AGI members and invited external experts including C-Path met in Bonn on Jan 24th and 25th 2023 to kick-off the AGI program on the development of a generally accepted ataxia rating scale.
After two days of discussion, consensus on the assessment of the current state of the art, unmet needs and work program could be reached. There was consensus on the following major points:
- Two major contexts of use for clinical outcome assessment (COA) should be considered: (1) capturing of global disease progression, and (2) capturing treatment response in early stage trials.
- Given the enormous heterogeneity of ataxia disease, Clinician Reported Outcomes (ClinROs) for the entire population of ataxia patients should be developed.
- For any ClinRO, the elicitation and use of a valid Concept of Interest has to be formally shown following procedures recommended by FDA
- Regarding SARA, there are strengths (e.g. large amount of data from natural history studies, good performance in item response theory analysis) and weaknesses (e.g. lack of anchoring in patient experience, questionable meaningfulness of single items)
- There was consensus that SARA or a modified version of SARA are not appropriate for capturing treatment response in early stage trials.
- AGI should consequently concentrate activities on two main areas:
- Modification of SARA for capturing global disease progression and anchoring of other COAs
- Development of a new ClinRO with optimised sensitivity for use in early-stage trial ClinRO
Results of this AGI consensus conference will be shared with C-Path (CPTA) to allow the coordination of AGI and CPTA activities.