FDA RWE Demonstration Project Expands to Live Trials

April 15, 2019

An FDA-private sector collaboration that aims to determine the validity of real-world evidence as a substitute for clinical trials has entered a new phase, the partners announced last week.

The RCT Duplicate project — a joint effort by the FDA, Brigham and Women’s Hospital and healthcare startup Aetion — began last spring by comparing the results of 30 completed trials to evidence gathered from healthcare databases to see if both approaches returned the same conclusions.

In the new phase, the project will attempt to predict the results of seven ongoing Phase IV trials, marking the first time researchers are estimating the results of randomized controlled trials that haven’t finished yet. Specifically, it will look to find where RWE can provide “robust estimates” of treatment safety and efficacy for supplemental drug applications — and potentially fill in for clinical trial results.

“This is going to, hopefully, really give solid evidence of how much we can rely on Big Data to replace the gold standard RCT, which could be really significant both for drug development and for comparative effectiveness studies that support the development of evidence-based medical guidelines,” Lindsay McNair, Chief Medical Officer with WCG Clinical told CenterWatch Weekly.

The project, which uses Aetion’s software platform to recreate randomized clinical trials through RWE plans to have interim results in mid-2019, with full results by 2020.

Brexit Delayed Again, Trials Still in Limbo
The UK has won a further delay from European leaders on its exit from the EU, leaving drug sponsors no clearer about the ultimate impact of Brexit on their clinical trials. The departure, originally scheduled for March 29 and then moved to April 12, now is slated for October 31.

UK regulators — who have promised to implement European drug guidelines even if the UK crashes out of Europe without a deal — would be excluded from Europe’s centralized IT portal for clinical trials and Europe’s single assessment model.

Drug Industry Asks FDA for Clarity on Natural Histories for Rare Disease Trials
The drug industry is asking the FDA for more clarity around whether natural histories should be included in rare disease trials — and two of the nation’s largest drug trade groups appear to be at loggerheads over the issue.

Among the proposals the FDA is kicking around is allowing natural history studies to substitute as control arms in special cases. Many commenters urged the agency to expand the idea beyond natural histories and to consider such things as patient registries as another way to speed drug development.

Roche, Takeda and BioMarin, among other drugmakers, suggested the agency be more specific about the ways in which natural histories can help speed a drug to market.

But BIO and PhRMA were split on the issue. BIO says it’s worried that the guidance will harden into a requirement that will stall life-saving drugs. Properly done natural histories, the group says, can take several years to complete.

PhRMA, on the other hand, said the draft guidance doesn’t go far enough. Natural histories “should not be the exception but rather common practice” in rare disease trials, the group said.

Read the full comments here:

Drugmakers Question Exclusion Criteria for Rare Esophageal Disease Trials
Two drug companies are pushing back against a proposed FDA guidance that would require drug sponsors to screen out patients in esophageal swelling trials who would respond to an alternate therapy.

The FDA’s draft guidance on eosinophilic esophagitis, published in February, would require clinical trials to exclude subjects who could benefit from proton pump inhibitor (PPI) therapy by holding a two-month pre-trial of PPI dosage.

In comments made public Monday, Adare said the proposal flies in the face of the most recent diagnostic consensus and suggested the agency require trials to examine patient histories to identify and assess those who already have tried PPI.

AstraZeneca noted that the scientific consensus on PPI was still “evolving.” The company currently is working with Children’s Hospital Medical Center in Cincinnati to recruit 26 esophagitis patients for a trial of Fasenra that’s scheduled to wrap up by next year. AZ won orphan disease status for Fasenra from the FDA last fall.

Read the full comments here:

Histogenics and Ocugen Agree to Merge
A merger agreement has been inked between cell therapy company Histogenics and eye disease drug developer Ocugen, the two companies jointly announced.

The merger would result in a new, clinical-stage biopharmaceutical company under the Ocugen name, with former Ocugen stockholders holding around 90 percent of the common stock of the combined company. Current Histogenics stockholders would retain the rest, the companies said.

Ophthalmology programs Ocugen currently has in development include a modifier gene therapy platform, ocular surface disease programs and retinal disease programs.

Histogenics’ technology platform, which has focused on restorative and pain relief cell therapies, has the potential to be used for a broad range of restorative cell therapy indications.

The merged companies will be headquartered in Malvern, Pennsylvania, under the leadership of Ocugen’s current management team.

New Jersey Site Network Acquires CNS Research Group
New Jersey-based sites company Evolution Research Group has acquired Finger Lakes Clinical Research, Evolution officials have announced.

Evolution already owns 13 sites and has a network of affiliates. The company focuses much of its work on early-to-late stage neuroscience drugs. Finger Lakes specializes in central nervous system trials.

Terms of the deal were not disclosed.

J&J Partners with Children’s Research Hospital in D.C.
Johnson & Johnson’s life sciences startup subsidiary JLABS has paired up with Children’s National Health System (CNHS) to develop a large pediatric research facility in the nation’s capital, the two announced last week.

The 32,000-square foot JLABS site will be located on the former Walter Reed Army Medical Center campus — which serves as CNHS’s Research and Innovation Campus — placing it close to academic research centers, universities, federal research institutions and government agencies.

The facility will be open to pharmaceutical, medical device, consumer and health tech companies working on new drugs, medical devices, precision diagnostics and health technologies.