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Califf: Rare Disease Hub Will Develop Methods To Guide Approvability Determinations

 
• By Sarah Karlin-Smith

The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads. 

FDA Commissioner Robert Califf speaks at a webinar sponsored by the Alliance for a Stronger FDA
FDA Commissioner Robert Califf discussed the FDA's new rare disease hub during a 27 September Alliance for a Stronger FDA event. • Source: Screenshot from Alliance for Stronger FDA webinar

The US Food and Drug Administration’s new Rare Disease Innovation Hub will have three main functions, including methods development work that should ultimately help better determine whether a drug meets the approval threshold, FDA Commissioner Robert Califf said.

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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.

Last Chance For Pharma To Respond To EU Strategy For Reforming Ecosystem For SMEs

 
• By Eliza Slawther

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.

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Early Randomization Among Advice On How To Meet Both EMA & HTA Needs

 
• By Neena Brizmohun

Newly published insights from a series of European Medicines Agency workshops can guide drug developers in designing development plans that meet the needs of both regulators and health technology assessment bodies.

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.

Pharma Exhales, Dodges Financial Blow With Tariff Exemption

 
• By Jessica Merrill

Industry lobbing for pharmaceuticals to be exempt from President Trump’s sweeping US tariffs appears to have paid off.