Health Technology Assessment Group Research Articles

Considerations for US coverage and reimbursement: Which digital health products to evaluate? Findings from a modified Delphi study.

The rapid growth of digital health tools, including digital applications, wearables, sensors, diagnostics, digital therapeutics (DTx), and prescription DTx, offers new ways to treat patients and close gaps in care. Payers need transparent, credible, and efficient processes to differentiate products for potential reimbursement from the larger universe of digital health products. To identify areas of agreement, disagreement, and rationale for payers to determine which digital health products should be evaluated for formulary consideration and to develop generalizable criteria for health care decision-makers developing policies and approaches for digital health products. Experts from the Academy of Managed Care Pharmacy DTx Advisory Group Payer Evaluation subcommittee rated whether a pharmacy and therapeutics committee, health technology assessment group, or an innovation center within a health plan or pharmacy benefit manager should consider 14 hypothetical products for potential formulary coverage. Using a 4-step modified Delphi approach, experts rated whether it was appropriate for a payer to evaluate each product on a scale of 1 (strongly disagree) to 9 (strongly agree). Quantitative agreement was assessed using terciles of responses, medians, and the distribution of appropriateness scores. The corresponding discussions are summarized to identify generalizable criteria for payers to consider as they develop approaches to determine which digital health products to evaluate. Among the 14 hypothetical products, 4 achieved quantitative agreement that payers should evaluate the product. 5 products had quantitative disagreement, and the remaining were indeterminant. Payers were most likely to review a product if it (1) was reviewed by the US Food and Drug Administration, (2) required a prescription, (3) was intended to be paid for using premium dollars, (4) treated rather than diagnosed or monitored a clinical condition, (5) had a low clinical opportunity cost, and (6) could address population health metrics. The rapid availability of digital health and DTx options can be daunting for health care decision-makers when determining which products to evaluate. These generalizable criteria can help payers develop a more efficient process.

PP27 The Private Healthcare Responses Against COVID-19 Emergencies In Brazil

IntroductionThe Corona Virus Disease 2019 (COVID-19) pandemic is the biggest public health crisis of all time. Private health care plays a major role in health globally. We conducted a study to document the engagement of a Brazilian private health care organization to influence decisions to private and public health emergencies of COVID-19.MethodsThis retrospective study evaluated the outputs of the health technology assessment (HTA) group of Unimed-BH, a private Health Maintenance Organization (HMO) with 1.5 million participants in Belo Horizonte, Minas Gerais state. The study evaluated the impacts on the overview of health local decisions in the municipality and the national supplementary health agency (ANS) during the period from March 2020 to December 2022.ResultsDuring the pandemic, Unimed-BH made all its sanitary decisions based on scientific research, such as the use of masks and appropriate medications. Even though some medicines, such as regdanvimab, were authorized for emergency use by the Brazilian Health Regulatory Agency (ANVISA), Unimed-BH did not recommend their use due to uncertain evidence, and months later, ANVISA withdrew the registration.Unimed-BH also conducted a systematic review of ivermectin for COVID-19 treatment, which showed no effect, and therefore did not recommend its use. Additionally, Unimed-BH provided weekly updates on COVID-19 data, including suspected and confirmed cases, hospitalizations, and deaths in their customer portfolio. The organization also actively supported the decisions made by ANS and municipal managers using evidence and statistics on the pandemic. The Unimed-BH HTA group produced a total of 167 reports from March 2020 to December 2022.ConclusionsBelo Horizonte had the lowest in-hospital mortality rates with COVID-19 in Brazil. Unimed-BH’s HTA reports provided evidence-based assessments for decision-making, proposed partnership with policymakers, fomented information transparency, and strict follow-up on pandemic numbers, which may have contributed to the lower fatality rate in our city. These findings underscore the importance of private healthcare organizations in responding to COVID-19 emergencies, and their potential to support evidence-based decision-making and minimize the impact of the pandemic.

Economic evaluations of mental health interventions in criminal justice.

