In an era of rapid scientific breakthroughs, global regulators are navigating new challenges—accelerating approvals, harmonising standards, and integrating digital tools—while balancing safety, efficacy, and access. Throughout 2024, PharmaBoardroom spoke with regulators worldwide to uncover the key regulatory trends shaping the future of healthcare in 2025 and beyond.
The Road Towards Global Regulatory Harmonisation
Regulatory fragmentation slows innovation and delays patient access to treatments. To address this, global regulatory agencies are intensifying efforts to standardise approval processes, clinical trial protocols, and compliance frameworks. Initiatives like the International Council for Harmonisation (ICH) are driving this transformation, ensuring greater consistency across markets.
For its part, Switzerland’s Swissmedic actively participates in several harmonisation efforts. “All these collaborations allow us to help shape standards and regulatory frameworks for innovations and new technologies worldwide, which in turn benefits Switzerland’s therapeutic product industry,” says executive director Raimund Bruhin. For Bruhin, being a part of the Access Consortium along with Canada, Australia, Singapore, and the United Kingdom, has been particularly important, as well as taking part in the FDA’s Orbis initiative. “This close international cooperation enables the rapid, targeted approval of medicines while fostering knowledge exchange and technological innovation,’ he asserts. “A standout feature of this collaboration is true work-sharing, where partner authorities assess specific modules of the scientific documentation. This sharing of work enables shorter processing times and minimises the submission gap, leading to quick and efficient approvals.”
The FDA clearly understands the role of harmonisation and collaboration with other agencies. Involved in the International Council for Harmonisation (ICH), International Pharmaceutical Regulators Programme (IPRP), the Pharmaceutical Inspection Co-operation Scheme (PIC/S), Asia-Pacific Economic Cooperation (APEC), and International Coalition of Medicines Regulatory Authorities (ICMRA), the FDA also operates several global offices. “The FDA leads standards and practices harmonisation efforts with other regulators across many regulated markets to reduce regulatory uncertainty and provide more predictable pathways for innovations to meet standards for safety, quality, and efficacy,” claims former country director of FDA India Sarah McMullen. “When regulations are applied consistently across multiple markets, that standardisation increases both access to markets by industry and to products by consumers and patients.”
Japan’s PMDA is looking to act as a facilitator for regional harmonisation and with its Bangkok office offer a gateway to Asia. “We hope to support the promotion of regulatory harmonisation among ASEAN countries directly, as well as drive the development of a regional clinical research network from regulator’s viewpoints,” says former senior executive director Shinobu Uzu.
The Power of Real-World Evidence
Regulators are tapping into real-world data to bridge the gap between clinical trials and everyday patient outcomes. By leveraging large-scale datasets, agencies can make more informed decisions on both new and existing therapies. The European Medicines Agency’s (EMA) DARWIN EU — a programme that encompasses several member states as well as non-EU countries, bringing together GP datasets, hospital data sets, and registries — has made some major strides. “DARWIN EU promises to be a great success. It is growing and delivering clinical evidence via real-world data studies,” says Peter Arlett, head of data analytics and methods at the EMA. “The current 20 data partners represent 130 million active patients who are continually contributing data, making this a massive and powerful system already.”
From 14 completed studies at the beginning of 2024 to an ambitious goal for 2025 and beyond, the programme represents a sweeping development for the use of RWE. “From 2025, we are contracted to a ceiling of 140 studies, which is an absolute game changer. As far as I’m aware, no other organisation in the world has ever done RWE studies in those kinds of numbers before,” Arlett asserts.
For Arlett, standardisation is again the name of the game, namely DARWIN EU’s Common Data Model. “The data partners and datasets we are onboarding are converted into a common structure with mapped terminologies, meaning that computer script can be run through the entire dataset despite the original data source being in different formats. Researchers can then upscale in terms of the number of data sources rather quickly.”
DARWIN examines an array of different types of studies and questions. “We can look at disease epidemiology, understanding who gets a particular disease, their age, gender distribution, their symptoms or mortality, how they are treated, the pathway in terms of therapeutics, and the natural population with a disease compared to the clinical trial population,” Arlett remarks. “We can also do causality studies: looking at associations between particular medical events and exposure to medicines. This is very well established in the drug safety area and has been done for over 30 years but is still relatively new in terms of efficacy and effectiveness. For example, there were some promising vaccine effectiveness studies using DARWIN conducted during COVID because high levels of testing meant that there were a lot of records of infection.”
Tackling Advanced Therapies
The rise of advanced therapies—mRNA, gene editing, CAR-T cell treatments—has pushed regulators to rethink approval processes. Swissmedic, for example, launched a dedicated department for Advanced Therapy Medicinal Products (ATMP) to accelerate approvals while maintaining safety standards. “To meet these demands, we have established a specialised department that allows us to quickly and safely approve these advanced medicinal products, as well as continuously refine our expertise and processes in this highly innovative field,” states Bruhin.
