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Rare Diseases: Collaboration in Orphan, Rare and Pediatric Drug Development by Nazim Kanji and Huw Jones

Child with her mother and a healthcare provider in a clinical setting

In this Pharmafile Magazine article Nazim Kanji and Huw Jones explore the orphan drug development and the benefit of CRO/CDMO collaboration, specifically in terms of pediatric rare diseases.

The development of therapeutics for the treatment of rare diseases can present several challenges to drug developers. The number of rare diseases that impact children make it clear why there is a demand for more pediatric medications that are appropriately designed and optimized for young patients.

Nazim Kanji and Huw Jones discuss benefits of CRO/CDMO collaboration and address the unique challenges of developing treatments for orphan, rare, and pediatric diseases. These partnerships help in creating patient-centric dosage forms, speeding up clinical trials, and customizing manufacturing to meet patient needs. Regulatory incentives in the US and EU support these efforts, ensuring timely and effective treatments for conditions that predominantly affect children.

To read more, check out the full article here on the Magazine Pharmafile website.

 

 

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