Pfizer, Gilead and others seek clarifications on trial designs outlined in FDA's UC and Crohn's draft guidance

2022-07-20

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Pharmas act to make sure their voices get heard — and before the FDA it’s no different. Especially when it relates to draft guidance. Multiple Big Pharma companies sought tweaks from the FDA on draft guidance related to the development of new drugs for ulcerative colitis and Crohn’s disease. Those two indications are key therapeutic areas for several of the major pharma players. The drafts explain the FDA’s current thinking around how to design clinical trials for these two disease areas, including recommendations for trial population, trial design, and efficacy and safety considerations. Many of the drugmakers’ comments sought additional info, with Pfizer calling on FDA to “[C]onsider including a statement or section regarding Ulcerative Colitis in the pediatric population as well as the potential applicability in this specific patient population. Additionally, consider creating a separate guidance for pediatric ulcerative colitis or IBD.” For Crohn’s disease, Pfizer “encouraged” that controlled active trials be designed to demonstrate superiority to an approved candidate “if that is a development goal.” Novartis followed up, wanting to replace the term “co-primary endpoints” in trials with “efficacy endpoints” in Crohn’s disease. On top of that, Novartis suggested removing co-primary analyses and replacing them with efficacy analyses to demonstrate statistical significance in the clinical endpoint and/or endoscopic endpoint (instead of originally both). Roche/Genentech also pitched in — and they had a bunch of suggestions. These included broadening a recommendation to pre-specify methods to handle missing data, aligning standards of remission based on study design, and recommending that sponsors also consider non-inferiority (NI) design against an approved therapy, after reaching an agreement with FDA on an acceptable NI margin. Next was Gilead, which had a bunch of clarifying questions — including how to distinguish between patients who are biologic naïve versus those patients who are advanced treatment naïve, clarify different types of terminology, and what exactly is the FDA’s expectation regarding “a balanced representation of patients (biologic experienced vs. naïve) as it relates to subgroup analyses.” Among others, Regeneron called on FDA to recognize different endotypes in Crohn’s disease and ulcerative colitis, plus balance the “representation of subjects who have never received treatment with a biologic and subjects who have failed prior therapy with one or more biologics or other advanced therapies.” Ulcerative colitis has been an indication once dominantly controlled by AbbVie, thanks to sales kingpin Humira.

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