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Antisense Oligonucleotide Clinical Guidance Emphasizes Individualized Nature Of Treatment

 
• By Sue Sutter

US FDA’ s latest ‘n of 1’ guidance describes clinical considerations for INDs to support dosing and monitoring of an individual with a life-threatening genetic disease who is eligible to receive an ASO; recommendations apply only when a few patients may be candidates for treatment.

Flowers in field
In FDA's view, a study for an individualized ASO therapy will involve 1 or 2 patients. • Source: Alamy

A US Food and Drug Administration draft guidance on clinical considerations for individualized antisense oligonucleotide drug products emphasizes the limited target population for such treatments.

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US FDA Rare Disease Innovation Hub’s Goals Align With Trump, Leader Says

 
• By Sue Sutter

The hub’s goals of efficiency, centralization and coordination fit with the Trump Administration’s current focus for the FDA, Amy Comstock Rick, director of strategic coalitions, told the Biopharma Congress.

AI Could Be Used ‘In The Deliberation’ Of HTA Reviews In England, Says NICE

 
• By Eliza Slawther

England’s health technology assessment institute, NICE, is looking to “reimagine” its evaluation process with the help of AI, rather than just using this technology to speed up its existing processes.

EU HTA Regulation: Companies Can Talk To National Authorities As ‘Substitute’ For EU-Level Advice

 
• By Eliza Slawther

Not all companies will be able to access joint scientific consultations under the EU Health Technology Assessment Regulation, but success is still possible for those that engage with national agencies early on, says EUCOPE’s Alexander Natz.

German, Dutch And Italian HTA Processes Not Supportive Of RWD, Say Companies

 
• By Eliza Slawther

Payers and health technology assessment bodies in the Netherlands, Germany and Italy are either unwilling to use real-world data in assessments or cannot due to their existing frameworks, say representatives from Gilead Sciences and Autolus Therapeutics.

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New Carbon Monitoring Initiative Could Help Future Proof Clinical Research

 
• By Francesca Bruce

A new European initiative will help trial sponsors better understand whether digitization and decentralized clinical trials of drugs generate less carbon than traditional methods.

Major Analysis To Help MHRA Fill Gaps In UK Clinical Trials Landscape

 
• By Vibha Sharma

A first-of-its-kind review of clinical trial applications submitted to the UK medicines regulator, the MHRA, shows there is a concentration of research in certain disease areas and identifies opportunities to enhance the representation of specific patient groups.

New Medicare Negotiation Program ‘Iterations’ Inevitable, Former CMS Official Blum Says

 
• By Cathy Kelly

Jonathan Blum, who was principal deputy administrator during the Biden Administration, explained the agency’s approach to setting up the price negotiation program and said changes likely will be implemented by the Trump Administration.