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10.1186/s13023-017-0597-1 http://scihub22266oqcxt.onion/10.1186/s13023-017-0597-1 C5335492!5335492 !28253932     free     free     free Warning: file_get_contents(https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&id=28253932 &cmd=llinks): Failed to open stream: HTTP request failed! 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Does the low prevalence affect the sample size of interventional clinical trials of rare diseases? An analysis of data from the aggregate analysis of clinicaltrials gov |pdf=|usr=}}{{28253932 }} gab.com Text Does the low prevalence affect the sample size of interventional clinical trials of rare diseases An analysis of data from the aggregate analysis of clinicaltrials gov JO: Orphanet J Rare Dis http://www.kidney.de/pget.php?&tw=1&pmid=28253932 #FOAvip BACKGROUND: Clinical trials are typically designed using the classical frequentist framework to constrain type I and II error rates. Sample sizes required in such designs typically range from hundreds to thousands of patients which can be challenging for rare diseases. It has been shown that rare disease trials have smaller sample sizes than non-rare disease trials. Indeed some orphan drugs were approved by the European Medicines Agency based on studies with as few as 12 patients. However, some studies supporting marketing authorisation included several hundred patients. In this work, we explore the relationship between disease prevalence and other factors and the size of interventional phase 2 and 3 rare disease trials conducted in the US and/or EU. We downloaded all clinical trials from Aggregate Analysis of ClinialTrials.gov (AACT) and identified rare disease trials by cross-referencing MeSH terms in AACT with the list from Orphadata. We examined the effects of prevalence and phase of study in a multiple linear regression model adjusting for other statistically significant trial characteristics. RESULTS: Of 186941 ClinicalTrials.gov trials only 1567 (0.8%) studied a single rare condition with prevalence information from Orphadata. There were 19 (1.2%) trials studying disease with prevalence <1/1,000,000, 126 (8.0%) trials with 1-9/1,000,000, 791 (50.5%) trials with 1-9/100,000 and 631 (40.3%) trials with 1-5/10,000. Of the 1567 trials, 1160 (74%) were phase 2 trials. The fitted mean sample size for the rarest disease (prevalence <1/1,000,000) in phase 2 trials was the lowest (mean, 15.7; 95% CI, 8.7-28.1) but were similar across all the other prevalence classes; mean, 26.2 (16.1-42.6), 33.8 (22.1-51.7) and 35.6 (23.3-54.3) for prevalence 1-9/1,000,000, 1-9/100,000 and 1-5/10,000, respectively. Fitted mean size of phase 3 trials of rarer diseases, <1/1,000,000 (19.2, 6.9-53.2) and 1-9/1,000,000 (33.1, 18.6-58.9), were similar to those in phase 2 but were statistically significant lower than the slightly less rare diseases, 1-9/100,000 (75.3, 48.2-117.6) and 1-5/10,000 (77.7, 49.6-121.8), trials. CONCLUSIONS: We found that prevalence was associated with the size of phase 3 trials with trials of rarer diseases noticeably smaller than the less rare diseases trials where phase 3 rarer disease (prevalence <1/100,000) trials were more similar in size to those for phase 2 but were larger than those for phase 2 in the less rare disease (prevalence ?1/100,000) trials. Twit Text FOAVip Does the low prevalence affect the sample size of interventional clinical trials of rare diseases An analysis of data from the aggregate analysis of clinicaltrials gov ????Orphanet J Rare Dis http://www.kidney.de/pget.php?&tw=1&pmid=28253932 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=28253932 #FOAvip English Wikipedia <ref>{{Cite pmid|28253932 }}</ref> Does the low prevalence affect the sample size of interventional clinical trials of rare diseases? An analysis of data from the aggregate analysis of clinicaltrials gov #MMPMID28253932 Hee SW ; Willis A ; Tudur Smith C ; Day S ; Miller F ; Madan J ; Posch M ; Zohar S ; Stallard N Orphanet J Rare Dis 2017[Mar]; 12 (1 ): 44 PMID28253932 show ga BACKGROUND: Clinical trials are typically designed using the classical frequentist framework to constrain type I and II error rates. Sample sizes required in such designs typically range from hundreds to thousands of patients which can be challenging for rare diseases. It has been shown that rare disease trials have smaller sample sizes than non-rare disease trials. Indeed some orphan drugs were approved by the European Medicines Agency based on studies with as few as 12 patients. However, some studies supporting marketing authorisation included several hundred patients. In this work, we explore the relationship between disease prevalence and other factors and the size of interventional phase 2 and 3 rare disease trials conducted in the US and/or EU. We downloaded all clinical trials from Aggregate Analysis of ClinialTrials.gov (AACT) and identified rare disease trials by cross-referencing MeSH terms in AACT with the list from Orphadata. We examined the effects of prevalence and phase of study in a multiple linear regression model adjusting for other statistically significant trial characteristics. RESULTS: Of 186941 ClinicalTrials.gov trials only 1567 (0.8%) studied a single rare condition with prevalence information from Orphadata. There were 19 (1.2%) trials studying disease with prevalence <1/1,000,000, 126 (8.0%) trials with 1-9/1,000,000, 791 (50.5%) trials with 1-9/100,000 and 631 (40.3%) trials with 1-5/10,000. Of the 1567 trials, 1160 (74%) were phase 2 trials. The fitted mean sample size for the rarest disease (prevalence <1/1,000,000) in phase 2 trials was the lowest (mean, 15.7; 95% CI, 8.7-28.1) but were similar across all the other prevalence classes; mean, 26.2 (16.1-42.6), 33.8 (22.1-51.7) and 35.6 (23.3-54.3) for prevalence 1-9/1,000,000, 1-9/100,000 and 1-5/10,000, respectively. Fitted mean size of phase 3 trials of rarer diseases, <1/1,000,000 (19.2, 6.9-53.2) and 1-9/1,000,000 (33.1, 18.6-58.9), were similar to those in phase 2 but were statistically significant lower than the slightly less rare diseases, 1-9/100,000 (75.3, 48.2-117.6) and 1-5/10,000 (77.7, 49.6-121.8), trials. CONCLUSIONS: We found that prevalence was associated with the size of phase 3 trials with trials of rarer diseases noticeably smaller than the less rare diseases trials where phase 3 rarer disease (prevalence <1/100,000) trials were more similar in size to those for phase 2 but were larger than those for phase 2 in the less rare disease (prevalence ?1/100,000) trials. |*Clinical Trials as Topic [MESH] |Humans [MESH] |Internet [MESH] |Prevalence [MESH] |Rare Diseases/*epidemiology/*therapy [MESH]Does the low prevalence affect the sample size of interventional clini(epmc).pdf DeepDyve Pubget Overpricing