Rare Diseases

PRA is an ideal partner to guide your rare disease clinical trials. With 80+ trials in this area, we possess a clear understanding of the challenges and critical success factors necessary to successfully develop drugs for rare disease patients. Our relevant experience encompasses Phase I-IV studies in 10 therapeutic areas and a wide range of indications.

 
PRA’s Approach to Rare Disease Development Programs

Rare disease trials require a customized approach based on the disease specifics and study goals. Through our experience, PRA has developed an effective planning and execution process that includes:

  • Understand patient pathways
  • Site Identification/Selection
  • Site Awareness and Support
  • Ongoing Patient Retention Support
 
Rare Diseases Study Experience

In the last five years, we have supported 73 clinical trials across all phases and in a variety of rare disease indications, involving 16,200+ patients at 3600+ sites. We’ve outlined our experience in the table below. 

 
Value Added Solutions in Rare Diseases

PRA has identified several critical success factors for rare disease studies:

  • PRA’s evidence-based methodology provides access to proprietary databases, feasibility studies and key investigator relationships to enhance patient recruitment.
  • Strong partnerships with relevant patient advocacy groups create stronger study awareness and support.
  • Quality subject support materials demonstrate the benefits of participation to subjects, leading to increased motivation to enroll.
  • A core set of study-branded tools help sites identify potential patients and educate potential patients about the study and its requirements.
  • PRA’s Patient Access and Retention Services (PARS) team partners with best-in-class patient recruitment organizations to develop customized programs.