The year 2025 has started off with the appearance of a year of uncertainty, however the regulatory landscape in 2025 will continue to reflect progress made by regulatory agencies fostering innovation in regulatory science in several key areas and beyond. 

While there are many predictions for the year 2025, there are five key regulatory trends that are laying the foundation for updated policies, risk-based regulatory frameworks, and forward-looking initiatives. These five key trends will likely shape the regulatory environment for the use of technology, scientific innovation, and drug development in 2025 and beyond.

Innovation in Clinical Trials:

In December of 2023, the US FDA published a draft guidance on the use of Master Protocols for drug and biological product development in December 2023. The proposed draft provides recommendations for designing and analyzing complex clinical trials, such as umbrella, platform, and basket trials, to streamline drug development. It emphasizes the use of concurrent control groups, randomization, and blinding to ensure robust and unbiased results. It will be important to watch if the FDA finalizes the guideline in 2025 what aspects are adopted following public consultation and if there becomes greater use of master protocols more commonly in therapeutic areas other than oncology, such as treatment of obesity and associated co-morbidities, and in neurodegenerative diseases.

Looking at the 2025 – 2026 workplan for Accelerating Clinical Trials in the European Union (ACT-EU) initiative – with a vision to have better, faster, and optimized clinical trials – efforts continue to support implementation of the Clinical Trials Regulation and work to address stakeholder needs and feedback. The Multi-stakeholder Platform continues to be a key feature of the initiative focusing on communication, understanding challenges and opportunities, and helping to define priorities. Finally, there are several activities planned for 2025 to facilitate work on clinical trial methodologies with similar themes as seen in the U.S. FDA guidance agenda (e.g., Bayesian statistics, pediatric trials, and platform trials).

Other novel areas to closely monitor in 2025:

• Use of Bayesian Methodology in Clinical Trials of Drug and Biologic Products, including use of Bayesian methods for Pediatric Clinical Trials.
• Clinical Pharmacogenomics – Evaluation, Study Design, and Analysis

Artificial Intelligence and other emerging technologies in drug development

The rapid proliferation of artificial intelligence (AI) and large language models (LLMs) is reshaping drug development and clinical research at an unprecedented pace. However, this explosive growth has also caught the attention of regulatory bodies. To ensure that these advanced technologies are ethically and safely integrated, regulators are actively engaging with industry stakeholders and advancing regulatory policies. Presenting both opportunity and uncertainty, these efforts are laying the foundation of a regulatory framework for a more technologically advanced healthcare and drug development environment in 2025 and beyond.

In January 2025, the US FDA published draft regulatory guidance regarding the Considerations for Use of Artificial Intelligence to Support Regulatory Decision-Making for Drug and Biological Products.  The guidance follows FDA’s 2023 discussion paper and aims to ensure that AI models are reliable and transparent in the application to regulatory processes.  There is a focus on developing a risk-based credibility assessment framework for establishing and evaluating the credibility of AI models used to support regulatory decision-making about the safety and efficacy of drugs and biologic products, with special considerations for life-cycle maintenance.

Additionally, the growing adoption of cloud-based technologies, alongside AI, is transforming regulatory submissions and data exchange by enabling real-time collaboration and a more agile approach to product development and lifecycle management. The FDA’s PRISM Project exemplifies this shift, offering a cloud-based platform to streamline regulatory submissions and scientific reviews. However, cloud adoption introduces challenges such as data security and IP protection, prompting the development of stringent guidelines to safeguard sensitive information. By adopting secure cloud-based frameworks, medical product developers can reduce submission bottlenecks, accelerate approval timelines, and enhance cross-border collaboration with regulatory agencies.

Oncology Drug Development

The US FDA’s Oncology Center of Excellence (OCE) continues to be active in advancing regulatory science in support of oncology drug development and regulation. OCE published an ambitious Guidance Agenda for 2025. Topics to watch closely include a revision to existing guidance on clinical trial endpoints for approval of cancer drugs and biologic products and approaches to assessment of overall survival in oncology clinical trials. Adaptations and evolutions for clinical trial endpoints in cancer trials, including overall survival, could have great impact for the future of oncological drug development.

Finally, OCE plans guidance for Oncology Therapeutic Radiopharmaceuticals – Dose Optimization during clinical development. As with OCE’s Project Optimus to encourage dose optimization early in Oncology Drug Development, further regulatory guidance specific to Radiopharmaceutical dose optimization could have similar effects for dose finding and clinical development planning.

Global Regulatory Harmonization

Another key trend shaping 2025 is regulatory bodies around the world continuing to work at the scientific and technical level to collaborate and advance convergence for robust regulatory standards. Additionally, work to modernize global drug development, including in areas such as training and adoption of Good Clinical Practices (GCPs), further implementation and use of multi-regional clinical trial principles and approaches will continue at an accelerated pace.

Broader adoption of the electronic common technical document (eCTD) format within ICH (International Council for Harmonization) member countries. This standardized approach to regulatory documentation reduces duplication, minimizes errors, and simplifies submissions across multiple regulatory agencies. As more regulatory bodies worldwide integrate eCTD standards, pharmaceutical companies will benefit from a more consistent and predictable submission process. This shift will not only enhance efficiency but also help reduce compliance risks.

Opportunity in a Time of Uncertainty

Change, transition, uncertainty, new leadership, different thinking are all terms that commonly can create anxiety and confusion. Fundamentally, science and disease remain the same, with investment and planning cycles that span decades. Patients are waiting. Many opportunities exist in the face of uncertainty. Ideas and ideologies to decrease bureaucracy and reduce or reform regulatory paradigms can coexist with ambitions to maintain and advance a “gold standard” for drug regulation around the world.

Consider fields such as Cell and Gene Therapy, or the many still untreated conditions identified as rare diseases. Regulatory frameworks and opportunities for new ways of thinking about the future of regulatory science are still to be developed along with the challenge and opportunity to consider new or different approaches. 

Preparing for the Future, Today

To stay ahead in this evolving regulatory landscape, pharmaceutical leaders must anticipate these changes and integrate compliance strategies into their long-term R&D and commercialization plans.  Further, comprehensive strategic and regulatory planning, when applied to drug development strategies and clinical development plans, can reduce evidentiary risk throughout a product’s lifecycle.

2025 presents a pivotal opportunity for the industry to embrace regulatory modernization, driven by AI-powered tools, cloud-based technologies, and expanded global harmonization efforts. At the same time, regulatory agencies will continue fostering innovation while reinforcing patient safety, data integrity, and ethical oversight. Organizations that align with these regulatory shifts early on will not only maintain compliance but also gain a competitive advantage in bringing transformative therapies to the market faster and more efficiently.

David Cameron, MPH, is the Sr. Director and Global Head, Novel Trial Design, within the Regulatory Affairs & Drug Development Solutions at IQVIA.  In this role he retains global responsibility for developing evidence generation, solution synthesis and messaging in support of innovative design approaches to improving the productivity of clinical research and development.  David has deep expertise in the industry with more than 18 years of experience consulting worldwide with biopharmaceutical organizations.