Top-down vs. Bottom-up
The traditional approach to establishing a patient registry comes from the top down. An interested party (say, a pharma company or a private research foundation) asks a healthcare system for access to patients that might fit the profile they’re seeking. For large-scale studies, this means reaching out to multiple potential partners, and then managing all of those contacts and homogenizing any differences in how they report their data.
But imagine if that interested party, having established their screening criteria, could apply that screen to a database of patient information derived from all of those contacts. For example, a healthcare system’s database could contain the records of all patients with a specific rare disease, who are pre-consented to participate in clinical trials, drawn from patient-reported data at the time of admission and from the provider’s electronic health record (EHR) system.
For the pharma company or research foundation, that unlocks access to a much greater number of potential participants for the main trial, plus major benefits in terms of patient engagement and recruitment for sub-studies or other related trials.
Now imagine that you’re a patient with a rare disease, desperate to learn more about possible treatments but unaware of any trials that might be relevant. As a member of a registry database, your anonymized profile can be screened to identify those treatments and facilitate enrollment in trials.
That enables not only a personalized course of treatment tailored to that patient’s condition, but also the opportunity for them to engage with the community of other patients with that disease.
Other patient facing technologies, including electronic patient-reported outcomes (ePRO), text messaging, and patient portals, are not only enabling better engagement between patients, but also between clinical trial investigators and participants. That’s especially important with long-term studies that run for years or even decades. Investigators can now better incentivize patients to stay involved via direct engagement, keeping them abreast of new developments across a variety of channels – not to mention that ePRO is much less expensive and time-consuming than older methodologies.
Additional Benefits
Clinical trial investigators can safely store and access large volumes of standardized data, unlocking unconsidered benefits. For example, at the conclusion of a trial, all the data generated for each patient can be uploaded back into the registry, creating a master registry that includes information on outcomes.
Patient registries can also greatly reduce the number of control participants required to conduct a clinical trial, because the population for control is already represented in the registry. That real-world data can be used to create a “synthetic” control arm of patients who received standard care for that condition, which can be compared to the experimental cohort without recruiting any actual patients.
And because it’s safely anonymized to ensure patient privacy, registry data can potentially be monetized to help offset the cost of establishing the registry. In addition to allowing interested parties to pay for access to their patients for enrollment in trials, registry operators can also provide paid access to their data, which not only adds to their coffers, but expands the ability to connect patients with treatments suited to their condition.
Example: I-SPY 2
One recent example is the groundbreaking I-SPY 2 trial, which used a database of genetic profiles of breast cancer patients and screening protocols to match participants with therapies that had the highest chance of a successful outcome.
I-SPY 2, which allows up to five therapeutics (or combinations thereof) to be evaluated in parallel, has to date led to the publication of 84 manuscripts and the presentation of more than 170 abstracts at medical conferences.
REDCap Cloud Can Help
REDCap Cloud’s unified data science platform delivers an automated, end-to-end solution that’s ideal for managing patient registries. Our solution empowers patients, life science companies, and healthcare organizations with effective data collection and management, allowing them to analyze and share data seamlessly that eliminates the barriers between research and care to deliver actionable, regulatory-grade data for any use case.
To learn more about getting involved with the rare disease community, visit the National Organization for Rare Disorders.
