FDA shifts trial standard
Reports say the FDA is moving to a default that one adequate and well‑controlled clinical trial will generally suffice for drug approval, replacing an expectation of two studies. (pharmexec.com) Travere Therapeutics secured FDA approval for Filspari in a rare kidney disease, showing the agency continues to approve niche indications. (medcitynews.com) Separately, the FDA cleared an at‑home HPV self‑collection kit and approved the BD Onclarity HPV assay to expand cervical‑cancer screening access. (cancernetwork.com)
The Food and Drug Administration is moving toward approving more drugs on one main clinical trial, not two, if other evidence backs the result. (pharmexec.com) A clinical trial is the formal test that asks whether a drug works in people, and “adequate and well-controlled” is the agency’s term for a study with a comparison group, preset endpoints, and methods meant to limit bias. Food and Drug Administration officials Martin Makary and Vinay Prasad said in February 2026 that one such study will be the agency’s default position for new drugs and other novel health products, with confirmatory evidence added around it. (fda.gov) (usnews.com) The legal opening for that approach is not new. The Food and Drug Administration’s 2019 draft guidance says the 1997 Food and Drug Administration Modernization Act allowed effectiveness to be shown with one adequate and well-controlled trial plus confirmatory evidence, even though the agency had generally interpreted the standard as two trials. (fda.gov) The new piece is the default. Makary and Prasad argued that better biomarkers, stronger statistical methods, and more targeted biology now let the agency judge some drugs without asking companies to run a second pivotal study that repeats the first. (pharmexec.com) (biopharmadive.com) The shift lands after years in which the agency already used single-study flexibility most often in oncology and rare diseases. Industry coverage of the February change said the practical effect could be larger for more common diseases, where sponsors had still expected to bring two pivotal trials. (pharmexec.com) (ajmc.com) This week’s approval of Travere Therapeutics’ Filspari shows the Food and Drug Administration is still clearing narrow, hard-to-study indications while it rewrites the broader rulebook. On April 13, 2026, the agency approved Filspari, or sparsentan, to reduce proteinuria in adults and children age 8 and older with focal segmental glomerulosclerosis without nephrotic syndrome. (ir.travere.com) Proteinuria means too much protein leaking into urine, a sign that the kidney’s filter is damaged, and focal segmental glomerulosclerosis is a rare scarring disease in that filter. Travere said Filspari is the first Food and Drug Administration-approved medicine for that disease, and said its Phase 3 DUPLEX study showed a 46% reduction in proteinuria at Week 108 versus 30% for irbesartan in the overall study population. (ir.travere.com) The agency also used its device pathway on April 8, 2026 to widen cervical-cancer screening outside a clinic. Waters said the Food and Drug Administration cleared the Onclarity human papillomavirus self-collection kit and approved the BD Onclarity human papillomavirus assay with extended genotyping for at-home use. (ir.waters.com) Human papillomavirus is the virus behind nearly all cervical cancers, and screening looks for the virus before cancer develops. Waters said about 60% of cervical cancer cases occur in people who are unscreened or under-screened, which is why at-home collection has become a target for public health programs. (ir.waters.com) The next test for the Food and Drug Administration is not whether it can cite one trial instead of two on paper, but how consistently it explains the extra evidence that makes one trial enough. That explanation will shape how drugmakers design studies, how doctors read approvals, and how patients judge what an approval means. (fda.gov) (pharmexec.com)
