November 4, 2019
As expected, President Trump on Friday nominated oncologist Stephen Hahn to be the next FDA Commissioner.
Hahn, chief medical executive of the MD Anderson Center in Houston, is a practicing radiation oncologist and a former researcher and clinician. He chaired the University of Pennsylvania’s Department of Radiation Oncology from 2005 until 2015.
Pending confirmation of Hahn’s nomination by the Senate, the agency will be led by HHS Assistant Secretary for Health, Brett Giroir, who also is senior advisor to HHS Secretary Alex Azar for opioid policy.
Ned Sharpless, who has been serving as acting commissioner since Scott Gottlieb’s resignation in April, will return to his former role as director of the National Cancer Institute.
FDA Sets New Requirements for Electronic Safety Reporting
Sponsors conducting trials under an investigational new drug application (IND) would have to file their adverse event reports electronically once a new draft guidance issued last week goes into effect.
The draft guidance outlines the proposed transition from the current standardized safety reporting form to a new electronic process for submitting reports directly to the FDA Adverse Event Reporting System (FAERS).
The new requirements would apply only to reports from sponsors testing a commercial IND that discover serious or unexpected adverse reactions, such as:
- A single adverse event that is uncommon and known to be strongly associated with drug exposure;
- One or more occurrences of an adverse event that is not commonly associated with drug exposure but is otherwise uncommon in the population exposed to the drug; or
- When an aggregate analysis of specific adverse events observed in a clinical trial that indicates the events occur more frequently in the drug treatment group than in a concurrent or historical control group.
Noncommercial sponsors, such as investigator-initiated and expanded access IND holders, would be exempt from the requirements, although the draft guidance encourages them to use the electronic system voluntarily.
Safety reports on anything other than serious and unexpected adverse reactions, including findings from animal or in vitro testing, would continue to be submitted in narrative form through the agency’s current portal for electronic common technical documents (eCTD).
The FAERS submission requirements would go into effect 24 months after the draft guidance is finalized. Until then, sponsors voluntarily may submit electronic IND safety reports to FAERS or use the eCTD portal.
The agency also issued a companion guidance that describes the technical specifications for electronic submission.
Comments on the draft guidance are due by Dec. 30.
Read the draft guidance here: https://bit.ly/2WFbsUV.
Read the technical conformance guidance here: https://bit.ly/2C2fZaq.
FDA Guidance Calls for Rapid Development of Hepatitis D Treatments
A new draft guidance from the FDA encourages drugmakers to speed up development of Hepatitis D treatments for use in the U.S., offering recommendations for innovative trial designs, target populations and endpoints.
Hep D is prevalent in third-world regions, but the draft guidance calls on sponsors to include U.S. populations in trials in anticipation of potential global spread of the virus.
Because there are no approved treatments for the life-threatening disease, the agency is interested in applications that qualify for accelerated approval through its fast-track, breakthrough therapy and priority review pathways.
Safety data from 300 to 500 patients may be adequate, the guidance says, but “the size of the safety database could be smaller for investigational drugs that demonstrate substantial efficacy and safety compared to other therapies.”
The FDA’s preferred phase 3 trial design is the traditional double-blind, placebo-controlled study, but the agency offers recommendations for acceptable alternative designs, including:
- Three-arm, randomized, controlled trial comparing the investigational drug to standard-of-care treatment and placebo;
- Randomized, controlled superiority trial comparing the investigational drug plus standard-of-care treatment to standard-of-care treatment alone;
- Randomized, controlled superiority trial that compares different doses and/or durations of treatment.
After approval of an investigational drug, the FDA suggests sponsors conduct a superiority or noninferiority trial comparing the drug to an appropriate drug already on the market.
The agency also expects that initial approvals of Hep D drugs will be based on trials using a surrogate endpoint such as decline in virologic replication with improvement in liver inflammation. However, approval of such drugs should be followed by “subsequent confirmation using a clinical endpoint,” the guidance says.
The FDA encourages sponsors to discuss enrollment strategies, eligibility criteria and other plans for phase 2 and 3 trials with the agency’s Division of Antiviral Products.
Comments on the draft guidance are due Jan. 1, 2020.
To read the draft guidance, click here: https://bit.ly/2ptmK2d.
FDA Temporarily Halts Zolgensma Trials for Spinal Injection
The FDA has placed a partial hold on clinical trials of the Novartis spinal muscular atrophy drug Zolgensma.
The hold calls a temporary halt to enrollment in a trial testing spinal injection of a high dose of the drug. The company already has completed a low-dose spinal injection trial.
The agency’s action was prompted by findings from a small, pre-clinical study of the drug initiated by the Novartis subsidiary AveXis, in which animal findings showed cell inflammation after spinal injection sometimes accompanied by neuronal cell body degeneration or loss.
Novartis said the trial will stop enrolling and administering the spinal injection until the hold is lifted. The hold will not impact marketed Zolgensma or clinical trials studying the drug for IV administration, the company said.
Deloitte Launches Platform for Clinical Trials
Deloitte’s ConvergeHealth has launched a two-part digital platform to connect patients with clinical trials and continuing care.
The MyPATH platform aims to create personalized care teams for patients that will provide support through a clinical trial and beyond.
The MyPATH for Health branch of the platform brings together patients, caregivers, providers and life sciences organizations to participate in virtual trials. It also offers trials support for electronic patient-reported outcomes and informed consent.
MyPATH for Connected Care will provide virtual care services for trial participants.
The platform offers access to around-the-clock support networks as well as remote health and risk monitoring services.
TrialScout Reaches International Audience
Site network hyperCORE International has partnered with Circuit Clinical’s TrialScout patient connection platform.
HyperCORE, a network of eight companies with more than 81 research sites around the world, is the first site network to use TrialScout’s platform, which allows trial participants to review and rate their experiences.
The network’s global base gives TrialScout its first access to international patients. HyperCORE’s Canadian partner, LMC Manna Research, already has joined the platform, as well as its U.S. partner, IACT Health.