Pinchon-Riviere and colleagues have made an extremely timely contribution that addresses the thorny issues of innovative, high-cost medicines, global health disparities, and the growing interest and application of HTA in low- and middle-income countries.

Objectives: Differential pricing, based on countries’ purchasing power, is recommended by the World Health Organization to secure affordable medicines. However, in developing countries innovative drugs often have similar or even higher prices than in high-income countries. We evaluated the potential implications of trastuzumab global pricing policies in terms of cost-effectiveness (CE), coverage, and accessibility for patients with breast cancer in Latin America (LA).

Methods: A Markov model was designed to estimate life-years (LYs), quality-adjusted life-years (QALYs), and costs from a healthcare perspective. To better fit local cancer prognosis, a base case scenario using transition probabilities from clinical trials was complemented with two alternative scenarios with transition probabilities adjusted to reflect breast cancer epidemiology in each country.

Results: Incremental discounted benefits ranged from 0.87 to 1.00 LY and 0.51 to 0.60 QALY and incremental CE ratios from USD 42,104 to USD 110,283 per QALY (2012 U.S. dollars), equivalent to 3.6 gross domestic product per capita (GDPPC) per QALY in Uruguay and to 35.5 GDPPC in Bolivia. Probabilistic sensitivity analysis showed 0 percent probability that trastuzumab is CE if the willingness-to-pay threshold is one GDPPC per QALY, and remained so at three GDPPC threshold except for Chile and Uruguay (4.3 percent and 26.6 percent, respectively). Trastuzumab price would need to decrease between 69.6 percent to 94.9 percent to became CE in LA.

Conclusions: Although CE in other settings, trastuzumab was not CE in LA. The use of health technology assessment to prioritize resource allocation and support price negotiations is critical to making innovative drugs available and affordable in developing countries.

Objectives: Local health technology assessment (HTA) to determine whether new health technologies should be adopted is now a common practice in many healthcare organizations worldwide. However, little is known about hospital-based HTA activities in France. The objective of this study was to explore hospital-based HTA activities in French university hospitals and to provide a picture of organizational approaches to the assessment of new and innovative medical devices.

Methods: Eighteen semi-structured interviews with hospital pharmacists were conducted from October 2012 to April 2013. Six topics were discussed in depth: (i) the nature of the institution concerned; (ii) activities relating to innovative medical devices; (iii) the technology assessment and decision-making process; (iv) the methodology for technology assessment; (v) factors likely to influence decisions and (vi) suggestions for improving the current process. The interview data were coded, collated and analyzed statistically.

Results: Three major types of hospital-based HTA processes were identified: medical device committees, innovation committees, and “pharmacy & management” processes. HTA units had been set up to support medical device and innovation committees for technology assessment. Slow decision making was the main limitation to both these committee-based approaches. As an alternative, “pharmacy & management” processes emerged as a means of rapidly obtaining a formal assessment.

Conclusions: This study provides an overview of hospital-based HTA initiatives in France. We hope that it will help to promote hospital-based HTA activities in France and discussions about ways to improve and harmonize practices, through the development of national guidelines and/or a French mini-HTA tool, for example.

Objectives: The Carpentier-Edwards pericardial (CEP) prostheses are the type of bioprostheses most used worldwide. Although they were designed to minimize the rate of valve deterioration and reoperation, their clinical superiority over other prostheses models still lacks confirmation. The objective of this study was to evaluate its effectiveness.

Methods: We performed a systematic review and meta-analysis in the PubMed, Embase, Cochrane, and Lilacs databases. Operative mortality, overall mortality and reoperation rates after heart valve surgery were compared between the use of CEP and other cardiac prostheses. Two independent reviewers screened studies for inclusion and extracted the data. Disagreements were resolved by consensus. The GRADE criterion was used to assess the evidence quality.

Results: A total of twenty-eight studies were selected, including 19,615 individuals. The studies presented a high heterogeneity and low quality of evidence what limited the reliability of the results. The pooled data from the selected studies did not demonstrate significant differences between CEP and porcine, pericardial or stentless prostheses regarding operative mortality, overall mortality and reoperation rates. However, the pooled data from 3 observational trials pointed out a higher risk for reoperation after valve replacement using CEP prostheses against mechanical prostheses (OR 4.92 [95 percent confidence interval 2.43–9.96]).

Conclusions: The current data present in the literature still does not support a clinical advantage for the use of CEP prostheses over other bioprostheses. The quality of the studies in the literature is limited and further studies are needed to address if CEP prostheses will have a clinical advantage over other prostheses.

