Picturing the hidden patient

Overcoming challenges in sizing and segmentation for rare diseases and niche treatments

Understanding the size and composition of addressable patient audiences is mission-critical for developers of niche treatments and drugs for rare diseases – and often highly difficult.

How can companies developing drugs for rare diseases and those afflicting smaller patient groups understand patient audiences that are highly underdiagnosed, miscoded and absent in the extant medical literature?

There’s a lot riding on this fundamental question, which can inform everything from go/no go decisions to clinical trial design to value generation and stakeholder engagement efforts.

Download this executive brief to learn:

  • Key challenges associated with sizing and segmenting smaller patient groups
  • The risks of having inaccurate or incomplete views
  • Creative analytical approaches to better understanding patients