The ultimate goal of using real-world data (RWD) to generate real-world evidence (RWE) is to improve patients' lives. But often, when dealing with medical data, it’s easy to get lost and forget that behind each data point, there’s a patient with their own lived experience, family and friends, and hopes and dreams for the future. There’s a need to bring the patient’s voice more prominently into RWD to capture the most relevant data and generate RWE that directly benefits them. Below, we discuss how insights can be derived by documenting the struggles of patients, patient- and clinician-reported outcomes, and how greater accessibility to data can positively impact patient care.
The Patient Experience
For many patients, the course of their disease is rocky and uncertain.
Patients living with inflammatory bowel diseases (IBD), such as Crohn’s disease or ulcerative colitis, for instance, describe the tension they face regarding symptom severity. Not knowing when the next flare will occur or when their disease will become so severe they’ll need surgery is a tremendous psychological burden.
The uncertainty doesn’t end there. Patients who require treatment wonder which treatment will work for them. The growing number of IBD treatment options, their vague indications, and the lack of standardized classification systems make it challenging to choose the right treatment for the right patient at the right time.
In addition, limited access to electronic health records (EHRs) puts patients at a disadvantage. If they want to get multiple opinions from different clinicians about their disease progression or treatment options, gathering medical records from many different sources is a struggle that can take a lot of patients weeks to months. With life-threatening diseases like cancer, waiting that long to assemble everything can slow access to life-saving treatment.
Introducing the Patient Voice to the RWD/RWE Conversation
Putting the patient's voice, experience, and needs at the center of RWD and RWE is essential for gaining insights into the disease course and accessing effective treatment options.
One example of how this can work comes from an initiative started by the Crohn’s and Colitis Foundation called IBD Plexus. Since launching in 2017, IBD Plexus has collected EHR data and patient samples, generating a wealth of 'omics data, and making it available to research partners in academia and industry, leading to numerous publications and active projects that benefit patients and clinicians. Many studies that use the IBD Plexus database shed new light on the basic pathophysiology of IBD, patterns of disease progression, treatments, access, and more.
From Database to Insight
With this data from IBD Plexus and input from the patients and clinicians, there can be real change in our understanding of IBD biology and how to identify severe patients in need of treatment.
Generating those insights requires continued collaboration and study, but enacting change for patients requires active communication of “lessons learned.”
The next step must be educating clinicians so that they can take action with patients on a severe disease course. From a drug development perspective, using the ‘omics data in IBD Plexus to identify biomarkers of severe disease can help focus planned trials on those patients with the greatest need for new treatments.
Lastly, collecting this type of patient-focused RWD and RWE can be leveraged to facilitate conversations with the FDA, payers, and the Centers for Medicare & Medicaid Services (CMS) to ensure affordable drug access.
Ultimately, this will help put the power of data into the hands of patients and their clinicians, enabling them to advocate for themselves.
Taking Action for Patients
How can we take action in the short term to improve the collection, sharing, analysis, and dissemination of multimodal data (i.e., data from different sources and formats)?
Advocating for strong data standards and making data collection for EHRs patient- and clinician-focused, rather than payer- and hospital-focused, is essential. There are glaring data gaps related to the patient experience in EHRs, such as responsiveness to treatment or descriptions of subjective symptoms (e.g., pain, fatigue, etc.).
Prioritizing patient-centered data collection and ensuring that the patient's voice is integral to RWD and RWE efforts can enhance our understanding of IBD and other diseases, improving treatment outcomes. Collaborative action around data collection, as seen with IBD Plexus, and focusing EHRs on patient and clinician needs will empower patients and healthcare providers to make informed decisions and advocate for optimal care.
If you’re curious about how you can leverage RWD and RWE to make a bigger impact on patient’s lives, contact us today.