When Amgen set out to generate real-world evidence for UPLIZNA® (inebilizumab-cdon) in neuromyelitis optica spectrum disorder (NMOSD), a debilitating rare autoimmune disease, they knew traditional site-based approaches couldn't deliver what they needed. Amgen partnered with PicnicHealth to launch the OBSERVE-NMO Study, an ambispective, non-interventional registry with minimal patient burden and an unconventional recruitment strategy that tapped into the patient’s existing treatment journey.

In this interview, we speak with Jenny Y. Park, PharmD, MS, a scientist in Amgen's Center for Observational Research, to learn more about why Amgen chose our approach and what it means for the future of rare disease evidence generation.

Disclaimer: The views expressed in this Q&A are Dr. Park’s personal opinions and do not necessarily reflect those of Amgen.


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Q: What made you choose PicnicHealth for your research needs?

A: Our primary objective was to robustly and rigorously characterize real-world treatment patterns, effectiveness, and safety in a rare disease population. Given the nature of these populations, traditional site-based infrastructure alone can be limiting and may introduce unnecessary burden for both patients and providers.

Collaborating with PicnicHealth offered a more patient-centric alternative. Participation is fully remote, eliminating the need for additional site visits, travel, or disruption to patients’ existing care. Rather than asking patients to generate new data, the research model focuses on curating and structuring information already being captured in their medical records as part of routine clinical care. This helps reduce participation burden while still enabling high-quality, longitudinal insights.

A key component of the approach is meeting patients where they already are. Recruitment is supported through an existing patient support program (PSP), where individuals may already have established, trusted relationships. This allows outreach to come from a familiar point of contact, rather than an unfamiliar third party, which can be critical for engagement. Leveraging that trust helps lower barriers to participation and enables more efficient enrollment.

Additionally, the model incorporates a meaningful value exchange for participants. Patients are given access to consolidated versions of their medical records, which can be difficult to obtain across fragmented healthcare systems. Providing something tangible in return reinforces a more respectful, partnership-oriented approach to research.

Overall, this approach reflects a broader shift toward designing studies that prioritize accessibility, minimize burden, and align more closely with patients’ real-world experiences while maintaining scientific rigor. 

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Q: How did the PSP recruiting channel perform relative to your expectations — and what surprised you most about the enrollment results?

A: Enrollment incredibly exceeded our initial expectations, particularly in the early phase of the study. We observed strong early engagement, suggesting that reducing participation burden and leveraging familiar points of contact can meaningfully improve recruitment efficiency.

One key takeaway was the importance of trust and accessibility in engaging patients, especially in rare disease settings. Approaches that feel integrated into patients’ existing care experiences may help lower barriers to participation.

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Q: What does "complete patient journey" data unlock for you that you couldn't get from claims data or site-based studies alone?

A: Claims data captures utilization but not clinical context, falling short of our evidence needs. Site-based studies add clinical depth but introduce selection bias toward patients near major academic centers. They also limit data retrieval to within the site or network.\

Remote, patient-mediated record collection addresses these limitations by obtaining longitudinal data from patients in real-world care settings, including pre-treatment history critical for understanding how UPLIZNA® is performing against each patient's own disease baseline, and prospective data that builds a complete picture of their treatment journey over time.

Q: Where is non-interventional research going in the future? Is patient-mediated a part of it?

A: Regulatory and payer pressure to generate robust post-approval evidence is only increasing, and I think the field is responding in a few important ways. Study designs are becoming more sophisticated, with ambispective approaches that capture both retrospective history and prospective follow-up becoming increasingly the norm. The integration of multiple data sources, from medical records to biomarkers to patient-reported outcomes, is enabling richer characterization of disease and treatment impact than any single source could support alone. And the question of how to reach patients outside of traditional academic medical centers — which has long been a structural limitation of observational research — is finally getting serious attention.

Patient-mediated collection is one meaningful answer to that last challenge, particularly in rare disease where site-based infrastructure can't deliver representative samples at pace. But it's part of a broader shift toward research designs that meet patients where they are, reduce burden across the board, and generate data that more accurately reflects real-world clinical practice. Overall, the field is moving toward more patient-centric, flexible research models that more comprehensively reflect real-world care and can scale to meet evolving evidence needs.

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Learn more about our approach to generating decision-grade evidence for treatments.