VeraSci attended ISCTM’s 2018 autumn conference, Advances in Clinical Trial Technology: Impact on methodology and Signal Detection, October 15-16 in Marina del Rey, California.

The conference highlighted many topics including:

  • Innovative uses of technology for measuring outcome in clinical trials
  • Designing trials using gene therapy for neurological disorders with a clear genetic basis
  • Biomarkers in psychiatric drug development
  • Novel technologies to identify underlying molecular pathology in neurodegeneration

The full-day session entitled “Innovative Uses of Technology for Measuring Outcomes in Clinical Trials” addressed a wide array of issues associated with the use of new technologies to assess cognition, symptoms and functioning in clinical trials. VeraSci CEO, Richard Keefe, PhD co-chaired this session alongside Alexandra S. Atkins, PhD, VP of Scientific Development at VeraSci, who participated as a panel speaker.

VeraSci presented two posters as part of the working groups and poster session.

There are many assessments of negative symptoms in schizophrenia with no clear consensus on the primary scale to use for this population as the constructs and general utility of each scale varies. Anzalee Khan, PhD, Senior Director of Data Science at VeraSci, presented “Evaluation of Relationship Between Negative Symptoms Scales in an IRT Framework.” The presentation used Item Response Theory to assess the quality and utility of the CAINS (Clinical Assessment Interview for Negative Symptoms), BNSS (Brief Negative Symptom Scale), PANSS Negative Factor (Positive and Negative Symptoms Scale), and the NSA-16 (Negative Symptom Assessment-16). Results showed that the BNSS, CAINS and NSA-16 were more informative for subjects with moderate severity, compared to those with low or severe severity levels. In contrast, the PANSS NSFS was equally informative for all levels of severity, although the BNSS and CAINS have distinct conceptual advantages over the PANSS NSF and NSA-16 and captures a broader spectrum of negative symptoms.

Carmen E. Sanchez, PhD, Senior Director of R&O at VeraSci presented “Common Rater Errors for Assessment in Pediatric Rare and Orphan Disease Trials.” The presentation investigated the impact of data quality review, by quantifying the number of scoring and administration errors typically found in a paper-based review of cognitive and adaptive behavior assessments, particularly tools that are sensitive to neurocognitive change in MPS disorders. Results showed that expert data review of cognitive and adaptive behavior assessments demonstrated the high frequency of administration and scoring errors committed by raters in R&O disease clinical trials. The impact of errors is considerable, leading to the inaccurate calculation of endpoints across the subtest and domain levels. A central rater serves a critical role in R&O disease trials to identify errors of scoring and correct them for accuracy. Common errors point to areas rater training could address. Specifically, VABS-II–The use of computer-based scoring programs to assist in score summation, BSID-III–Explicit training on the double basal guideline and its application to scoring, KABC-II–Provision of clear guidelines for scoring. Data quality review and remediation can have large positive effects on signal detection in R&O trials.

Poster: Evaluation of Relationship Between Negative Symptoms Scales in an IRT Framework

Poster: Common Rater Errors for Assessment in Pediatric Rare and Orphan Disease Trials

Presentation: Real-World Challenges in Developing Digital Cognitive Tools: Two Recent Examples

Presentation: Innovative Uses of Technology for Measuring Outcomes in Clinical Trials: Panel Comments