Mental health interventions targeting crime perpetrators are available. An overview of the current scenario of their economic benefits will help policy decisions. To provide an update on economic evidence for mental health interventions in criminal justice, and to identify challenges and responses in using economic evidence to inform policy. Narrative review with an analysis frame that organises evidence around four points on the criminal justice system pathway: (a) point of contact; (b) post-arrest; (c) incarceration/punishment and (d) post-incarceration. There is a paucity of high-quality economic evidence, especially from cost-benefit analyses. However, there is some evidence of cost-effectiveness in support of interventions at the point of incarceration, such as cognitive behavioural therapy, multisystemic therapy for juvenile delinquents, therapeutic communities, electronic monitoring and telepsychiatry in forensic psychiatry settings. There is also evidence that post-incarceration interventions such as assertive community treatment can be cost-effective. There remain large evidence gaps. There are also challenges in turning economic evidence on mental health interventions in criminal justice into policy changes and improved practice, such as hidden costs, silo budgeting and delayed pay-off. Research incorporating multi-sectoral costs and benefits recommended by health economics and health technology assessment groups should be prioritised to support difficult resource allocation decisions faced by policy makers.

Abstract 5963: A framework for extrapolating evidence for molecularly targeted therapies from common to rare cancers - bridging the gap

Abstract BACKGROUND: Advances in understanding of potentially targetable molecular pathways driving carcinogenesis coupled with rapid development of tumor profiling technologies are partitioning cancers into rare biomarker-defined diseases. Assessing biomarker-targeted therapies within these small populations in adequately powered randomized trials is becoming unfeasible, leading to reliance on lower quality evidence from uncontrolled studies using unvalidated surrogate outcomes. When robust evidence for a targeted therapy being considered already exists in common cancers, this evidence may potentially be extrapolated to support the use of this therapy in rare cancers sharing the same biomarker and reduce additional evidence requirements for regulatory or reimbursement approval. However, extrapolation may not be appropriate in some settings due to biological differences between cancer types. METHODS: Building on recommendations for core components of extrapolation identified from a scoping review of methodological guidance, we constructed a framework proposing criteria for extrapolating evidence for targeted therapies from common to rare cancers. RESULTS: Criteria reflect key assumptions of disease similarity when defined by the biomarker and similarity of treatment response. Criteria are judged under five extrapolation components: (1) Analytical validity of the test used to identify the biomarker and criteria used to define biomarker status in the rare cancer, (2) Strength of evidence supporting biomarker actionability in the rare cancer and evidence that actionability may differ from the common cancer, (3) Quantitative estimation of the natural history of the biomarker-defined rare cancer as control group, (4) Validity of surrogate endpoints used to extrapolate treatment effect from the common cancer and predictions of clinical benefit in the rare cancer, and (5) Similarity of the safety profile between cancers and methods to augment safety data in the rare cancer. Using these criteria, decision-makers can judge whether sufficient evidence exists to support extrapolation or identify specific knowledge gaps to better target further research to be able to judge whether extrapolation is appropriate or not. Residual uncertainties for fulfilling criteria can also define post-approval commitments. CONCLUSIONS: We outline a pragmatic and systematic approach for selecting and evaluating existing evidence to judge when extrapolation is appropriate. This framework can be used to promote standardized, comprehensive, and transparent decision-making and facilitate discussion between stakeholders in drug development and clinical guideline and health technology assessment groups. Citation Format: Doah Cho, Sarah J. Lord, John R. Simes, Wendy Cooper, Saskia Cheyne, Chee Khoon Lee. A framework for extrapolating evidence for molecularly targeted therapies from common to rare cancers - bridging the gap [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2022; 2022 Apr 8-13. Philadelphia (PA): AACR; Cancer Res 2022;82(12_Suppl):Abstract nr 5963.

