Rare Daily Staff

U.S. Food and Drug Administration Commissioner Scott Gottlieb went before lawmakers earlier this week to make the case for the Trump administration’s proposed $5.8 billion budget for the agency in fiscal 2019.

The Trump administration’s budget request includes represents a $663 million increase or 13 percent more than the annualized continuing resolution for fiscal 2018. Of that increase, $190 million would come from industry user fees.

Should Gottlieb get the funding he seeks, rare disease patients may be one of the beneficiaries. The commissioner is seeking the additional funding to invest in a variety of areas including regulatory science, public health, and advancing the regulator model for digital health technology. But it also includes advances to new approaches to support the development of treatments for rare pediatric diseases.

The agency is investing in developing new guidance to reflect the state of science and the agency’s current thinking on the development of drugs targeted for such things as ulcerative colitis; rare pediatric cancers; pediatric HIV; and serious, life-threatening and non-cancer blood disorders like aplastic anemia. The goal is to apply current principles to make drug development more efficient by focusing on the most effective ways to prove safety and efficacy.

“There’s a common theme here. One aim is to focus more guidance on laying out the pathway for developing drugs targeted to less common and serious conditions where there’s a lack of available therapy and drug development pathways can be more challenging,” Gottlieb said during testimony before the U.S. House Subcommittee on Agriculture, Rural Development.

As an example, Gottlieb pointed to the agency’s efforts to develop guidance describing the evidence needed to demonstrate the effectiveness of new drugs intended for certain slowly progressive, low prevalence rare diseases for which clinical trial design can be particularly hard. Guidance, he argues, will reduce regulatory uncertainty and provide clarity to drug developers working in this area.

“The focus of this policy work mirrors the general direction of drug development, where new science is enabling us to discover novel, safe and effective treatments for previously intractable illnesses,” he said.

Gottlieb also hopes to harness technology to do a better job of knowledge management within the agency. One initiative highlighted by the commissioner is plans to build a knowledge management platform as part of the agency’s drug and medical device review programs. He said the knowledge management system is essential to modernizing medical product review programs and establishing scientific precedents that are established every day.

The platform would enable the FDA to store and manage “the collected experience” of its medical review staff and to have a means of identifying how decisions are made across different functions, the scientific precedents the agency establishes during its review process, and the knowledge it develops.

“Right now, if you asked me how we made a particular review decision in the past, I’d begin by asking our review staff if they’ve confronted a similar clinical circumstance, how it was decided and why,” he said. “We have limited options to query review decisions to extract how we reached certain conclusions. We can’t store and interrogate the scientific precedent we establish every day.”

April 19, 2018
Photo: FDA Commissioner Scott Gottlieb