A market research report issued at the beginning of the year forecast that the market for ALS drugs in several key countries would exceed $1 billion over a 10-year period.
The study, by Personal Genome Diagnostics and academic researchers, was able to predict responses to checkpoint inhibitor therapy in patients with MSI-H and high tumor mutational burden.
Hear executives from Quantum Health, Surescripts, EY, Clinical Architecture and Personify Health share their views on digital transformation in healthcare.
In a guidance issued Wednesday, the agency said randomized, controlled trials of cancer drugs should only use placebo in cases like when surveillance is standard of care. A bioethics expert said such principles have long existed, but it was good to see them in an official guidance.
The agency warned against use of AbbVie's Mavyret, Gilead Sciences' Vesovi and Merck's Zepatier in patients with moderate to severe liver disease amid reports of 63 patients with liver decompensation, with some cases being fatal.
The Wall Street Journal reported that the agency was focusing on the two-month delay between the March 14 discovery of data manipulation by Novartis' AveXis unit regarding the therapy Zolgensma and its May 15 launch of a formal inquiry into the matter.
Several FDA approvals - including ones going against AdCom votes - have attracted controversy and a perception of lowered standards at the agency. However, some see a long-standing institutional tension between patient protection and speedy approval, with the latter having a net positive effect.
Closing cancer health equity gaps require medical breakthroughs made possible by new funding approaches.
Among Kaiser union workers in California, more than 37,000 ballots were cast in support of a walkout compared to only 867 in opposition.
In a statement Tuesday, an FDA official wrote that subsidiary AveXis disclosed manipulation of mouse data for the gene therapy Zolgensma, which the agency approved in May for spinal muscular atrophy. The official wrote that civil or criminal penalties were possible.
Pfizer, which had been running the trial of rivipansel in SCD, reported that it had failed on its primary and key secondary endpoints.
The administration proposed two pathways, one that would allow importation of some drugs from Canada via pilot projects, and another that would allow manufacturers to import from other countries and sidestep distribution contracts. PhRMA's president criticized the proposal as "dangerous."
The progress in artificial intelligence (AI) is reshaping patient care, across various dimensions, from facilitating faster discharge to curating treatment plans and suggesting lifestyle changes.
Although the agency goes against about one-fifth of AdCom recommendations, a study published Sunday only found one reliable variable predicting when that would happen.
An expert pointed to the draft guidance's lack of distinction between immune-activating bispecifics and those that do not activate the immune system, as well as potentially burdensome trials industry may have to conduct. The comment period ended Tuesday.
The DOJ claims that IBM subsidiary Cúram Software misrepresented its ability to meet specified technical requirements in its contract to develop Maryland's health insurance exchange website and IT platform.
The agency said it would likely require such drugs to undergo the traditional approval pathway, as there is insufficient evidence to support giving drugs accelerated approval based on surrogate endpoints.
Gottlieb previously worked at NEA from 2007 to 2017, when he was appointed as commissioner of the FDA. He stepped down from that role in March, also returning to another previous employer, the American Enterprise Institute.