P.29 Accelerating clinical development of new therapeutics with patient data: evidence from the collaborative Trajectory Analysis Program (cTAP) in DMD

Abstract

Patients with DMD and their families are generous in giving their consent to their data being used to advance research that they hope may help lead to new treatments. Although it is widely believed that curated patient data are invaluable to both research and drug development, the literature on the use and value of patient data in drug development is sparse. We conducted a retrospective analysis of studies conducted on longitudinal data from 3,000+ patients conducted by cTAP and collaborators over a 6 year period from 2015 –2021. Studies were categorized by i) the scientific purpose of the study, ii) the clinical trial problem that studies were designed to solve. A total of 78 analytical studies had been conducted. The annual number increased almost an order of magnitude from 2 in 2014 to 18 in 2021 paralleling the growth of cTAP membership which increased more than 3-fold. The types of analyses conducted on cTAP are: a) characterizing trajectories of disease progression (14%), b) developing prognostic models to predict progression (27%), c) determining the minimal clinical effect and clinical meaningfulness of endpoints commonly used in clinical studies (22%), d) assessing the consistency of progression between placebo arms and real world data natural history (16%), e) quantifying the impact of dystrophin genotype on disease progression over the course of a trial (11%), and f) development of tools (11%). The majority of studies were focused on clinical trial design (65%) and interpretation (24%). Overcoming trial design and analysis challenges can make the difference between regulatory approval of a new therapeutic and acceptance by heath authorities and payors versus the requirement to conduct a new clinical trial. The value of analyzing patient data in cTAP studies has the potential to accelerate clinical development by markedly improving the probability that a clinical trial will deliver unequivocal evidence of efficacy (or lack thereof).