Sponsors making drugs for rare diseases in Australia who fail to file for approval within six months of being granted orphan designation will see their designation disappear, the Therapeutic Goods Administration has decided. The new procedure will be problematic for orphan drug sponsors and might even make it more difficult for them to use the new expedited pathways the agency... 

A public consultation on a proposed version of the reforms in 2016 had raised concerns among drug makers who were worried about designations lapsing too soon; previously, orphan designations lasted indefinitely. The TGA’s original proposal was for designations to lapse automatically after three to six months, but in April this year it settled on a six-month deadline after considering the feedback to the consultation.

According to the responses to the consultation, drug sponsors generally agreed with the concept of orphan drug designations lapsing after a set period of time, but most of them said three to six months would be too short. Industry body Medicines Australia said: “We suggest instead that the period before which designations lapse be 12 months at a minimum.”

From the editors of Scrip Regulatory Affairs.