EURODIS Europe Rare diseases in general Patient Patient organizations help to adapt the design of Not specified • (2011) organizations clinical trials to meet expectations, patients’ [21] facilitating adherence and ensuring quality of life is taken into consideration EURODIS Europe Rare diseases in general Patient Patient organizations discuss results with trial Not specified • (2011) organizations sponsors to contribute to the assessment of [21] treatment benefits EURODIS Europe Rare diseases in general Patient Patient organizations provide early information Not specified • (2011) organizations to potential participants to ensure inclusion in a [21] trial Boon Netherlands NMDs Patient The VSN is involved in assisting and initiating Not specified • (2010) organizations clinical trials [46] Black United States Severe myoclonic epilepsy Patient The IDEA League provides researchers with Not specified • (2011) of infancy (SMEI or Dravet organizations access to a large cohort of patients for research [22] syndrome) Black United States SMEI Patient Centres within the IDEA Not specified • League’s Collaborative (2011) organizations Clinical Research and Comprehensive Care [22] Network collaborate to develop clinical research protocols Groft United States Rare diseases in general Patient The Rare Diseases Clinical Research Network Not specified • (2013) organizations (RDCRN) obtains input from patient organizations [23] on the development of informed consent statements, recruitment strategies, and protocols for research conducted within the Network Groft United States Rare diseases in general Patient Patient organizations help the RDCRN to Researchers in the RDCRN rank help with • • (2013) organizations identify patient cohorts and recruit patients for patient recruitment as one of the top three [23] studies conducted by the Network benefits of interactions with patient organizations [154] Seminara United States Urea Cycle Disorders Patient The National Urea Cycle Disorders Foundation Not specified • (2010) (UCDs) organizations is involved in the Urea Cycle Disorders [24] Consortium (UCDC) of the RDCRN The foundation is directly involved in the • development of protocols, consents, content evaluations, and progress reporting in RDCRN studies Patient Not specified Rare diseases in general Patient Patient organizations inform patients of Not specified • Partner organizations opportunities for taking part in clinical trials [20]
EURODIS (2011) [21] Europe Rare diseases in general Patient organizations • Patient organizations help to adapt the design of clinical trials to meet patients’ expectations, facilitating adherence and ensuring quality of life is taken into consideration Not specified EURODIS (2011) [21] Europe Rare diseases in general Patient organizations • Patient organizations discuss results with trial sponsors to contribute to the assessment of treatment benefits Not specified EURODIS (2011) [21] Europe Rare diseases in general Patient organizations • Patient organizations provide early information to potential participants to ensure inclusion in a trial Not specified Boon (2010) [46] Netherlands NMDs Patient organizations • The VSN is involved in assisting and initiating clinical trials Not specified Black (2011) [22] United States Severe myoclonic epilepsy of infancy (SMEI or Dravet syndrome) Patient organizations • The IDEA League provides researchers with access to a large cohort of patients for research Not specified Black (2011) [22] United States SMEI Patient organizations • Centres within the IDEA League’s Collaborative Clinical Research and Comprehensive Care Network collaborate to develop clinical research protocols Not specified Groft (2013) [23] United States Rare diseases in general Patient organizations • The Rare Diseases Clinical Research Network (RDCRN) obtains input from patient organizations on the development of informed consent statements, recruitment strategies, and protocols for research conducted within the Network Not specified Groft (2013) [23] United States Rare diseases in general Patient organizations • Patient organizations help the RDCRN to identify patient cohorts and recruit patients for studies conducted by the Network • Researchers in the RDCRN rank help with patient recruitment as one of the top three benefits of interactions with patient organizations [154] Seminara (2010) [24] United States Urea Cycle Disorders (UCDs) Patient organizations • The National Urea Cycle Disorders Foundation is involved in the Urea Cycle Disorders Consortium (UCDC) of the RDCRN • The foundation is directly involved in the development of protocols, consents, content evaluations, and progress reporting in RDCRN studies Not specified Patient Partner [20] Not specified Rare diseases in general Patient organizations • Patient organizations inform patients of opportunities for taking part in clinical trials Not specified
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