US FDA Plans Rare Disease Center Of Excellence, But Not "That" Kind Of COE

A COE modeled after the Oncology Center of Excellence, which stakeholders had proposed, is not in the works, but rather a more virtual structure.

Source: Shutterstock

After the OND reorganization, the rare disease program will continue helping assessors as needed.

The reorganization of the US Food and Drug Administration's Office of New Drugs will set the stage for another center of excellence, although not under the construct used for oncology.

OND's rare disease staff will be placed into its own division as part of the organizational flattening now under way. Center for Drug Evaluation and Research Director Janet Woodcock said the move will eventually allow for a rare disease center of excellence.

"They'll have the lead. They'll be these groups' point of contact for a lot of these diseases," Woodcock said on 22 October during the National Organization for Rare Disorders' Rare Diseases and Orphan Products Breakthrough Summit. "That wouldn't be true necessarily for rare cancers or rare neurologic syndromes and so forth because they'll have their own place, but we will have sort of the beginnings of a center of excellence in rare diseases."

Woodcock's comment drew applause from the audience of rare disease patients, advocates and other stakeholders. But she clarified after the session that the agency does not plan to create a center of excellence similar to the Oncology Center of Excellence.

A more "virtual" center is envisioned that will ensure rare disease experts within OND can collaborate, but they will remain in their respective divisions. Rare disease-related applications also will not be routed through the new office and division.

OND's rare disease staff will continue to consult with the various assessment divisions to ensure policies are followed consistently and answer questions when necessary.

The FDA plans to increase the number of offices within OND to eight and the number of clinical assessment divisions to 27 as part of a move to a more disease-focused structure. The reorganization was announced in September and will be completed in four phases. Also among the changes are an expansion of the Office of Biostatistics and Office of Clinical Pharmacology and a restructuring of the Office of Pharmaceutical Quality. Organizational charts are here and here. (Also see "US FDA's Office Of New Drugs Reorganization Approved, But Not Happening Overnight" - Pink Sheet, 26 Sep, 2019.)

Moving the rare disease staff into a separate office was seen as a promotion and recognition of the increasing influence of orphan drugs on the OND workload. (Also see "US FDA's Rare Diseases Program Not Expected To Change With Drug Office Reorg" - Pink Sheet, 27 Jun, 2018.)

The model used to establish the OCE was created as part of the 2016 21^st Century Cures Act, and is considered a success. The agency was empowered to create multiple COEs in the legislation. (Also see "US FDA's Cancer Review Staff Should All Move To Oncology Center of Excellence, Advocates Say" - Pink Sheet, 15 Mar, 2018.)

Advocates Pushed For OCE-Like Center Of Excellence

While Woodcock's statement about creating a rare disease center of excellence may have been applauded, her actual intentions may not be quite as expected.

Advocates had pushed for a formal rare disease COE to be created, but Woodcock said a rigid structure may not improve application assessments or the rare disease program's visibility within the agency. She also argued that having the rare disease program report directly to the commissioner's office may actually decrease oversight. (Also see "Center Of Excellence Not Best Model For US FDA Rare Disease Program, Woodcock Says" - Pink Sheet, 13 Sep, 2018.)

Among the goals for the advocates' rare disease center of excellence proposal was to improve the consistency with application assessments. While some divisions are well-versed with agency rare disease policy, such as its flexibility with evidentiary requirements, others are not, in part because they do not see many orphan applications.

The FDA wants to broaden the understanding of rare disease policies within the assessment divisions, especially as the number of rare disease applications submitted continues to increase. In 2018, orphan drugs constituted the majority of new molecular entity approvals for the first time. (Also see "Orphan Drugs Compose Majority Of Novel US Approvals For First Time Ever In 2018" - Pink Sheet, 3 Jan, 2019.)

Indeed, OND Director Peter Stein recently said that increased training within FDA, along with the growing popularity of the rare disease space among drug developers has overcome the perceived experience gap. (Also see "US FDA's New Drug Assessors More Experienced With Rare Disease Issues" - Pink Sheet, 11 Sep, 2019.)

Along with the center of excellence proposal also was a request to create a pool of experts that could be added to FDA's advisory committees when an orphan application is considered. (Also see "US FDA May Create Rare Disease Expert Pool For Advisory Committee Reviews" - Pink Sheet, 29 Oct, 2018.) The agency has noted that recruitment for advisory committees, particularly ones involving orphan drugs, has been challenging because of conflict of interest rules.  (Also see "Top US FDA Officials Continue To Lament Advisory Cmte. Recruitment Difficulties" - Pink Sheet, 18 Oct, 2019.)