Variation in market access decisions for cell and gene therapies across the United States, Canada, and Europe

Transformative cell and gene therapies have now launched worldwide, and many potentially curative cell and gene therapies are in development, offering the prospect of significant health gains for patients. Access to these therapies depend on decisions made by health technology assessment (HTA) and payer organizations. We sought to describe the emerging cell and gene therapies market access landscape by analyzing 17 US commercial payer medical policies, and HTA reports from five European countries and Canada. We found that some US health plans applied coverage restrictions more often than others (four plans applied restrictions in all decisions, while four plans applied restrictions in <30% of decisions). The European and Canadian HTA bodies recommend access to fewer therapies than US health plans, reflecting a more stringent approach in the context of limited evidence and high scientific uncertainty that is commonly associated with these treatments. Our findings suggest that patient access to approved cell and gene therapies is restricted in all regions studied, though the nature of these restrictions differs between US health plans and the European/Canada HTA recommendations. Payers, HTA groups, pharmaceutical companies, and other stakeholders should collaborate to more clearly define the “uncertainties” and develop market access policies that balance benefits of early access with ongoing data collection to close evidence gaps over time.

OP93 Collaboration Between Health Technology Assessment And Procurement: A Rapid Mixed-Methods Study

IntroductionThe Irish Health Service (HSE) Health Technology Assessment Group (HTAG) aims to maximise the impact of its work by collaborating with HSE Procurement, formalised through an evidence-based Memorandum of Understanding (MOU). This study aims to inform the MOU.MethodsA sequential mixed-methods study design was used. A rapid review of the literature identified no substantive body of evidence on collaboration between independent national health technology assessment (HTA) and procurement bodies. Personnel involved in HTA or procurement were invited by email to complete a survey, take part in an interview, or both. The quantitative and qualitative data were analysed using descriptive statistics and thematic analysis, respectively. Findings were integrated using a conceptual framework that examined the complementarity of HTA and procurement processes relevant to an MOU.ResultsThirteen surveys were completed (response rate was 13 percent). Eleven interviews (five Ireland, two Canada, three UK, one New Zealand) were conducted between August and November, 2017. No formalised collaboration between independent national HTA and procurement bodies was identified. However in New Zealand, HTA and procurement are an integrated function of the Pharmaceutical Management Agency (PHARMAC). In other jurisdictions, successful ad hoc collaborations occurred where there was a clear need expressed by Procurement for additional evidence required for decision-making, and where HTA personnel tailored their research approaches accordingly. Key themes to successful collaboration were relationships, communication, clear roles, rigorous research and ‘system support’. Good individual relationships and ready access/communication promoted successful outcomes. Successful outcomes included improved clinical practice, and major cost savings. Collaboration may be focussed on: innovative or established devices; specific types of HTA/research products; specific categories/specialties; or specific procurement departments.ConclusionsAll participants considered collaboration to be beneficial but requiring good relationships and ‘system support’. Furthermore, successful collaboration requires clarity regarding the purpose, parties involved, their roles, responsibilities, modes of communication, information to be shared, and the expected outcomes.

OP91 Developing A Celtic Connections Regional Health Technology Assessment Alliance

IntroductionThe Irish, Scottish and Welsh national Health Technology Assessment (HTA) bodies (Health Information and Quality Authority, Health Technology Assessment Group, Scottish Health Technologies Group, Health Technology Wales) have recently (2018) established a ‘Celtic connections’ regional HTA alliance on non-medicine technologies. The primary purpose is to add value by realizing potential economies of scale and scope in non-medicine HTA efforts.MethodsA Memorandum of Understanding (MoU) was agreed to: formalize collaboration and partnership working; improve shared understanding of work programs and processes; collaborate on and co-produce evidence reviews of mutual interest; increase both the volume and range of technology topics for which advice is developed in each nation; promote knowledge exchange; and enhance professional and personal development for each agency's staff.ResultsEarly benefits include: collaboration on one technology topic resulting in the production of bespoke guidance in three countries; an update of a partner's rapid review; identification of a further potential topic collaboration (sacral nerve stimulation); a six month senior staff secondment; and reciprocal observer membership on each country's national committees. Other general benefits have included: reduced duplication of effort; improved quality assurance through ‘critical friend’ peer review; enhanced access to methodological advice and a broader range of stakeholders; and development of a forum for discussion and peer support.ConclusionsThe alliance offers real potential to optimize use of the scarce resources for non-medicine technologies across the three countries and increase evidence review and guidance volume through adapting or co-producing outputs. Longer term benefits are anticipated to include: improved knowledge exchange; advancing skills of staff; building and broadening capacity through shared learning and access to a wider professional peer group; improved staff recruitment and retention; production of joint publications and other modes of dissemination; and increased profile for each country's work.

