FDA Plans to Ramp Up Disease-Focused Guidance

Regulatory NewsRegulatory News | 17 April 2018 |  By 

FDA Commissioner Scott Gottlieb on Tuesday told a House Appropriations subcommittee that the agency plans to “sharply increase” its release of disease-focused guidance documents to help drugmakers develop new treatments.

“Among some of the many areas we’re working on right now are new guidance to lay out modern criteria for the development of drugs targeted to ulcerative colitis; rare pediatric cancers; pediatric HIV; and serious, life-threatening and non-cancer blood disorders like aplastic anemia,” according to Gottlieb’s written testimony on the 2019 budget.

The new guidance documents will aim to make drug development more efficient and focus more on developing drugs targeted to less-common conditions where there’s a lack of available therapy and development pathways can be challenging.

“For example, the guidance on blood disorders will allow drug developers to reduce the use of animal testing and will outline ways to measure benefit that may permit more efficient development programs and earlier approvals,” his testimony said.

He also noted how clinical trial design "can be particularly hard and guidance in this area will reduce regulatory uncertainty and provide clarity to drug developers. 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.”

In addition to the guidance, he said FDA is looking to set up a policy office‎ inside the Office of New Drugs in CDER that will “distill and align regulatory, clinical and scientific reasoning of review divisions to promote policy transparency and consistency.”

Discussions between representatives and Gottlieb in Tuesday’s hearing centered around FDA's work to regulate electronic cigarettes, the opioid epidemic, continuous manufacturing innovation and medical device safety.


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