The approval of eflornithine (brand name Iwilfin) for pediatric high-risk neuroblastoma offers an interesting example of how the FDA evaluates single-arm trials supported by external control arms (ECAs). External controls are increasingly common in oncology and rare disease drug development, but they remain a source of careful regulatory scrutiny. In this case, the FDA’s assessment… Continue reading Regulatory Insights from FDA Review of the External Control Arm in a Pediatric Rare Disease Trial
Tag: Regulatory
EHDS Section 60: The Illusion of Neutral Data Sharing?
Reading the EHDS regulation carefully, and in particular Section 60, leaves a sense of discomfort that is not really about legal drafting, but about how knowledge is produced and legitimized. The text is cautious, layered with safeguards, and repeatedly reassures the reader that intellectual property, trade secrets, and commercial confidentiality remain protected. And yet, between… Continue reading EHDS Section 60: The Illusion of Neutral Data Sharing?
European Health Data Space (EHDS): Status, Implications for Patients and Doctors
What the EHDS is and why it was established The European Health Data Space (EHDS) is a European Regulation (EU 2025/327) that entered into force in March 2025. It creates a common framework for using electronic health data across the European Union (EU). The main motivation behind the EHDS is the increasing need for… Continue reading European Health Data Space (EHDS): Status, Implications for Patients and Doctors
The Role of Real-World Data in HTA Reassessments
Health technology assessments (HTAs) are the foundation of evidence-based decision-making for reimbursement, and coverage of medicines and medical technologies. Traditionally, the process has been heavily anchored in evidence from randomized controlled trials (RCTs), assessed at the time of market entry. However, healthcare systems have been evolving and are increasingly shifting toward a lifecycle approach to… Continue reading The Role of Real-World Data in HTA Reassessments
Label Expansion and the Role of RWD
In the lifecycle of a pharmaceutical product, initial regulatory approval is often just the beginning. A newly authorized drug typically enters the market with a carefully defined label specifying the disease, population, dose, and setting for its use based on data from randomized controlled trials (RCTs). But often label specifications evolve. Real world clinical needs… Continue reading Label Expansion and the Role of RWD