology, clinical effectiveness and safety.

Results: The level of detail in the required evidence varies considerably across countries. Some countries include specific questions while others request information under general headings. Some countries include all information in a single document, which may or may not include guidance on how to complete the template. Others have specific guidance documents or methods and process manuals that help with the completion of the submission templates. Despite differences in quantity and detail, the content of the evidence requirements is broadly similar. All countries ask for information on the health technology, target disease, and clinical effectiveness and safety. However, one country only requests clinical effectiveness information as part of cost-effectiveness analyses. We found twenty-six evidence requirements for which generic answers may apply across borders and nineteen in which countries requested nationally specific information.

Conclusions: This work suggests that it would be possible to put together a minimum set of evidence requirements for HTA to support reimbursement decisions across Europe which could facilitate collaboration between jurisdictions.

Objectives: The aim of this study was to conduct a review of health technology assessments (HTAs) in cervical cancer screening to highlight the most common metrics HTA agencies use to evaluate and recommend cervical cancer screening technologies.

Methods: The Center for Reviews and Dissemination (CRD), MedLine, and national HTA agency databases were searched using keywords (“cervical cancer screening” OR “cervical cancer” OR “cervical screening”) and “HTA” from January 2000 to October 2014. Non-English language reports without English summaries, non-HTA reports, HTAs unrelated to a screening intervention and HTAs without sufficient summaries available online were excluded. We used various National Institute for Health and Care Excellence (NICE) methods to extract key assessment criteria and to determine whether a change in screening practice was recommended.

Results: One hundred and ten unique HTA reports were identified; forty-four HTAs from seventeen countries met inclusion criteria. All reports evaluated technologies for use among women. Ten cervical screening technologies were identified either as an intervention or a comparator. The most common outcome metric evaluated was diagnostic accuracy, followed by economic effectiveness. Additional outcome metrics such as the use of adjunct testing, screening intervals, and age-specific testing were commonly evaluated. Nearly one-third (fifteen of forty-four) of HTAs recommended a change in practice.

Conclusions: This review highlights popular metrics used in HTAs for cervical cancer screening. Clinical and economic effectiveness metrics have been consistently assessed in HTAs, while the use of adjunct testing, screening intervals, and age-specific screening became increasingly prevalent from after 2007. Moreover, we observed an increase in optimized recommendations after 2007.

Objectives: The headroom approach to medical device development relies on the estimation of a value-based price ceiling at different stages of the development cycle. Such price-ceilings delineate the commercial opportunities for new products in many healthcare systems. We apply a simple model to obtain critical business information as the product proceeds along a development pathway, and indicate some future directions for the development of the approach.

Methods: Health economic modelling in the supply-side development cycle for new products.

Results: The headroom can be used: initially as a ‘reality check’ on the viability of the device in the healthcare market; to support product development decisions using a real options approach; and to contribute to a pricing policy which respects uncertainties in the reimbursement outlook.

Conclusions: The headroom provides a unifying thread for business decisions along the development cycle for a new product. Over the course of the cycle attitudes to uncertainty will evolve, based on the timing and manner in which new information accrues. Within this framework the developmental value of new information can justify the costs of clinical trials and other evidence-gathering activities. Headroom can function as a simple shared tool to parties in commercial negotiations around individual products or groups of products. The development of similar approaches in other contexts holds promise for more rational planning of service provision.

Objectives: This review aims to assess the state of the science around the potential impact of certain patient characteristics on the safety and effectiveness of in vitro fertilization (IVF).

Methods: Following Cochrane Collaboration guidelines and the PRISMA statement, a comprehensive systematic review of reviews and recent primary studies examining the impact of paternal age and maternal age, smoking, and body mass index (BMI) on the safety and effectiveness of IVF was performed. Papers, published between January 2007 and June 2014, were independently reviewed and critically appraised by two researchers using published quality assessment tools for reviews and primary studies. Due to heterogeneity across papers (different study designs and patient selection criteria), a qualitative analysis of extracted information was performed.

