Patients and Caregivers Play an Active Role in MPN Research

  • MPNRF | March 18, 2024

    Today, it is essential for patients to be involved with research and drug developers as they progress through the various stages of bringing a new therapy to market. MPN Research Foundation (MPNRF) is committed to ensuring that patient participation is more than a ticked box for drug approval.

    We actively work to incorporate the patient voice and experience early enough in the discovery and drug development process to allow for insights and input that might influence a clinical trial design, or how participants monitor their symptoms, or something about the treatment protocol itself that could lead to more meaningful benefit of the treatment.

    Our collaborative approach: bring academia, clinicians, industry, and patients together to interact throughout the research and development process, including offering their input to the FDA and other drug regulatory bodies. 

    As a group of rare diseases, this team approach is particularly important, as broader awareness is necessary about the often life-altering effects of living with ET, PV, or MF. 

    The MPNRF Patient Impact Council (PIC) is one way we are incorporating patient and caregiver input into where the gaps are in MPN research. We want to better understand what the priorities are from a patient perspective, and widely share their expertise. And we want to identify what might be missing from the drug development process that hasn’t been previously considered. 

    Members of the PIC are active participants in the review process for our 2024 MPN Challenge awards, MPNRF’s open RFP research initiative that allows us to fund the most pressing MPN science through 2-year funding cycles. 

    How MPNRF Patient Impact Council Members Are Involved 

    • 2024 project applications are currently in the review process, including by a peer review committee and members of the PIC.  
    • Patient/Caregiver reviewers are asked to score the potential patient impact of each proposed project. 
    • They offer insight into why each project might be important to patients – regarding how they feel, function, or survive. 
    • They underscore how understandable the project’s abstract is, helping researchers communicate their science in ways the general patient population will comprehend.
    • They comment on whether the accomplishments of a specific project’s aims advance our knowledge of MPN causes, prevention, or cure. 
    • Will the research add additional care burdens for a patient? 
    • Is it clear from the narrative that the research has the potential to impact patients?

    These and other opportunities for comment are part of the PIC members’ participation in both the scoring analysis – using the NIH (National Institutes of Health) Grant Application Scoring System – and the live review process of final candidates.