INTERVAL (investigation of NICE technologies for enabling risk-variable-adjusted-length) dental recalls trial: a multicentre randomised controlled trial investigating the best dental recall interval for optimum, cost-effective maintenance of oral health in dentate adults attending dental primary care

BackgroundTraditionally, patients at low risk and high risk of developing dental disease have been encouraged to attend dental recall appointments at regular intervals of six months between appointments. The lack of evidence for the effect that different recall intervals between dental check-ups have on patient outcomes, provider workload and healthcare costs is causing considerable uncertainty for the profession and patients, despite the publication of the NICE Guideline on dental recall. The need for primary research has been highlighted in the Health Technology Assessment Group’s systematic review of routine dental check-ups, which found little evidence to support or refute the practice of encouraging 6-monthly dental check-ups in adults. The more recent Cochrane review on recall interval concluded there was insufficient evidence to draw any conclusions regarding the potential beneficial or harmful effects of altering the recall interval between dental check-ups. There is therefore an urgent need to assess the relative effectiveness and cost-benefit of different dental recall intervals in a robust, sufficiently powered randomised control trial (RCT) in primary dental care.MethodsThis is a four year multi-centre, parallel-group, randomised controlled trial with blinded outcome assessment based in dental primary care in the UK. Practitioners will recruit 2372 dentate adult patients. Patient participants will be randomised to one of three groups: fixed-period six month recall, risk-based recall, or fixed-period twenty-four month recall. Outcome data will be assessed through clinical examination, patient questionnaires and NHS databases. The primary outcomes measure gingival inflammation/bleeding on probing and oral health-related quality of life.DiscussionINTERVAL will provide evidence for the most clinically-effective and cost-beneficial recall interval for maintaining optimum oral health in dentate adults attending general dental practice.Trial registrationISRCTN95933794 (Date assigned 20/08/2008).

Critical appraisal of nonrandomized studies-A review of recommended and commonly used tools.

When randomized controlled trial data are limited or unavailable, or to supplement randomized controlled trial evidence, health technology assessment (HTA) agencies may rely on systematic reviews of nonrandomized studies (NRSs) for evidence of the effectiveness of health care interventions. NRS designs may introduce considerable bias into systematic reviews, and several methodologies by which to evaluate this risk of bias are available. This study aimed to identify tools commonly used to assess bias in NRS and determine those recommended by HTA bodies. Appraisal tools used in NRS were identified through a targeted search of systematic reviews (January 2013-March 2017; MEDLINE and EMBASE [OVID SP]). Recommendations for the critical appraisal of NRS by expert review groups and HTA bodies were reviewed. From the 686 studies included in the narrative synthesis, 48 critical appraisal tools were identified. Commonly used tools included the Newcastle-Ottawa Scale, the methodological index for NRS, and bespoke appraisal tools. Neither the Cochrane Handbook nor the Centre for Reviews and Dissemination recommends a particular instrument for the assessment of risk of bias in NRS, although Cochrane has recently developed their own NRS critical appraisal tool. Among HTA bodies, only the Canadian Agency for Drugs and Technologies in Health recommends use of a specific critical appraisal tool-SIGN 50 (for cohort or case-control studies). Several criteria including reporting, external validity, confounding, and power were examined. There is no consensus between HTA groups on the preferred appraisal tool. Reviewers should select from a suite of tools on the basis of the design of studies included in their review.

Medicines Adaptive Pathways to Patients (MAPPs): A Story of International Collaboration Leading to Implementation.

