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Using A Natural History Study As An External Control In Rare Diseases: Ipsen Offers An Example

 
• By Ramsey Baghdadi

Although the advisory committee was uncomfortable with the external control, US FDA was reassuring over the approach – which eventually secured a ‘yes’ vote from the panel on palovarotene for FOP.

Dinosaur and child hand
How close a match do natural history studies need to be? • Source: Shutterstock

Ipsen SA was facing an uphill battle last month at the Endocrinologic & Metabolism Drugs Advisory Committee's review of palovarotene for a rare bone tissue condition.

Key Takeways

  • Ipsen didn’t follow FDA’s advice and conduct a placebo-controlled trial, and the study ended up failing.

  • But the natural history study (NHS) the sponsor used as an external control met many of the criteria that the agency has for such studies.

  • FDA’s satisfaction with the NHS encouraged advisory committee endorsement of palovarotene for heterotopic ossification in patients with fibrodysplasia ossificans progressiva

The company is seeking approval of the drug for treatment of heterotopic ossification (HO) in adults and children with fibrodysplasia ossificans progressiva (FOP)

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