Review Pathways
The FDA’s accelerated approval draft guidance has left stakeholders seeking clarification of the process for determining a surrogate marker or intermediate clinical endpoint is reasonably likely to confirm clinical benefit.
Experts working in the advanced therapy space say the US has less strict criteria for regulatory pathways for cell and gene therapies than the EU, particularly for products in early development.
Levodopa/carbidopa (ND0612), Mitsubishi Tanabe Pharma’s investigational drug-device combination therapy for the treatment of motor fluctuations in people with Parkinson’s disease, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.
An enhanced role for patients in the European Medicines Agency was a key proposal within the EU pharmaceutical legislation overhaul – but patient groups warn this provision could be scrapped or weakened due to ongoing negotiations.
The UK drug regulator has made a number of improvements to its innovative licensing and access pathway, including a more “core” role for National Health Service partners, which is expected to facilitate flexible commercial discussions around drug reimbursement.
The European Medicines Agency is considering whether apitegromab, brensocatib and diazoxide choline, from Scholar, Insmed and Soleno respectively, are drugs that are of potential major public health interest, particularly from the point of view of therapeutic innovation.
The agency sent the neuroscience-focused company a refuse-to-file letter for Wakix as a treatment for Idiopathic Hypersomnia, a disease with only one approved treatment and few others in development.
The US FDA is receiving more requests for regenerative medicine advanced therapy designation and granting more of them, according to recently reported agency data.
Companies that are admitted to the UK’s revised innovative licensing and access scheme will be mandated to work with the regulator on developing a target development profile for their product.
The inspector general said the council should be more involved in specific approval decisions when there is internal disagreement, but FDA said that would preclude some individuals from adjudicating a subsequent withdrawal proceeding.
Two recent accelerated approval guidances describe the FDA’s policy expectations for trial timeliness, but recognize the need for case-specific determinations. Regulatory actions in 2024 show the FDA is willing to delay application filing or approval over confirmatory trial status.
The European Medicines Agency’s chief medical officer, Steffen Thirstrup, said the surge in positive opinions adopted by the agency in 2024 for new marketing approvals signals a post-COVID recovery.
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.
Vanda’s fight against a complete response letter for its gastroparesis drug results in a notice of opportunity for hearing on a formal FDA proposal to refuse to approve the NDA.
Regenerative medicines using expedited review pathways dominate novel approvals at the Center for Biologics Evaluation and Research, while the Center for Drug Evaluation’s higher volume comes with lower first-cycle approval rates and more standard reviews.
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.
The US FDA drugs center cleared 50 novel agents and the biologics center contributed 11 novel biologic approvals. The agency also acted on 77 novel applications, including 16 complete response letters.
A Catalyst fix, a pediatric rare disease voucher reauthorization that should prevent future workload imbalances for FDA, and a boost for out-of-state coverage in Medicaid are among Congress’s year end commitments to pediatric and rare disease patients that now seem in doubt.