This week, we heard from health authority leaders from the United States, Europe, the United Kingdom, and beyond at the DIA 2025 Global Annual Meeting in Washington, DC, and one theme stood out: modernizing regulation to match innovation, with leading health agencies at the forefront.
In the Opening Plenary and Keynote Address: From Personal Journeys to Global Solutions – Collaborating for Innovation and Patient Impact, agency leaders were asked to reflect on how they are keeping pace with fast-moving advancements in healthcare. Leadership from the FDA, the European Medicines Agency (EMA), and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) emphasized the urgency of aligning regulatory systems with therapeutic innovation and shared what their agencies are doing to stay ahead.
FDA: Commissioner Makary’s Push for Reform
FDA Commissioner Dr. Martin Makary calls for meaningful transformation both within the FDA and in how we approach public health in the U.S., including raising our children. In Monday’s panel and numerous public forums, Makary underscored the need to dismantle legacy systems that create unnecessary barriers to innovation.
He advocates for integrating real-world evidence (RWE) and external data sources, such as healthcare databases, to inform and refine drug approval thresholds. Makary also calls for a shift away from outdated evaluation models, promoting the use of computational modeling and organoids instead of relying solely on traditional animal studies.
His vision goes beyond drug approvals: future FDA pilot programs will address broader public health issues such as food systems, pediatric health, and upstream drivers of disease. Central to his approach is an outcomes-based mindset focusing on faster access to treatment, more adaptable regulatory pathways, and decisions grounded in population-level health impact.
EMA: Adapting to Innovation Through Internal Transformation
Emer Cooke, Executive Director of the EMA, highlighted the rapid progress in healthcare technologies, including cell and gene therapies, and reinforced the agency’s responsibility to enable timely patient access. The EMA’s strategy is multifaceted.
The agency is expanding accelerated scientific advice and regulatory support to reduce development and approval delays. EMA is also establishing frameworks to incorporate RWE into regulatory decisions, using platforms such as DARWIN EU[i] to support data access and analysis. Significant investments have been made in technology, including artificial intelligence, speech-to-text tools, and internal systems designed to streamline assessments.
The EMA is also enhancing patient and caregiver engagement, using AI to synthesize lived experience through collaboration with EURORDIS[ii]. Cooke described the agency’s outlook as rooted in “bold thinking,” with a goal of authorizing medicines that make a genuine difference based on real-world evidence and clinical trial data.
Shared Direction: A Mindset Shift in Regulation
The FDA and EMA recognize that regulatory science must evolve to match the pace and complexity of modern therapeutics. From AI-enabled tools and RWE integration to data-sharing platforms and systemic pilots, today’s global regulators are laying the foundation for a more responsive, patient-centered future.
These changes reflect more than procedural reform they mark a mindset shift. Regulators are no longer trying to keep up with innovation; they are embracing a shared role in enabling it.
What Does Success Look Like in Five Years?
In response to a final panel question, leaders outlined what success could look like by 2030:
FDA (Dr. Makary, Commissioner):
- Universal access to transformative treatments for rare diseases.
- Regulatory systems that are agile, adaptive, and informed by RWE, AI, and computational modeling.
- Greater reliance on electronic health data, patient-reported outcomes, and external evidence in decision-making.
- A proactive role in public health, addressing chronic disease prevention, and leveraging the agency’s unique oversight of both food and medicine.
EMA (Emer Cooke, Executive Director):
- Modernized internal systems supported by AI and automation for faster reviews.
- Accelerated access to medicines through structured pathways without compromising safety.
- Expanded use of RWE via DARWIN EU to evaluate real-life therapy performance.
- Deepened collaboration with rare disease organizations and patient communities.
MHRA (Lawrence Tallon, CEO):
- Continued leadership in advanced therapies and cancer vaccines.
- A proactive, partnership-based approach to innovation across stakeholders.
- A regulatory model that is flexible, transparent, scalable, and designed to support scientific progress.
While each agency brings its own context, all three are aligned on the fundamentals. Regulatory success in the next five years is not simply about speeding up approvals or embracing new technologies but building systems that evolve alongside innovation, rooted in real-world impact, collaboration, and a shared commitment to public health.
At RareMoon Consulting, we welcome this direction. Our work with advanced therapies and rare diseases has shown firsthand how transformative regulatory alignment can be when it prioritizes both urgency and integrity.
[i] DARWIN EU is a federated network that connects the EMA and national regulators to real-world data sources across Europe, including Electronic health records, Claims databases, Disease registries, and Hospital data.
[ii] EURORDIS is a non-governmental alliance of over 1,000 patient organizations from more than 70 countries, representing people living with rare diseases in Europe and working with the European Commission, EMA, and national governments to shape rare disease policies and access frameworks.
