trending Market Intelligence /marketintelligence/en/news-insights/trending/6I15UrUnzqapdfsFdoEtvw2 content esgSubNav
In This List

Capitol Checkup: US FDA drug review revamp; cancer pilots; right-to-try claims

Blog

A Pharmaceutical Company Capitalizes on M&A Activity with Brokerage Research

Blog

2021 Year in Review: Highlighting Key Investment Banking Trends

Blog

Insight Weekly: US stock performance; banks' M&A risk; COVID-19 vaccine makers' earnings

Blog

Global M&A By the Numbers: Q3 2021


Capitol Checkup: US FDA drug review revamp; cancer pilots; right-to-try claims

The U.S. Food and Drug Administration this week is expected to unveil its much-anticipated plans to overhaul the reviewing process for bringing new medicines to the U.S. market quicker, Commissioner Scott Gottlieb said.

He revealed the news during a June 2 address in Chicago at the annual meeting of the American Society of Clinical Oncology.

Gottlieb and other top FDA officials have been hinting for months that the restructuring of the agency's Office of New Drugs was coming.

Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research — who has also been filling in as the head of the new drugs office since early 2017 — noted in January that agency had been working on the plan for some time to modernize its new drug review process.

"We're really now deeply into that and I think that'll be a central theme for this year," she said in a January podcast. "We're looking to react and be able to manage the new science that's coming forward."

On May 3, at the annual meeting of the Food and Drug Law Institute in Washington, Woodcock said the FDA was seeking to "flatten" the new drugs office by splitting up its divisions to form smaller groups with more concentrated expertise.

Speaking at the same meeting, Gottlieb said the goal was to make the new drug review process "even more team-based, so that the skills of FDA staff who have expertise in discrete areas like statistics and modeling and simulation and advanced manufacturing can be more easily leveraged when it comes to novel trial designs and more novel, but promising, development approaches are brought to us."

"Our professional staff members are thought leaders in their disciplines and we want to give them more time to collaborate with academic medical scientists and patients, to analyze scientific and commercial developments and to strategically foster drug development," the FDA chief added.

On May 4, at the annual meeting of the Reagan-Udall Foundation for the FDA — a congressionally created nonprofit formed to advance the agency's mission — Gottlieb explained that one division regulators planned to split was the Division of Gastroenterology and Inborn Errors Products, which he said would be separated into three distinct areas: gastroenterology, liver diseases and inborn errors.

Even though there are now expected to be more divisions at the FDA's new drugs office, Gottlieb said the agency plans to use a common review template for new drug applications, which he said would reduce the multiple and sometimes redundant memos that are generated by statistical, clinical and other reviewers. He said that action would allow medical and scientific staff to have more time to advance policy in their fields.

Ahead of the public announcement, Woodcock explained the specific aspects of the new drugs office reorganization to her staff during a June 1 internal, non-public town hall meeting, the details of which were leaked to BioCentury.

Under the plan, the number of divisions in the new drugs office is expected to grow from 19 to 30, with each varying in size and workload, according to a June 2 BioCentury article, which Gottlieb promoted on Twitter.

The FDA's plan to revamp the new drugs office and its review process must have congressional approval, which the agency is hoping to seek sometime in December, BioCentury reported.

Pilots to speed cancer drugs

Meanwhile, at the American Society of Clinical Oncology meeting, Gottlieb revealed two new pilot programs intended to speed cancer drugs to the market.

For the first pilot program, the FDA's Oncology Center of Excellence — established under the Obama administration's Cancer Moonshot initiative to employ a more integrated approach to speed new cancer therapies to the market — is testing a process known as real-time oncology reviews.

Under the program, as soon as a drugmaker has decided it wants to file for FDA approval, the company starts sharing the bottom-line data with the agency, which conducts a sort of "pre-review" of the information, Gottlieb said.

The partial package submission includes key raw and derived datasets, including safety and efficacy tables and figures, the study protocol and amendments and a draft of the package insert, Gottlieb said.

The pre-analysis gives reviewers and manufacturers an early opportunity to address data quality issues and allows regulators to provide early feedback. The FDA has estimated that using the real-time review approach could cut reviewers' time on applications by as much as 30%, leaving more opportunity for engagement with product developers and resulting in more efficient application reviews, the commissioner said.

Currently, the FDA is testing the real-time review process on applications for supplemental uses of cancer drugs that are already approved for the U.S. market.

"If the pilot shows the efficiencies we expect, it could be expanded to drugs and biologics being considered for initial approval," Gottlieb said.

The FDA is also piloting a new assessment tool, in which regulators layer their reviews over the manufacturer's drug file rather than creating a separate document that recapitulates many of the same data tables.

Gottlieb said the assessment aid would reduce the administrative burden on FDA reviewers and allow them to focus on key results and perform critical analyses that may have been omitted by the company.

"Importantly, the new format leads to a more dynamic review process where key regulatory questions can be answered more thoroughly and effectively," he said.

The assessment aid pilot program is also only being used currently for applications involving additional uses of approved drugs.

Right-to-try claims

Gottlieb was also on Twitter over the weekend promoting the Right To Try Act, despite being told in a May 31 letter by its author, Sen. Ron Johnson, R-Wis., the law was intended to "diminish the FDA's power."

The law, signed May 30 by President Donald Trump — who had long-advocated for the legislation's enactment — permits Americans to bypass the FDA's compassionate-use process, also known as expanded access, for seeking experimental drugs.

While the FDA plans to further strengthen its expanded-access program, Gottlieb said the "message is clear: Patients and families facing terminal illness want to be able to access promising drugs when they've exhausted available options."

He said there was "clearly a consensus among American families that patients want these options."

But Gottlieb provided no evidence for his consensus claim.

The FDA did not respond to questions from S&P Global Market Intelligence about the data on which the commissioner relied.

Alison Bateman-House, an assistant professor in the Division of Medical Ethics at New York University Langone Medical Center, said that while there has been anecdotal support for the ability of patients with no other options to try experimental medicines, she told S&P Global Market Intelligence in a tweeted response that "there's certainly no consensus over the mechanism by which such drugs" ought to be obtained.

"There is no consensus on right to try," Bateman-House added.

She noted that about 100 patient groups actively opposed the legislation, calling it dangerous.

The right-to-try bill was also opposed by some consumer protection organizations and "lots of bioethicists," Bateman-House said.

Steven Joffe, a pediatric oncologist and professor of medical ethics and health policy and pediatrics at the University of Pennsylvania, said he was not aware of any consensus data supporting the right-to-try legislation.

"I'm sure, if polled, majority would support some form of access. But polling that gets people to deal with tradeoffs and complexities of issues is devilishly hard," Joffe said in a tweeted response.

"It all depends on how you ask the question," said Holly Fernandez Lynch, an assistant professor of medical ethics and health policy at the University of Pennsylvania.

Fernandez Lynch noted that from one perspective, the question may be whether dying patients should be able to access unapproved medicines with FDA providing information on safety, but from another standpoint, the question may be whether patients should be taken advantage of by charlatans.

In a June 1 op-ed in The New York Post, Health and Human Services Secretary Alex Azar said the Right To Try Act has opened up an opportunity for patients to "try a potential cure."

But speaking on June 2 on C-Span's "Washington Journal," Joffe said he was concerned the law is a new pathway for unscrupulous companies and doctors to go around the FDA and exploit patients for financial gain.

"I actually worry that the right-to-try law is a step backwards," he said.