Gulfstream Daily Brief

Regulatory intelligence and policy · Issue 010

FDA opens rare disease statistics docket, European regulators update safety and application information

FDA is seeking public feedback on statistical considerations for rare disease clinical investigations. In Europe, EMA documented an application withdrawal, while MHRA published safety notices and ADR reporting guidance.

By Scott Morvay, Founder September 15, 2026 Official sources linked

FDA

FDA seeks input on rare disease clinical investigation statistics

FDA established a public docket alongside its RISE Workshop to collect feedback on pre-read documents and workshop content concerning statistical considerations for rare disease clinical investigations.

Read the official source

EMA

EMA publishes Q&A on Zumrad application withdrawal

EMA published questions and answers concerning the withdrawal of the marketing authorisation application for Zumrad (sasanlimab).

Read the official source

MHRA

MHRA details how holders should submit ADR information

MHRA published guidance on how marketing authorisation holders should inform the agency of a signal or an adverse drug reaction to a medicine.

Read the official source

MHRA

MHRA posts its latest Field Safety Notices

MHRA published a list of Field Safety Notices covering 7 September through 11 September 2026.

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Why this matters

The FDA docket gives stakeholders an opportunity to inform discussion of statistical approaches in rare disease investigations. EMA’s Q&A adds transparency around a withdrawn application, while MHRA’s publications provide current reference points for ADR communication and device safety monitoring.

Scott Morvay

Founder, Gulfstream Intelligence

This brief provides general regulatory news and educational information. It is not legal or regulatory advice.