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What is the importance of real-world data within early access for rare diseases?

We spoke to Karen Klein of Clinigen on the importance of real-world data and evidence within early access. Karen has been working at Clinigen for four years, she is vice president of Real-World Evidence and she feels passionately about harnessing the power of data from Early Access Programmes (EAPs) to help patients.
What is real-world data in relation to early access?

For Karen, “Real-world data is any data that’s collected and represented outside of a randomised controlled trial.” Clinigen are using electronic data capture systems to collect data points of interest  to develop a clearer picture of medicine. The focus is on collecting data that would be usual to collect during standard of care in a particular condition, and EAPs provide a unique opportunity to collect such data prospectively from patients that are not able to participate in a clinical trial and gain initial insight into the real-world usage and performance of a medicine.

What is the vital role of data in rare diseases where patient numbers are low? 

Data collection is extremely valuable: it creates a broader picture for small patient populations. Every bit of information goes towards providing patients with better quality of care. In some disease areas there is little historical research, so it can provide a brand new road map for managing a disease at different stages, helping generations to come. Karen expressed how exciting it is to think “what it could offer to not just that patient but the patient down the road.”

Engaging as soon as possible with the healthcare professionals (HCPs) who are likely to participate in your EAP is vital to help them understand how data will be used and what insight you are looking to gain. This allows them to make an informed decision regarding participation in data collection. Armed with this information, they can tell patients why this information is valuable and makes sense to collect. If you are looking at small patient populations from a clinical trial perspective, that ability to treat patients and collect data is valuable, but finer details are case by case and need to be engaged upon. Karen’s advice for patient groups looking to start data collection is to “start small, understand how you are using the data to ensure you aren’t trying to achieve too many end results, work towards it” and understand that “you might not achieve your end goal on the first go”. But not meeting your goal first time does not mean stop doing it because “the more companies collect and report on data collected in the real-world setting, the more people are listening.” 

Questions are being asked around the limitations and quality of real-world data. Karen thinks “the health authorities and the payers are starting to recognise the value of this type of data, and in some instances these agencies are requesting data from real world sources.” 

Karen is honest about the reality of data collection and explains, “Unless we are doing the analysis on behalf of our clients, we don’t always get to see how our clients ultimately use the data.” She believes total transparency between sponsors and patients is necessary and makes it clear to sponsor companies that providing that data back to the patients and back to the advocacy group” is essential.

 If they took the time to provide it, they should get to see the results of it.

Karen Klein, Vice President of Real-World Evidence, Clinigen

Communication between everyone involved at the start of the process is one of the keys to success! Clinigen work with sponsor companies at the outset to create a clear statement which acts as a driving force. There is a tight focus  on goals and  making the process accessible. 

Karen clarifies, “One of the first things we ask a sponsor company is: What are you looking to do with your data? What are you looking to achieve?” Karen also believes in the power of the patients’ voice; “You’re really starting to get that patient perspective, not just the clinical perspective.” 

Talk to the relevant patient advocacy group(s), see what they’re looking for, what’s valuable to them, understand what data they feel is important to collect.​ What pieces of insights do they feel are missing?

Karen Klein, Vice President of Real-World Evidence, Clinigen

​It is important to hear from key opinion leaders alongside patient advocacy groups and physicians. For instance, Karen explains, ​​“You don’t want physicians to come back and say they don’t understand why the data is being collected—this leads to decreased compliance with data entry; so, it is really important to have stakeholder buy-in when developing your data collection plan.” 

​Joined-up data from early access can be merged into existing registries used by patient populations. When successful, joined-up data can enable medicines to be approved in the most time efficient way. This means Clinigen can work with companies to mediate access to their groundbreaking new treatments for patients who need them the most and at the same time show how that medicine operates in the real-world. 

​Why collect real-world data as part of early access?

​When it comes to real-world data, we have only begun to see the tip of the iceberg in terms of the impact it could have: “We are still learning about real-world data and the power of it and what it can create. The more programs that collect it, the more HCPs will become accustomed to providing data, which ultimately provides more evidence for everyone. This means that competent health authorities who don’t have clear guidance on real-world data will begin to follow suit from those that do.” Karen is passionate that data and EAPs provide value for patients, advocates and pharmaceutical companies.

I want companies to gain knowledge each time they work on an EAP where real-world data is being collected with Clinigen. It’s important to apply learnings to each future programme. This space is growing and changing so quickly, and at Clinigen we are committed to staying at the forefront of using real-world data and sharing that with our clients. 

​Karen Klein, Vice President of Real-World Evidence, Clinigen

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