All clinical trials are challenging. But when the protocol is evaluating the safety and efficacy of rare disease treatment, the challenges are even greater. This applies to all aspects of the trial, especially the logistics around randomization and trial supply management (RTSM).
For example, while it’s true that in all clinical trials, every patient matters, this takes on new meaning in studies of rare diseases. As there are not many people living with the diagnosis, identifying, enrolling, and keeping patients engaged in clinical trials is critical. Trial sponsors can’t risk failed visits, which could lead to a patient dropping out because the drug wasn’t available for a dispensing visit.
And rare disease trials often require many investigative sites to recruit a small number of patients. Combine this with the reality that rare disease drugs are typically very expensive to produce, and it’s easy to understand the importance of reducing excessive drug wastage during development.
So, ensuring the right sites have the right amount of drug at the right time – without over-producing and shipping drugs to all sites – is critical to a rare disease development program’s success.
In this first series installment, Calyx’s Malcolm Morrissey outlines these and other RTSM factors sponsors should consider and demonstrates how working with a reliable interactive response technology (IRT) provider with subject matter expertise, customizable designs, and dedicated study support can make a big difference in rare disease trials.