Rare diseases are almost certain to remain one of the biggest areas for novel drug development in 2024, but the orphan drug pipeline will be making space for some novel therapies for common conditions over the next six months.
Rare Diseases Rule, But Some Common Conditions Stand Out In Pending US Applications
Half of the FDA’s upcoming 2024 user fee goals target rare diseases, but highly prevalent conditions like uncomplicated urinary tract infections and atopic dermatitis are also up for agency action in the second half of the year.
More from Rare Diseases
• By
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.
• By
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.
• By
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.
• By
Companies have until 17 March to respond to an EU consultation on a new strategy that will seek to simplify the regulatory framework and make it easier for innovative small and medium-sized enterprises to “access the capital they need” to scale up in the bloc.
More from Pink Sheet
• By
Pink Sheet reporter and editors discuss how the massive layoffs will reshape the FDA and its impact on the drug and biologics industries.
• By
While the pharma industry appears to be exempt from US tariffs imposed by President Trump, a member of the UK House of Lords says the details are unknown and warned that uncertainty “leads to less investment” in business as a whole.
• By
The EU Clinical Trials Information System has achieved primary registry designation in the International Clinical Trials Registry Platform in a move that is expected to reduce regulatory burden for companies and help them lower compliance costs by aligning with publication requirements in medical journals.