After nearly a decade of discussion, analysis, and development, the Medicines Adaptive Pathways to Patients (MAPPs) initiative is beginning to see acceptance from regulators, industry, patients, and payers, with the first live pilot project initiated under the guidance of the European Medicines Agency in 2014. Although it is a significant achievement to see the first asset being placed into human trials under an adaptive pathway, there is much to be learned regarding the multinational and multi-stakeholder effort that has driven the growing acceptance of MAPPs as a methodology and concept, as well as the need for continued and increasing international collaboration to foster the wider adoption of MAPPs. Changes in available science and technology, as well as a number of challenges in the current system, outlined in this paper, are transforming approaches to medicines development and approval. It is these challenges that have led directly to the groundbreaking MAPPs collaboration between the Massachusetts Institute of Technology Center for Biomedical Innovation's New Drug Development Paradigms Initiative, the EMA, patient, payer and health technology assessment groups, the European Federation of Pharmaceutical Industries and Associations, and the Innovative Medicines Initiative-a European public-private partnership. This article examines the development of MAPPs, from inception of the concept, to the establishment of this trans-Atlantic initiative, and examines challenges for the future.

PAYER PERSPECTIVES ON FUTURE ACCEPTABILITY OF COMPARATIVE EFFECTIVENESS AND RELATIVE EFFECTIVENESS RESEARCH.

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. 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. 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). 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.

Pazopanib for the First-Line Treatment of Patients with Advanced and/or Metastatic Renal Cell Carcinoma

The National Institute for Health and Clinical Excellence (NICE) invited the manufacturer of pazopanib hydrochloride (GlaxoSmithKline) to submit evidence of the clinical and cost effectiveness of the drug for the first-line treatment of advanced and/or metastatic renal cell carcinoma, as part of the Institute's single technology appraisal (STA) process. The Aberdeen Health Technology Assessment Group were commissioned to act as the Evidence Review Group (ERG). This article provides a description of the company submission, the ERG review and NICE's subsequent decisions. The objective of this paper is to summarize the independent review and critique of the evidence submitted for the consideration of the NICE Appraisal Committee and NICE's subsequently issued guidance. The ERG produced a critical review of the evidence for the clinical and cost effectiveness of the technology based upon the manufacturer's submission to NICE. The ERG also independently searched for relevant evidence and modified the manufacturer's decision analytic model to examine the impact of altering some of the key assumptions. For progression-free survival (PFS), there was a statistically significant longer survival for pazopanib compared with placebo (as assessed by the ERG, based upon the original manufacturer submission with a clinical cut-off date of 23 May 2008) [median 11.1 vs. 2.8months; hazard ratio (HR) 0.40; 95 % CI 0.27, 0.60]. Data from the indirect comparison suggested that pazopanib had a greater survival than interferon alpha (IFN-α) [HR 0.512; 95 % CI 0.326, 0.802] but provided no evidence of any difference compared with sunitinib (HR 0.949; 95 % CI 0.575, 1.568). With regard to overall survival, 64 % (n=99) of patients in the pazopanib arm and 63 % (n=49) of patients in the placebo arm had died and a total of 51 % (n=40) of placebo patients had crossed over to receive pazopanib. Although data were provided on an intention-to-treat basis, crossover between therapies made such data difficult to interpret. There was no evidence of any statistically significant difference between pazopanib and best supportive care (HR 0.501; 95 % CI 0.136, 2.348). In the indirect comparison, there were no statistically significant differences between pazopanib and IFN-α (HR 0.627; 95 % CI 0.173, 2.269) or between pazopanib and sunitinib (HR 0.969; 95 % CI 0.359, 2.608). Based upon the work presented including a 12.5 % discount for pazopanib, sunitinib was extendedly dominated by a combination of pazopanib and IFN-α. As a consequence, the incremental cost per QALY for pazopanib versus IFN-α was £38,925. The results were not greatly altered over the range of univariate deterministic sensitivity analyses conducted by the manufacturer but pair-wise probabilistic sensitivity analyses suggested that given a threshold value of £30,000, there is a 54 % probability that pazopanib was preferred to sunitinib, 40 % chance against IFN-α and 47 % chance against best supportive care. The Appraisal Committee concluded that pazopanib should be recommended as a first-line treatment option for people with advanced renal cell carcinoma who have not received prior cytokine therapy and have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1, and if the manufacturer provides pazopanib with a 12.5 % discount on the list price and provides a possible future rebate linked to the outcome of the head-to-head COMPARZ trial, as agreed under the terms of the patient access scheme and to be confirmed when the COMPARZ trial data are made available.

