PHARMAVOICE – Selecting the right endpoint for a clinical trial can make or break a drug’s success. But it can also exclude unique patient groups with high unmet needs, especially in the rare disease space.
For example, drug developers often exclude non-ambulatory patients from neuromuscular studies because they can’t measure their progress using common endpoints like the six-minute walk test or the four-stair climb. BridgeBio took a different approach, however, when the company designed its phase 2 study for BBP-418, an oral investigational treatment for limb-girdle muscular dystrophy type 2I/R9. At the urging of the LGMD patient community, the trial’s participation criteria included both ambulatory and non-ambulatory patients, according to patient advocate Kathryn Bryant Knudson.
That change might not have happened without high involvement with a patient advocacy group.
“That is a clear example of where they were listening to the patient voice,” said Knudson, CEO and founder of the LGMD patient advocacy organization The Speak Foundation who is also an LGMD patient herself. “We wanted to have non-ambulatory patients to have the opportunity to take BBP-418.”
With more than two decades of experience in public relations and journalism, Mike is a strategic communications leader who focuses on media relations and reputation management.