Claire Packer

Cross-national comparability of burden of disease estimates: the European Disability Weights Project

OBJECTIVE To investigate the sources of cross-national variation in disability-adjusted life-years (DALYs) in the European Disability Weights Project. METHODS Disability weights for 15 disease stages were derived empirically in five countries by means of a standardized procedure and the cross-national differences in visual analogue scale (VAS) scores were analysed. For each country the burden of dementia in women, used as an illustrative example, was estimated in DALYs. An analysis was performed of the relative effects of cross-national variations in demography, epidemiology and disability weights on DALY estimates. FINDINGS Cross-national comparison of VAS scores showed almost identical ranking orders. After standardization for population size and age structure of the populations, the DALY rates per 100000 women ranged from 1050 in France to 1404 in the Netherlands. Because of uncertainties in the epidemiological data, the extent to which these differences reflected true variation between countries was difficult to estimate. The use of European rather than country-specific disability weights did not lead to a significant change in the burden of disease estimates for dementia. CONCLUSIONS Sound epidemiological data are the first requirement for burden of disease estimation and relevant between-countries comparisons. DALY estimates for dementia were relatively insensitive to differences in disability weights between European countries.

International diffusion of new health technologies: A ten-country analysis of six health technologies

OBJECTIVES The objective of this study was to examine and explain the differential international diffusion of six health innovations. METHODS A retrospective diffusion study was undertaken of sildenafil, cyclooxygenase-II (COX II) inhibitors, beta interferon, verteporfin, deep brain stimulators, and drug-eluting coronary stents in ten countries-Australia, Canada, Denmark, France, The Netherlands, Norway, Spain, Sweden, Switzerland, and the United Kingdom. We plotted diffusion curves of daily defined doses per quarter, vials or implants per million population, and examined the association between diffusion and five key variables. RESULTS Canada, Switzerland, and Sweden are generally high users of new technologies; Spain, Denmark, and particularly the United Kingdom are low users. Almost all countries experienced rapid adoption of sildenafil with diffusion to a similar level; there was variable adoption and diffusion of COX II inhibitors, verteporfin, and interferon beta; drug-eluting stents penetrated the market in a similar way in all but one country; and two countries had very different adoption patterns for deep brain stimulators. Above average health spending and the presence of health technology assessment (HTA) or other guidance reports are consistently associated with increased diffusion. Early warning activity and a national coverage decision being taken are more likely to be associated with a reduced diffusion. CONCLUSIONS The significant differences in diffusion between different countries are not consistent with a neat evidence-based world. The tools available to policy makers to control diffusion (early warning systems, HTA, and a fourth hurdle) play some part in influencing diffusion but need close scrutiny of how successfully they operate.

Effective early warning systems for new and emerging health technologies: Developing an evaluation framework and an assessment of current systems

OBJECTIVES The aim of this study was to define an effective early warning system, to identify and rank the characteristics of an effective early warning system for emerging health technologies, and to evaluate current early warning systems against these characteristics. METHODS An iterative Delphi-type process with the thirteen members of the International Information Network on New and Changing Health Technologies (EuroScan). We synthesized key characteristics that network members had graded. Members were then asked whether these characteristics were present or fulfilled in their system. RESULTS The definition of an effective early warning system developed was the following: a system that identifies innovations in the field of health technology likely to have a significant impact; and disseminates information relevant to the needs of the customer which is timely, so as to enable appropriate decision making (such as resource allocation), facilitate appropriate adoption, and identify further research requirements. Five primary and eleven secondary components of effective early warning systems were identified. The five primary characteristics concerned relevance, independence, resourcing, a clear pathway for the outputs to reach decision makers, and defined customers. Although the primary characteristics were present or fulfilled to some extent in the majority of evaluated early warning systems, there was considerable variability in the presence of the secondary characteristics in the evaluated systems. CONCLUSIONS Our study provides a definition for an effective early warning system and a shared understanding of the important characteristics and components of such systems. This work should provide guidance to those setting up new early warning systems as well as for those managing and reviewing current systems.

