Lysosomal Storage Disorder Charitable Access Program (LSD CAP)

Rare diseases are often severe, life-threatening and complex conditions. Every day, Takeda works to provide treatments for the estimated 350 million people living with rare diseases around the world.
Our Charitable Access Program (CAP), established in partnership with several 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 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 identify the greatest areas of unmet patient needs around the world.
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 i) Takeda’s products are not available; ii) some patients have been diagnosed; iii) no local regulatory filing is planned (or is more than 12-24 months away); and iv) in-country capabilities exist to safely and effectively administer treatments.
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, locally sustainable patient care.
The key objective of this patient-centric program focuses on not only ensuring stable and continuous access to treatment for underserved patient communities, but also at the same time, supporting country capacity building for sustainable long-term patient care by understanding local challenges and unmet needs in underserved communities. Capacity building includes: supporting with regular Educational training, with focus on practical and sustained (digital based) knowledge building around disease awareness, clinical skills & management and diagnostic challenges.
LSD Cap current operating model
Takeda is the primary provider of LSD product donation, funding, and educational medical resources and support.The program is centralized through not-for-profit logistics partners (Direct Relief and Project Hope), who collaborate with external partners that have a shared common vision (such as Care Beyond Diagnosis, Centogene, FYMCA Medical Ltd, patient association groups, and governmental bodies). The Direct Relief managed program is 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, all who volunteer their time. The MEC, with the exception of patient association group representatives, is responsible for patient selection and monitoring of patient follow-up. The Project Hope managed program is supported by a separate MEC comprising a different group of medical experts. Project Hope supports 12 patients with GD in Egypt as part of the LSD CAP.
Patient treatment and monitoring
Applications for participation in the LSD CAP are submitted by the treating physician. The application may also include a short video of the patient’s clinical state as appropriate, to allow reviewers to more accurately assess patient clinical severity and thus potential to benefit from treatment.
Once a patient is accepted into the program, the operators focus on ensuring that the Enzyme Replacement Therapy (ERT) drug supply can reach the approved treatment center and patient efficiently, following all local regulations. Patients enrolled in the program must adhere to guidelines that include:
  • Attending the treatment center to receive their infusions every week or every other week (dependent on disease requirement),
  • Agreeing to regular physician follow-up visits and routine blood tests to allow for monitoring of response to treatment,
  • Completing an annual quality-of-life survey.

Patients may choose to discontinue treatment at any time. Treatment through the program may also be stopped at the discretion of the independent MEC if the patient is deemed non-compliant with treatment, shows no response to treatment, develops another life-threatening condition, develops a serious adverse reaction, or if no patient-follow-up information is provided within the specified period. This is done on a case-by-case basis and in agreement with the local treating clinician. Every effort is made to explore options to facilitate compliance prior to discontinuing ERT.
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