Existing US Food and Drug Administration programs intended to foster innovative and streamlined drug development may be the answer for the ultra-rare disease sector instead of new incentives, said Kerry Jo Lee, associate director for rare diseases in the Center for Drug Evaluation and Research’s Office of New Drugs.
Existing Innovative Trial Design, Endpoint Tools Could Be Help Ultra-Rare Sponsors Need
Amid a push for new incentives, the US FDA’s Kerry Jo Lee, associate director for rare diseases in the Center for Drug Evaluation and Research’s Office of New Drugs, said in an interview with the Pink Sheet that the tools ultra-rare sponsors need already are available.
More from Rare Diseases
• 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.
• By
Center for Biologics Evaluation and Research Director Peter Marks expects no fundamental changes in support for FDA initiatives and said the Rare Disease Innovation Hub, which promotes cross-center collaboration, is “consistent with what we're hearing in the current environment.”
More from Conferences
• By
Sponsors should consider the DTC ad's audio as the major statement about a prescription drug’s side effects and then choose strategically how to display the accompanying text. TV ads are now employing banners and larger text to satisfy the rule’s “dual modality” requirement.
• By
Given the Trump Administration’s intense focus on cutting costs and increasing efficiency, industry and the FDA should focus on what is and is not working in PDUFA VII when negotiating the next agreement, lobbyists said at the BIO CEO and Investor Conference.
• By
Biotech execs and lobbyists try to explain the Trump Administration's policy strategy and how to thrive in it.