A draft FDA guidance for heart failure endpoints could spark investment and accelerate development of cardiovascular therapies. But companies still don’t have a clear sense of when and whether they’ll need to gather mortality data -- a difference-maker for whether they should enter the space.
Settling the confusion should be a top priority for the July 26 meeting FDA is holding to discuss clinical endpoints for trials in heart failure.
FDA issued the draft guidance on June 27 to spur development of new cardiovascular agents, a field that has been stymied by the misconception that new heart failure treatments must show a mortality benefit to be approved. Clinical trials can require more than 10,000 patients and take over five years to demonstrate that the number of deaths in the treatment arm is significantly lower than the control.
In the guidance, FDA set the record straight and stated that new heart failure therapies do not have to show a mortality benefit or a reduction in hospitalizations to be approved. “A drug that improves symptoms or function when added to standard of care would be valuable even if it did not improve survival or hospitalization,” FDA stated in the guidance.
But the guidance left a gray area where approval is based on improvement of symptom or function.
In some cases, data still need to be collected to show that even if a therapy produces no benefit on mortality, it doesn’t do any harm.
Although FDA signaled there might be circumstances where it can accept a certain level of mortality risk, depending on the magnitude of symptomatic or functional benefits, it didn’t define clear boundaries on how much risk was