Approval Standards
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The US Food and Drug Administration updated obesity drug development draft guidance for the first time in almost 20 years and although a lot has changed in the field, the FDA’s fundamental expectations to support approval did not.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among other factors, in determining whether a confirmatory trial is underway prior to accelerated approval, but the new draft guidance does not specify the proportion of patients that must be enrolled at the time of approval.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.
When the agency decided biosimilarity standards would be high, it likely ensured the interchangeability designation eventually would become unnecessary.
New US FDA draft guidance attempts to address sponsor confusion about the different types of regulatory meetings under PDUFA, as well as frequently asked questions on nonclinical testing, CMC, pharm/tox and clinical studies.
In the second part of the Pink Sheet’s interview with the former senior FDA official, Woodcock says her push to create a rare disease drug approval pathway that wouldn’t require a randomized controlled trial will not return the agency to “an anecdote standard” or cause a downward creep in the evidentiary standard for approval.
The former CDER Director spoke to the Pink Sheet about why a new statutory standard is needed for certain rare disease drug approvals. We lay out some of her thinking in part one of a multi-part series on the topic.
Regulators do not have the resources to “double check” that companies are using AI appropriately, meaning that manufacturers must ensure the AI tools they use meet relevant standards, says an EU regulatory expert.