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Precision Medicine and Rare Diseases: Innovative Approaches to Clinical Trials



Hari Radhakrishnan of Medicover gave a presentation at COG Europe 2023 highlighting innovative approaches to conducting clinical trials for precision medicine in rare diseases.



Here are some of the key takeaways:


Rare diseases present unique challenges for clinical trials due to small patient populations, disease heterogeneity, and difficulties with patient recruitment and retention. Creative trial designs are needed. Basket trials allow testing a drug or therapy across different rare diseases that share common genomic or other biomarkers. This increases sample size and efficiency. Umbrella trials test multiple therapies within one disease population, accelerating drug development and requiring smaller sample sizes.



Alternative trial designs like crossover, delayed start, and randomized withdrawal can be more ethical and economical with small patient pools. Surrogate biomarkers, continuous outcomes, and composite outcomes help address difficulties in measuring efficacy with small heterogeneous samples.


Patient engagement through advocacy groups and decentralized at-home trials improves recruitment and retention. Natural history studies identify responsive subgroups. Regulatory incentives like breakthrough designation and compassionate use programs accelerate market access for rare disease therapies.


A "one-stop shop" model with comprehensive in-house capabilities helps rare disease drug developers navigate unique complexities. Harmonization, patient-centricity, and collaborations will further advance precision medicine for rare diseases.



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