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Disease-Specific Guidances Reflect US FDA's 'Nimble, Collaborative, Patient-Focused' Approach

 
• By Brenda Sandburg

Patient groups helped with draft guidance on ALS drug development and revision of Alzheimer's draft guidance; agency also issues draft guidance on pediatric seizure, final guidances on DMD and migraine.

Guidance word in red keyboard buttons

The US FDA's five new guidances on development of drugs for neurological diseases showcase several changing aspects of the agency's regulatory approach, from how it works with patients to how it formats documents. 

"FDA recognizes the urgent need for new medical treatments for many serious conditions including neurological disorders such as muscular dystrophies,...

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More from Approval Standards

US FDA’s ‘Plausible Mechanism’ Pathway: A Platform-Based Approach Not Just For Rare Diseases

 
• By Sue Sutter

Manufacturers who succeed in treating several patients with bespoke therapies could leverage platform data to gain marketing approval for similar products in additional conditions, FDA leaders say in a NEJM article that describes five prerequisites for use of the new pathway.

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The British Pharmacopoeia: Setting standards that are trusted worldwide

In this exclusive article with Peter Crowley, Secretary and Scientific Director of the BP Commission, you’ll discover how the BP is preparing for its landmark 2026 edition while strengthening its influence as a global bridge between science, regulation, and industry.

UK Targets Contamination Tests & Pharmacokinetic Studies In Animal Testing Phase Out

 
• By Eliza Slawther

Pharmaceutical companies will be expected to phase out certain animal tests in the drug development process under a UK roadmap aimed at pivoting towards using alternative models, such as organ-on-a-chip systems and artificial intelligence.

Novel Oncology Endpoints Take Time, Require Broad Outreach, US FDA Says

 
• By cole werble

The US FDA’s oncology team is emphasizing a methodical approach to developing novel endpoints like MRD or ctDNA and that community buy-in is important, not just regulatory standards.

More from Pathways & Standards

UK Targets Contamination Tests & Pharmacokinetic Studies In Animal Testing Phase Out

 
• By Eliza Slawther

Pharmaceutical companies will be expected to phase out certain animal tests in the drug development process under a UK roadmap aimed at pivoting towards using alternative models, such as organ-on-a-chip systems and artificial intelligence.

Proposed Shutdown-Ending Agreement Would Cut US FDA Non-User Fee Funding

 
• By Derrick Gingery

But the Senate’s reduction would not be as much as the House of Representatives and President Trump proposed earlier this year, which is likely a win for the agency.

How Regulatory Horizon Scanning Works In The EU, UK & Switzerland

 
• By Eliza Slawther

Horizon scanning is a tool used by regulators to prepare for forthcoming medicines that might challenge existing processes. Senior figures from the European Medicines Agency, Swissmedic and the UK MHRA explain how they conduct horizon scanning activities in their respective jurisdictions.