Results: Seventeen papers (ten systematic reviews and seven primary studies) were included. They comprised evidence from retrospective observational studies in which maternal age, BMI, and smoking status were explored as part of secondary analyses of larger studies. The majority of papers found that the likelihood of achieving a pregnancy was lower among women who were >40 years, had a BMI ≥ 25 and smoked. Advanced maternal age and BMI were also associated with higher rates of preterm birth and low birth weight.

Conclusions: Based on available evidence, it may be appropriate to consider “maternal age” and “morbid obesity” in public funding policies that aim to maximize the effectiveness of IVF. However, given inconsistencies in the effect of smoking across different pregnancy-related outcomes, support for incorporating it into funding conditions appears weak.

Background: “Mapping” onto generic preference-based outcome measures is increasingly being used as a means of generating health utilities for use within health economic evaluations. Despite publication of technical guides for the conduct of mapping research, guidance for the reporting of mapping studies is currently lacking. The MAPS (MApping onto Preference-based measures reporting Standards) statement is a new checklist, which aims to promote complete and transparent reporting of mapping studies.

Methods: In the absence of previously published reporting checklists or reporting guidance documents, a de novo list of reporting items was created by a working group comprised of six health economists and one Delphi methodologist. A two-round, modified Delphi survey with representatives from academia, consultancy, health technology assessment agencies, and the biomedical journal editorial community was used to identify a list of essential reporting items from this larger list.

Results: From the initial de novo list of twenty-nine candidate items, a set of twenty-three essential reporting items was developed. The items are presented numerically and categorized within six sections, namely: (i) title and abstract, (ii) introduction, (iii) methods, (iv) results, (v) discussion, and (vi) other. The MAPS statement is best applied in conjunction with the accompanying MAPS explanation and elaboration document.

Conclusions: It is anticipated that the MAPS statement will improve the clarity, transparency. and completeness of reporting of mapping studies. To facilitate dissemination and uptake, the MAPS statement is being co-published by seven health economics and quality of life journals, and broader endorsement is encouraged. The MAPS working group plans to assess the need for an update of the reporting checklist in five years’ time.

Objectives: The aim of this study is to analyze the quality and transferability issues reported in published peer-reviewed English-language economic evaluations based in healthcare settings of the Central and Eastern European (CEE) and former Soviet countries.

Methods: A systematic search of economic evaluations of healthcare interventions was performed for Armenia, Azerbaijan, Belarus, Bulgaria, Estonia, Georgia, Turkmenistan, Kazakhstan, Lithuania, the former Yugoslav Republic of Macedonia, Republic of Moldova, Romania, the Russian Federation, Serbia, Slovenia, and Ukraine. The included studies were assessed according to their characteristics, quality (using Drummond's checklist), use of local data, and the transferability of inputs and results, if addressed.

Results: Most of the thirty-four economic evaluations identified were conducted from a healthcare or payer perspective (74 percent), with 47 percent of studies focusing on infectious diseases. The least frequently and transparently addressed parameters were the items’ stated perspectives, relevant costs included, accurately measured costs in appropriate units, outcomes and costs credibly valued, and uncertainties addressed. Local data were often used to assess unit costs, baseline risk, and resource usage, while jurisdiction-specific utilities were included in only one study. Only 32 percent of relevant studies discussed the limitations of using foreign data, and 36 percent of studies discussed the transferability of their own study results to other jurisdictions.

Conclusions: Transferability of the results is not sufficiently discussed in published economic evaluations. To simplify the transferability of studies to other jurisdictions, the following should be comprehensively addressed: uncertainty, impact of influential parameters, and data transferability. The transparency of reporting should be improved.

Background: Despite ongoing policy debate, little is known about the growth in orthopedic surgery practices with onsite magnetic resonance imaging (MRI) capacity, or practice characteristics associated with the acquisition of in-office MRI equipment.

Methods: In July 2012, American Academy of Orthopaedic Surgeons (AAOS) member practices received a web-based survey requesting general information about their practice, such as number practice providers authorized to order MRIs, the type of onsite MRI capacity present (if any), and the date of acquisition for the MRI equipment. Survey responses were augmented with county-level measures of practice area characteristics as of the year of first onsite MRI acquisition (or 2012 for practices without an onsite MRI).

