Our orphan team is led by PSR Orphan Experts (PSR), Ergomed’s rare disease subsidiary. PSR is one of the few companies exclusively focused in orphan disease drug development and recognised as a leading expert in assisting biotech and pharma companies in the rare disease niche.  Through our Site Management model and Study Physician Team Support, we find the hard to locate patients around the globe and work with the investigative sites to create the best designs to maximise clinical programs and registries.

Orphan drug development is Ergomed’s core strength, and we are committed to helping those in need. Our expert team allows our clients to implement patient driven solutions in their clinical trials, and we believe that patients will define our future landscape, including the rise of both precision and preventative medicine.

Following are the key factors that set Ergomed and PSR’s joint expertise apart:

  • Unique insight into orphan drug programs to accurately address the specific challenges and project needs that rare disease trials require in order to provide an innovative and solid infrastructure
  • Extensive experience in innovative regulatory & clinical development (Pharma, biotech, CRO, Academia)
  • Global access to hard-to-find patients in under-served regions
  • A regulatory consultancy service to provide complete support for orphan drug related interactions with the EMA and FDA (e.g. Orphan Drug designations, PIP, Protocol Assistance/Scientific Advice)
  • A unique site management model that allows for faster recruitment of quality patients and site coordination
  • Expertise in Natural History Studies to better understand the etiology and progression of the disease, which allows for the setting up of crucial endpoints in the development process.
  • Complete staffing solutions specialising in placing team members with orphan drug experience
  • Unique access to patients in the Middle East and North African regions, which has the highest prevalence of genetic disorders in the world