FDA Plans Clinical Trial Regulatory Changes

May 27, 2019

The FDA has unveiled its agenda for upcoming proposed and final rulemakings, including several noteworthy proposals related to clinical trials.

The agency’s plans for the remainder of 2019 include a proposed rule on responsibilities for initiating and conducting clinical trials; a proposed rule harmonizing several provisions of FDA regulations on human subject protection; proposed new reporting requirements under the Right to Try Act; and a proposed rule adding development safety update reports to IND application annual reporting requirements.

Additionally, a proposed rule is being considered that would require any U.S. institution participating in multi-site cooperative research to rely on approval by a single IRB for research conducted in the U.S. “with some exceptions.”

Read the Unified Agenda here:

Feds Dole Out $9 Million for Alzheimer’s Candidate Trial
Federal officials have awarded $9 million to a North Carolina drugmaker to help bring an anti-Alzheimer’s drug through a clinical trial.

Pharma firm T3D Therapeutics says the money will be doled out over the next four years to support its T3D PIONEER study, a Phase II trial designed to test the safety and efficacy of its candidate pill, T3D-959.

PIONEER hopes to recruit 252 adults with mild-to-moderate Alzheimer’s disease and give them either one of three doses of T3D-959 or a placebo for 24 weeks. The trial is supposed to start next year.

The money comes from a grant through the National Institute on Aging.

Drug Giants Partner with Data Company to Ramp Up Clinical Trials
Verily, a subsidiary of Google parent company Alphabet, is partnering with drug giants Novartis, Otsuka, Pfizer and Sanofi to explore novel methods for generating real-world data.

Verily’s evidence generation platform, Project Baseline, will help the partners collect health information from electronic health records, sensors and other digital sources. The companies plan to use the data to inform future trials in various therapeutic areas, including cardiovascular disease, oncology, mental health, dermatology and diabetes.

The collaboration aims to make clinical trials more accessible to patients and accelerate the clinical research process.

Drugmakers Comment on FDA’s Guidance on Natural Histories for Rare Diseases
Ionis, Merck, Lundbeck and six other companies offered feedback on the FDA’s guidance for developing natural history studies for rare disease drug development, praising some parts of the guidance but calling for more details.

Responding to the agency’s call for comments on the guidance, which closed on May 24, Ionis suggested that the agency add three additional uses for natural history studies to the guidance — establishing the burden of disease, characterizing genetic mutations and biological consequences and reducing trial burden in interventional studies.

The guidance should strongly encourage sponsors to share with the public and community “not only methods, practical aspects and results, but also subject-level data in a timely fashion,” the drugmaker said. It also called for greater emphasis on interim analyses.

Lundbeck noted that many rare diseases have established disease registries made available by clinicians and institutions, calling on the agency to acknowledge that it may not always be feasible for sponsors to influence registry development. Establishing a free-standing prospective natural history study can be challenging in terms of recruitment as well, the drugmaker added.

The company also suggested electronic health records as a potential source for creating a control group, and requested a separate section on methods to deal with bias. The agency should also look into how the guidance’s principles could apply to other vulnerable populations and pediatric development, it said.

Merck asked the agency to clarify its expectations for data submissions. The company said it’s unclear if sponsors should submit only external control data or the whole natural history study.

It also stressed the importance of outside organizations and programs running natural history studies to improve the characterization of rare diseases, and urged the agency to emphasize that industry collaboration with them could be beneficial.

Read all nine comments here:

MAGI, ACRP Call for Proposals and Speakers
Clinical research professionals looking for speaking opportunities can look to two upcoming national conferences.

MAGI has issued a call for proposals for sessions at its October 2019 conference in Las Vegas. The conference will feature more than 100 sessions and workshops in six tracks.

ACRP will open a call for proposals on June 3 for its ACRP 2020 conference to be held in Seattle next May.

Learn more about MAGI speaking opportunities here:

Get information on ACRP’s 2020 conference here:

Genentech Added to Corrona Multiple Sclerosis Registry
Roche’s Genentech has become the first pharmaceutical company to have its data be included in a national registry of multiple sclerosis patients.

The Multiple Sclerosis Registry is one of six efforts created by Corrona to collect data on diseases ranging from rheumatoid arthritis to inflammatory bowel disease. The registries collaborate with more than 400 participating investigator sites and the data have been used in more than 140 peer-reviewed manuscripts. More than 700 patients have enrolled in the multiple sclerosis registry alone.  

es and the data have been used in more than 140 peer-reviewed manuscripts. More than 700 patients have enrolled in the multiple sclerosis registry alone.