Radiopharmaceuticals: Aligning FDA and EMA Guidance for Global Development

Harpreet Singh, MD, former Director of the Division of Oncology 2 at the FDA, noted the importance of considering the global regulatory environment in the development of radiopharmaceuticals. This is particularly relevant as the European Medicines Agency (EMA) also issued a position or concept paper in late 2024 regarding these novel agents.

The simultaneous release of significant regulatory documents from major global bodies—the FDA's draft guidance and the EMA's concept paper—creates a critical need for alignment. Sponsors developing these assets operate internationally and prefer regulatory requirements to be as harmonized as possible to streamline clinical development and eventual market access. Dr. Singh emphasized that stakeholders are currently hoping and expecting some form of discussion or collaboration between the EMA and FDA to achieve convergence on their expectations for sponsors.

The core challenge lies in the slight misalignment that currently exists between the 2 documents. While both agencies are focused on ensuring appropriate dose justification and patient safety for radiopharmaceuticals, differences in specific requirements—such as the required duration of long-term safety follow-up, the weight given to dosimetry data versus clinical toxicity, or precise trial design expectations—can force sponsors to adopt dual development paths. Such duality inevitably leads to increased complexity, cost, and time for drug development.

For pharmaceutical sponsors, awareness of this regulatory landscape is paramount. The first step for any developer is to thoroughly analyze both regulatory documents to identify areas of divergence and overlap. This comparative analysis is crucial for designing a global clinical development plan that has the highest probability of satisfying both agencies.

Secondly, Dr Singh suggested that stakeholders have a responsibility to actively participate in the harmonization process. This involves providing constructive feedback during the open comment periods for the FDA's draft guidance. The industry's feedback should aim to highlight areas of conflict and propose scientifically sound solutions that could bridge the gap between the two regulatory bodies. By proposing harmonized approaches to aspects like dosimetry data submission or long-term safety monitoring protocols (eg, suggesting a more flexible, risk-adapted follow-up schedule instead of a rigid 5-year requirement), sponsors can help shape the final guidance documents toward a more globally acceptable standard.

Ultimately, the successful and efficient development of these complex radiopharmaceutical assets—which offer new hope for patients with cancer—depends on the regulators' ability to establish a unified, robust, and predictable framework. Without this harmonization, regulatory uncertainty risks slowing down innovation and hindering global access to these novel, highly targeted therapies.

Watch part 1, part 2, and part 3 of this interview with Harpreet Singh.