- Adjusts based on unique rare disease study conditions
- Addresses challenges via anticipation and adaptation
- Collaborates to further and reshape best practices
ARG knows from experience that the willingness to be flexible has fueled our success in rare disease clinical trials, resulting in approvals with over $3B in sales.
Challenges are likely to emerge in projects designed to develop groundbreaking treatments for rare conditions. Often, these disease states have limited established standards of care and diagnostic procedures. Flexible study design as well as staff who anticipate and quickly resolve issues are keys to consistent study management.
In an ongoing ARG study of a drug for a rare eye disease in which blinding fibrous membranes grow under children’s eyelids, a small number of patients were treated at two large referral centers in the US and Europe.
The first interim analysis revealed an issue: measuring membranes manually on squirming children was producing inconsistent readings between sites, and even from the same patients at different visits.
For this trial, the potential for greater variability was not anticipated in the protocol given the lack of an established scale for fibrous membrane growth. An idea was used to require digital photographs at every patient visit. Further, a central reader was brought in to evaluate the images by measuring the lesion areas electronically.
The resulting data enabled a robust statistical analysis, supporting the study’s original lesion mass reduction endpoint. Additionally, the data highlighted differences in baseline disease states between the eyes of many patients and separate treatment responses.
Analyzing both eyes effectively doubled the study sample, which was initially defined as individual patients rather than individual eyes. This yielded an unexpectedly detailed and valuable understanding of the relationship between disease severity and treatment response.
ARG fosters collaboration among experts in clinical and research disciplines to successfully address and overcome challenges.