François Houÿez: Enhancing the use of real-world data in non-interventional studies
November 2024
In today’s rapidly evolving healthcare landscape, the utilisation of real-world data (RWD) – information collected from everyday medical care or routine medical practice – has become increasingly significant.
The recent Reflection Paper on the Use of Real-World Data in Non-Interventional Studies to Generate Real-World Evidence issued by the European Medicines Agency is a timely document that underscores the importance of using RWD to enhance clinical research and support regulatory decisions.
What is Real-World Data, and why is it important?
Real-world data (RWD) refers to information gathered from sources like electronic health records, patient registries, or insurance claims, rather than from clinical trials. This type of data helps researchers understand how treatments work in the real world, outside the controlled environment of a clinical trial.
The EMA’s reflection paper highlights the value of using RWD to answer important questions about the effectiveness and safety of treatments, and how they are actually used in medical practice, such as at which dose. For example, RWD can help doctors and regulators monitor the use of medicines taken during pregnancy, as pregnant women are rarely included in clinical trials.
Target Trial Emulation: a step forward in reducing bias and confounding factors
One method discussed in the paper is called Target Trial Emulation. This technique aims to mimic the design of a clinical trial using RWD. By applying the right analytical methods, it helps researchers draw conclusions about cause-and-effect relationships between treatments and outcomes while reducing the risk of bias.
Additionally, advanced technologies like machine learning (a type of artificial intelligence) can further improve the accuracy of these analyses, as demonstrated by the HTx project. For instance, Super Learner algorithms help reduce errors in predicting how well a treatment will work, making the findings more reliable.
The challenge of accessing Real-World Data
One of the primary challenges in using RWD is accessing the data itself. Often, data holders impose overly restrictive controls, limiting researchers’ ability to utilise the most relevant and reliable data. A common justification for these restrictions is that patients did not explicitly consent to the secondary use of their data or its sharing with commercial entities. This raises a significant barrier to research that could benefit public health.
Proposals for raising re-consent requirements
To address these challenges, it is proposed that in cases where the secondary use of data aligns with the original research purpose, re-consenting should not be necessary. This would streamline the process and reduce the burden on both researchers and patients. However, if the secondary use differs largely from the initial purpose, an ethics committee should evaluate the need for re-consent, considering the risks of re-identification and the impact on the validity and reliability of the analysis.
Regardless of whether re-consent is required, it might be more important that patients are kept informed about how their data is used and the outcomes of any analyses. Transparency is key to maintaining trust and ensuring that patients feel secure in how their information is being utilised.
Learning from experience: the role of organisations
Organisations such as the Get-Real Institute play an essential role in this process. By providing a platform for all stakeholders to share experiences and develop expertise in analysing RWD, these organisations help bridge the gap between research and practical application.
The potential of Real-World Data in repurposing medicines
Another important use of RWD is in the repurposing of medicines – finding new uses for existing drugs. For example, patient registries, which collect data over time on specific patient groups, can provide the evidence needed to confirm that a medicine is safe and effective for a new condition. This could potentially save time and money compared to running new clinical trials, especially when the treatment is already widely used.
In conclusion, while the EMA’s reflection paper outlines the many benefits of using RWD, it also recognises the challenges that need to be addressed. By making it easier to access data, simplifying consent processes, and using advanced analysis methods, we can make sure that RWD plays a key role in advancing medical research and improving patient care. As we continue to explore the possibilities of RWD, it’s essential that we keep patients’ rights and needs at the centre of our efforts.
By François Houÿez, Director of Treatment Information and Access
Disclaimer: As a Staff Blog, the opinions – including possible policy recommendations – expressed in this article are those of the author and do not necessarily represent the views or opinions of EURORDIS. The publication of this article on the EURORDIS website does not equate to endorsement.