Objectives: Differences in the process of using liver imaging technologies might be important to patients. This study aimed to investigate preferences for scanning modalities used in diagnosing focal liver lesions.

Methods: A discrete choice experiment was administered to 504 adults aged ≥25 years. Respondents made repeated choices between two hypothetical scans, described according to waiting time for scan and results, procedure type, the chance of minor side-effects, and whether further scanning procedures were likely to be required. Choice data were analyzed using mixed-logit models with respondent characteristics used to explain preference heterogeneity.

Results: Respondents preferred shorter waiting times, the procedure to be undertaken with a handheld scanner on a couch instead of within a body scanner, no side-effects, and no follow‑up scans (p ≤ .01). The average respondent was willing to wait an additional 2 weeks for the scan if it resulted in avoiding side-effects, 1.5 weeks to avoid further procedures or to be told the results immediately, and 1 week to have the scan performed on a couch with a handheld scanner. However, substantial heterogeneity was observed in the strength of preference for desirable imaging characteristics.

Conclusions: An average individual belonging to a general population sub‑group most likely to require imaging to characterize focal liver lesions in the United Kingdom would prefer contrast‑enhanced ultrasound over magnetic resonance imaging or computed tomography. Insights into the patient perspective around differential characteristics of imaging modalities have the potential to be used to guide recommendations around the use of these technologies.

Objectives: There have been multiple calls for explicit integration of ethical, legal, and social issues (ELSI) in health technology assessment (HTA) and addressing ELSI has been highlighted as key in optimizing benefits in the Omics/Personalized Medicine field. This study examines HTAs of an early clinical example of Personalized Medicine (gene expression profile tests [GEP] for breast cancer prognosis) aiming to: (i) identify ELSI; (ii) assess whether ELSIs are implicitly or explicitly addressed; and (iii) report methodology used for ELSI integration.

Methods: A systematic search for HTAs (January 2004 to September 2012), followed by descriptive and qualitative content analysis.

Results: Seventeen HTAs for GEP were retrieved. Only three (18%) explicitly presented ELSI, and only one reported methodology. However, all of the HTAs included implicit ELSI. Eight themes of implicit and explicit ELSI were identified. “Classical” ELSI including privacy, informed consent, and concerns about limited patient/clinician genetic literacy were always presented explicitly. Some ELSI, including the need to understand how individual patients’ risk tolerances affect clinical decision-making after reception of GEP results, were presented both explicitly and implicitly in HTAs. Others, such as concern about evidentiary deficiencies for clinical utility of GEP tests, occurred only implicitly.

Conclusions: Despite a wide variety of important ELSI raised, these were rarely explicitly addressed in HTAs. Explicit treatment would increase their accessibility to decision-makers, and may augment HTA efficiency maximizing their utility. This is particularly important where complex Personalized Medicine applications are rapidly expanding choices for patients, clinicians and healthcare systems.

Objectives: Developing a search strategy for use in a systematic review is a time-consuming process requiring construction of detailed search strings using complicated syntax, followed by iterative fine-tuning and trial-and-error testing of these strings in online biomedical search engines.

Methods: Building upon limitations of existing online-only search builders, a user-friendly computer-based tool was created to expedite search strategy development as part of production of a systematic review.

Results: Search Builder 1.0 is a Microsoft Excel®-based tool that automatically assembles search strategy text strings for PubMed (www.pubmed.com) and Embase (www.embase.com), based on a list of user-defined search terms and preferences. With the click of a button, Search Builder 1.0 automatically populates the syntax needed for functional search strings, and copies the string to the clipboard for pasting into Pubmed or Embase. The offline file-based interface of Search Builder 1.0 also allows for searches to be easily shared and saved for future reference.

Conclusions: This novel, user-friendly tool can save considerable time and streamline a cumbersome step in the systematic review process.

Objectives: A rapid scoping review was performed to support the development of a new clinical technology platform. An iterative sifting approach was adopted to address the challenges posed by the nature of the review question and the extremely large volume of search results to be sifted within the timescales of the review.

Methods: This study describes the iterative sifting approach applied in the scoping review and a preliminary validation of the methods applied.

Results: The searches performed for the rapid scoping review retrieved 27,198 records. This was the full set of records subjected to the staged, iterative sifting approach and the subsequent validation process. The iterative sifting approach involved the screening for relevance of 17,354 (i.e., 63.8 percent) of the 27,198 records. A list of fifty-three potential biomarker names was generated as a result of this iterative sifting method, of which nineteen were selected by clinical specialists for further scrutiny. The preliminary validation involved the exhaustive sifting of the remaining 9,844 previously unsifted records. The validation process identified sixteen additional potential biomarker names not identified by the iterative sifting process. The clinical specialists subsequently concluded that none were of further clinical interest.