Denosumab per la prevenzione delle fratture osteoporotiche in donne in post-menopausa

The National Institute for Health and Clinical Excellence (NICE) invited the manufacturer of denosumab (Amgen Inc., UK) to submit evidence for the clinical and cost effectiveness of denosumab for the prevention of fragility fractures in post-menopausal women, as part of the Institute’s single technology appraisal (STA) process. The University of Aberdeen Health Technology Assessment Group were commissioned to act as the Evidence Review Group (ERG); the role of the ERG being to appraise the manufacturer’s submission and to produce an independent report. This article provides a description of the company submission, the ERG review and NICE’s subsequent decisions.

A cost-utility study of the use of pregabalin in treatment-refractory neuropathic pain

Objectives:A small but significant proportion of patients with peripheral neuropathic pain (NeP) are refractory to the typical treatments applied in clinical practice, including amitriptyline and gabapentin. Thus, they continue to suffer the debilitating effects of NeP. This study aimed to evaluate the cost-effectiveness of pregabalin in comparison to usual care, in patients with refractory NeP, from a third party payer’s perspective (NHS).Methods:A stochastic simulation model was constructed, using clinical data from four non-randomized studies, to generate pain pathways of patients receiving usual care and pregabalin. Treatment effect (pain reduction) was converted to quality-of-life (QoL) data, using a regression analysis based on new utility data, collected from a survey of refractory NeP patients presenting to pain clinics in Cardiff, Wales. All relevant direct costs were estimated using resource use from the survey data (where available) and unit costs from the British National Formulary (BNF). The analysis was run over a 5-year time horizon, with costs and benefits discounted at 3.5%.Study limitations:The use of non-randomized (observational) data to characterize the effectiveness of treatments for NeP. Exclusion of productivity costs and consequences from the analysis.Results:In the base case analysis, an incremental cost-effectiveness ratio (ICER) of £10,803 per quality adjusted life year (QALY) was attained. This result was found to be reasonably insensitive to variations in the key input parameters, with ICERs ranging from £8505 to £22,845 per QALY gained.Conclusions:The analysis shows that pregabalin is a cost-effective alternative to usual care in patients with refractory NeP, with an ICER well below the threshold typically adopted by UK health technology assessment groups, such as NICE.

