Baltimore, MD (January 28, 2020) – The Green Park Collaborative (GPC) is partnering with the National Hemophilia Foundation (NHF) to develop a publicly available, content-validated, patient-reported outcome measure (PROM) that measures the impact on one’s mental health outlook associated with receiving gene therapy for hemophilia A and B. The instrument will be designed for patients 15 or older.
This project follows work completed in coreHEM, where a multi-stakeholder group achieved consensus on a core outcome set (COS) for gene therapy in hemophilia. In the coreHEM COS, a mental health outcome was included, and the proposed definition included elements of psychological and mental health status, emotional functioning, and positive and negative experiences due to the transformational change of a potentially one-time treatment. Because gene therapy treatment will serve as a durable therapy for hemophilia, the voters on the coreHEM Delphi panel stated that it was important to measure the changes in mental and emotional health in relation to this fundamental change in a patient’s life. No current measures exist to effectively evaluate this outcome.
“Developing a new instrument to measure the transformative potential of gene therapy on an individual’s overall mental health, whether positive or negative, will further enable our understanding of the entire spectrum of functional and social impact of living with hemophilia,” stated Mark Skinner, co-investigator of the coreHEM study.
“Mental health in the bleeding disorders community has always been vital to the individuals and families we serve,” said Dawn Rotellini, Interim Chief Executive Office at NHF. “As we move towards enhanced and innovative treatments in hemophilia it will become imperative to measure how the community responds and how NHF can support them. We are excited to continue our partnership with the Green Park Collaborative to better serve the bleeding disorders community.”
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About the Green Park Collaborative (GPC)
GPC is a major initiative of CMTP, an independent 501(c)(3) non-profit organization dedicated to improving the quality, relevance, and efficiency of clinical research. GPC is a multi-stakeholder forum for developing condition- and technology-specific study design recommendations to guide the creation of evidence needed to inform both clinical and payment decisions.
About the National Hemophilia Foundation (NHF)
The National Hemophilia Foundation (NHF) is a 501(c)(3) non-profit organization dedicated to finding better treatments and cures for inheritable bleeding disorders and to preventing the complications of these disorders through education, advocacy and research. NHF’s programs and initiatives are made possible through the generosity of individuals, corporations and foundations, as well as through a cooperative agreement with the Centers for Disease Control and Prevention (CDC).
