Clinical Trials
A US FDA Advisory Committee responded enthusiastically to the potential use of challenge models to enable pivotal trials of novel pertussis vaccines, but most members suggested more refinements are needed before they are ready to use.
Members of the Cardiovascular and Renal Drugs Advisory Committee repeatedly challenged Stealth’s assertion that a new randomized trial in Barth syndrome was not possible, but also said potential functional unblinding and other challenges could compromise a new study.
Slow adoption of alternatives to animal testing in the current decentralized regulatory framework shows the need for a ‘one-stop shop’ at FDA that can provide advice, precedents and qualification programs.
The Cardiovascular and Renal Drugs Advisory Committee voted 10-6 that efficacy had been shown for the ultra-rare disease, but even panelists in the majority questioned whether the product satisfied the threshold requirement for an adequate and well-controlled study.