Results: The survey obtained usable responses from 740 orthopedic practices, which were geographically representative of AAOS member practices. Forty percent (298) reported onsite MRI capacity. Onsite MRI acquisition occurred at a steady pace over 2000–2012, with no dramatic increase occurring in any particular year over that period. Multivariate logistic regression indicated that practice size (number of providers) was the most important factor affecting the likelihood of onsite MRI acquisition. There was no association between onsite MRI acquisition and any of the county-level practice area characteristics included in the analysis.

Conclusions: Orthopedic practices acquiring onsite MRI equipment on average are much larger than practices without onsite MRI capacity. Larger practices may be more likely to attain the economies of scale necessary to absorb the fixed costs associated with onsite MRI acquisition.

Objectives: There is widespread commitment—at least in principle—to “evidence-informed” clinical practice and policy development in health care. The intention is that only “appropriate” care ought to be delivered at public expense. Although the rationale for an appropriateness agenda is widely endorsed, and methods have been proposed for addressing it, few published studies exist of contemporary policy initiatives which have actually led to successful disinvestment. Our objective was to explore whether the direct involvement of policy stakeholders could advance appropriateness and disinvestment.

Methods: Several collaborative engagements with policy stakeholders were undertaken to adapt and combine conceptual and empirical material related to appropriateness and disinvestment from the literature to create tools and processes for use in Canada and the province of Ontario in particular.

Results: By combining inputs from the literature with colloquial evidence from policy stakeholders, a definition of appropriateness was developed and, importantly, endorsed by all the provincial and territorial ministers of health in Canada. Second, a reassessment framework was successfully implemented for identifying priorities for selective disinvestment.

Conclusions: When scientific evidence was combined with colloquial evidence from policy stakeholders, progress was made on the design and successful implementation of policies for appropriateness and disinvestment.

Objectives: The aim of this study was to evaluate the diagnostic accuracy of sensitivity and specificity of cervical lesions by the low-cost, portable Gynocular colposcope and a stationary colposcope, in women referred for colposcopy with abnormal cervical cytology.

Methods: A randomized cross-over clinical trial for evaluating the diagnostic accuracy in detecting cervical lesions by the Gynocular and a stationary colposcope. The Swede score systematic colposcopy system was used for evaluation of colposcopic abnormalities. Directed punch biopsy and excisional cone biopsy were used as the “gold-standard” by histologically confirmed high grade cervical lesions CIN2+ (CIN2, CIN3, CIN3+). In total, 123 women referred for colposcopy due to abnormal cervical cytology were recruited at the Department of Obstetrics and Gynecology, Danderyd Hospital, Stockholm, Sweden. The percentage agreement and the kappa statistic were calculated for Swede score by the Gynocular and a stationary colposcope. Swede scores were compared with the results from directed punch biopsy and excisional cone biopsy.

Results: The Gynocular and the stationary colposcope had a high agreement of Swede scores with a Kappa statistic of 0.947, p < .0001. Punch biopsy diagnosed CIN2+ (CIN2, CIN3, and invasive cancer) in 44 (35.7 percent) women while cytology detected CIN2+ in 34 (27.6 percent) women. There were no significant differences of the sensitivity and specificity for different Swede scores by the Gynocular or a stationary colposcope in detecting CIN 2+.

Conclusions: There were no significant differences in sensitivity or specificity in detecting cervical lesions by the Gynocular or stationary colposcope. The Gynocular is as accurate in diagnosing cervical lesions as a stationary colposcope.

Objectives: The aim of this study was to explore stakeholders’ points of views regarding the applicability and relevance of a framework for user involvement in health technology assessment (HTA) at the local level. We tested this framework in the context of the assessment of alternative measures to restraint and seclusion among hospitalized adults and those living in long-term-care facilities.

Methods: Twenty stakeholders (health managers, user representatives, and clinicians) from seven regions of Quebec participated in a semi-structured interview. A thematic analysis of the transcribed interviews was performed.

