For years we’ve relied on randomized controlled trials (RCTs) as the gold standard for proving safety and efficacy of new treatments. RCTs are still important for drug development but they’re not without their flaws. Trials are conducted in controlled, idealised environments that don’t always reflect how treatments work in real life. To fill this gap researchers are turning to real world evidence (RWE) to make trials more effective and get new treatments to patients faster and sooner.
What is Real-World Evidence?
Real world evidence is data gathered from real life settings such as electronic health records (EHRs), insurance claims, patient registries and even wearable health devices. Unlike the controlled environment of clinical trials RWE captures information on how treatments work in diverse populations in everyday life. This data can give insight into long term effects of therapies, how they work in different demographic groups and how they are used in practice.
RWE can complement traditional clinical trials by giving a more complete view of a treatment’s impact. For example an RCT may focus on a specific outcome such as reducing disease symptoms RWE can help assess how the treatment affects patients’ quality of life, overall health and long term outcomes.
Recruitment and Trial Design
One of the ways RWE is improving clinical trials is by improving patient recruitment and trial design. Recruiting patients for clinical trials is one of the most time consuming and costly parts of research. Traditional trials struggle to enrol a diverse enough population, leading to delays and biased results.
By using RWE researchers can find potential participants who match the trial’s inclusion criteria faster. Data from EHRs for example can help find patients with the right medical history or demographic characteristics, faster recruitment.
Approximately 80% of clinical trials fail to meet their patient enrollment timelines. Leveraging RWE can significantly reduce this bottleneck. Studies show that using RWE can increase the diversity of trial participants by up to 30%. RWE can also ensure trials include a broader more representative population, so the results are more generalisable.
RWE can also be used to design more flexible and adaptive trials. Adaptive trial designs can reduce costs by up to 20% compared to traditional RCTs. By looking at real world data researchers can adapt trial protocols to reflect how treatments are used in real life. This can mean adjusting dosages, monitoring different health outcomes or expanding the trial to include patients with co-morbidities that would have been excluded from traditional RCTs. These adaptive trial designs can save costs and get new treatments to market sooner.
Real-World Evidence and Post-Market Surveillance
Even after a treatment is approved, long term safety and efficacy must be monitored. Real-World Evidence (RWE) plays a key role in post-market surveillance by tracking how treatments perform over time in large populations. Unlike clinical trials which have defined timelines and controlled populations, RWE provides continuous insight into how a treatment performs in real world settings.
Identifying Rare Side Effects
RWE can identify rare side effects that were not seen in clinical trials. By analysing data from insurance claims or Electronic Health Records (EHRs) researchers can spot trends or adverse events that emerge after a treatment is widely prescribed.
This information can inform regulatory decisions such as updating treatment guidelines or issuing safety warnings so patients are safe in the long term. Approximately 10-20% of drugs approved by the FDA are found to have serious side effects that were not seen in clinical trials. Insurance claims and EHRs are gold dust for post-market surveillance, providing data on millions of patients over many years.
Regulatory Decision Support
RWE gives you a bigger picture of a medicine’s performance which is critical for regulatory bodies. Continuous monitoring leads to better decisions on safety and efficacy of treatments. RWE has led to changes in treatment guidelines in over 30% of cases where new safety information was uncovered.
The FDA and other regulatory agencies have issued safety warnings or updated labels for many medicines based on RWE findings. With global health data, RWE can show how treatments perform across different populations and healthcare systems. RWE can empower patients with more information on the long term effects of their treatments so they can make informed decisions about their care.
RWE in post marketing surveillance is key to long term safety and efficacy of treatments. Continuous monitoring of real world data means better patient health and better care.
RWE Challenges and Opportunities
Using Real-World Evidence (RWE) has many opportunities but also challenges. We need to address these challenges to get the most out of RWE in clinical research and beyond.
Data Quality
One of the biggest is data quality. Real world data is collected for purposes other than clinical research, such as billing or routine medical care. So the data may be incomplete or inconsistent. We need to make sure the data is accurate and relevant for it to be used in clinical trials.
- Statistics: Up to 30% of real world data can be incomplete or inaccurate.
- Solutions: Implementing robust data validation and cleaning processes can help. Advanced analytics and AI can also help to identify and correct inconsistencies.
Privacy and Security
Privacy is another big one. Collecting and analyzing huge amounts of real world data requires patient privacy and data security to be top of mind. Researchers need to follow strict ethical and legal guidelines to protect individual’s health information.
- Regulations: Compliance with regulations such as GDPR in Europe and HIPAA in the US is key to protecting patient data.
- Technological Measures: Encryption, anonymization and secure data storage solutions are important for data privacy and security.
Technological Infrastructure
Integrating RWE into clinical trials requires robust technological infrastructure. Platforms that allow researchers to access and analyze huge datasets in a secure and user friendly way is key to getting the most out of real world evidence.
- Investment: Significant investment in IT infrastructure is required to support RWE integration. This includes cloud computing, big data analytics and AI technologies.
- Interoperability: Ensuring different data systems can talk to each other and share information seamlessly is critical for RWE integration.
Ethical Considerations
Ethics is key when using RWE. Researchers need to make sure the use of real world data respects patient autonomy and consent. This means transparent communication with patients about how their data will be used and getting informed consent where needed.
- Informed Consent: Patients need to be fully informed about how their data will be used and get consent.
- Transparency: Being transparent with patients and stakeholders about the purpose and outcome of using RWE helps to build trust and ethical integrity.
Bottom-line
Real world evidence (RWE) is changing the clinical trial landscape, giving us more insight into treatment efficacy and patient safety. By using real world data, we can overcome traditional trial limitations, improve recruitment, adapt trial designs and post market surveillance. While there are still challenges in standardising and analysing this data, the benefits – more efficiency, accuracy and relevance – are clear.
As technology and data sources evolve, RWE will play a bigger and bigger role in shaping the future of clinical research, leading to more patient centric and evidence based healthcare.