Early identification and assessment of new and emerging health technologies : Actions, progress, and the future direction of an international collaboration-EuroScan

OBJECTIVES To report on a workshop, and subsequent discussions, that reviewed the achievements and progress of the EuroScan collaboration since its establishment in 1999 to share information on the methods and results of early identification and assessment of new and emerging health technologies; considered challenges to the collaboration; and discussed its possible future direction. METHODS A workshop was held in Stockholm in September 2006, with thirty-two participants from ten countries and representatives from EuroScan member agencies, policy makers involved in policy or decision making relating to new technologies, and invited external commentators from international HTA networks. The workshop used a mix of presentations, panel and audience discussions, and small group work to consider the achievements and challenges put forward. RESULTS EuroScan has developed as a sustainable network, and has made progress on all tasks in its initial action plan, with the EuroScan information sharing database on new and emerging technologies being one of the collaborations key achievements. Identified immediate concerns for the network included consideration of the impact of its current name and membership model; acknowledgement and publication of the full range of benefits of membership; contribution to and development of the database to encourage increased information sharing; and EuroScans ongoing interaction with the wider HTA world. CONCLUSIONS The workshop was a useful mechanism for reviewing the work of EuroScan and for creating a platform to take the collaboration forward. The workshop affirmed the benefits of the network to individual members; posed some significant challenges to the network to consider; and acted as a stimulus for an interim name change to better represent the global membership, and a major review of the EuroScan database of identified and assessed emerging health technologies.

Bringing regenerative medicines to the clinic: the future for regulation and reimbursement.

Significant investments in regenerative medicine necessitate discussion to align evidentiary requirements and decision-making considerations from regulatory, health system payer and developer perspectives. Only with coordinated efforts will the potential of regenerative medicine be realized. We report on discussions from two workshops sponsored by NICE, University of Alberta, Cell Therapy Catapult and Centre for Commercialization of Regenerative Medicine. We discuss methods to support the assessment of value for regenerative medicine products and services and the synergies that exist between market authorization and reimbursement regulations and practices. We discuss the convergence in novel adaptive licensing practices that may promote the development and adoption of novel therapeutics that meet the needs of healthcare payers.

Health technology diffusion rates. Statins, coronary stents, and MRI in England.

OBJECTIVE To analyze the rates and influences on the adoption of three selected health technologies: statins, coronary stents, and magnetic resonance imaging (MRI). METHODS A retrospective diffusion study using primary care prescribing data and questionnaire responses from acute hospital trusts in the West Midlands region (population 5.3 million or 10% of England). RESULTS The selected technologies had markedly different diffusion curves. Statins diffused rapidly soon after launch. Coronary stents were initially used 6 years after first availability, but within 2 years all responding hospitals reported using them. MRI scanners were initially purchased 6 years after first availability with a subsequently slow rate of diffusion, and are still absent from some hospitals. Influences on the adoption of each technology were different. Commercial marketing was reported as a major influence on the diffusion of statins but not at all on MRIs. Cost impact was a major negative influence on the diffusion of MRI scanners and statins, whereas enthusiastic individuals were key to the diffusion of stents. CONCLUSIONS Influences on adoption and consequent diffusion rates are very different for different health technologies. It is not at all clear that such diffusion patterns relate well to an optimum timing rate. This has important implications for technology gatekeepers in health care.

Assessing the accuracy of forecasting: Applying standard diagnostic assessment tools to a health technology early warning system

OBJECTIVES Early warning systems are an integral part of many health technology assessment programs. Despite this finding, to date, there have been no quantitative evaluations of the accuracy of predictions made by these systems. We report a study evaluating the accuracy of predictions made by the main United Kingdom early warning system. METHODS As prediction of impact is analogous to diagnosis, a method normally applied to determine the accuracy of diagnostic tests was used. The sensitivity, specificity, and predictive values of the National Horizon Scanning Centres prediction methods were estimated with reference to an (imperfect) gold standard, that is, expert opinion of impact 3 to 5 years after prediction. RESULTS The sensitivity of predictions was 71 percent (95 percent confidence interval [CI], 0.36-0.92), and the specificity was 73 percent (95 percent CI, 0.64-0.8). The negative predictive value was 98 percent (95 percent CI, 0.92-0.99), and the positive predictive value was 14 percent (95 percent CI, 0.06-0.3). CONCLUSIONS Forecasting is difficult, but the results suggest that this early warning systems predictions have an acceptable level of accuracy. However, there are caveats. The first is that early warning systems may themselves reduce the impact of a technology, as helping to control adoption and diffusion is their main purpose. The second is that the use of an imperfect gold standard may bias the results. As early warning systems are viewed as an increasingly important component of health technology assessment and decision making, their outcomes must be evaluated. The method used here should be investigated further and the accuracy of other early warning systems explored.