Denosumab for the Prevention of Osteoporotic Fractures in Post-Menopausal Women

The National Institute for Health and Clinical Excellence (NICE) invited the manufacturer of denosumab (Amgen Inc., UK) to submit evidence for the clinical and cost effectiveness of denosumab for the prevention of fragility fractures in post-menopausal women, as part of the Institute's single technology appraisal (STA) process. The University of Aberdeen Health Technology Assessment Group were commissioned to act as the Evidence Review Group (ERG); the role of the ERG being to appraise the manufacturer's submission and to produce an independent report. This article provides a description of the company submission, the ERG review and NICE's subsequent decisions. The manufacturer considered that denosumab would be appropriate for patients unable to take, comply with or tolerate oral bisphosphonates. Comparator treatments selected for the submission were, therefore, 'no treatment', raloxifene, strontium ranelate, intravenous zoledronic acid, intravenous ibandronate and teriparatide. The main effectiveness evidence for denosumab was derived from a large randomized controlled trial comparing denosumab with placebo. Given by subcutaneous injection at 6-monthly intervals for 3 years, denosumab reduced the incidence of hip fracture by 40%, and reduced the incidence of clinical vertebral fracture by 69%. An indirect treatment comparison was used to derive adjusted relative risk (RR) estimates for different types of fracture for each comparator versus placebo. The RRs (95% CI) applied for denosumab were 0.316 (0.208, 0.478) for clinical vertebral fracture, 0.605 (0.373, 0.983) for hip fracture and 0.842 (0.638, 1.110) for wrist fracture. Despite a number of concerns surrounding the methodology of the indirect comparison, the ERG was satisfied with the robustness of the effect estimates. The RR estimates were applied in a good-quality Markov model that took account of drug costs, administration and monitoring costs, costs associated with fractures, and long-term nursing home costs. Utility weights were used to adjust time spent in fracture states, allowing QALYs to be estimated. The base-case analysis was conducted for women aged 70 years with a T-score of -2.5 or less and no prior fracture, and women aged 70 years with a T-score of -2.5 or less with a prior fragility fracture. Subgroup analyses based on T-score and independent clinical risk factors were also undertaken. Applying a willingness-to-pay (WTP) threshold of £30 000 per QALY, the manufacturer's results suggested that denosumab would offer a cost-effective alternative to all treatment comparators for the primary and secondary prevention of fractures. The ERG was concerned about an assumption that denosumab would be administered in general practice at the average cost of two standard GP visits a year. As a result, the ERG requested some further sensitivity analysis and undertook some further modelling, applying an assumption that denosumab would be provided primarily in secondary care. This modification altered the cost effectiveness of denosumab versus 'no treatment' (in women with no prior fragility fracture) and zoledronic acid. The NICE Appraisal Committee concluded that, as a treatment option for the prevention of osteoporotic fractures, denosumab should be recommended only in post-menopausal women at increased risk of fracture who cannot comply with the special instructions for administering oral bisphosphonates, or have an intolerance of, or contraindication to, those treatments. For primary prevention, the Appraisal Committee also stipulated specific levels of fracture risk at which denosumab is recommended.

SCOPE for social inclusion?

This paper sets out to look at social inclusion among users within services, asking the questions is there a general measure of agreement about what social inclusion means to people, and is there an accepted way of measuring these views? The initiative was funded by the NIHR Research Methods programme now part of the Health Technology Assessment group at Southampton. Nine focus groups explored what people understand by ‘social inclusion’ and concept mapping was used to try and define the term. The research methods are reported here, along with plans for the future.

Opioid Treatment for Chronic Back Pain and Its Association with Addiction

Letters4 September 2007Opioid Treatment for Chronic Back Pain and Its Association with AddictionMeredith Noble, MS and Karen Schoelles, MD, SMMeredith Noble, MSFrom ECRI Institute Evidence-based Practice Center and Health Technology Assessment Group, Plymouth Meeting, PA 19462.Search for more papers by this author and Karen Schoelles, MD, SMFrom ECRI Institute Evidence-based Practice Center and Health Technology Assessment Group, Plymouth Meeting, PA 19462.Search for more papers by this authorAuthor, Article, and Disclosure Informationhttps://doi.org/10.7326/0003-4819-147-5-200709040-00018 SectionsAboutFull TextPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissions ShareFacebookTwitterLinkedInRedditEmail TO THE EDITOR:The poor quality and limited generalizability of the 9 studies included in Martell and colleagues' (1) assessment of substance abuse or addiction prevalence among patients with chronic back pain limits the applicability of their findings to clinical practice. The authors acknowledge some of these limitations in their paper. We will draw attention to additional problems and propose an alternative method for estimating abuse or addiction risk among individuals taking opioids for chronic back pain.First, many of the studies determined prevalence of substance abuse or addiction in populations that are likely to have higher rates than the ...

