CRITICAL CONSIDERATIONS FOR RARE & ORPHAN DISEASE TRIAL PLANNING
For groundbreaking treatments on rare conditions for which limited or no standards of care or diagnostic procedures have been established, challenges are likely to emerge. Flexible study design and staff who can anticipate and quickly resolve possible issues are keys to success.
This white paper illustrates the value of foresight, flexibility and collaboration among experts in clinical and research disciplines to successfully address challenges, and then benefit from them.
Conducting A Robust Medical Protocol Review:
To properly balance the varied and often competing factors in a trial, a robust protocol feasibility assessment should be conducted. It should include input not only from key opinion leaders and a medical science advisory board, but also from study coordinators; potential investigators; any support clinicians such as radiologists, lab managers and pharmacists; and potential patients or patient advocacy groups.
Conducting A Robust Site Feasibility Review:
With a feasible study design in hand, sponsors can begin evaluating potential study sites. Having the protocol worked out will help evaluators identify needed site personnel and facility capabilities. It will also help potential investigators understand your needs and whether they can meet them