{"entity":{"id":"paper-davis-bmj","kind":"paper","name":"Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13","aka":[],"tldr":"Paper cited by two bottleneck pages and 15 idea pages, indexed on Europe PMC as PubMed record 28978555 and published in BMJ; the citing pages link this DOI, which is how the record was matched.","summary":"Objective To determine the availability of data on overall survival and quality of life benefits of cancer drugs approved in Europe. Design Retrospective cohort study. Setting Publicly accessible regulatory and scientific reports on cancer approvals by the European Medicines Agency (EMA) from 2009 to 2013. Main outcome measures Pivotal and postmarketing trials of cancer drugs according to their design features (randomisation, crossover, blinding), comparators, and endpoints. Availability and magnitude of benefit on overall survival or quality of life determined at time of approval and after market entry. Validated European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) used to assess the clinical value of the reported gains in published studies of cancer drugs. Results From 2009 to 2013, the EMA approved the use of 48 cancer drugs for 68 indications. Of these, eight indications (12%) were approved on the basis of a single arm study. At the time of market approval, there was significant prolongation of survival in 24 of the 68 (35%). The magnitude of the benefit on overall survival ranged from 1.0 to 5.8 months (median 2.7 months). At the time of market approval, there was an improvement in quality of life in seven of 68 indications (10%). Out of 44 indications for which there was no evidence of a survival gain at the time of market authorisation, in the subsequent postmarketing period there was evidence for extension of life in three (7%) and reported benefit on quality of life in five (11%). Of the 68 cancer indications with EMA approval, and with a median of 5.4 years' follow-up (minimum 3.3 years, maximum 8.1 years), only 35 (51%) had shown a significant improvement in survival or quality of life, while 33 (49%) remained uncertain. Of 23 indications associated with a survival benefit that could be scored with the ESMO-MCBS tool, the benefit was judged to be clinically meaningful in less than half (11/23, 48%). Conclusions This systematic evaluation of oncology approvals by the EMA in 2009-13 shows that most drugs entered the market without evidence of benefit on survival or quality of life. At a minimum of 3.3 years after market entry, there was still no conclusive evidence that these drugs either extended or improved life for most cancer indications. When there were survival gains over existing treatment options or placebo, they were often marginal.\n\nIndexed on Europe PMC as PubMed record 28978555 (DOI 10.1136/bmj.j4530). Matched by DOI alone: two bottleneck pages and 15 idea pages cite this DOI among their external links (the pages are listed under Related), and this page was written so that the citation resolves inside OnCo. No figure has been checked by an editor.","asOf":"2026-09-22","links":[{"label":"BMJ 2017","url":"https://doi.org/10.1136/bmj.j4530"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/28978555/"},{"label":"Europe PMC","url":"https://europepmc.org/article/MED/28978555"}],"tags":["europepmc-ingest"],"related":["b-toxicity-qol","b-trial-design","idea-tr1-window-of-opportunity-default","idea-tr1-tumour-agnostic-approval-standard","idea-tr1-aggregated-n-of-1-supportive-care","idea-tr1-ai-central-imaging-reads","idea-tr1-shrinkage-subgroup-analysis","idea-tr1-shared-control-arms-across-sponsors","idea-tr1-target-trial-emulation-to-prioritise-rcts","idea-tr1-crossover-adjusted-survival-standard","idea-tr1-immunotherapy-stop-trials","idea-tr1-randomised-phase-2-before-phase-3","idea-tr1-seamless-2-3-with-prespecified-go","idea-tr1-smart-designs-for-adaptive-strategies","idea-tr1-aggressive-futility-boundaries","idea-tr1-ctdna-guided-stop-in-metastatic-disease","idea-tr1-validate-real-world-progression-endpoints"],"cancers":[],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["bmj"],"dependsOn":[],"notes":[],"journal":"BMJ","year":2017,"doi":"10.1136/bmj.j4530","pmid":"28978555","authors":"Davis