Analyzing 10 years of early awareness and alert activity in the United kingdom.

OBJECTIVES The aim of this study was to assess the accuracy of the English National Horizon Scanning Centre (NHSC) in identifying and filtering pharmaceutical developments using end user and international collaborator databases of emerging technologies as proxies for new drugs of likely significance to health services and/or patients. METHODS We used the NHSC information system and the list of National Institute for Health and Clinical Excellence (NICE) technology appraisals to estimate the false positive rate for NHSC identification, filtration, and reporting. We assessed the sensitivity of NHSC identification and filtration of pharmaceuticals for NICE technology appraisals from 1999 to the end of December 2010, and for pharmaceuticals entered into the EuroScan International Network database. RESULTS We estimate that overall NHSC identification, filtration and reporting had a positive predictive value of 0.39 (95 percent CI, 0.36 to 0.43) and a false positive rate of 60 percent. Using NICE appraisals and EuroScans database as proxies for pharmaceuticals of significance, we estimate the NHSC sensitivity over the 10-year period at 0.92 (95 percent CI, 0.89 to 0.95) and 0.89 (95 percent CI, 0.82 to 0.96) respectively. CONCLUSIONS Our results suggest that the NHSC has performed well in terms of sensitivity over the past decade, but that the false positive rate of 60 percent may indicate that the filtration criteria for pharmaceuticals could be tightened for increased efficiency. Future evaluations of EAA systems should include an element of external review and explore the level of accuracy acceptable to funders and customers of such systems.

Prioritisation criteria for the selection of new diagnostic technologies for evaluation

BackgroundCurrently there is no framework for those involved in the identification, evaluation and prioritisation of new diagnostic technologies. Therefore we aimed to develop prioritisation criteria for the assessment of new diagnostic technologies, by gaining international consensus on not only which criteria should be used, but also their relative importance.MethodsA two-round Delphi process was used to generate consensus amongst an international panel of twenty-six experts on priority criteria for diagnostic health technology assessment. Participants represented a range of health care and related professions, including government, industry, health services and academia.ResultsBased on the responses to the first questionnaire 18 criteria were placed into three categories: high, intermediate and moderate priority. For 16 of the 18 criteria, agreement with the categorisation of the criteria into the high, intermediate and moderate categories was high at ≥ 70% (10 had agreement ≥ 80%). A further questionnaire and panel discussion reduced the criteria to 16 and two categories; seven were classified as high priority and nine intermediate.ConclusionsThis study proposes an objective structure of prioritisation criteria to use when assessing new diagnostic technologies, based on an expert consensus process. The value of these criteria is that no one single component should be used as the decisive driver for prioritisation of new diagnostic technologies for adoption in healthcare settings. Future studies should be directed at establishing the value of these prioritisation criteria across a range of healthcare settings.

The future role of genetic screening to detect newborns at risk of childhood-onset hearing loss

Abstract Objective: To explore the future potential of genetic screening to detect newborns at risk of childhood-onset hearing loss. Design: An expert led discussion of current and future developments in genetic technology and the knowledge base of genetic hearing loss to determine the viability of genetic screening and the implications for screening policy. Results and Discussion: Despite increasing pressure to adopt genetic technologies, a major barrier for genetic screening in hearing loss is the uncertain clinical significance of the identified mutations and their interactions. Only when a reliable estimate of the future risk of hearing loss can be made at a reasonable cost, will genetic screening become viable. Given the speed of technological advancement this may be within the next 10 years. Decision-makers should start to consider how genetic screening could augment current screening programmes as well as the associated data processing and storage requirements. Conclusion: In the interim, we suggest that decision makers consider the benefits of (1) genetically testing all newborns and children with hearing loss, to determine aetiology and to increase knowledge of the genetic causes of hearing loss, and (2) consider screening pregnant women for the m.1555A> G mutation to reduce the risk of aminoglycoside antibiotic-associated hearing loss.