Participant-funded clinical trials on rare diseases.
Sign Up to like & getrecommendations! Published in 2020 at "Anales de pediatria"
DOI: 10.1016/j.anpede.2020.03.005
Abstract: The development of medicines for certain rare diseases can be cut short by lack of funding. In certain cases the patients themselves, or their relatives, occasionally fund the clinical trial in which they will be… read more here.
Keywords: funded clinical; rare diseases; clinical trials; trials rare ... See more keywords
A Bayesian adaptive design for clinical trials of rare efficacy outcomes with multiple definitions
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DOI: 10.1177/17407745221118366
Abstract: Introduction: Bayesian adaptive designs for clinical trials have gained popularity in the recent years due to the flexibility and efficiency that they offer. We consider the scenario where the outcome of interest comprises events with… read more here.
Keywords: clinical trials; adaptive design; bayesian adaptive; trials rare ... See more keywords
Bayesian adaptive trials for rare cardiovascular conditions.
Sign Up to like & getrecommendations! Published in 2018 at "Future cardiology"
DOI: 10.2217/fca-2017-0040
Abstract: Escalating costs of cardiovascular trials are limiting medical innovations, prompting the development of more efficient and flexible study designs. The Bayesian paradigm offers a framework conducive to adaptive trial methodologies and is well suited for… read more here.
Keywords: cardiovascular conditions; study; bayesian adaptive; trials rare ... See more keywords
A survey of registered pharmacological clinical trials on rare neurological diseases in children in 2010–2020
Sign Up to like & getrecommendations! Published in 2022 at "Frontiers in Pediatrics"
DOI: 10.3389/fped.2022.963601
Abstract: Objective To clarify the current state of methodology of clinical trials for rare neurological diseases in children, and to provide a basis for the further optimization of the trial design. Methods Data of clinical trials… read more here.
Keywords: clinical trials; methodology; trials rare; rare neurological ... See more keywords
Characteristics of Early Phase Clinical Trials for Rare Cancers: Insights From Interviews With Stakeholders
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DOI: 10.3389/fphar.2022.775217
Abstract: Background: Rare cancers occur with an incidence of no more than six cases per 100,000 people according to the definition used by the Surveillance of Rare Cancers in Europe project. For a variety of reasons… read more here.
Keywords: phase rare; trials rare; rare cancer; phase ... See more keywords