Building Better Bleeding Disorders Therapies With NBDF Patient Panels

NBDF’s Advisory Panels give biopharma direct access to patients, caregivers, and clinicians within the bleeding disorders community

NEW YORK--(BUSINESS WIRE)--Pathway to Cures (P2C), the venture philanthropy fund of the National Bleeding Disorders Foundation (NBDF), encourages biotech and pharmaceutical companies that are developing treatments and cures for blood and bleeding disorders to connect directly with the bleeding disorders community through NBDF’s Advisory Panel program. Since its launch in 2019, the program has convened approximately 36 panels for 18 organizations across the pharmaceutical and biopharmaceutical industries, specialty pharmacy, and nonprofits, producing insights that have shaped clinical trial design, regulatory strategy, and product development.

“We have partnered with leading industry and biotech organizations to ensure the authentic voices of patients are integrated into every stage of clinical development,” said Maria Santaella, PhD(c), MSN, RN-BC, CPHON, vice president of research strategy for NBDF “Through structured engagement and insights drawn from real lived experience, we help our partners design trials that are more feasible, more inclusive, and more aligned with the needs of the bleeding disorders community.”

Why Patient Engagement Can’t Wait

The earlier companies incorporate patient perspectives, the more effectively therapies can meet real-world needs. At the earliest stages, input from lived experience experts can shape study design and product development. Research shows that incorporating patient perspectives into clinical trials can improve study design, focus on outcomes that matter most to patients, and increase participant enrollment. (CDER) (BMJ) Conversely, therapies that reach the market without this input risk poor adoption, a costly outcome for companies.

“We have seen promising therapies receive approval, only to be withdrawn from the market due to lack of adoption. The kind of insights that companies could gain through our Advisory Panels could help them avoid these pitfalls.” said Santaella.

How It Works

Each customized Advisory Panel is designed to help the pharma or biotech sponsor to better understand and gain a better understanding of a specific topic related to a bleeding or blood disorder challenge, such as assessing clinical trial feasibility, understanding barriers to participation, evaluating if a therapy meets an unmet need, or gauging potential quality-of-life impact. Sponsors can request a Virtual Advisory Panel (two hours) or an in-person Advisory Panel (up to a day and half). Sessions can be repeated to ask participants follow-up questions or convene a different participant group.

Advisory Panels consist of six to eight participants screened for eligibility and may include patients, researchers, or health care providers. Participants are identified by the administrator of NBDF’s Community Voices in Research patient registry. The program is designed to maintain confidentiality. Sponsors receive a comprehensive report with expert recommendations.

Real-World Impact

Insights from NBDF’s Advisory Panels have already had impact on the development of bleeding disorders therapies:

• Input helped simplify gene therapy and immunology trials
• Panel findings sparked submission of an application requesting a new ICD-10 code for an ultra-rare disorder

To learn more about NBDF’s advisory panels, patient registry, data queries, and sponsored surveys, please contact Samantha Carlson, MSW, LMSW, director of research operations, at scarlson@bleeding.org or 212-328-3700.

Biopharma companies developing treatments for bleeding and blood disorders can also explore investment and collaboration opportunities through Pathway to Cures (P2C), NBDF’s venture philanthropy fund. Learn more at www.pathwaytocures.org.

About National Bleeding Disorders Foundation

The National Bleeding Disorders Foundation (NBDF) is dedicated to finding cures for inheritable blood and bleeding disorders and addressing and preventing these disorders’ complications through research, education, and advocacy, enabling people and families to thrive. Today, NBDF serves people across the United States with all bleeding disorders, including hemophilia, von Willebrand disease, rare factor deficiencies, and platelet disorders. The foundation also supports a network of 50 chapters across the country. To learn more, visit www.bleeding.org and follow NBDF across social media @nbd_foundation.

About Pathway to Cures

Pathway to Cures (P2C) is a venture philanthropy fund created to accelerate development of cures across all inheritable blood disorders. In collaboration with other organizations, P2C invests in innovative therapies and technologies, leveraging the deep resources and scientific community relationships of the National Bleeding Disorders Foundation. By reinvesting proceeds from investments back into the fund, P2C will amplify investment impact, support promising companies and build portfolio of investments that further the mission of the National Bleeding Disorders Foundation. For more information, visit www.pathwaytocures.org.

Jay Patel
jpatel@bleeding.org
National Bleeding Disorders Foundation