Washington, D.C. – U.S. Reps. Randy Weber (TX-14), Brian Babin (TX-36), and Dan Crenshaw (TX-02) sent a letter to the Food and Drug Administration’s (FDA) Division of Rare Disease and Medical Genetics (DRDMG) to urge the FDA to reconsider clinical trial designs within the population of certain fatal heterogeneous degenerative diseases so that patients do not have to choose between medication that is slowing the progress of their disease and participating in FDA trials.
“Placebo requirements are necessary in most trials, however, in the case of certain fatal heterogeneous degenerative diseases—such as CLN3 Batten Disease, Pompe Disease, and Fabry Disease—with limited patient populations and no available treatments, the use of placebos can discourage patient involvement,” the letter states. “We have heard from parents of children with CLN3 Batten Disease and other rare diseases that would be willing to participate in clinical trials but are hesitant to remove their children from drugs that are significantly slowing down the progression of this disease.”
“We are urging FDA to reconsider the clinical trial for these children,” said Rep. Weber. “They shouldn’t have to choose between medication and participating in FDA trials. These children don’t have years to wait for treatment; instead, we should do everything we can to allow them a chance to fight this battle. I look forward to their response.”
Read the letter here.
Original source can be found here.