The cost-effectiveness of drug-eluting stents: a systematic review

Drug-eluting stents have been seen as an attractive alternative to bare-metal stents for percutaneous coronary interventions (PCIs) because of the decreased need for revascularization. However, comparative clinical trials have shown no difference in patient outcomes, and drug-eluting stents are considerably more expensive than their bare-metal counterparts. We conducted a systematic review of all published comparative cost-effectiveness analyses to identify the factors contributing to the heterogeneity of their conclusions. We retrieved all articles published between Jan. 1, 2000, and July 31, 2006, in which the cost-effectiveness, from a third-party payer perspective, of drug-eluting stents was compared with that of bare-metal stents for PCI in unrestricted patient populations. Electronic databases, Web sites from health technology assessment groups and references of identified articles were searched. Our outcome variable was whether the study's conclusions favoured widespread use of drug-eluting stents, as assessed by 4 independent reviewers. Study characteristics such as quality, funding source, country and year of publication were extracted. Two-by-2 tables and Fisher's exact test were used to study the association between covariates and the outcome variable. A classification and regression tree (CART) model was used for multivariate analysis. We identified 19 cost-effectiveness analyses. Ten were in favour of widespread use of drug-eluting stents, and 9 favoured more restrained use. Only 1 of 9 high-quality studies supported widespread use, as compared with 9 of 10 lower quality studies (p < 0.001). All of the 7 sponsored studies argued in favour of widespread use, as compared with 3 of the 12 studies without sponsorship (p = 0.003). Studies from the United States were more likely than those from other countries to endorse unlimited use (p = 0.032). A CART model with 2 covariates--study quality and sponsorship--provided the best fit (error rate 10.5%). Conclusions drawn by cost-effectiveness analyses of drug-eluting stents for PCI are associated with the study's quality, funding source and country of origin. Vigilance regarding these study characteristics is required when interpreting findings from cost-effectiveness analyses.

Consultant supported intermediate care - a model for remote and island hospitals

Providing local consultant-delivered hospital services in remote and island communities in the United Kingdom is increasingly problematic due to difficulties with recruitment and retention of staff, statutory restrictions to hours worked by health professionals and the expectation each clinician must manage an externally defined volume of cases to maintain clinical standards. This article describes a before-and-after evaluation of a novel method of providing consultant support for acute internal medicine to an island grouping off the Scottish coast. Under the scheme, local GPs provided acute medical care of inpatients. A consultant general physician was appointed in a district general hospital on the mainland, approximately 100 miles from the island group, to provide a lead clinician role for inpatient services at the island hospital, visiting the island on a twice-monthly basis, undertaking educational sessions and developing local guidelines and care pathways for the management of individual medical conditions. In addition, two junior doctors were appointed to the island hospital to support inpatient care. A prospective recording system for case mix was established with agreed evidence-based protocols, developed as integrated care pathways (ICP), for indicator conditions. General case mix was determined during two 6-month periods, June-November 2001 and June-November 2002, before and after implementation of the new arrangements. Performance against an ICP for management of suspected cardiac chest pain was evaluated in detail, examining the process of management, clinical outcome and economics. Data from the clinical literature were used to estimate the potential health gains from observed changes in clinical practice. Total admissions rose by 25% in the second time period, with particular increases noted for cardiovascular, cerebrovascular disease, and cancer. Total air ambulance transfers between the islands and the mainland within these time periods increased by 31%, from 88 to 115 transfers. Recording specific details from the history and frequency of appropriate blood investigations increased and initial steps in management changed considerably after introduction of the ICP. The number of transfers to the mainland teaching hospital increased from 3/37 (8%) in 2001 to 15/56 (27%) in 2002. Based on an estimated 100 patients per year, of whom 15 would receive thrombolysis, total additional patient costs would be 64,000 pounds sterling. The annual cost of the additional resource input into the medical service was 148,000 pounds sterling. Approximately 16 adverse events would be avoided at a combined cost of 212,000 pounds sterling (148,000 pounds sterling direct costs of intervention + 64,000 pounds sterling additional treatment costs) or 13,250 pounds sterling per event avoided. This is a conservative estimate of benefit as all the direct costs of the intervention have been included. This study shows that appropriate standards of care can be delivered in the setting described. Costs of care increased, but the level of service provided increased concomitantly, and the health benefits were achieved at costs that compare favourably with other interventions recommended by health technology assessment groups. An estimate of notional costs involved in alternative models for the delivery of hospital medical services in a remote area suggests that costs would be similar for a three-consultant service, the present model, and a triage and transfer system. In the future, the models chosen by remote and island communities and healthcare providers are therefore likely to be determined by viability, sustainability and public acceptability rather than cost. Our study indicates that consultant supported intermediate care is a viable model.