Gottlieb Calls it Quits

March 11, 2019

FDA Commissioner Scott Gottlieb will leave the agency within a month, the commissioner said in a surprise announcement last week.

Some sources speculated that Gottlieb’s Washington job was putting a strain on his family — he still lives in Connecticut and commutes to D.C. — but Gottlieb himself dismissed rumors of his departure as recently as January. He had just testified before a key House appropriations subcommittee last week, discussing the FDA’s plans for 2019 (CenterWatch Weekly, March 4, 2019).

Gottlieb, a veteran pharma executive who returned to the FDA in May 2017, took a light touch with clinical trials, but his agency was still discomfited by the advance of Right-to-Try legislation which — while only nominally offering drug sponsors liability immunity for patients in clinical trials — was seen by some as a first march against the FDA itself.

Under his leadership, the agency also revised guidance that allowed drug sponsors or sites to help defray some of the costs of participation to some trial’s patients. It was a seemingly small measure that will have a lasting impact, said Dana Dornsife, founder and president of the Lazarex Cancer Foundation, a patient advocacy group.

“His patient-centric, insightful and visionary leadership and guidance will continue to be beneficial to cancer research and cancer patients as we continue this vital effort,” Dornsife said of Gottlieb.

The FDA has not yet announced who will fill Gottlieb’s shoes until the new commissioner can be nominated by the president and confirmed by the Senate, a process that could take weeks to months.

FDA, ICH List Clinical Guidances in the Works
Both the FDA and the International Council on Harmonisation (ICH) have big plans for new clinical trial guidances in the coming months.

The FDA’s Center for Drug Evaluation and Research plans to release more than two dozen clinical trial-related draft guidances in 2019, according to the center’s guidance agenda released last week.

In addition to a new Q&A guidance on risk-based trial monitoring, CDER will address such issues as key information and informed consent, enhancing the diversity of clinical trial populations, multiple endpoints in clinical trials, patient-focused drug development, use of real-world data, endpoints for heart failure drug development and adjusting for covariates in randomized experiments.

CDER does not specify when it will issue the guidances, but the center already has released some, including documents on eosinophilic esophagitis drug development, assessing the effects of food on drugs in INDs and NDAs and bioavailability studies in NDAs for INDs.

ICH has been working on revising its 20-year-old guideline on general considerations for clinical trials for more than a year and plans to make the update final in mid-2020. ICH E8 addresses internationally accepted trial practices and principles.

The revision is part of ICH’s initiative to modernize its clinical guidelines, which began with an update to ICH E6-Good Clinical Practices in 2017.

Clinical trial design and conduct have become more complex since ICH E8 was first adopted in 1997, ICH says, and a wide range of trial designs and data sources are not adequately addressed in the current version. The revision will put emphasis on how design or planning considerations can optimize trial and data quality. ICH plans to release the new guideline in mid-2020.

Read CDER’s guidance agenda here:

Advarra to Acquire Quorum and Consulting Division Kinetiq
Advarra has acquired Seattle-based IRB Quorum, along with its research and technology consulting division Kinetiq.

The acquisition will give Advarra access to Kinetiq’s team of regulatory attorneys and compliance professionals, who offer expert guidance on international regulatory standards for medical devices, drugs, biologics and good clinical practice.

The Quorum headquarters in Seattle will support the West Coast customers it shares with Advarra as well as boosting existing Advarra locations in Columbia, Md., Cincinnati, Malvern, Pa., Research Triangle Park, N.C. and Ontario.

UK Researchers Claim New Hope for Parkinson’s Research
British researchers say they may have found new hope for Parkinson’s disease sufferers after a futuristic trial that used robots to implant restorative proteins directly into patients’ brains.

Researchers at the North Bristol NHS Trust recruited 41 Parkinson’s patients and had tubes placed in their brains. Half were randomly given either a placebo or an infusion of Glial Cell Line Derived Neurotrophic Factor (GDNF) once per month for nine months.

The trial didn’t find any significant difference in the outer symptoms of the Parkinson’s group compared to the placebo group, but the researchers nonetheless say they’re cheered by signs of repaired brain cells in patients in the GDNF group. The patients had their brains scanned before the trial started and again after nine months to check how their dopamine-producing brain cells were working. After nine months, GDNF patients showed improvement in 100 percent of a “key area” of the brain.

Over the next nine months, patients in each group were offered a chance to continue with the GDNF therapy, and both groups showed “moderate to large improvements in symptoms,” the research team found.

The team’s findings were published last week in Brain and the Journal of Parkinson’s Disease.