A Comprehensive Algorithm for Approval of Health Technologies With, Without, or Only in Research: The Key Principles for Informing Coverage Decisions

Claxton, Karl; Palmer, Stephen; Longworth, Louise; Bojke, Laura; Griffin, Susan; Soares, Marta; Spackman, Eldon; Rothery, Claire

doi:10.1016/j.jval.2016.03.2003

Cited by 43 publications

(51 citation statements)

References 28 publications

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“…10 If there is an ongoing trial for regulatory purposes it may be more efficient to delay a NICE decision pending evidence on long term outcomes from the regulatory trial. For drugs for which a trial is judged unethical or impractical, careful non-randomised studies should be conducted to minimise confounding, by collecting longitudinal data on all relevant prognostic characteristics and outcomes for patients receiving and not receiving the new drug.…”

Section: Editorialsmentioning

confidence: 99%

Cancer Drugs Fund requires further reform

Grieve

Abrams

Claxton

et al. 2016

BMJ

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Section: Editorialsmentioning

confidence: 99%

Cancer Drugs Fund requires further reform

Grieve

Abrams

Claxton

et al. 2016

BMJ

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“…Sensitivity analysis, and the display of uncertainty using cost‐effectiveness acceptability curves/frontiers, plays a key role in quantifying uncertainty in cost‐effectiveness appraisals (Claxton, ; Heintz et al, ). Even though these are standard parts of most cost‐effectiveness assessments today, the way in which these are used as decision criteria, and ultimately determine the proportion of false positive and false negatives, needs further clarification and consideration, informed by recent work in this area (Claxton et al, ; Claxton et al, ; Walker et al, ).…”

Section: Discussionmentioning

confidence: 99%

How to design the cost‐effectiveness appraisal process of new healthcare technologies to maximise population health: A conceptual framework

et al. 2017

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This paper presents a conceptual framework to analyse the design of the cost-effectiveness appraisal process of new healthcare technologies. The framework characterises the appraisal processes as a diagnostic test aimed at identifying cost-effective (true positive) and non-cost-effective (true negative) technologies. Using the framework, factors that influence the value of operating an appraisal process, in terms of net gain to population health, are identified. The framework is used to gain insight into current policy questions including (a) how rigorous the process should be, (b) who should have the burden of proof, and (c) how optimal design changes when allowing for appeals, price reductions, resubmissions, and re-evaluations. The paper demonstrates that there is no one optimal appraisal process and the process should be adapted over time and to the specific technology under assessment. Optimal design depends on country-specific features of (future) technologies, for example, effect, price, and size of the patient population, which might explain the difference in appraisal processes across countries. It is shown that burden of proof should be placed on the producers and that the impact of price reductions and patient access schemes on the producer's price setting should be considered when designing the appraisal process.

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“…• Between 2000-2016, 22 of the 489 (4%) individual pharmaceutical technologies assessed by NICE were based upon comparative clinical effectiveness estimates calculated using non-RCT data. Out of these:…”

Section: Key Findingsmentioning

confidence: 99%

“…Although whilst the 'Only In Research' designation may be appropriate in some cases, this must be balanced against delayed access to medicines for patients. (22) Secondly, patient access schemes offer another option to mitigate uncertainty regarding value of new technologies. Interestingly, there was only a small difference between technologies with and without RCT data utilising a patient access scheme.…”

Section: A C C E P T E D Accepted Manuscriptmentioning

confidence: 99%

A review of NICE appraisals of pharmaceuticals 2000–2016 found variation in establishing comparative clinical effectiveness

Anderson

Naci

Morrison

et al. 2019

Journal of Clinical Epidemiology

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To date, a small number of appraisals by NICE based on non-RCT data did not result in substantially different treatment decisions. The majority of the technologies appraised on the basis of non-RCT data either received a positive recommendation or a positive recommendation with restrictions. The methods used to calculate comparative clinical effectiveness estimates varied, highlighting the need to establish clear guidance.

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A Comprehensive Algorithm for Approval of Health Technologies With, Without, or Only in Research: The Key Principles for Informing Coverage Decisions

Abstract: These principles demonstrate that cost-effectiveness is a necessary but not sufficient condition for approval. Even when research is possible with approval, OIR may be appropriate when a technology is expected to be cost-effective due to significant irrecoverable costs.

Cited by 43 publications

References 28 publications

Cancer Drugs Fund requires further reform

Cancer Drugs Fund requires further reform

How to design the cost‐effectiveness appraisal process of new healthcare technologies to maximise population health: A conceptual framework

A review of NICE appraisals of pharmaceuticals 2000–2016 found variation in establishing comparative clinical effectiveness

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