EMA RWE Framework

The EMA has published a report on it’s Real-world evidence framework to support EU regulatory decision-making: Report on the experience gained with regulator-led studies from September 2021 to February 2023 [Link].

According to the EMA, real-world evidence (RWE) from studies led by regulators can complement evidence from other sources including clinical trials. RWE can support both pre-authorisation and post-approval assessments of EMA’s scientific committees, working parties and national competent authorities. However, more effort is needed to better anticipate the need for such studies and to speed up their initiation to ensure that regulators have access to RWE in a timely manner [EMA RWE Framework News - June 2023].

While established in the safety monitoring of medicines, there is less experience in using RWD/RWE in addressing evidentiary gaps at earlier stages of the lifecycle of medicines including pre-authorisation and for evaluating the effectiveness of medicinal products. Further work is needed to make full use of its potential. Consequently, EU regulators have recently set out their vision to have enabled the use of RWE and established its value for regulatory decision-making by 2025 (Arlett et al., 2022) [Section 1 of the EMA RWE Framework, June 2023].

This vision is embedded in the wider EU policy context, in particular the European Commission’s proposal for a European Health Data Space as well as with a view to the Agency’s commitment towards international collaboration to overcome common challenges in using RWD/RWE for regulatory decisions (Global regulators call for international collaboration to integrate real-world evidence into regulatory decision making | European Medicines Agency) [Section 1 of the EMA RWE Framework, June 2023].

The current framework include 3 evidence generation pathways: (1) In-house database, (2) DARWIN-EU, and (3) ‘Traditional’ studies.

80% of the database studies, also referred to as ‘rapid data analytics’ , were delivered in less than 90 days. Whereas, delivery of the DARWIN-EU studies averaged 215 days.

The majority of the studies focused on safety, rare diseases, and paediatrics. 

Conclusions

• Overall, during the period of the report, 27 regulator-led RWD studies have been completed, primarily to support PRAC (Safety), COMP (Rare diseases), SAWP (Scientific advice) and the PDCO (Paediatrics) in their regulatory assessments.
• Studies were performed using all three available RWE generation pathways and included safety, drug utilisation, and disease epidemiology studies, as well as studies to inform the design and feasibility of clinical trials and clinical management.
• Most studies were descriptive using simple analytics to describe incidence or prevalence rates of clinical outcomes or adverse events with or without prior exposure to specific medicinal products, or describing the extent and duration of exposure to medicinal products, their indications, as well as characterising the treated population.
• Comparative analyses were also performed, e.g., to explore the association of an adverse event with the use of (a class of) medicinal products, some of these employing more complex approaches such as self-controlled case series.
• Both the number and the breadth of the types and designs of the studies performed shows the versatility and the potential of the available RWE resources and pathways, as well as the Agency’s ability to address a range of research questions for regulatory decision making.
• There is room to improve in order to better leverage the full potential of the EMA RWD study framework

29 August 2023 - EU | Lynxcare - A Bridge into European RWE Data

The European medicine regulators have already taken first steps towards fulfilling their vision of harnessing the potential of RWE and recognizing its value across regulatory use cases by 2025. As such, the European Medicine Agency (EMA) is actively working on creating a framework that will facilitate the use and establishes the value of RWE in decision-making throughout the entire product lifecycle. 

Join Stuart McCully and go further with more clarity into the journey of RWE Regulations in Europe and explore what lies ahead. How can RWE be used in regulatory submissions? How can RWE help to improve clinical trial designs? How can I expand my RWE strategy following the recent EMA guidance updates?

Shahbaz Pervaiz, RWE Services Director at LynxCare, will present our solution designed to address your research questions and fulfill your real-world data needs in Europe [Register Here].

26 - 27 SEPTEMBER 2023 - USA | FDA/PQRI Workshop on the Regulatory Framework for the Utilization of Artificial Intelligence in Pharmaceutical Manufacturing

FDA and the Product Quality Research Institute (PQRI) are inviting organizations and individuals interested in the use of artificial intelligence (AI) in pharmaceutical manufacturing to attend a public workshop on September 26-27. During the workshop, the public can provide FDA with input on these new technologies including input on the topics of FDA’s discussion paper on the use of AI in drug manufacturing [Register Here].

Real World Evidence (RWE) 101 - The Complete Series [Link]

Real World Research (RWR) refers to the collection of patient-related data in a real-world environment (real-world data) and obtaining clinical evidence (real-world evidence) of the value and potential benefits or risks of the medical products through analysis [Link].

ABOUT STUART MCCULLY...

Stuart is a real world research (RWR) industry expert and thought leader. With his background as a pharmacologist working on traditional pre-approval clinical trials, he became frustrated with the operational inefficiencies and support gaps for non-interventional (observational) studies. As a result, Stuart created CHCUK which became a global leader in supplying regulatory and operational intelligence for non-interventional studies. Currently, Stuart is a co-founder of RWR-Regs, a regulatory compliance solutions platform for real world research.

Stuart regularly provides RWR regulatory science advice, guidance and training to top 10 Pharma, Biotech, Medical Device companies and CROs.