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FDA to Accelerate Gene, Cell Therapy Approvals

January 21, 2019

The FDA expects to receive some 200 new gene and cell therapy INDs in the next two years and plans to expedite their processing by hiring up to 50 extra staffers to handle review and other tasks. The agency also is considering new guidances focusing on regenerative therapies.

“The accelerated approval pathway may offer a faster route to approval for new treatments, including potentially curative benefits in significant, unmet medical needs. But the pathway also offers additional authorities for FDA to require postmarket follow-up studies,” the agency said.

“Since many of the risks associated with gene therapy products relate to questions about the product’s durability and potential for rare instances of off-target effects, it may not be feasible to conduct pre-market trials that address all these theoretical risks in any reasonably sized study,” the agency said. Robust postmarket tools, such as those afforded to the FDA under the accelerated approval pathway, particularly for products granted [Regenerative Medicine Advanced Therapy (RMAT)] designation, can help achieve the goal of developing a larger data set to address these theoretical risks in a timely fashion.”

FDA Trial Data Transparency Pilot Has Stalled
An FDA pilot program that aimed to improve transparency in clinical trials appears to have stalled less than a year after its launch.

In 2018, the FDA created a pilot project to test the feasibility of releasing sponsor-generated summaries of trial methods and results. The agency enlisted nine sponsors in the pilot and shared one summary in March — the reports for Janssen’s Erleada (apalutamide) trials. Since then, the pilot seems to have gone dark.

In an open letter to the FDA this month, a group of nearly two dozen clinical trial specialists from academic institutions and organizations in the U.S. and UK question the fate of eight other trials — whose names FDA has not disclosed —that were participating in the pilot.

The academics — who range from Matthew Herder, director of Dalhousie University’s Health Law Institute, to Peter Lurie, president of Science in the Public Interest — say in their letter that making the records public “can help better explain the basis for FDA’s decisions, and in turn, inform prescribers and patients about the evidence underlying a given drug.”

Sharing data also has “enormous potential to improve public health by identifying unpublished, underreported, and misreported trials in the published medical literature,” the letter states.

The letter urges the FDA to recommit to the pilot so the public and clinical trials experts can:

  • “Learn more about safety and efficacy evidence supporting FDA’s approval decisions;”
  • Add the data to systemic reviews; and
  • Engage in “methodological research” to describe how the records can help synthesize evidence and to make sure that trial reports in biomedical literature are “reported fully and accurately.”

Read the Erleada clinical trial study here: https://bit.ly/2EWFn4X.

EMA Seeks Comment on Revised Antibiotic Guidelines
For the first time in five years European regulators are proposing to revise antibiotic trial standards.

The EMA released a 30-page guidance document last week that contains revised language on primary endpoints, primary analysis populations and non-inferiority margins in trials. It focuses on superiority trials for specific diseases, such as acute otitis media, acute bacterial sinusitis, bacterial-exacerbated forms of bronchitis and cystic fibrosis and superficial skin infections.

The agency has been working on revisions since last summer and says its new proposals adapt to the realities of antibiotic-resistant bugs.

When considering patients for trials, the “criteria should maximise the likelihood that patients have the type of bacterial infection under study and minimise enrollment of patients with infections that are likely to resolve rapidly without antibacterial therapy,” the guidance states.

Generally, regulators will accept non-inferiority trial designs if there is a licensed treatment for a disease “for which the magnitude of the treatment effect over placebo is known or can be estimated from the existing data.”

Comments on the draft proposals are due July 31. Read the EMA proposals here: https://bit.ly/2sxf393.

French Regulators Greenlight Ultrasound Trial for Glioblastoma
French regulators have given the go-ahead for a Phase I-II trial of an ultrasound device to treat recurrent glioblastoma.

Sponsor CarThera, also headquartered in France, is hopeful that its implantable SonoCloud-9 will help open up the blood-brain barrier in patients with recurrent glioblastoma who are eligible for carboplatin chemotherapy.

The SonoCloud-9 issues low-intensity pulsed ultrasound and it’s already proven to be safe and effective for patients in the short-term. The Phase I/Phase II open-label dose escalation study will enroll 20 patients over the next year and be followed by an extension.

CarThera officials also are hoping for FDA approval to extend the trials to the U.S., in the MD Anderson Cancer Center in Houston and Northwestern Memorial Hospital in Chicago.