United States
At the advisory committee review of Stealth’s Barth syndrome treatment elamipretide, Office of New Drugs Director Peter Stein clarified the circumstances where clinical data in a related indication could serve as confirmatory evidence for a single adequate and well-controlled study.
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.
A growing number of future Part B candidates for Medicare price negotiation may avoid price controls in the program with injectable versions.
FDA law experts do not buy the outsourcers’ argument that the agency must go through notice-and-comment rulemaking to remove a drug from the shortage list.
But the prospects for attaching the legislation to a federal spending package in December will be dependent on the outcome of the upcoming elections.
Big corporations fought to scuttle a California PBM reform bill, despite favoring federal efforts, in part from a desire to be held to one national standard.
Decentralized manufacturing methods for cell and gene therapies will be critical for improving patient access to treatments, but sponsors must prepare to demonstrate “comparability” with centralized manufacturing.
Teva resolved two US Department of Justice civil suits accusing the firm of violating the US Anti-Kickback Statute and the False Claims Act by allegedly conspiring to fix the price of three generic drugs and paying Medicare patients’ copays for its multiple sclerosis brand product Copaxone.
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.
Pink Sheet reporter and editors discuss experts’ experience helping drug sponsors negotiate prices with CMS and the FDA’s proposal for a new pathway to update vaccines before a pandemic is declared.
The proposed CMS innovation center's $2 drug list model will not address the barriers to newer generics getting on Part D formularies and plans may have little incentive to participate in the demo, an industry group said.
The FDA’s vaccine advisory committee endorsed a new plan to update pandemic influenza vaccines “inter-pandemic,” but encouraged the agency to consider how to make the approach feasible for novel vaccine technologies.
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.
The head of the US FDA’s drug center suggests that economic factors, including the Inflation Reduction Act, are potentially more important pathways shaping the focus of research and development than regulatory flexibility.
A US National Academy of Sciences, Engineering and Medicine panel is considering whether biomedical innovation is aligned with public health needs. The resulting report could impact FDA and federal policy on rare disease and research and development incentives.
Policy experts and consultants recount their experiences shepherding manufacturers through the first round of Medicare price negotiations during a recent webinar.
The US FDA is finalizing a guidance intended to encourage the use of decentralized clinical trial techniques. After the COVID-19 pandemic necessitated adoption of the more patient-friendly approaches, the agency is clearly eager to see them become routine.
The threat of a highly pathogenic avian influenza pandemic spurred the FDA to have its vaccine advisory committee comment on a new process to update licensed prototype pandemic flu vaccines.
The Cardiovascular and Renal Drugs Advisory Committee will consider whether open-label extension data from a randomized trial that failed its primary endpoint, along with a historical control comparison, are enough to support approval in the ultra-rare disease.