A cluster of CRLs for rare disease applications based on one trial plus confirmatory evidence may represent a shift away from the regulatory flexibility that had come to characterize ultra-rare drug development
New approach methodologies are increasingly shaping the future of medicine development by making drug testing less reliant on animals.
India is advancing regulatory rationalization efforts, refining procedures and pathways. Guidelines for biosimilars are expected to align with international standards, while early steps towards PIC/S compliance have been initiated.
Horizon scanning is a tool used by regulators to prepare for forthcoming medicines that might challenge existing processes. Senior figures from the European Medicines Agency, Swissmedic and the UK MHRA explain how they conduct horizon scanning activities in their respective jurisdictions.
Real-world evidence (RWE) studies in India face challenges like lack of data reliability and uniformity and absence of clear guidelines but firms like Bharat Serums have scored a regulatory win. Pink Sheet examines the RWE landscape and the BSV case study for lessons in beating the odds
FDA reviewers cited several limitations of the videos, including a lengthy recall period, use of the term “today” in questions, and a lack of standardization in interview conduct.
The Pink Sheet’s Drug Review Profile looks at the clinical development and US FDA review timeline for Stealth's elamipretide for Barth syndrome.
Stealth BioTherapeutics' diligent response to the FDA’s accelerated approval suggestion, its justification for proposed confirmatory trial milestones, and the rarity of Barth syndrome gave the agency confidence to grant approval before the postmarketing study began.
Recently departed CDER Director George Tidmarsh publicly touted his own role in reviewing Stealth's elamipretide data and guiding the division on the last-minute accelerated approval, though his name is absent from the review documents.
Flu vaccines are specifically mentioned in the CBER director's internal email, but all new and some older vaccines seem to be affected. The impact on development programs is unclear.
While there is a general shift towards alignment and collaboration in health technology assessments in the EU under the new HTA Regulation, member states still have “their own recipe” when it comes to making reimbursement decisions.
While the volume of questions that companies have received from the EU member states under the Health Technology Assessment Regulation has been lower than expected, some firms are struggling with the comparator analysis required to answer some questions, an expert says.
Association for Accessible Medicines CEO John Murphy told the Pink Sheet that the FDA may need to consider phasing in the requirements for the new ANDA priority voucher incentive program if it wants sponsors to apply in the near-term.
