The increasing importance of real-world data for the EMA
Over recent years, real-world data (RWD) has become a critical element of the development and commercialization of medications. RWD includes a whole range of data collected outside of clinical trials and can show us how patients respond to treatment regimes in routine clinical practice. RWD can come from various sources such as patient health records, prescription data, digital platforms, and even wearable technology.
The European Medicines Agency (EMA) is increasingly relying on RWD and insight to support regulatory decision-making, particularly in assessing the safety and effectiveness of medicines in everyday use. Here we explore what that means for the launch of new treatments for rare and ultra-rare diseases.
Why is real-world data important for orphan drugs?
For rare diseases, patient populations are naturally small and often varied. This means that data generated in clinical trials, with their strict eligibility criteria, rarely gives us the deep understanding we need of how the therapy will perform in the real world. RWD, therefore, provides invaluable insight that can be vital in building evidence and understanding of a drug’s efficacy and safety among a more diverse patient population.
RWD is increasingly being used to support the launch of vital new orphan drugs
For example, it can:
- Supplement clinical trial data to give regulators additional evidence on the drug’s safety and effectiveness across a wider patient population.
- Be used to inform the development of clinical guidelines and best practices for using the drug in a real-world setting. For example, the side effects noted in a clinical trial may have more of an impact on a patient’s quality of life than previously thought.
- Influence reimbursement decisions and support faster market access and uptake.
The role of the European Medicines Agency (EMA)
The EMA has been an advocate for the use of RWD in ongoing monitoring, safety surveillance, and improving access to medication for some time.
With an understanding that clinical trials alone are unable to tell us everything we need to know about a drug’s performance in the real world, the EMA has been turning to RWD to improve its regulatory decision-making. Some of the steps it has taken include:
- Issuing new guidance and frameworks to support and encourage the use of RWD, including recommendations on successfully incorporating RWD into regulatory submissions (e.g. marketing authorization applications and post-approval studies).
- Exploring the quality, reliability, and relevance of RWD in different contexts through pilot studies.
- Developing standards for RWD in collaboration with national authorities, industry, patient groups, and academics.
- Launching the Data Analytics and Real World Interrogation Network (DARWIN EU) in 2022 to set standards and support access to data from across the EU.
Five key steps to ensuring success
Building RWD collection into your plans from an early stage can help ensure success. It can also maximize the value of the data collected, allowing you to use it to support regulatory submissions and market access.
Based on our experience, we recommend these following five steps:
1. Understand your objectives You must understand what types of data will be the most valuable for your product such as efficacy, safety, quality of life measures, or health economic data. A deeper understanding of a drug’s impact on a patient’s quality of life can support the design of interventions that increase adherence and persistence.
2. Establish what you already know Look at your clinical trial data and decide if it is enough or if there are gaps that may matter to patients and assessors when it comes to pricing and reimbursement negotiations.
3. Find out what your stakeholders want By collaborating with healthcare providers, patient advocacy groups, and regulators you can make sure you are aligned in terms of data collection and that the data will be relevant and actionable.
4. Ensure data quality and compliance Clear processes for data management are essential, such as data quality checks, privacy protections, and compliance with data protection regulations, such as GDPR.
5. Partner with a specialist service provider Partnering with local experts who can offer proven experience in managing product launches across Europe, such as Sciensus, can help you successfully navigate the many regulatory, logistical, and cultural challenges associated with a European launch.
Find out more about how Sciensus can support the success of your early access program in Europe.
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