United States
The injection for radicular leg pain appears likely to be approved, but with a very prescriptive label to help balance safety concerns and a lack of long-term outcomes data.
Cavazzoni, who led the drugs center four-and-a-half years, decided on her own to retire at this time, sources said. The decision may have been a preemptive move knowing her time as head of CDER would have been limited, or at the very least highly challenging, under the new administration.
The draft guidance clarifies that sex and gender are not interchangeable, but could be vulnerable in the Trump Administration as Republicans have argued against transgender medical care and that sex and gender must be congruent.
Pink Sheet editors discuss Center for Drug Evaluation and Research Director Patrizia Cavazzoni’s surprising retirement announcement, the importance of the large bolus of guidance documents that the FDA released 6 January, and the FDA’s decision to continue reviewing and granting rare pediatric disease designations even though the program lapsed.
Amid complaints about the challenges of following gene therapy patients for up to 15 years, OTP Director Nicole Verdun said the FDA is considering how to conduct long-term postmarket studies more efficiently.
Any Trump Administration policy involving international pricing should include foreign governments paying higher prices, as well as lowering US prices, CEOs say.
The risk-based framework described in a new draft guidance starts with defining the question of interest and context of use and includes development and execution of a credibility assessment plan. The guidance is limited to AI models used to support regulatory decisions about drug safety, effectiveness or quality.
An FDA advisory committee will consider modifications to the indicated patient population, as well as the appropriate health professionals and settings to administer the proposed treatment for radicular leg pain.
6 January 2025 will be remembered in Washington, DC, as the date Congress certified Donald Trump’s second presidential election victory and a snowstorm shut down most of the city, but for FDA watchers it will be the day the agency released more than two dozen new draft and final guidances.
A TD Cowen survey explored expectations about the impact of the Inflation Reduction Act, access to 340B discounts and general price trends.
The agency’s actions may signal its optimism about near-term reauthorization despite the PRV program and other bills aimed at tackling rare and childhood diseases not making the December 2024 government spending bill.
Viatris’ oral finished dose manufacturing facility in Indore, India, was slapped with a US Food and Drug Administration import alert and warning letter.
Final guidance on communicating scientific information on unapproved uses (SIUU) allows firm-generated presentations to be based on sources other than reprints, but says communications based on nonscientific content are not protected from enforcement.
The agency will consider study enrollment, accrual rates and the number of clinical site activations, among other factors, in determining whether a confirmatory trial is underway prior to accelerated approval, but the new draft guidance does not specify the proportion of patients that must be enrolled at the time of approval.
Data illustrates the 2025 Medicare Part D plan landscape, including changes in premiums and plan options.
The agency’s compliance directors also discussed expectations for greater inspection and review efficiencies as part of the 1 October reorganization during a recent FDLI conference.
Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
Despite GOP criticism of the Part D stabilization program and its budgetary impact, the Trump Administration may decide to keep it in part to avoid blame for a premium increase.
The development of a joint document is not iterative and there is no discussion between the agency and sponsor, according to new guidance seemingly aimed at differentiating the frequent use of joint backgrounders in oncology from the single document developed for the Alzheimer's drug Aduhelm.
Retiring CDER Deputy Director Douglas Throckmorton spoke to the Pink Sheet ahead of his early 2025 retirement about regulatory flexibility, AI and other topics.