Results: The findings highlighted the relevance and applicability of the framework to this specific HTA. According to interviewees, direct participation of users in the HTA process allows them to be part of the decision-making process. User consultation makes it possible to consider the views of a wide variety of people, such as marginalized and vulnerable groups, who do not necessarily meet the requirements for participating in HTA committees. However, some user representatives emphasized that user consultation should be integrated into a more holistic and participatory perspective. The most frequent barrier associated with user involvement in HTA was the top-down health system, which takes little account of the user's perspective.

Conclusions: The proposed framework was seen as a reference tool for making practitioners and health managers aware of the different mechanisms of user involvement in HTA and providing a structured way to classify and describe strategies. However, there is a need for more concrete instruments to guide practice and support decision making on specific strategies for user involvement in HTA at the local level.

Objectives: The Centers for Medicare and Medicaid Services (CMS) issues National Coverage Determinations (NCDs) for medical interventions expected to have a significant impact on Medicare, the health insurance program for US citizens aged 65 years and older and certain people with disabilities under the age of 65 years. The objective of this study was to evaluate NCDs issued from 1999 to 2013 to identify key trends, and to discuss implications for future CMS policy.

Methods: We used the Tufts Medical Center Medicare National Coverage Determination Database to examine characteristics of NCDs from 1999 through 2013. We examined various characteristics of NCDs, including: whether the intervention under review is used for prevention or treatment of disease, the type of intervention considered, evidence limitations cited by CMS, and coverage determination outcome. We evaluated longitudinal trends in categorical and continuous variables in the database, using Cochran-Armitage trend tests and linear regression, respectively.

Results: We found that NCDs increasingly focus on preventive care (p = 0.072), pertain to diagnostic imaging (p = 0.033), and evaluate health education/behavioral therapy interventions (p = 0.051). CMS increasingly cites the lack of relevant outcomes (p = 0.019) and the lack of applicability of study results to the Medicare population (p < 0.001) as evidence limitations. CMS less often restricts coverage to certain population subgroups in NCDs (p < 0.001), but increasingly applies coverage with evidence development policies (p < 0.001).

Conclusions: Identified trends reflect broader changes in Medicare as CMS shifts its focus from treatment to prevention of disease, addresses potentially overutilized technologies, and attempts to issue flexible coverage policies.

Objectives: The EuroScan International Network is a global network of publicly funded early awareness and alert (EAA) systems for health technologies. We describe the EuroScan member agency systems and methods, and highlight the potential for increased collaboration.

Methods: EuroScan members completed postal questionnaires supplemented with telephone interviews in 2012 to elicit additional information and check equivalence of responses. Information was updated between March and May 2013.

Results: Fifteen of the seventeen member agencies responded. The principal purpose of agencies is to inform decisions on coverage or reimbursement of health services and decisions on undertaking secondary research. The main users of information are national governments; health professionals; health services purchasers, commissioners, and decision makers; and healthcare providers. Most EuroScan agencies are small with almost half having fewer than two whole time equivalent staff. Ten agencies use both active and passive identification approaches, four use only active approaches. Most start identification in the experimental or investigational stages of the technology life cycle. All agencies assessed technologies when they are between the investigational and established, but under diffusion stages. Barriers to collaboration revolve around different system aims, purposes, and requirements; a lack of staff, finance, or opportunity; language differences; and restrictions on dissemination.

Conclusions: Although many barriers to collaboration were identified, the majority of agencies were supportive of increased collaboration either involving the whole EuroScan Network or between individual agencies. Despite differences in the detailed identification processes, members thought that this was the most feasible phase to develop additional collaboration.

Objectives: The aim of this study was to determine the role of community preference information from discrete choice studies of colorectal cancer (CRC) screening in health technology assessment (HTA) reports and subsequent policy decisions.

Methods: We undertook a systematic review of discrete choice studies of CRC screening. Included studies were reviewed to assess the policy context of the research. For those studies that cited a recent or pending review of CRC screening, further searches were undertaken to determine the extent to which community preference information was incorporated into the HTA decision-making process.

