FDA official discusses agency’s new Rare Disease Innovation Hub

The US Food and Drug Administration’s (FDA) new Rare Disease Innovation Hub will serve as a main focal point for the rare disease community to raise issues related to developing drugs in this space and will take a different approach in reviewing these drugs compared to the center for excellence model in the center for drugs.

So asserted Julie Tierney, the newly appointed deputy director for strategy, policy and legislation at the Center for Biologics Evaluation and Research, who discussed the hub at a recent meeting sponsored by American Society of Gene and Cell Therapy (ASGCT) in Washington.

In July, FDA announced the creation of the Rare Disease Innovation Hub to speed development of new treatments, integrate regulation of drugs and biologics, and engage patient advocacy communities. (RELATED: FDA unveils rare disease innovation hub, plans public meeting this fall, Regulatory Focus 18 July 2024).

The session’s moderator, Keith Wonnacott, executive director of gene and cell therapy for Lexeo Therapeutics, said the hub is an “exciting new development that I’m hoping will really accelerate innovation through collaboration” in the rare disease space. He asked Tierney to address how the hub will lead the agency in developing policy for rare diseases.

Tierney responded that “I am very really excited about the products in CBER’s remit, particularly gene editing, which have the real potential to change the face of rare diseases.” She added that CBER is in “growth mode” and is set up more as a “start-up instead of a stand-alone” center as the leadership team questions the status quo and is open to taking a new approach to developing drugs for rare diseases.

She considers a hub structure the best model for regulating treatments for rare diseases rather than establishing a separate center of excellence “where there are questions about turf and who is in charge.”

Tierney said this structure “is going to leave review decisions within the office where they sit and where they review common and rare diseases.”

The hub will also serve as a central point of contact for the rare disease community to raise general issues related to drug development that are not related to an application. Currently, these issues are presented to the different centers in an ad hoc way.

When asked to address her long-term vision for the hub, Tierney said there are plans to set new goals for the hub after the public meeting on 16 October. The upcoming meeting will be sponsored by the Reagan-Udall Foundation and will bring together rare disease patient advocates, researchers and industry to establish a set of priorities governing its work.

Tierney explained that the center is now in the process of hiring a new director of rare disease strategy who will “identify the sticky spots” and develop a policy agenda.

Wonnacott then asked Tierney to elaborate on some of the administrative differences between the hub and centers for excellence, such as the Oncology Center of Excellence (OCE), saying there has been confusion between the two structures.

Tierney said, “there are different models, many are familiar with the OCE, led by Rick Pazdur, and that model works for cancer where there are many different types of cancer but it is a type of disease so you pull together the oncologists and that model has a consistent and a collaborative approach to reviews for oncology products across the agency and there is a separate body that exists, the OCE.”

Also, applications are reviewed within the different divisions, but the OCE director also has a say in the final approval decisions.

The hub is a type of organization that exists virtually that builds on the work of CDER’s Accelerating Rare disease Cures (ARC) program and CBER’s rare disease program and rather than have one leader that is separate from the centers, is instead led by the center directors, Patrizia Cavazzoni at the Center for Drug Evaluation and Research (CDER) and Peter Marks at CBER.

An audience member from the rare disease community told Tierney that initially “our hopes were dashed when we did not get a center for excellence” but said he was encouraged by the news that the approval decisions will be retained within the review divisions and that “putting it in the disciplinary areas really makes sense.”

In other areas, Tierney was asked to address the timetable for issuing a draft guidance on accelerated approval for gene therapy products for rare diseases. 

Tierney said the accelerated approvals guidance is a “work in progress” and was unsure if the guidance will be issued this year. She added that the Marks has personally been involved in developing the guidance.