Approval Standards
The European Commission’s new biotech and biomanufacturing hub will make it easier for innovative biotech companies to find legislative documents, funding information and guidance to help them bring new products to the market.
The UK’s drug regulator is developing a “clear and streamlined” regulatory pathway for individualized cancer mRNA immunotherapies.
The US FDA faces many communications challenges with vaccine skepticism rampant. Is doing everything right going to be good enough?
The US FDA wants a smoother transition to newer “low global warming potential” propellants in metered dose inhalers than the prior transition away from CFCs.
Medicines containing a new active substance (NAS) approved for pan-EU marketing last year were again led by cancer drugs, with treatments for blood disorders following closely behind. Meanwhile, three NAS-containing drugs have been approved so far this year, and 12 other products are expected to be authorized shortly, having recently been recommended for approval by the European Medicine Agency.
The MHRA’s new scientific dialog program will offer drug developers “closed-door meetings” that will offer “confidential, commercially sensitive discussion” with its staff to help them “refine” their real-world evidence generation strategies.
Former FDA commissioners Mark McClellan and Scott Gottlieb, former acting commissioner Janet Woodcock and current commissioner Robert Califf offered advice on successfully implementing reforms and preventing a mass exodus of FDA employees as inklings emerge that the Trump team is already engaged on this front.
The US Food and Drug Administration updated obesity drug development draft guidance for the first time in almost 20 years and although a lot has changed in the field, the FDA’s fundamental expectations to support approval did not.
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.
2024 saw important regulatory changes in India including in areas such as GMP, clinical trials and efforts to rein in unethical marketing practices. Further action is expected to play out in the new year as well.
The policies for China’s biopharma industry in 2024 centered around innovative small molecules, biologics and cell and gene therapies. Regulation changes for the industry in 2025 could be a continuation of that.
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.
Many of the recommendations listed in a new report, which aims to prepare the European medicines regulatory network (EMRN) for Alzheimer’s disease development challenges, will form part of EMRN’s future plans.
Incorporating pragmatic and decentralized approaches into postapproval studies presents less risk for data collection and reliability than in the premarket setting, an expert panel said at a Friends of Cancer Research meeting.
AZ’s DAPA-MI trial showed the feasibility of R-RCT designs to support new indications in drug development and more established uses to inform medical practice.
Convening an advisory committee before a formal proposal to withdraw has been the standard practice to date, but under FDORA it allows the agency to streamline the withdrawal process. The new accelerated approval draft guidance also codifies the practice of tapping other product center leaders to serve as decision-makers in withdrawal disputes.
Strategies championed by the Oncology Center of Excellence, including early planning and use of a single randomized study to support accelerated approval and later verify clinical benefit, are reflected in a new draft guidance broadly applicable to drugs and biologics.
New draft guidance discusses the data necessary for deciding whether an effect on a surrogate or intermediate clinical endpoint is reasonably like to predict clinical benefit and can support accelerated approval.
The agency also will not expand the guidance categories that may be issued “for immediate implementation” without prior public comment, but more guidance documents could be issued in Q&A or bulleted formats, the agency said.
The agency reaffirmed support for a hemoglobin A1C endpoint for glycemic control in type 1 diabetes, but the diabetic ketoacidosis risk with sotagliflozin is so concerning that it is looking for additional benefits in patients with chronic kidney disease, a targeted subpopulation.