{"corpus_id":44162853,"paper_sha":"2399cbd044a4a25abf5e36c72a59e8b1a574f10a","doi":"10.1186/s12913-018-3162-2","arxiv_id":null,"pmid":29855313,"pmcid":"5984433","mag_id":2807109147,"dblp_id":null,"acl_id":null,"title":"Will the reformed Cancer Drugs Fund address the most common types of uncertainty? An analysis of NICE cancer drug appraisals","year":2018,"publication_date":"2018-05-31","venue":"BMC Health Services Research","journal":{"name":"BMC Health Services Research","pages":null,"volume":"18"},"journal_issn":null,"journal_title":null,"publication_types":["JournalArticle","Review"],"pubmed_pub_types":["Journal Article","Research Support, Non-U.S. Gov't"],"s2_fields_of_study":["Medicine","Economics"],"reference_count":54,"citation_count":22,"influential_citation_count":0,"is_open_access":true,"arxiv_categories":null,"arxiv_license":null,"arxiv_journal_ref":null,"mesh_headings":[{"d":"Advisory Committees","mj":false,"ui":"D026683"},{"d":"Antineoplastic Agents","mj":false,"qs":[{"q":"economics","mj":true,"ui":"Q000191"},{"q":"therapeutic use","mj":false,"ui":"Q000627"}],"ui":"D000970"},{"d":"Clinical Trials as Topic","mj":false,"qs":[{"q":"economics","mj":false,"ui":"Q000191"}],"ui":"D002986"},{"d":"Cost-Benefit Analysis","mj":false,"ui":"D003362"},{"d":"Drug Approval","mj":false,"qs":[{"q":"economics","mj":false,"ui":"Q000191"}],"ui":"D017277"},{"d":"Drug Costs","mj":false,"ui":"D016527"},{"d":"England","mj":false,"ui":"D004739"},{"d":"Financial Management","mj":false,"ui":"D005376"},{"d":"Humans","mj":false,"ui":"D006801"},{"d":"Neoplasms","mj":false,"qs":[{"q":"drug therapy","mj":false,"ui":"Q000188"},{"q":"economics","mj":true,"ui":"Q000191"}],"ui":"D009369"},{"d":"Quality of Life","mj":false,"ui":"D011788"},{"d":"Quality-Adjusted Life Years","mj":false,"ui":"D019057"},{"d":"Sample Size","mj":false,"ui":"D018401"},{"d":"Technology Assessment, Biomedical","mj":false,"qs":[{"q":"economics","mj":false,"ui":"Q000191"}],"ui":"D013673"},{"d":"Uncertainty","mj":true,"ui":"D035501"}],"chemicals":[{"n":"Antineoplastic Agents","ui":"D000970","reg":"0"}],"comments_corrections":null,"source_flags":5,"s2_open_access_pdf_url":"https://bmchealthservres.biomedcentral.com/track/pdf/10.1186/s12913-018-3162-2","s2_open_access_landing_url":"https://www.semanticscholar.org/paper/2399cbd044a4a25abf5e36c72a59e8b1a574f10a","s2_open_access_license":"CCBY","s2_open_access_status":"GOLD","pmc_open_access_pdf_url":null,"pmc_open_access_landing_url":null,"pmc_open_access_license":null,"pmc_open_access_status":null,"unpaywall_open_access_pdf_url":null,"unpaywall_open_access_landing_url":null,"unpaywall_open_access_license":null,"unpaywall_open_access_status":null,"abstract":"BackgroundOne of the functions of the reformed Cancer Drugs Fund in England is as a managed access fund, providing conditional funding for cancer drugs where there is uncertainty in the economic case, and where that uncertainty can be addressed by data collection during two years’ use in the NHS. Our study characterises likely sources of such uncertainty, through a review of recent NICE Technology Appraisals.MethodsDiscussions of uncertainty in NICE Appraisal Committees were extracted from published Single Technology Appraisals of cancer drugs, 2014–2016, and categorised inductively. The location of the comments within the structured Appraisal document was used as a proxy for the degree of concern shown by the Committee.ResultsTwenty-nine appraisals were analysed, of which 23 (79%) were recommended for funding. Six main sources of uncertainty were identified. Immaturity of survival data, and issues relating to comparators, were common