EMA Unveils 2025 Regulatory Science Strategy to Advance Innovation in Medicines

Recently, the European Medicines Agency (EMA) announced its strategic objectives for pharmaceutical regulation through 2025. We have compiled and translated these objectives to outline how regulatory authorities should respond to technological advancements in the future, translating scientific and technological innovations into better patient-centered healthcare services.

In recent years, the pace of innovation has accelerated significantly, requiring regulatory agencies to prepare to support the development of increasingly complex drugs. A growing number of pharmaceutical products are integrating diverse technologies to promote and safeguard human and animal health, thereby delivering medical solutions.

For regulatory agencies, how should they prepare to address these emerging technological innovations? Do regulatory bodies possess the necessary skills and capabilities to facilitate the utilization and review of such innovations, including revolutions in fields such as big data, precision medicine, advanced manufacturing, novel clinical trial designs, cell and gene therapies, artificial intelligence, and synthetic biology?

The EMA’s Draft Strategic Objectives for 2025 is a plan designed to enhance the EMA’s engagement with regulatory science over the next five to ten years, covering both human and veterinary medicines. In December 2018, the EMA released a draft of the strategic objectives for a six-month public consultation.

EMA Executive Director Guido Rasi stated, “The Regulatory Science Strategy to 2025 aims to establish a more adaptive regulatory framework to encourage innovation in both human and veterinary medicines. This strategy, which addresses developments and challenges in drug development, was jointly formulated by EU Member States and expert committees. We now seek stakeholders’ views on whether they have sufficient confidence in this strategy.”

The European Medicines Agency’s strategic objectives primarily focus on scientific and technological innovation to advance regulatory science. Revolutionary advances in technologies such as cell and gene therapies, combination products, novel clinical trial designs, real-world evidence, big data, and artificial intelligence (AI) pose significant challenges to regulatory authorities. Therefore, in 2016, the EMA formally established a Regulatory Science Observatory to monitor emerging scientific and technological trends and guide resource allocation and external collaborations to promote regulatory science. The EMA’s vision is to catalyze and enable the translation of science into patient-centered care and its delivery within evolving healthcare systems. To achieve this vision, the EMA has formulated five major strategic objectives for 2025.

• Promote the integration of science and technology in drug development;

• Promote the generation of collaborative evidence to enhance the scientific quality of assessments;

• Collaborating with healthcare systems to ensure patient-centered medication accessibility;

• Addressing emerging health threats and challenges in treatment accessibility;

• Promoting and Leveraging Research and Innovation in Regulatory Science.

The ultimate goal of public health is to ensure that regulation supports the development of new drugs and innovative technologies, thereby better meeting patient needs through safe, effective, and clinically appropriate treatments. This requires regulatory frameworks to evolve toward patient-centered healthcare, precision medicine, and personalized medicine. Therefore, the European Medicines Agency (EMA) seeks to integrate the latest scientific and technological knowledge into pharmaceutical development to benefit public health.

This necessitates specific objectives aimed at encouraging and enabling the integration of the latest scientific and technological knowledge into the drug development phase, ensuring continuous dialogue among regulatory agencies, academic research centers, and developers throughout all stages of the product lifecycle. Specific recommendations include:

• Support the development of precision medicine, biomarkers, and “omics.” Strengthen early engagement with developers of novel biomarkers to facilitate regulatory qualification, address the impact of emerging “omics,” and promote their application throughout the development lifecycle. Collaborate with health technology assessment (HTA) bodies, payers, and patients to evaluate the impact of therapies on clinical outcomes measured by biomarkers.

• Supporting evidence generation for advanced therapy medicinal products (ATMPs). The EMA recommends enhancing early interactions among developers, regulatory authorities, health technology assessment (HTA) bodies, and payers to address regulatory issues related to ATMPs at an early stage and facilitate faster patient access to treatments.

• Identify bottlenecks and propose recommendations for modernizing relevant regulations to facilitate the adoption of new manufacturing technologies in production. Recruit experts in new manufacturing technologies to strengthen the evaluation process. Address management challenges in production management, as advances in manufacturing may not align well with traditional regulatory models. This may require adjusting the requirements and standards of Good Manufacturing Practice (GMP) for pharmaceuticals and developing specific regulatory guidelines and oversight measures.

• Establish a Comprehensive Evaluation Pathway for Medical Devices, In Vitro Diagnostic Reagents, and “Borderline” ProductsAn increasing number of complex products are emerging that combine pharmaceuticals and medical devices through mixed mechanisms of action. Innovative drugs may also rely on the use of associated in vitro diagnostic reagents. To support the development and regulation of such “borderline” products, the European Medicines Agency (EMA) requires comprehensive capabilities and expertise, and must collaborate with agencies responsible for regulating medical devices.

