Table of Contents
Karim Mikhail, director of the US Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER), used a keynote conversation at the Cell & Gene Meeting on the Mesa in Phoenix on Monday, October 5, 2026 to say the agency wants eventually to pull its separate rare disease efforts into one package, and to say the FDA’s new early-trial pilot, which is meant to speed up first-in-human studies, borrows from Australia’s approach without adopting either the Chinese or the Australian system.
On rare diseases, Mikhail said that in practice the FDA has been “applying flexibilities right and left”, and pointed to separate efforts, including a workshop the agency has held on statistical approaches to rare diseases. “At a certain point in time, we want to bring this all together into one package,” Mikhail said. “And if we don’t do it, honestly, no other regulatory agency is going to do it.”
Asked how to balance oversight with trusting review teams, Mikhail described taking regular half-hour briefings on applications under review so that any discussion with reviewers happens as a file progresses. A center director “cannot come at the 11th hour”, Mikhail said; trying to “move 180 degrees” in the last few weeks before a decision is “impossible and inappropriate”.
On the consistency of FDA advice, Mikhail said that “if the rules keep changing” it becomes “very difficult for anybody to operate”, and expressed the hope that future meetings will leave people with “100% clarity” on what was agreed, while cautioning that development programs run for years and the science moves during them. Sponsors have a part in that, Mikhail said: “It’s not only the FDA part, honestly. I think the sponsor side carries a lot of responsibility in terms of clarity, vision.”
The closing section dealt with competition for early-stage trials. “We will never have a China-like system, and we will never have or go to an Australian-like system,” Mikhail said, adding that the FDA believes both have gaps but that it can learn from them. Mikhail said the pilot started from one feature of the Australian model: “qualified third-party groups that are able to help a sponsor”.
That became the Expedited Investigational New Drug (IND) Pilot, which pairs sponsors with third-party qualified research institutions (QRIs). For sponsors in the pilot who are advised by a QRI, Mikhail said, “we are willing to engage with you frequently” before an IND is filed. “There is no shortcut, but there is external support that will help the sponsor be ready”, Mikhail said.
“We’ll have to see if that pilot is successful,” Mikhail said. Only after that, and “maybe one day”, could QRIs with a track record “actually approve your low risk files”, Mikhail said, adding that “for us to do this, we have to go to Congress”. Mikhail gave no timeline. The FDA says it retains full regulatory authority throughout the pilot.
Mikhail, who the session moderator said is also acting head of CBER’s Office of Therapeutic Products (OTP), said there had been no hurry to fill the OTP director post. “I did not rush to hire this,” Mikhail said.
About CBER and Karim Mikhail
CBER is the FDA center that regulates biological products, including cell and gene therapies, which are reviewed by OTP. According to the FDA, Mikhail joined the agency in 2025 as a senior advisor in the Office of the Commissioner after more than two decades leading pharmaceutical companies and biotechnology startups.
The FDA launched the Expedited IND Pilot on September 15, 2026. The agency says it will review individual IND components on a rolling basis during the pre-IND phase, is accepting applications until October 30 and expects to select eight to 10 sponsor-QRI pairs. The FDA says it will prioritize candidates under the Center for Drug Evaluation and Research’s Office of New Drugs, CBER’s OTP or the Oncology Center of Excellence.
More Onyx coverage from the meeting is here.
Sources: Karim Mikhail keynote conversation, Cell & Gene Meeting on the Mesa, October 5, 2026; Cell & Gene Meeting on the Mesa agenda; FDA biography of Karim Mikhail; FDA press release; FDA Expedited IND Pilot Program
Comments