C, Naci H, Gurpinar E, et al.","paperType":"observational","findings":[],"whatItMeans":"Two bottleneck pages and 15 idea pages on OnCo cite this paper by its DOI; this record gives the citation a page of its own so a reader can follow it without leaving OnCo. Read the abstract above alongside the citing pages listed under Related; the record was created automatically from the Europe PMC entry and its figures have not been checked by hand.","caveats":["Matched to the citing OnCo records by DOI alone; the summary reproduces the Europe PMC abstract and no figure has been verified against the full paper."]},"route":"/key-papers/paper-davis-bmj/","neighbours":{"bottleneck":[{"id":"b-toxicity-qol","kind":"bottleneck","name":"Toxicity and quality of life are undervalued","route":"/bottlenecks/b-toxicity-qol/"},{"id":"b-trial-design","kind":"bottleneck","name":"Trial design, endpoints and cost","route":"/bottlenecks/b-trial-design/"}],"idea":[{"id":"idea-tr1-window-of-opportunity-default","kind":"idea","name":"A short pre-surgery drug window as the default early test of new agents","route":"/ideas/idea-tr1-window-of-opportunity-default/"},{"id":"idea-tr1-tumour-agnostic-approval-standard","kind":"idea","name":"A standard for tumour-agnostic approvals: minimum histologies and hierarchical modelling","route":"/ideas/idea-tr1-tumour-agnostic-approval-standard/"},{"id":"idea-tr1-aggregated-n-of-1-supportive-care","kind":"idea","name":"Aggregated single-patient crossover trials for symptom and supportive treatments","route":"/ideas/idea-tr1-aggregated-n-of-1-supportive-care/"},{"id":"idea-tr1-ai-central-imaging-reads","kind":"idea","name":"AI-assisted central imaging reads to cut endpoint cost and variability","route":"/ideas/idea-tr1-ai-central-imaging-reads/"},{"id":"idea-tr1-shrinkage-subgroup-analysis","kind":"idea","name":"Bayesian shrinkage for subgroup claims to stop false 'works in this group' stories","route":"/ideas/idea-tr1-shrinkage-subgroup-analysis/"},{"id":"idea-tr1-shared-control-arms-across-sponsors","kind":"idea","name":"Competing sponsors share one control arm in the same indication","route":"/ideas/idea-tr1-shared-control-arms-across-sponsors/"},{"id":"idea-tr1-target-trial-emulation-to-prioritise-rcts","kind":"idea","name":"Emulate the trial in real-world data first to decide which trials to run","route":"/ideas/idea-tr1-target-trial-emulation-to-prioritise-rcts/"},{"id":"idea-tr1-crossover-adjusted-survival-standard","kind":"idea","name":"Pre-specified crossover-adjusted survival in every trial that allows crossover","route":"/ideas/idea-tr1-crossover-adjusted-survival-standard/"},{"id":"idea-tr1-immunotherapy-stop-trials","kind":"idea","name":"Randomised trials of stopping immunotherapy after one year versus continuing","route":"/ideas/idea-tr1-immunotherapy-stop-trials/"},{"id":"idea-tr1-randomised-phase-2-before-phase-3","kind":"idea","name":"Require a randomised phase 2 before any phase 3","route":"/ideas/idea-tr1-randomised-phase-2-before-phase-3/"},{"id":"idea-tr1-seamless-2-3-with-prespecified-go","kind":"idea","name":"Seamless phase 2/3 with pre-registered go rules as the default for new agents","route":"/ideas/idea-tr1-seamless-2-3-with-prespecified-go/"},{"id":"idea-tr1-smart-designs-for-adaptive-strategies","kind":"idea","name":"SMART designs to test treatment strategies, not just single drugs","route":"/ideas/idea-tr1-smart-designs-for-adaptive-strategies/"},{"id":"idea-tr1-aggressive-futility-boundaries","kind":"idea","name":"Stop failing trials earlier with pre-registered aggressive futility rules","route":"/ideas/idea-tr1-aggressive-futility-boundaries/"},{"id":"idea-tr1-ctdna-guided-stop-in-metastatic-disease","kind":"idea","name":"Use tumour DNA in blood to decide when to pause treatment in metastatic cancer","route":"/ideas/idea-tr1-ctdna-guided-stop-in-metastatic-disease/"},{"id":"idea-tr1-validate-real-world-progression-endpoints","kind":"idea","name":"Validate real-world progression endpoints so pragmatic trials can use them","route":"/ideas/idea-tr1-validate-real-world-progression-endpoints/"}],"journal":[{"id":"bmj","kind":"journal","name":"BMJ","route":"/journals/bmj/"}]}}