Convening RUNX1-FPD Stakeholders to Advance PCOR

Background: RUNX1 familial platelet disorder (RUNX1-FPD) is a rare hereditary blood disease predisposing individuals to develop hematologic malignancies. Like other rare diseases, many patients are left undiagnosed and misdiagnosed, leading to slow growth of the patient community and minimal research attention. This exacerbates silos and results in very few patient voices being included in the development of research questions and priorities.

Proposed Solution to the Problem: The “Convening RUNX1-FPD Stakeholders to Advance PCOR” project will directly address this problem by bringing together patients with key stakeholders to support the discussion and development of patient-centered outcomes research (PCOR)/comparative clinical effectiveness research (CER)-aligned research topic priorities, based on patient-identified gaps in diagnostics, management, treatment, and care. It will build upon a previous PCORI Engagement Capacity Building-funded initiative that launched the Research Guided by Patients Committee (RGPC)—a RUNX1-FPD patient-driven research advisory body trained on PCOR/CER principles. This project will allow the RGPC to implement their learnings by identifying critical research gaps, developing patient-driven research questions, and enhancing current RUNX1-FPD research plans with PCOR/CER strategies.

The proposed solution involves planning and implementing a series of convenings that augment the RGPC’s engagement work by providing a unique forum to engage all stakeholders and provide ample discussions around new and emerging research questions, identifying ways to incorporate the patient voice and perspective into research plans and incorporating the patient journey. The convenings will build upon one another to develop a five-year PCOR/CER strategic plan for RUNX1-FPD.

Objectives: The primary objectives of the project are to promote collaboration among all stakeholders regarding how to best incorporate the patient voice in research, with a focus on developing the first-ever PCOR/CER priorities and agenda; educate the broader patient, scientist, and clinician communities on PCOR in order to facilitate future implementation of PCOR; and develop a strategic framework to drive continued engagement among all stakeholders.

Activities: Four workshops will be held in total, with two held at the 2022 RUNX1 Week Scientific and Patient Conference and two outside of the conference to facilitate discussion and engagement around the development of a PCOR/CER strategic plan. One workshop will engage the patient and family community in PCOR/CER to develop patient-driven research priorities, one workshop will foster new partnerships among the scientific and clinician communities, one workshop will outline PCOR/CER future agenda among all stakeholders, and a final workshop with all stakeholders will refine the strategic plan.

Projected Outcomes and Outputs:

The short-term outcome during the project period includes a strategic plan identifying PCOR/CER research agenda and priorities.

The medium-term outcome (0-2 years post-project period) is to have the RGPC actively integrated in the RUNX1 Research Program (RRP’s) research strategy.   

The long-term outcome (3+ years post-project period) is to have defined PCOR studies as part of the larger RUNX1-FPD research field, and to have successfully implemented PCOR/CER engagement principals throughout the research continuum.

Patient and Stakeholder Engagement Plan: Patients and stakeholders will be key partners in the development and implementation of the convenings. Two RUNX1-FPD patients and a researcher, (all of whom are RGPC members), will be co-leads in the planning and implementation of the convenings. The overall concept and design of the workshops will be planned and executed together with the RRP and the RGPC.