A new draft guidance on rare disease trials may help sponsors ease the tensions between the need for robust, scientifically sound data and the small patient populations that they’re hoping to treat.
The guidance offers sponsors advice on filling the gap by using such tools as natural histories, surrogate biomarkers, external controls and early randomization but perhaps most importantly, offers a new section on safety.
As the partial government shutdown entered a record-setting fourth week, FDA Commissioner Scott Gottlieb said it was no longer “business as usual” at the agency — calling the shutdown “one of the most significant operational challenges in FDA’s recent history.”
Over the past few weeks, Gottlieb has announced multiple shifts from normal at the FDA, including that new INDs sent to the FDA won’t be considered “accepted” while the shutdown continues. Normally, INDs are considered automatically approved if the FDA doesn’t respond within 30 days. The FDA announcement stops the legal clock.
The FDA is challenging drug sponsors to come up with their own ideas for identifying and gathering the kind of patient experience data that can bring ordinary people closer to the clinical trial process without sacrificing safety or efficacy.
The agency, in a draft guidance released in late December, is encouraging sponsors to propose new draft guidances that focus on points in a treatment or device’s lifecycle that “could be particularly informed by” patient’s experiences.
The FDA last week set out a path for clinical researchers aiming to use biomarkers as a way to increase their trials’ timeliness and efficiency.
A new draft guidance, “Biomarker Qualification: Evidentiary Framework,” explains what steps researchers must take to prove to the agency that their proposed biomarkers are valid indicators of trial results.
The FDA has issued its long-awaited plan on real-world evidence, a document that many in the industry read as the beginning of a conversation.
The FDA doesn’t see real-world evidence as a substitute for clinical trials but as a possible augmentation to them, the document, released last week, shows. It allows for the possibility of “hybrid” trials where data extracted from medical claims, electronic health records or lab or pharmacy databases could be used for “certain elements” of clinical trials.
The FDA is encouraging fatty liver disease researchers to come up with new biomarkers for clinical trials to avoid invasive — and potentially dangerous — biopsies that drive away many would-be participants.
“The use of liver biopsies in clinical trials poses significant logistical challenges,” the agency says in new draft guidance. “Therefore, noninvasive biomarkers are needed (including imaging biomarkers) to supplant liver biopsy and provide a comparable or superior ability to accurately diagnose and assess” chronic inflammation or nonalcoholic steatophepatitis (NASH).
Two influential clinical industry groups are focusing on new training efforts to prevent principal investigators from burning out and it appears that the FDA is willing to work with them on the effort.
Sponsors, CROs and sites have to move away from repetitive, “one-size fits all training” for trial investigators and toward a targeted approach that addresses the needs of investigators and the protocols themselves, says the Clinical Trials Transformation Initiative.
The FDA says it wants to modernize device clinical trials by using more up-to-date technologies as comparators to make sure only the safest, most effective devices get to market.
Low- to moderate-risk medical devices have long been able to coast to approval by demonstrating they’re equivalent to already-approved or “predicate” devices. But regulators say too many predicate devices are decades old.