Real-World, Natural History Data May Supplement, Replace Placebo Data in DMD Trials
The largest-ever multinational, multi-collaboration study in Duchenne muscular dystrophy (DMD) has found that real-world data (RWD) and natural history data (NHD) are comparable to data from placebo-controlled trials, suggesting RWD and NHD could either supplement or replace placebo controls in DMD studies.
The DMD study was conducted by the multistakeholder, global research coalition Collaborative Trajectory Analysis Project (cTAP), which was established in 2015. A total of 383 patients with DMD randomly assigned to receive placebos in six clinical trials were compared with data from a real-world setting comprising 430 patients from five clinical registries in the U.S. and Europe.
Outcomes, including a six-minute walk distance assessment, were fairly consistent between patients in the placebo-controlled trials and patients from the real-world and natural history studies. According to the cTAP investigators, the potential for bias was low, suggesting that RWD and NHD in trials could be considered valid and may be usable in drug trials for other diseases.
The study can be found here: https://bit.ly/3mdEl6G.