“At the European Medicines Agency, we work hard on trying to make sure that we can enable innovation by putting in place mechanisms where developers will come talk to us, explain what we're doing, and we can try and advise them on how we make sure that that innovation will actually eventually reach innovations,” Cooke said.
She also highlighted some current areas of focus for EMA, including the use of AI and patient experience data in rare disease research. The agency has set up the Data Analysis and Real-World Interrogation Network (Darwin EU), which encompasses 30 data partners in 18 countries, to help answer questions about the way medicines perform in real-life settings and further advance rare disease research.
Makary then highlighted areas of focus for FDA. He said: “We have to be willing to learn from data other than randomized, controlled trial data. We preserve that gold standard methodology, but at the same time, we can learn from real-world data, we can learn from synthetic controls.”
Under Makary’s leadership, the FDA is also putting an emphasis on reducing animal testing and taking a closer look at food ingredients that could potentially be toxic.
“We have to study the environmental exposure to ingredients in our food supply, not just chemo and radiation to treat cancer,” Makary explained.
Tallon then highlighted the need for more collaboration between all the global regulatory bodies. He explained that some areas of the world may have more expertise in a certain area of medical research than others, making it crucial that all regulatory bodies work together to bring life-changing therapies to patients around the globe.
“We need more international reliance and recognition. That will be a key priority,” he said.
Future of medical research: Inspiring the next generation
As the panel concluded, each member shared their closing thoughts. Melton placed an emphasis on the future of medical research and getting more young people involved.
He said: “I hope as a community, we can continue to encourage curious young people that doing science and medicine is a kind of call. It's something that's both interesting and fun and has to get the benefit of doing good in the world.”
Olaghere, who had earlier shared his own story of battling sickle cell disease and receiving Casgevy (exagamglogene autotemcel) as treatment, concluded: “I think we need to start moving away from this value-based healthcare system that has dictated for decades, and turn it in to something new. We need to fix that. We need to break the wheel and start to imagine the ways to get therapies to people as quickly as possible.”
Reference
Cooke E, Makary M, Melton D, Olaghere J, Parikh S, Tallon L, Vassileva M. Powering Progress: Global Collaboration to Derisk Innovation and Improve Patient Lives. June 16, 2025. 2025 DIA Global Annual Meeting.