sources of uncertainty regardless of degree of concern. Uncertainties relating to quality of life, and the patient population in the trial, were discussed frequently but rarely occurred in the more uncertain appraisals. Concerns with trial design, and cost uncertainty, were less common, but a high proportion contributed to the most uncertain appraisals. Funding decisions were not driven by uncertainty in the evidence base, but by the expected cost per QALY relative to acceptance thresholds, and the resultant level of uncertainty in the decision.ConclusionsThe reformed CDF is an improvement on its predecessor. However the main types of uncertainty seen in recent cancer appraisals will not readily be resolved solely by 2 years’ RWD collection in the reformed CDF; where there are no ongoing trials to provide longer-term data, randomised trials rather than RWD may be needed to fully resolve questions of relative efficacy. Other types of uncertainty, and concerns with generalisability, may be more amenable to the RWD approach, and it is these that we expect to be the focus of data collection arrangements in the reformed CDF.","claims":[{"public_id":"cl_50f79482a0c0ce49639d7297657ba38a","status":"active","text":"Immaturity of survival data and comparator issues were common sources of uncertainty regardless of the degree of committee concern.","confidence":0.93,"contributors":[{"id":136,"public_id":"3c2apqe3ut","public_label":"Anonymous (3c2apqe3ut)","roles":["extraction"],"url":"https://sah.borca.ai/u/3c2apqe3ut"},{"id":2,"public_id":"4715169a40","public_label":"AK (4715169a40)","roles":["review"],"url":"https://sah.borca.ai/u/4715169a40"},{"id":17,"public_id":"322360f1c1","public_label":"Killer Whale 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uncertainty were identified in NICE Appraisal Committee discussions of cancer drug appraisals.","confidence":0.96,"contributors":[{"id":136,"public_id":"3c2apqe3ut","public_label":"Anonymous (3c2apqe3ut)","roles":["extraction"],"url":"https://sah.borca.ai/u/3c2apqe3ut"},{"id":2,"public_id":"4715169a40","public_label":"AK (4715169a40)","roles":["review"],"url":"https://sah.borca.ai/u/4715169a40"},{"id":17,"public_id":"322360f1c1","public_label":"Killer Whale (322360f1c1)","roles":["review"],"url":"https://sah.borca.ai/u/322360f1c1"}],"url":"https://sah.borca.ai/claims/cl_6d189eee07e0d9a6010921a920062a66"},{"public_id":"cl_31747d798fb98473b903bb2878b8aad7","status":"active","text":"Twenty-nine NICE Single Technology Appraisals of cancer drugs from 2014 to 2016 were analyzed, and 23 of them were recommended for funding.","confidence":0.98,"contributors":[{"id":136,"public_id":"3c2apqe3ut","public_label":"Anonymous (3c2apqe3ut)","roles":["extraction"],"url":"https://sah.borca.ai/u/3c2apqe3ut"},{"id":2,"public_id":"4715169a40","public_label":"AK (4715169a40)","roles":["review"],"url":"https://sah.borca.ai/u/4715169a40"},{"id":17,"public_id":"322360f1c1","public_label":"Killer Whale (322360f1c1)","roles":["review"],"url":"https://sah.borca.ai/u/322360f1c1"}],"url":"https://sah.borca.ai/claims/cl_31747d798fb98473b903bb2878b8aad7"},{"public_id":"cl_9daeb26b8002f76797cf853082296518","status":"active","text":"Two years of real-world data collection in the reformed Cancer Drugs Fund is unlikely to resolve the main types of uncertainty seen in recent cancer appraisals by itself.","confidence":0.94,"contributors":[{"id":136,"public_id":"3c2apqe3ut","public_label":"Anonymous (3c2apqe3ut)","roles":["extraction"],"url":"https://sah.borca.ai/u/3c2apqe3ut"},{"id":2,"public_id":"4715169a40","public_label":"AK 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