Health Innovators – Jennifer Schranz

In the rare and ultra-rare disease space, the paucity of both data and patients makes everything about research and development more complicated. In today’s Health Innovators, Jennifer Schranz, SVP and global head of rare diseases at Ipsen, talks to editor-in-chief Jonah Comstock about those challenges and how Ipsen approaches them.

Because patient populations are so small, traditional study designs are not only hard to conduct, but they also require a disproportionate number of patients to be in the placebo group, preventing them from getting the benefits of new treatments.

They dig into the specific example of fibrodysplasia ossificans progressiva (FOP), a disease area in which Ipsen has recently had challenges getting through FDA approval, taking a look at what’s happened there and what lessons can be learned. They also touch on the reimbursement challenges in rare disease, especially with novel one-time therapies.

Check out the video below for a nuanced conversation about a complex topic, but one that’s increasingly important for patients and pharma companies alike.