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Patient organisations are increasingly involved in HTA. Given this, it is important to understand what these organisations contribute and how their voices are accounted for in the decision-making process. This study characterises inputs from patient organisations and/or their nominated patient experts in technology appraisals for ultra-rare diseases in England and Wales and seeks to understand how these are considered in NICE final recommendations.

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Prevalence of Immature Survival Data for Anti-Cancer Drugs Presented to the National Institute for Health and Care Excellence between 2018-2022.

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Sheffield Centre for Health and Related Research, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA; Delta Hat Limited, Nottingham, UK.

Objectives: Between 2015 and 2017, 41% of NICE cancer single technology appraisal (STA) decisions relied upon immature survival data. This occurs when clinical trials that form the evidence base in support of new or existing technologies suffer from limited follow-up. During this period, NICE did not negatively recommend any cancer technologies that used immature data.

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Critical Appraisal of Evidence: Synthesis and Recommendations.

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Susan Farus-Brown is an associate professor at the Ohio University School of Nursing in Athens. Ellen Fineout-Overholt is national senior director, Evidence-Based Practice & Implementation Science, at Ascension in St. Louis. Deana Hays is an associate professor at Oakland University in Rochester, MI. Mary C. Zonsius is an associate professor at the Rush University College of Nursing in Chicago. Kerry A. Milner is a professor in the Davis and Henley College of Nursing at Sacred Heart University in Fairfield, CT. Contact author: Kerry A. Milner, The authors have disclosed no potential conflicts of interest, financial or otherwise.

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