— Lack of choices for fallen back illness versus unpredictability about supporting information
by Charles Bankhead, Senior Editor, MedPage Today
October 3, 2023
An FDA advisory committee on Wednesday will wade into a complex contrast of a high unmet requirement in oncology versus unpredictability about whether a drug has “significant proof” of efficiency.
At the center of the balancing act is an approval application for eflornithine (difluoromethylornithine; DFMO) for high-risk pediatric neuroblastoma. The Oncologic Drugs Advisory Committee (ODAC) will weigh in on whether a single, properly designed potential research study with an external control group– plus nonclinical, exploratory, and expanded-access information– makes up adequate proof to advise approval.
Randomized regulated trials are the gold requirement for examining time-to-event endpoints, FDA team member kept in mind in their instruction file. Information from the big potential single-arm trial produced information recommending “significant enhancement over a benchmark historical control.”
“The usage of an externally managed trial to support a marketing application might be appropriate in the setting of an unusual illness with a distinct nature and bad diagnosis if the anticipated treatment result is approximated to be big, especially in a setting where conduct of an RCT [randomized controlled trial] might be infeasible,” FDA personnel composed.
“There are intrinsic restrictions in translating the approximated treatment impact of a single nonrandomized, externally managed trial,” they included. “These prospective predispositions might affect the projected treatment impact in manner ins which might not be straight measurable.”
In 2019, the FDA provided market assistance for showing “significant proof of efficiency.” The assistance specified that “in basic, substantiation of a drug’s efficiency acquired with 2 trials, specifically with complementary style, will supply more persuading proof of efficiency than would a single trial … Under specific scenarios and constant