“Swissmedic is prepared to support cutting-edge technologies and expedite the approval of these products. This initiative sends a strong signal to the industry: we have the competencies needed to regulate and promptly approve these novel therapies, and we are committed to taking a leading role in this area. This includes cell and gene therapies, CAR-T cell therapies, mRNA-based vaccines, and tissue engineering,” executive director affirms.
“Areas like cell and gene therapy are certainly new frontiers for the CDSCO,” Santosh Indraksha, deputy drugs controller at India’s Central Drugs Standard Control Organisation (CDSCO) confirms. To adapt, CDSCO is learning from international counterparts. “We’ve collaborated with Japan’s PMDA and the US FDA on CAR-T therapies and have more regulatory partnerships planned with the UK, Denmark, and the Netherlands,” explains Indraksha.”
Ramping Up Digital Transformation
Regulators have long recognised the value of digitalisation and its impact on efficiency. The Danish Medicines Agency (DKMA) was an early adopter of digital tools and has since had to update systems to keep pace and accelerate its processes. “Given that Denmark was one of the countries at the early forefront of digitalisation, some of our IT systems are now becoming outdated. We have needed to make infrastructure investments,” Lars Bo Nielsen, former director general of DKMA contends. These updates have led to some major advances. “One of the things that we are particularly proud of is the installation of several robotic process automations (RPAs); product solutions for handling some of our document and case flows. Additionally, we have adopted an AI-based solution for our information centre,” says Nielsen.
Swissmedic is also looking to gain efficiency through digitalisation and take its place among the world’s more digitally advanced agencies. “Our goal is to soon adopt state-of-the-art digital technology and become a data-centric authority. For example, in August 2024, we successfully introduced a new cloud-based medical device database. This is one step toward becoming one of the top five digitally advanced regulatory authorities. This modernisation is essential not only for interoperability with other regulatory authorities but also for effective collaboration with the industry.”
India’s CDSCO has already made some major advances, deputy drugs controller Indraksha points out. “We currently have a 100 percent online system for all processes relating to drugs, devices, cosmetics, and imports, meaning that applications can be made from anywhere in the world. If deficiencies are found, they are communicated online, and approvals are also given online.” Digitalisation has also led to standardisation across India’s individual states. “CDSCO has taken the initiative to develop and deploy a unified Online Drugs Licensing System (ONDLS) to all the State/UT Drug Control Authorities and many States have already on-boarded this platform,” he says.
For the DKMA’s Nielsen, keeping up with technology also goes beyond a country level. “We are looking at how to use AI as part of the European system. Together with the EMA, the Heads of Medicines Agencies (HMA) is part of the Big Data Steering Group Initiative, where we now have an AI workplan for the coming years.”
New Role as Pro-active Facilitators
Some regulators are shifting from gatekeepers to facilitators, actively engaging with industry to attract innovation. Japan’s PMDA, for example, is making regulatory processes more transparent and accessible to encourage early-stage R&D investment. “We are proactively engaging internationally with other national regulators, international bodies, and individual companies – both big and small – to disseminate information about the transparent and efficient regulatory processes that exist in Japan,” says PMDA’s Uzu. “We are engaging with companies earlier to show them how flexible and friendly PMDA is, and ultimately ensure that Japan is not losing out on innovation.”
Breakthrough Therapies and EU Reform
Fast-tracking breakthrough therapies is becoming a top priority for regulators worldwide. The US FDA’s Breakthrough Therapy Designation and EMA’s PRIME initiative are setting the pace, helping life-saving treatments reach patients faster. A recent International Coalition of Medicines Regulatory Authorities (ICMRA) Symposium on Orphan Diseases “brought together experts from around the world to discuss and reassess the definition and regulation of orphan drugs,” recounts Swissmedic’s Bruhin. “A key topic was distinguishing between truly rare diseases and so-called “orphan-like” diseases, which may require new criteria to update regulations in this area.”
An important development in Europe is of course the new EU Pharma legislation aimed at increasing availability, access and affordability of medicines in Europe. At last year’s DIA Europe, the EMA’s chief executive, Emer Cooke, said the moment was “exciting and inspiring,” noting other important reforms like the European Health Data Space and HTA reform. Rainer Becker of the European Commission directorate-general for health and food safety stressed that the new legislation, after 50 years without reform, paves the way for a “fairer and more competitive” ecosystem.
As regulatory agencies worldwide push for harmonisation, digitalisation, and real-world evidence integration, the industry stands at a pivotal moment. The challenge remains: how to balance speed and safety while ensuring equitable access to medical innovation. The next few years will determine how successfully regulators navigate these shifting dynamics.
