Marden, Lora

Introduction: In conjunction with pericardial disease physician specialists, industry experts, and recurrent pericarditis (RP) patients the RESONANCE Registry was designed to collect retrospective and prospective, longitudinal, observational data in real-world clinical practice across the United States (US). We aimed to collect data that were complete and meaningful in terms of patient experience and health related quality of life (HRQoL). Herein, we describe a novel patient-centric research approach to obtain feedback on evaluation tools for incorporation in the RESONANCE Registry (NCT04687358). Methods: Ten adult patients with a self-reported duration of disease ranging from 8 months to 25 years participated in an advisory board during which they provided feedback through a virtual platform. Their feedback informed development of multiple registry aspects. These included selection of patient reported outcomes measures (PROMs), to be collected every three months at prospective data collection timepoints, and design of several questionnaires, including evaluation of specific RP episode characteristics. Results: Prior to finalization, registry case report forms were optimized based on feedback provided by patients. Areas of data-capture informed by patient input included selection of PROMs to be assessed and patient-centric tools used for self-assessment of disease experience and HRQoL. For example, sections of a patient questionnaire were modified to include documentation of the presenting symptom, breathlessness, fear of episodes, and patient management of each episode. Conclusions: This patient-centered research collaboration resulted in enhancements to the RESONANCE Registry that enabled capture of information meaningful to patients’ daily experiences, preferences, and expectations. Patient participation in tangible aspects of research, including registry tools and design, can increase engagement with the communities that have a vested interest in improving patient care. In addition, patient involvement can help to ensure research efforts reflect the needs of not only the healthcare community but of patients alike, with an ultimate goal of improving clinical outcomes.

Abstract Background Annually in the United States (US), an estimated 80–90,000 patients are diagnosed with acute pericarditis and 15–30% experience recurrent pericarditis (RP), resulting in increased morbidity and reduced health-related quality of life (HRQoL). Treatment options include non-steroidal anti-inflammatory drugs (NSAIDs) and colchicine. Corticosteroids (CS) are often added to the treatment plan in RP despite CS-associated adverse events and inherent potentiation of recurrence with long-term treatment. A recent Phase 3 clinical trial RHAPSODY (NCT03737110) demonstrated efficacy and safety of rilonacept, an interleukin-1 α and β cytokine trap, in patients with RP. RHAPSODY data helped support FDA approval of the first therapy for RP. With the emergence of this targeted therapy, there is increased interest to learn more about this disease with the goal to better inform treatment and management decisions and improve long-term outcomes. Purpose RESONANCE Registry aims to evaluate the natural history of RP by collecting retrospective and prospective, longitudinal physician- and patient-reported outcomes data in real-world clinical practice across the US. Methods RP patients with active disease (recurrence within 3 years) will have both retrospective and prospective data collected (Figure 1) for as long as their RP is managed up to 5 years. For patients with inactive disease (no recurrence within 3 years), data collection will be retrospective (Figure 2). Up to 500 patients in the US are planned for enrollment at pediatric and adult medical centers, with the potential for expansion to European sites. Additionally, patients will be recruited through a novel, internet-based technology platform and screened for eligibility at a “decentralized” trial site. The registry will include variables obtained from health records, including baseline characteristics and medical history, as well as patient reported outcome (PRO) measures collected every 3 months. The RESONANCE protocol is designed to include a broad population of pediatric and adult patients, regardless of etiology or treatment course, including patients treated with rilonacept. Data will be analyzed to understand disease heterogeneity, variability in treatment and management, and impact on HRQoL. The protocol and Case Report Forms (CRFs) were developed in collaboration with physicians, patients, and patient advocates. Conclusions Registries utilize real-world data to fill knowledge gaps in the management of less common diseases such as RP. The RESONANCE Registry is the first RP registry designed to collect data across a broad range of patients regardless of treatment. The registry will also serve as a connection point for physicians to further educate and empower patients with information about their disease. In addition, PRO data may enable greater insights into the understanding of the burden of RP from the patient's perspective. Funding Acknowledgement Type of funding sources: Private company. Main funding source(s): Kiniksa Pharmaceuticals