Gottlieb Successor Promises to Maintain Current FDA Priorities
Acting FDA Commissioner Ned Sharpless pledged to stay the course set by his predecessor, Scott Gottlieb, in his first address to agency staff.
“I plan to maintain FDA’s current course of action in every area and proceed full speed ahead,” he said. “I promise you ... we’ll continue to do everything we can to make the development of new treatments and cures more efficient across our medical product centers, while ensuring that we maintain FDA’s gold standard of safety and efficacy.”
An oncologist by training, Sharpless said he considered himself a champion of real-world evidence when he served at the National Cancer Institute and said he was excited by plans at the National Evaluation System for Health Technology to use real-world evidence.
White House Review Will Delay New Guidances, Experts SaySponsors and investigators awaiting new draft guidances promised by the FDA in its 2019 guidance agenda will have to wait a little longer thanks to a new White House policy.
The Trump administration last week ordered federal agencies — including the FDA — to submit all pending guidance documents to the White House budget office, a move that some experts say could stall drug policy changes.
Draft guidances on the agenda include Enhancing the Diversity of Clinical Trial Populations — Eligibility Criteria, Enrollment Practices and Trial Designs, and Multiple Endpoints in Clinical Trials.
The White House will review drafts it considers to have a “minor” impact within 10 days, but if it’s potentially a major proposal, it’ll take 30 days.
The added review stage will create “a real bottleneck for new guidances,” says Kurt R. Karst, a director at Hyman, Phelps, and McNamara in Washington, D.C., who concentrates his practice on drug regulation.
In addition, Karst says, “Having OMB review these, comment on them, will certainly give the White House some oversight or control over these issues.”
Read the White House memo here: https://bit.ly/2PbDbbJ.
Canadian Regulators Lay Out Quality Standards for Real-World EvidenceCanadian regulators laid out guidance last week that they hope will help sponsors use real-world evidence to speed life-saving drugs to those who need them most.
Health Canada is eager to bring real-word evidence into drug reviews, especially for the most vulnerable patients, the agency says. But it points out that data can be compromised by bias or poor study design. For now, it recommends using either prospective or retrospective data.
Prospective data collection requires similar rigor to a clinical trial, with “transparency and clearly defined procedures” essential, the agency says.
For retrospective data studies, researchers should understand the limits of looking back on previously collected data and try to mitigate the risks of poor quality. “All data should be traceable to the source, and source data should be available whenever possible,” the document states. “Curated data will be preferred in all instances.”
Data elements should be well-defined to “identify and assess exposures, outcomes, endpoints and covariates of interest, and validation of these study aspects should be included,” the agency says.
Statistical assumptions in a retrospective real-world evidence study may have to be tested for validity and the validity of data sets should be “clearly described and include information addressing data completeness,” the guidance states.
Read the Health Canada guidance here: https://bit.ly/2XlUXfn.
Health Canada Adopts ICH Guidance on Multi-Regional Clinical TrialsCanadian regulators have adopted ICH’s E17 guidelines on multi-regional trials.
The agency said current Health Canada guidance may not be entirely consistent with the ICH document, but the Health Canada-implemented ICH guidance will take precedence in such cases.
The adoption comes nearly two years after ICH finalized the E17 document. European and American regulators adopted the guidelines last year.
Read the guidance here: https://bit.ly/2PfYSrr.
First U.S. CRISPR Trial LaunchesThe first U.S. trial of gene-editing treatments is underway as part of a 15-year project that researchers hope will change everything about cancer treatments.
Researchers at the University of Pennsylvania Parker Institute and the private, for-profit CRISPR company Tmunity hope to enroll 18 patients between now and 2033 for the Phase I trial that aims to determine the safety profile of a single infusion of edited T-cells as well as the feasibility of manufacturing the edited cells.
The partners have been working towards the trial — the first to administer gene editing therapy in U.S. cancer patients — for more than two years.
Tmunity was established in 2017 by legendary CAR T-cell scientist Carl June and former Novartis executive Usman Azam. The group announced last year that it had received $100 million in startup funding, most of it from Gilead and Facebook founder Sean Parker, whose family names adorns Penn’s institute.