Results: Eight discrete choice studies that evaluated preferences for CRC screening were identified. Four of these studies referred to a national or local review of CRC screening in three countries: Australia, Canada, and the Netherlands. Our review of subsequently released health policy documents showed that while consideration was given to community views on CRC, policy was not informed by discrete choice evidence.

Conclusions: Preferences and values of patients are increasingly being considered “evidence” to be incorporated into HTA reports. Discrete choice methodology is a rigorous quantitative method for eliciting preferences and while as a methodology it is growing in profile, it would appear that the results of such research are not being systematically translated or integrated into HTA reports. A formalized approach is needed to incorporate preference literature into the HTA decision-making process.

Objectives: Health technology reassessment and disinvestment can be difficult due to uncertainties regarding available evidence. Pathology testing to investigate cobalamin (vitamin B12) deficiency is a strong case in point. We conducted a 3-month economic evaluation of five strategies for diagnosing and treating cobalamin deficiency in adult patients hypothetically presenting with new unexplained fatigue in the primary care setting. The first consultation per patient was considered. Screening tests other than serum cobalamin were not included.

Methods: A cost-effectiveness analysis was undertaken using a decision tree to represent the diagnostic / treatment pathways, with relevant cost and utility scores assigned to different stages in the evaluation process. Input parameter values were estimated from published evidence, supplemented by expert opinion, with sensitivity analysis undertaken to represent parameter uncertainty.

Results: Ordering serum vitamin B12 to assess cobalamin deficiency among patients with unexplained fatigue was not cost-effective in any patient population, irrespective of pretest prevalence of this deficiency. For patients with a pretest prevalence above 1 percent, treating all with oral vitamin B12 supplements without testing was most cost-effective, whereas watchful waiting with symptoms monitoring was most cost-effective for patients with lower pretest prevalence probabilities.

Conclusions: Substantial evidence gaps exist for parameter estimation: questionable cobalamin deficiency levels in the fatigued; debatable treatment methods; unknown natural history of the condition. Despite this, we reveal a robust path for disinvestment decision making in the face of a paradox between the evidence required to inform disinvestment compared with its paucity in informing initial funding decisions.

Background: Decision makers in middle-income countries are using economic evaluations (EEs) in pricing and reimbursement decisions for pharmaceuticals. However, whilst many of these jurisdictions have local submission guidelines and local expertise, the studies themselves often use economic models developed elsewhere and elements of data from countries other than the jurisdiction concerned. The objectives of this study were to describe the current situation and to assess the challenges faced by decision makers in transferring data and analyses from other jurisdictions.

Methods: Experienced health service researchers in each region conducted an interview survey of representatives of decision making bodies from jurisdictions in Asia, Central and Eastern Europe, and Latin America that had at least 1 year's experience of using EEs.

Results: Representatives of the relevant organizations in twelve countries were interviewed. All twelve jurisdictions had developed official guidelines for the conduct of EEs. All but one of the organizations evaluated studies submitted to them, but 9 also conducted studies and 7 commissioned them. Nine of the organizations stated that, in evaluating EEs submitted to them, they had consulted a study performed in a different jurisdiction. Data on relevant treatment effect was generally considered more transferable than those on prices/unit costs. Views on the transferability of epidemiological data, data on resource use and health state preference values were more mixed. Eight of the respondents stated that analyses submitted to them had used models developed in other jurisdictions. Four of the organizations had a policy requiring models to be adapted to reflect local circumstances. The main obstacles to transferring EEs were the different patterns of care or wealth of the developed countries from which most economic evaluations originate.

Conclusions: In middle-income countries it is commonplace to deal with the issue of transferring analyses or data from other jurisdictions. Decision makers in these countries face several challenges, mainly due to differences in current standard of care, practice patterns, or gross domestic product between the developed countries where the majority of the studies are conducted and their own jurisdiction

Objectives: The aim of this study was to assess effect of adult diapers on health-related quality of life (HRQOL) and the independent level of performing activities of daily living (ADLs) in people with urinary or fecal incontinence. Psychological consequences of patients’ caregivers were also measured.

