Rare Disease Day

R&D

Net Treatment Benefit in rare disease trials: Aligning trial...

Net Treatment Benefit provides a formal framework to integrate multiple prioritised outcomes into a single measure of overall treatment effect.

R&D

How innovative trial approaches are advancing rare disease r...

Innovative trial designs that harness data augmentation, RWD, and the power of AI are enabling a new era of rare disease research.

Market Access

What rare disease commercialisation in Europe teaches us - a...

Rare disease launches are the ultimate stress test for European commercialisation.

Oncology

Collaboration & personalisation in rare disease R&D, with AO...

For Rare Disease Day, in an AOP Health-sponsored podcast Melissa Fellner, VP of Global Therapeutic Areas, Commercial Operations, discussed their work.

News

FDA maps new regulatory route for ultra-rare diseases

The FDA has sketched out its 'plausible mechanism' regulatory pathway for individualised therapies that treat ultra-rare diseases.

News

FDA starts review of Regeneron's drug for rare disease FOP

Can Regeneron's drug for ultra-rare bone disease FOP pick up the baton from Ipsen's Sohonos, whose growth has stalled?