Conclusions: This study describes an approach to the screening of search records that can be successfully applied in appropriate review and decision problems to allow the prioritization of the most relevant search records and achieve time savings. Following further refinement and standardization, this iterative sifting method may have potential for further applications in reviews and other decision problems.

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 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 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: 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: Our objective was to gather perspectives from payers on how comparative effectiveness research (CER) in the United States and relative effectiveness (RE) research in Europe will impact evidentiary standards for access decisions of new drugs by 2020.

Methods: We conducted semi-structured interviews with fourteen senior officials representing public and private payers, health technology assessment groups, and pricing and reimbursement bodies in the United States and Europe. An online survey assessed current use of CER/RE evidence and potential trends that might influence its use for decision making by 2020. A semi-structured interview elicited payers' definitions of CER/RE and was structured around four hypothetical cases resembling drugs expected to be more common or poised to create policy challenges by 2020. Topics included acceptance of study designs and analytic methods associated with CER/RE. A systematic content review was done to extract relevant information.

Results: According to key informants, randomization will remain an essential component for assessing comparative or relative effectiveness. They anticipate greater use of policy levers such as conditional reimbursement or prior authorization to manage diffusion of new drugs. Case studies provided important insights into situations when certain types of CER evidence may be acceptable (e.g., observational data when differences between drugs are largely convenience).

Conclusions: Industry perceptions that CER/RE will change payers' evidentiary requirements in the future are consistent with our findings. Growing investment in payers' own data and increased reliance on policy tools to control diffusion of new drugs may also influence the type of evidence industry will be required to produce by 2020.

Objectives: Clinical research data are often collected on paper and later inputted onto an electronic database. This method is time consuming and potentially introduces errors. Therefore, to make primary data collection more efficient and less error prone we aimed to develop a touch-screen application for data collection in a psoriatic arthritis research clinic and compared it with the pre-existing paper-based system.

Methods: We developed a Web application using Java and optimized it for the iPad®. It highlights missing fields for physicians in real time, and only permits submission of data collection form after corrections are made. For its evaluation, seven physicians participated, and before each patient visit they were randomly assigned paper or iPad® data entry. Number of errors, length of visit, and time between clinic visit and completion of data entry were measured.

Results: A total of 106 patients seen in the clinic who agreed to participate were randomly assigned to be evaluated by clinic physicians using the iPad® (fifty-three patients) or a paper protocol (fifty-three patients). On average, 3.34 omissions were found per paper form, of which 2.24 would have been detected on the iPad®. The iPad® increased the mean patient encounter time from 37.2 minutes to 46.5 minutes, but eliminated delay between a clinic visit and its data entry.

Conclusions: Entering data using the iPad® application makes the patient encounter slightly longer, but reduces “missing fields.” It also eliminates the delay between clinic visit and data entry thus improving the efficiency of clinical data capture in a research setting.

Objectives: To describe the first hospital-based health technology assessment (HTA) program in a public hospital in Argentina, and report some clinical, educational, economic and organizational results after 10 years of its implementation.

Methods: A hospital-based HTA program was created in March 2001 at Hospital Garrahan (Buenos Aires, Argentina), a national pediatric facility with a self-managed budget. Its main goal is to promote a rational and evidence-based technologic development. The program consists of HTA reports for technology acquisition, clinical practice guidelines (CPG), capacity building in research and management, and technical support for health services research (HSR). The evaluation cycle comprises: prioritization, evidence synthesis, dissemination and monitoring. We report program performance, comment educational and organizational effects, and discuss unresolved issues and future challenges.

Results: During the first 10 years the program produced 18 HTA reports on drugs (6 = 33 percent), therapeutic (6 = 33 percent), preventive (2 = 11 percent) or diagnostic (2 = 11 percent) procedures and institutional programs (3 = 17 percent). The scope covered effectiveness (12 = 67 percent), safety (10 = 56 percent), budget impact (6 = 33 percent), cost-effectiveness (2 = 11 percent) and organizational impact (3 = 17 percent). Mean time from request to report was 10 months. Eleven pediatric CPGs were submitted to expert consensus and disseminated for full-text Web access. A 1-year course on research and management was completed by 225 professionals in 6 years, and twenty-two projects for HSR were coached.

Conclusions: Our experience shows that an HTA program is both feasible and useful in a public hospital of a developing country. Promotion of hospital-based HTA, professional integration in HTA activities and network collaboration to discuss unresolved issues with colleagues can multiply the benefits and optimize the use of hospital budgets.