Europe – Convergence: EMA official urges transition of existing clinical trials to CTIS portal
Sponsors should plan to transition existing clinical trials into the new Clinical Trial Information System (CTIS) portal by 30 January 2025, Anabela Marcal, of...
UK – Recommendation from NICE for PTC Therapeutics gene therapy
Eladocagene exuparvovec, the only gene therapy for children with the ultra-rare genetic disorder, aromatic L-amino acid decarboxylase (AADC) deficiency, has been recommended by the...
USA – FDA offers details on PDUFA VII STAR review pilot
The US Food and Drug Administration (FDA) has announced details on its Split Real Time Application Review (STAR) pilot, a program designed to shorten...
UK – New T-cell study demonstrates ‘powerful’ potential for tailored cancer medicine
The clinical study involved 16 patients with either colon, breast or lung cancers that had failed to respond to other treatments, and was made...
USA – FDA issues final guidance on AML drug development, two oncology draft guidances
The US Food and Drug Administration has published a final guidance and two draft guidances that detail its thinking on developing certain types of...
USA – New FDA guidance offers ethical roadmap for including children in clinical trials
The US Food and Drug Administration (FDA) has issued draft guidance outlining a series of ethical considerations for conducting clinical trials that involve children.
The guidance,...
Europe – Patient registries: EMA officials highlight opportunities in orphan drug development
Officials from the European Medicines Agency (EMA) tout the benefits of patient registries to support regulatory decision making for orphan medicinal products in a...
USA – CERSI Summit: Wider use of real-world evidence continues to face hurdles
The widespread use of real-world evidence (RWE) in regulatory decisions continues to face challenges, including problems with data quality and methodologies, and a disconnect...
Europe – Actions to support the development of medicines for children
Regulators in the European Union (EU) have taken several initiatives in the past four years to increase the efficiency of paediatric regulatory processes and...
USA – FDA issues draft guidance on mpox drug development
In a much-anticipated draft guidance issued on Thursday, the US Food and Drug Administration (FDA) said that unlike smallpox therapeutics, treatments for mpox cannot...
