Development of a Transparent Interactive Decision Interrogator to Facilitate the Decision-Making Process in Health Care

Bujkiewicz, Sylwia; Jones, Hayley E; Lai, Monica C.W.; Cooper, Nicola J.; Hawkins, Neil; Squires, Hazel; Abrams, Keith R.; Spiegelhalter, David; Sutton, Alex J.

doi:10.1016/j.jval.2010.12.002

Cited by 22 publications

(21 citation statements)

References 25 publications

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“…Given the potential complexity in both undertaking these analyses and communicating the results, more efforts should be made to ensure that informal judgements can be more explicitly incorporated in a timely and transparent manner. 209 A key assumption employed within both models is that from year 5 onwards all patients who remained alive were assumed to experience a similar mortality risk profile as that of a long-term survivor of ALL. Hence, the mortality risks assumed in both models after year 5 were based on matched general population estimates of the all-cause risk of mortality adjusted for excess morbidity and mortality reported in cohorts of long-term survivors of ALL.…”

Section: The Exemplar Economic Modelmentioning

confidence: 99%

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

Hettle

Corbett

Hinde

et al. 2017

Health Technol Assess

103

143

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BackgroundThe National Institute for Health and Care Excellence (NICE) commissioned a ‘mock technology appraisal’ to assess whether changes to its methods and processes are needed. This report presents the findings of independent research commissioned to inform this appraisal and the deliberations of a panel convened by NICE to evaluate the mock appraisal.MethodsOur research included reviews to identify issues, analysis methods and conceptual differences and the relevance of alternative decision frameworks, alongside the development of an exemplar case study of chimeric antigen receptor (CAR) T-cell therapy for treating acute lymphoblastic leukaemia.ResultsAn assessment of previous evaluations of regenerative medicines found that, although there were a number of evidential challenges, none was unique to regenerative medicines or was beyond the scope of existing methods used to conceptualise decision uncertainty. Regarding the clinical evidence for regenerative medicines, the issues were those associated with a limited evidence base but were not unique to regenerative medicines: small non-randomised studies, high variation in response and the intervention subject to continuing development. The relative treatment effects generated from single-arm trials are likely to be optimistic unless it is certain that the historical data have accurately estimated the efficacy of the control agent. Pivotal trials may use surrogate end points, which, on average, overestimate treatment effects. To reduce overall uncertainty, multivariate meta-analysis of all available data should be considered. Incorporating indirectly relevant but more reliable (more mature) data into the analysis can also be considered; such data may become available as a result of the evolving regulatory pathways being developed by the European Medicines Agency. For the exemplar case of CAR T-cell therapy, target product profiles (TPPs) were developed, which considered the ‘curative’ and ‘bridging to stem-cell transplantation’ treatment approaches separately. Within each TPP, three ‘hypothetical’ evidence sets (minimum, intermediate and mature) were generated to simulate the impact of alternative levels of precision and maturity in the clinical evidence. Subsequent assessments of cost-effectiveness were undertaken, employing the existing NICE reference case alongside additional analyses suggested within alternative frameworks. The additional exploratory analyses were undertaken to demonstrate how assessments of cost-effectiveness and uncertainty could be impacted by alternative managed entry agreements (MEAs), including price discounts, performance-related schemes and technology leasing. The panel deliberated on the range of TPPs, evidence sets and MEAs, commenting on the likely recommendations for each scenario. The panel discussed the challenges associated with the exemplar and regenerative medicines more broadly, focusing on the need for a robust quantification of the level of uncertainty in the cost-effective estimates and the potential value of MEAs in limiting the exposure of the NHS to high upfront costs and loss associated with a wrong decision.ConclusionsIt is to be expected that there will be a significant level of uncertainty in determining the clinical effectiveness of regenerative medicines and their long-term costs and benefits, but the existing methods available to estimate the implications of this uncertainty are sufficient. The use of risk sharing and MEAs between the NHS and manufacturers of regenerative medicines should be investigated further.FundingThe National Institute for Health Research Health Technology Assessment programme.

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Section: The Exemplar Economic Modelmentioning

confidence: 99%

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

Hettle

Corbett

Hinde

et al. 2017

Health Technol Assess

103

143

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“…In order to do so, these hospitals must at least meet the costs of the procedures they perform (5) . However, the decision to use high technology in healthcare is often based on complex economic models and uncertain and informal judgments (6) .…”

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confidence: 99%

Direct cost of connecting, maintaining and disconnecting patient-controlled analgesia pump

Gouvêa¹,

Lima²

2014

Rev. esc. enferm. USP

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RESUmoPesquisa quantitativa que objetivou identificar o custo total médio (CTM) da instalação, manutenção e desligamento da bomba de analgesia controlada pelo paciente (PCA) no manejo da dor. A amostra não probabilística correspondeu à observação de 81 procedimentos em 17 Unidades do Instituto Central do Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo. Calculou-se o CTM multiplicando-se o tempo despendido pelas enfermeiras pelo custo unitário da mão de obra direta, somando-se ao custo dos materiais e medicamentos/soluções. O CTM da instalação foi de R$ 107,91; da manutenção R$ 110,55 e do desligamento R$ 4,94. Os resultados obtidos subsidiarão discussões acerca da necessidade de repasse monetá-rio do Sistema Único de Saúde às unidades hospitalares que realizam essa técnica de terapia antálgica e contribuirão para o gerenciamento de custos visando à tomada de decisão eficiente e eficaz na alocação dos recursos disponíveis.

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“…Access to this type of information requires balancing safeguards to avoid breaches on proprietary information with the need for the public and partners to be fully informed of the deliberations. The use of innovative tools for decision-making transparency could be of help [46–48]. …”

Section: Resultsmentioning

confidence: 99%

Contributions and challenges for worldwide vaccine safety: The Global Advisory Committee on Vaccine Safety at 15 years

Asturias

Wharton

Pless

et al. 2016

Vaccine

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In 1999, the Global Advisory Committee on Vaccine Safety (GACVS) was established by the World Health Organization (WHO) to provide independent scientific advice on issues relating to the safety of vaccines and immunization. Fifteen years onward, we conducted a multi-faceted review to evaluate the impact, reach and challenges facing GACVS, including the role GACVS plays in informing global, regional and WHO member state vaccine policy. The methods included measures of organizational structure, citation impact, themes approached, and a discussion by previous and current members to evaluate past, present and future challenges. Given the increasing range of data sources and the deployment of many new vaccines, the Committee is facing the complex task of identifying the best available evidence for recommendations on vaccine safety. To help meet the increased demand for public transparency in decision making, GACVS-structured methodology for evidence-based decisions is evolving. GACVS also promotes best practices and capacity building for timely and accurate risk assessment; risk communications; outreach to help countries maintain and, if needed, rebuild public trust in vaccines; and advocacy for bridging the major gaps in vaccine safety capacity globally.

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Development of a Transparent Interactive Decision Interrogator to Facilitate the Decision-Making Process in Health Care

Cited by 22 publications

References 25 publications

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

Direct cost of connecting, maintaining and disconnecting patient-controlled analgesia pump

Contributions and challenges for worldwide vaccine safety: The Global Advisory Committee on Vaccine Safety at 15 years

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