Disease natural history data is critical to rare disease medical product development where information may be severely limited. In fact, this type of data is often required or encouraged by regulators as it can help with interpretation of both clinical trials and real-world experience post-approval.
drug development for rare diseases
Pharmaceutical and biotech sponsors still face hurdles when seeking FDA approval of new medications for rare diseases. UBC examined the FDA’s draft guidance related to the development of these drugs and presented the results in a poster at ISPOR.