GENETIC RARE DISEASE PROGRAM TO SUPPORT REGULATORY SUBMISSION

CASE STUDY

PHASTAR provided a team of statisticians and programmers to deliver the analysis and reporting of a program of four trials in a genetic rare disease, and the corresponding integrated summary of efficacy and safety, to support a regulatory submission to the FDA. There were four studies comprising twelve, one, two, and three patients. The one and two patient studies were pilot studies that included historical data from hospital notes that had been transcribed into a new clinical trials database (after obtaining informed consent from the patients). The three patient study was a compassionate use study that had been initiated due to patient requests.

There were a number of time windows of interest. Initially PHASTAR reported the first three years from the 12-patient study and was subsequently requested to report on time windows of both 4-8 and 0-8 years. PHASTAR initially created analysis plans and mock output shells for the studies. All statistical work including programming was by PHASTAR. Understanding all biomarkers collected for the study was crucial and everyone was briefed on this aspect.

Endpoints were also derived such as the presence of genetic modified cells and presence of lymphocyte ADA enzyme activity to name a few. A Poisson regression analysis was run on the number of severe infections comparing infection rates before gene therapy and after gene therapy, the definition of before and after was critical in ensuring the correct results were obtained. All safety data was summarized including exposure to gene therapy. Growth charts were plotted for all subjects, including individual laboratory and biomarker data. All work was independently checked for quality and verified. An integrated CDISC dataset was created comprising all 18 patients, and all studies and integrated reports were created from the integrated datasets. PHASTAR additionally provided all FDA data packages to support the submission.

As well as providing all reports and data required for the submission, PHASTAR also provided a team to help answer questions from the regulators.

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