Methods: This quasi-experimental study was conducted at two rehabilitation centers in Thailand. People aged 15 years or greater with chronic urinary or fecal incontinence were eligible. Study participants received adult diapers for 10 weeks after recruitment. Thai EuroQol Five Dimensions (EQ-5D) and the Barthel Index were measured at baseline and weeks 2, 6, and 10 to evaluate HRQOL and the independent level of performing ADLs, respectively. The Braden Scale was used to assess the risk of having pressure ulcers. Mean differences in the Thai EQ-5D, the Barthel Index, and the Braden Scale, before and after receiving adult diapers, were estimated using a multilevel linear regression model.

Results: There were ninety patients and forty-eight caregivers who took part in this study. HRQOL and independent level of performing ADLs had improved significantly by week 10 after receiving adult diapers with mean differences of 0.102 (95% confidence interval [CI], 0.046–0.158) and 4.40 (95% CI, 1.74–7.07), respectively. The risk of having pressure ulcers had significantly decreased by 67 percent (95% CI, 16 percent–78 percent) by week 10 after receiving adult diapers.

Conclusions: The results indicate a significant improvement of HRQOL and the independent level of performing ADLs among incontinent patients after receiving adult diapers. These results were used to inform the development of the health benefits package under the Universal Health Coverage Scheme in Thailand.

Objectives: Economic evaluations, although not formally used in purchasing decisions for medical devices in Canada, are still being conducted and published. The aim of this study was to examine the way that prices have been included in Canadian economic evaluations of medical devices.

Methods: We conducted a review of the economic concepts and implications of methods used for economic evaluations of the eleven most implanted medical devices from the Canadian perspective.

Results: We found Canadian economic studies for five of the eleven medical devices and identified nineteen Canadian studies. Overall, the device costs were important components of total procedure cost, with an average ratio of 44.1 %. Observational estimates of the device costs were obtained from buyers or sellers in 13 of the 19 studies. Although most of the devices last more than 1 year, standard costing methods for capital equipment was never used. In addition, only eight studies included a sensitivity analysis for the device cost. None of the sensitivity analyses were based on actual price distributions.

Conclusions: Economic evaluations are potentially important for policy making, but although they are being conducted, there is no standardized approach for incorporating medical device prices in economic analyses. Our review provides suggestions for improvements in how the prices are incorporated for economic evaluations of medical devices.

Objectives: The aim of this study was to explore whether wealth effects of health interventions, including productivity gains and savings in other sectors, are considered in resource allocations by health technology assessment (HTA) agencies and government departments. To analyze reasons for including, or not including, wealth effects.

Methods: Semi-structured interviews with decision makers and academic experts in eight countries (Australia, France, Germany, Italy, Poland, South Korea, Sweden, and the United Kingdom).

Results: There is evidence suggesting that health interventions can produce economic gains for patients and national economies. However, we found that the link between health and wealth does not influence decision making in any country with the exception of Sweden. This is due to a combination of factors, including system fragmentation, methodological issues, and the economic recession forcing national governments to focus on short-term measures.

Conclusions: In countries with established HTA processes and methods allowing, in principle, the inclusion of wider effects in exceptional cases or secondary analyses, it might be possible to overcome the methodological and practical barriers and see a more systematic consideration of wealth effect in decision making. This would be consistent with principles of efficient priority setting. Barriers for the consideration of wealth effects in government decision making are more fundamental, due to an enduring separation of budgets within the public sector and current financial pressures. However, governments should consider all relevant effects from public investments, including healthcare, even when benefits can only be captured in the medium- and long-term. This will ensure that resources are allocated where they bring the best returns.

Many countries in the world have tried to examine the possible methods for import and logical use of health technologies to manage their budgets on one hand and to prevent the entry of uncertain, inefficient, and insecure technologies on the other hand (1). The “health technology assessment” (HTA) is one of the dominant methods in most developed countries (2). HTA is a multidisciplinary field which studies the medical, social, and ethical aspects, as well as economic outcomes of production, diffusion, and application of health technologies (3).