Gene and Innovative Therapies Education

The treatment landscape for bleeding disorders is evolving rapidly, with new therapies offering innovative solutions and significant hope for the community. Gene therapy, once a distant prospect, has become a tangible treatment option. Recent FDA approvals have brought gene therapies to market, designed to offer a one-time treatment with the potential to reduce or eliminate the need for regular infusions. However, many important questions remain:

• Who qualifies for gene therapy, and what are the eligibility criteria?
• How accessible and affordable will this treatment be for patients?
• What is the long-term durability and efficacy of these therapies?
• Are they safe for widespread use?

Beyond gene therapy, non-factor therapies continue to revolutionize treatment options. Since the release of the first non-factor therapy in 2017, additional advancements have expanded the range of therapies available, offering prophylaxis options that don't rely on traditional factor replacement. This era of innovation has brought an abundance of choices for those affected by bleeding disorders, enabling more personalized treatment plans.

At NBDF, we recognize the complexity of these new therapies and their potential impact on the community. As a trusted resource, our mission is to provide accurate, non-biased educational content that explores the science, promise, and potential challenges of these technologies. Our goal is to empower individuals with bleeding disorders, in collaboration with their health care providers, to make informed decisions about their care.

NBDF’s educational efforts include:

• Developing clear, accessible resources for patients and families.
• Offering tools and guidance for payers, policymakers, and health care providers to understand the value of innovative treatments.
• Supporting informed advocacy for access to all FDA-approved therapies.

The rare disease community is stronger than ever, united by a shared commitment to patient education and advocacy. Partnerships and collaborations across sectors have fostered unprecedented support and celebration of these advancements, bringing hope for better treatments for some and first-time treatments for many.

As innovation accelerates, our focus remains on ensuring equitable access to these groundbreaking therapies. We work diligently to:

• Advocate for policies that reflect the value of these innovations to patients and caregivers.
• Educate payers and policymakers about the life-changing impact of these therapies.
• Uphold rigorous standards for the safety and efficacy of all treatments.

NBDF remains steadfast in our mission to advance treatments, support cures for inheritable bleeding disorders, and prevent complications through education, advocacy, and research. Together, we will continue to champion a future of improved health and quality of life for our community.