As breakthrough therapies are under development, particularly for diseases for which limited or no treatment is currently available, more patients are requesting access to medications in the window between the end of registrational trials and marketing approval. Often biopharmaceutical companies launch Expanded Access (EAP) or Compassionate Use Programs (CUP) to address this unmet need. The implementation of EAPs can be considered in a variety of stages and situations during the lifecycle of the drug. They can be considered in the early stages of product development where there is a likelihood of the product showing promising results for patients who cannot take part in clinical trials, or, once a clinical trial is fully enrolled, making the product available to patients until market approval. For sponsors, there are numerous operational logistics and regulatory approvals required in order to provide access to their medications through an EAP or CUP.
UBC was engaged to assume responsibilities for a named-patient EAP by a sponsor company that is developing a product to treat a rare infectious disease. Named-patient EAPs involve pre-approval access to treatments in response to requests by physicians on behalf of specific, or “named”, patients before those medicines are licensed in the patient’s home country. Whereas site-based EAPs are initiated by sponsor companies and allow physicians to offer the treatments to a cohort of patients, named-patient EAPs are limited only to the requested named patients. The sponsor had initiated the named-patient EAP but required dedicated operational support due to a high patient demand and the need to expand globally.