There are an estimated 350 million people living with rare diseases around the world (1), and they are often severe, life-threatening and complex life-long conditions.

Takeda’s Charitable Access Program (CAP), established in partnership with non-governmental organizations, aims to improve access to therapies for patients with rare and genetic diseases regardless of where they may live. Through the CAP, we provide structured charitable donations of our products for patients with rare genetic conditions. We continue to explore options for charitable donations through strong partnerships with third-party organizations that help support patients with this disease, regardless of where they live and their ability to pay for treatment.

In addition to product donation, the LSD CAP provides support for local physicians with ongoing educational programs about rare diseases, diagnosis, and management to support long-term patient care. The key objective of this patient-centric program focuses on ensuring stable and continuous access to treatment for patients who might not otherwise have access,  and supporting country capacity building for sustainable long-term patient care, by understanding local challenges and unmet needs in underserved communities. Capacity building efforts include: educational training, with focus on practical and sustained (digital based) knowledge building around disease awareness, clinical and diagnostic skills & management.

The CAP is currently available for patients with three of the most common lysosomal storage disorders (LSD), Fabry disease, Gaucher disease and Hunter syndrome. The program provides contributions of medical products to non-profits and non-governmental organizations in select countries where:

• Takeda’s products are not available
• Some patients have been diagnosed
• No local regulatory filing is planned (or is more than 12-24 months away)
• In-country capabilities exist to safely and effectively administer treatments.

Takeda is the primary provider of LSD product donation, funding, educational medical resources and support. The program is delivered in partnership with expert not-for-profit organizations including Direct Relief and Project HOPE, who collaborate with partners who have a shared mission to support patient access to innovative and often life-saving treatment. This includes patient association groups and governmental bodies. The programs are supported by an independent medical expert committee (MEC) consisting of leading LSD medical experts, representatives from rare disease patient association groups (International Gaucher Alliance, Fabry International Network, and MPS Europe), and an ethicist, who all volunteer their time.

References: (1) Global Genes. Global Genes Factsheet on Rare Diseases. https://globalgenes.org/rare-diseases-factsstatistics/