2014
DOI: 10.1186/s13023-014-0164-y
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A methodological framework for drug development in rare diseases

Abstract: IntroductionDeveloping orphan drugs is challenging because of their severity and the requisite for effective drugs. The small number of patients does not allow conducting adequately powered randomized controlled trials (RCTs). There is a need to develop high quality, ethically investigated, and appropriately authorized medicines, without subjecting patients to unnecessary trials.Aims and ObjectivesThe main aim is to develop generalizable framework for choosing the best-performing drug/endpoint/design combinati… Show more

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Cited by 21 publications
(13 citation statements)
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“…Strategies that have been proposed for enhancing statistical efficiency at the design phase for clinical evaluative studies of rare disease treatments include factorial trials and adaptive designs. Factorial trials are designed to test multiple treatments simultaneously using the same study population, thus reducing the overall number of participants needed [ 2 , 33 , 39 , 40 , 46 , 49 , 53 , 57 ]. For example, in a 2 × 2 factorial design participants are randomized to either treatment A or control group A, and then randomized again to treatment B or control group B, which effectively reduces the sample size needed to test these two treatments by 50% because the same participants are being randomized [ 40 ].…”
Section: Resultsmentioning
confidence: 99%
“…Strategies that have been proposed for enhancing statistical efficiency at the design phase for clinical evaluative studies of rare disease treatments include factorial trials and adaptive designs. Factorial trials are designed to test multiple treatments simultaneously using the same study population, thus reducing the overall number of participants needed [ 2 , 33 , 39 , 40 , 46 , 49 , 53 , 57 ]. For example, in a 2 × 2 factorial design participants are randomized to either treatment A or control group A, and then randomized again to treatment B or control group B, which effectively reduces the sample size needed to test these two treatments by 50% because the same participants are being randomized [ 40 ].…”
Section: Resultsmentioning
confidence: 99%
“…Furthermore, combining the results using techniques from meta-analysis can help estimate the average treatment effect and heterogeneity of treatment effects in the population as well as the individual treatment effects for single patients [2022, 2527]. …”
Section: Discussionmentioning
confidence: 99%
“…This has negative impacts, including delayed diagnosis, suboptimal patient care and limited access to treatments, either because they have not been developed, or where available due to high costs and/or lack of government reimbursement. Registries are systems of uniform data collection to evaluate specified outcomes for a population defined by a disease, condition, or exposure, typically spanning different clinical or academic partnerships over an extended time period …”
Section: European Organisation For Rare Diseases the National Organimentioning
confidence: 99%
“…Registries are systems of uniform data collection to evaluate specified outcomes for a population defined by a disease, condition, or exposure, typically spanning different clinical or academic partnerships over an extended time period. 8,9 Registries are particularly important for rare or poorly understood diseases with small patient numbers, complex delayed diagnoses, a propensity for variable standards of care and limited treatment options. 1,2 Registries act as a central networking point for all stakeholders around a particular RD, including patient advocacy groups, researchers, clinicians, industry and Government.…”
mentioning
confidence: 99%