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Jul. 11, 2019

TOPIC: Events, Get Involved, Patients & Members

Upcoming Externally-Led Patient Focused Drug Development Meeting for PKD Community

Posted by Lisa Sencen

NORD and the Foundation for Rare Blood Diseases (SZB) invite you to register for an externally-led Patient Focused Drug Development (EL-PFDD) meeting on Pyruvate Kinase deficiency (PKD), taking place Friday, September 20. Scholarships are available to eligible applicants to assist with travel and accommodation expenses.

If you are a patient, family member or caregiver affected by PKD, this is an important opportunity to have your voice heard. 

EL-PFDD meetings provide an opportunity for patients, their families and caregivers to share critical information about the impact of their disease on their daily lives and their experiences with currently available treatments. Patients’ experiences provide valuable insight for the U.S. Food and Drug Administration (FDA) and other key stakeholders, including researchers, medical product developers and health care providers. 

PKD is a rare condition with no targeted treatment currently available, and existing medical interventions (mainly routine transfusions and splenectomy) introduce significant risks and burdens to patients. NORD and SZB believe this rare, genetic hemolytic anemia disorder is one with an unmet need and a severe burden of disease, especially in the pediatric population. Together, NORD and SZB strive to collect rigorous patient experience data from PKD patients and caregivers that can guide medical product development and assist regulatory agencies in conducting assessment and review of potential therapies for patients with PKD, given the lack of available treatment options for these patients.

The goals of this EL-PFDD meeting are to provide researchers, drug developers and the FDA with a robust understanding of patients’ and caregivers’ experiences with PKD, including how individuals with PKD view their quality of life, what aspects of the disease are most problematic for them, and what actions they currently perform to treat and cope with this disease. The results of this meeting will be shared publicly in a “Voice of the Patient” report in an effort to inform the development of potential therapeutics that are impactful to patients living with PKD.

The meeting will take place on Friday, September 20, 2019 from 9:30a.m. – 3:30p.m. ET Participants can attend in-person or virtually. The in-person meeting will take place at the College Park Marriott Hotel and Conference Center, Hyattsville, MD. For more information, to apply for a scholarship and to register, please click here.