Neonatal Drug Development Could Benefit From Rare Disease Experience
Early parent involvement in drug development and clinical trial design could improve recruitment and outcomes, stakeholders argue.
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Rare disease advocates push for National Academies of Sciences, Engineering and Medicine study on how or whether ultra-rare should be defined, which could create legislative pressure. In an interview, Ultragenyx CEO Emil Kakkis suggests patient thresholds, accelerated approval triggers.
Advocates also worry that long follow-up times could prevent participation in future clinical trials.
Just because a measure is relevant to the patient community does not mean it is capable of capturing meaningful change in a clinical trial, US FDA officials say at a meeting on mitochondrial disease drug development.