Whether in the corridors of the FDA or the fast-evolving frameworks of ANVISA in Brazil, regulators around the world are undergoing a quiet revolution. While mature markets like the US and Europe double down on digitalisation and real-world evidence, emerging players like Brazil and China are accelerating access and joining global harmonisation efforts at full speed. In 2025, a clear trend is emerging: regulatory agencies—regardless of geography or GDP—are embracing agility, collaboration, and innovation to reshape how medicines reach patients. This article dives into the global shift, from decentralised trials and smarter approvals to real-time data and cross-border alignment.

 

Accelerated Approval Pathways – Speed with Accountability

Regulators across the globe are working to expand accelerated approvals and conditional pathways, especially for breakthrough, orphan, and personalised therapies. The US FDA, initially cautious, has significantly increased its use of the accelerated approval pathway, greenlighting around 330 drugs by the end of 2024. Meanwhile, the European Medicines Agency’s (EMA) PRIME programme has helped reduce evaluation timelines from approximately 210 days to as few as 150.

National agencies are also adopting more flexible approaches. In Switzerland, Swissmedic enables early market access for treatments with promising, althoug incomplete, clinical data. “Swissmedic can grant conditional approval when preliminary data suggest a favourable benefit-risk profile,” explains Julia Djonova, head of its Advanced Therapy Medicinal Products Division.

In Latin America, Brazil’s ANVISA is prioritising therapies with high public health impact through its Resolution 204. “These include medicines for emergencies, diseases with no treatment options, and innovations that significantly improve patient outcomes,” says director Romison Rodrigues Mota.

However, these accelerated pathways are not without controversy. The FDA has faced criticism over delayed confirmatory trials, with an US Department of Health and Human Services OIG report revealing that nearly 40 percent of drugs approved through expedited channels between 1992 and 2021 had yet to complete post-approval studies. In response, the FDA now requires that confirmatory trials be underway at the time of accelerated approval—except in cases involving orphan drugs or public health emergencies.

Improving Efficiency – Smarter Approvals, Global Collaboration

Regulators are also working to streamline their internal review mechanisms. Brazil’s ANVISA, for example, has joined collaborative evaluation networks like Project ORBIS, which enables simultaneous reviews of oncology drugs by multiple regulatory authorities while maintaining independent decision-making. “Since 2020, ANVISA has approved over 40 applications collaboratively through ORBIS,” confirms Rodrigues Mota.

The UK’s MHRA launched its International Recognition Procedure in 2024, which expedites approvals by recognising decisions made by trusted foreign regulators. In a bold move, the agency also plans to outsource up to 40 percent of applications to further accelerate reviews.

China is embracing similar efficiencies by accepting clinical trial data generated outside its borders—an important shift aimed at reducing duplication and speeding up local approvals.

 

Real World Evidence (RWE) – from Nice-to-Have to Standard

Real-world evidence (RWE) is rapidly becoming a critical tool in the regulatory toolkit. The FDA’s Advancing Real-World Evidence Programme contributed to at least four approvals between 2023 and 2024. Similarly, the EMA’s DARWIN EU network is harnessing patient data across Europe to inform regulatory decisions.

Denmark is leading among European regulators in institutionalising RWE. With a strong national health data infrastructure and alignment with EU initiatives like the European Health Data Space (EHDS), the Danish Medicines Agency (DKMA) is scaling up its RWE initiatives. “Denmark is uniquely positioned to incorporate real-world data into both regulatory decisions and scientific advancement,” says DKMA Director General Nils Falk Bjerregaard.

The number of RWE-based projects in Denmark has more than doubled year-over-year. Bjerregaard acknowledges that RWE cannot fully replace clinical trials but sees value in regulatory sandboxes and pilot projects that build methodologies and governance structures.

In China, the National Medical Products Administration (NMPA) is increasingly incorporating RWE into approval decisions. However, challenges remain, particularly around standardisation and data quality. In Japan, limited infrastructure and industry scepticism have slowed broader RWE adoption, with notable resistance to proposals for RWD-only approval pathways.

Decentralised Clinical Trials

Decentralised clinical trials (DCTs) are redefining how studies are conducted by enabling remote participation and broadening patient access. With regulators like the FDA and EMA issuing clear guidance on decentralised elements, DCTs are becoming an integral part of modern clinical trial design.

By mid-2024, more than 2,350 decentralised trials were underway globally—a 45 percent increase in just two years. In Denmark, the DKMA has actively promoted DCTs to reach underserved patient populations and reduce geographic barriers to participation. “Denmark was among the first to fully operationalise the EU’s Clinical Trials Information System (CTIS),” says Bjerregaard.

Other major markets are also moving in this direction. China’s Center for Drug Evaluation (CDE) supports DCTs as a way to enhance efficiency and patient engagement. Japan, however, lags behind—only 18.6 percent of companies had implemented any DCT component by 2023, indicating a slower pace of adoption.

 

Global Harmonisation – Aligning Standards for Shared Impact

International harmonisation is gaining momentum as agencies strive to align trial protocols, safety standards, and submission formats across jurisdictions. Bodies such as the International Council for Harmonisation (ICH), the Pharmaceutical Inspection Co-operation Scheme (PIC/S), and the World Health Organization (WHO) are playing central roles in unifying global regulatory practices.

For ANVISA, harmonisation is not optional. “We must align with global best practices to avoid technological isolation,” says Rodrigues Mota. Brazil actively participates in international forums, including ICH, PIC/S, and the International Coalition of Medicines Regulatory Authorities (ICMRA), and also collaborates regionally within Latin America.

In Europe, the DKMA is deeply integrated into the EU regulatory framework and contributes to initiatives like the COMBINE project, which fosters consistency across EU member states. “This helps ensure that applicants receive coherent, non-conflicting advice,” says Bjerregaard.

A major step forward in EU-level harmonisation is the implementation of the Health Technology Assessment Regulation (HTAR), which took effect in January 2025. It introduces Joint Clinical Assessments (JCAs) for oncology and advanced therapy medicinal products (ATMPs), with plans to expand to orphan and all centrally authorised drugs by 2030.

According to Birgitte Klindt Poulsen, Chair of Denmark’s Medicines Council, the HTAR will improve both the efficiency and quality of assessments. “Earlier access to shared data and better methodological alignment across member states allows for stronger evaluations, while preserving national flexibility on cost-effectiveness and health policy,” she says.