CASE STUDIES
Delivering Hope through Expanded Access: Custom-Compounded Medication & Direct-To-Patient Supply
Study: Expanded Access Program with Direct to Patient Needs
Participants: 5 patients with brain tumors, who were unable to join the sponsor’s phase I clinical trial
Solution: Oral medication compounding and Direct-To-Patient within the USA
Challenge
The FDA has approved an Expanded Access Program (EAP) for an experimental therapy for patients with recurrent glioblastoma. The EAP allows patients with serious or immediately life-threatening diseases or conditions to gain access to an investigational medical product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Fast and effective treatment for glioblastoma patients can improve quality-of-life and potentially prolong life. This drug candidate has demonstrated early efficacy for patients with various malignancies.
Thus, the sponsor required a partner proficient in compounding oral medication while also being licensed to manage Direct-to-Patient (DtP) shipments, particularly in North Carolina. The Expanded Access Program (EAP) necessitated highly customized services, including small batch compounding, precise dosage formulation, and rapid turnaround to meet urgent patient needs.
The Myonex Solution
Saveway, Myonex’s fully owned compounding pharmacy, provided an end-to-end solution tailored to the EAP’s requirements. Saveway’s experts handled small-batch production, ensuring the requested 500 capsules were manufactured to precise specifications and in a very short time.
Licensed for DtP shipments in North Carolina, Saveway ensured each patient received their compassionate use medication directly at home, for a better patient experience and convenience.
The Results:
Compounding Excellence applied to Small-Batch Manufacturing: The 500 capsules successfully compounded at Saveway’s facility met the required exacting standards and the sponsor’s needs.
Efficiency and Responsiveness: The entire process – from Active PharmaceuticaI Ingredient (API) receipt from the sponsor to capsule delivery – was completed in just one week, minimizing delays for patients in urgent need.
Patient-Centric Delivery: Patients participating in the EAP received their custom-compounded medication directly at home, enhancing convenience and accessibility.
Providing Hope: For these five individuals with brain tumors and no other treatment options, the experimental drug offered a pathway of hope, delivered with care and precision.
Conclusion:
This Expanded Access Program, governed by the FDA, provided a vital lifeline to patients with limited or no other treatment options. By leveraging its compounding capabilities and Direct-to-Patient delivery expertise and licenses, Myonex enabled the sponsor to fulfill the program’s requirements with speed, flexibility, and reliability.
Through its tailored approach, Myonex delivered not only the required medication but also a message of hope to patients battling brain tumors, contributing to empower them with access to promising experimental therapies – all from the comfort of their homes.