• Establish complementary and flexible consultation mechanisms to support innovative product development.

• Enhance awareness and regulation of nanotechnology and novel pharmaceutical materials, establish safety guidelines for pharmacokinetics and pharmacodynamics (PK/PD) requirements and efficacy, and develop regulatory pathway guidance.

Its goal is to provide regulators and payers with better evidence to strengthen the foundation of regulatory assessments, enabling patients to access beneficial treatments more promptly. There is a substantial burden in special populations (e.g., children, patients with rare diseases, or those posing significant individual and/or public health burdens). By integrating new digital tools into preclinical development, clinical trials, drug manufacturing, and real-world settings, data can be collected more broadly and effectively throughout the drug lifecycle. This presents an opportunity to better incorporate patient preferences during the assessment process, making clinical development and regulation more cost-effective and effectively alleviating the burden on healthcare systems. Specific recommendations include:

• Promote the adoption of new clinical trial practices to facilitate clinical trial approvals, adherence to Good Clinical Practice (GCP) for drug trials, and acceptance by Health Technology Assessment (HTA) bodies.

• Establish a dedicated artificial intelligence testing “laboratory” to explore the application of innovative digital technologies in support of data-driven decision-making.

• Develop the capability to evaluate technologies such as wearable devices for collecting complex datasets.

• Cultivate the ability to analyze individual patient data to support decision-making.

Patients and healthcare institutions should be at the center of the regulatory framework, aiming to ensure that patients have timely access to affordable, high-quality medicines and that healthcare stakeholders receive the information necessary to guide appropriate prescribing and use. Specific recommendations include:

• Co-develop health-related core quality of life measures and patient-reported outcomes (PROs) with HTAs for implementation in clinical trials, thereby bridging gaps through comparative effectiveness assessment. There is a growing consensus that more comprehensive assessments of the impact of drugs on patients’ quality of life are essential to generate critical insights.

• Engage in information exchange with HTAs to support bridging from benefit-risk assessment to relative effectiveness evaluation. New standards and guidelines should be developed among stakeholders to avoid discrepancies in evidence standards.

• Create a sustainable, quality-assured, and flexible framework that enables rapid access to and analysis of representative, longitudinal real-world data throughout the product lifecycle. As such data sources are often heterogeneous, it is necessary to develop enhanced analytical and epidemiological methods to generate robust evidence.

• Involve payers’ requirements in the anticipated discussions on evidence generation plans.

This objective aims to ensure that the European Medicines Agency (EMA) can effectively address challenges related to medicine demand and accessibility in response to existing and emerging health threats (such as the need for new antibiotics and approaches to managing antimicrobial resistance, as well as initiatives to improve communication and build public understanding of and trust in vaccines). Another area requiring attention is the issue of uneven medicine supply across the European Union (including medicines not being marketed or experiencing supply disruptions). Specific recommendations include:

• Implement the EMA's health threat plan, identify resources, and improve preparedness methods.

• Develop regulatory guidelines and support the development of new antimicrobial agents and innovative approaches for the prevention and treatment of infections.

• Exploring Mechanisms to Enhance Production Capacity in Europe and Internationally.

• Develop methods and tools, such as biomarkers, to characterize immune responses and support the definition of vaccine quality attributes.

• Use evidence-based tools to actively communicate the benefit-risk profile of vaccines to stakeholders, addressing vaccine confidence issues.

• Support the development and implementation of a “drug repurposing” framework, which has the potential to reduce development time and costs while providing patients with additional treatment options.

Achieving this objective is key to realizing the four strategic goals outlined above, and can be accomplished by collaborating with academic research centers to establish new regulatory science and innovation platforms. This will provide a mechanism for scientists within the EMA regulatory network and academia to collaborate in identifying and addressing highly relevant fundamental research issues, such as patient-reported outcomes (PROs), omics-based diagnostics, combination products, modeling and simulation, big data, and artificial intelligence. It will also help cultivate new regulatory competencies in emerging areas of innovation, including precision medicine and wearable devices. Furthermore, achieving this strategic objective will enhance access to regulatory science expertise, thereby improving the ability to track innovations and deepen insights into new drug product development.

The policy will be officially released later this year, with its core recommendations implemented through a series of concrete action steps. In addition to human medicines, it includes four objectives for veterinary drugs. The draft document, titled *Regulatory Science 2025*, outlines these five strategic goals and was opened for public comment in 2019. After considering the feedback received, the final version of the document will be publicly released in the fourth quarter of 2019.

Shenyang Pharmaceutical University, Feng Yingying

References

EMA Regulatory Science to 2025 Strategic reflection

Philip A. Hines, Richard H. Guy, Anthony J. Humphreys，et al.The European Medicines Agency’s goals for regulatory science to 2025.Nature Reviews Drug Discovery 18, 403-404 (2019)