Providing Pre-Commercial Access Globally
Published by Idis, 2012
A mid-size US biotechnology company with a diverse pipeline had an orphan drug in development, and the phase III clinical trials were completed. Once approved, the drug would represent a major advancement as it would be the first approved treatment for this rare disease.
The company was focused on obtaining approval and the subsequent commercial launch in the US. At the same time they started to receive high demand from patient advocacy groups for access to the medicine outside the US, where commercial availability would take longer. Unfortunately, providing such access would require significant internal resources that the company needed for management of the US launch.
Additionally, the drug was only suitable for certain patients with the disease, and the company could not afford to make the drug available free of charge outside the US prior to commercial availability.
The company wished to facilitate access for patient without the operational burden due to their focus on the US launch. Since this would be the first experience for most of the physicians with the company and drug, the company wanted to assure the best possible experience for them and their patients via provision of educational materials on appropriate use of the medicine. They also needed to confirm that appropriate patients were treated and that each patient received an infection prophylaxis prior to initiation of therapy. Finally, they needed to charge for the drug.
How best to provide access outside the US prior to commercial launch?
Managed Access Programs: Enabling Access Across the Product Lifecycle
Patient access to medicines across the lifecycle from Phase I testing through to discontinuation follows a typical pattern (A; figure on right). The number of patients gaining access grows as the trial process moves forward and continues to increase following first (B) and subsequent launches (C).
Actual patient demand for medicines that address an unmet medical need (D) often exceeds the level of access that is typically delivered. The gap between actual patient access and the total demand for access reflects the fact that not all patients who may benefit from the drug are able to gain access via traditional routes.
Managed Access Programs bridge these gaps along the entire lifecycle, allowing patients to access new therapies prior to their approval and launch, or when commercial access is not an option, all in a fully compliant manner.
They encompass a variety of regulatory approaches used to provide access to medicines outside the clinical trial or commercial setting for medicines that:
- Are still in clinical development and have yet to be approved
- May never be approved, but still have value for a very small population (for example, an orphan drug)
- Are approved in one country, but not another
- Have been discontinued in a particular market or globally
Idis designed and implemented a customized Managed Access Program to respond to the demand from patients outside the US. Idis provided physicians with company-supplied medical information and screened patients to assure they received infection prophylaxis prior to initiation of treatment. A plan for charging for the drug was also implemented during this period.
Pre-approval access was provided to 400 patients in 10 countries outside the US. More than 300 physicians participated in the program. The company was able to charge for the drug and ensure payment was received for all patients.
- Patients with a life-threatening condition were able to access the drug prior to commercial availability
- Physicians were given a significant new treatment option for their patients prior to commercial availability in their country
For the Company
- The program enabled the company to respond to patient demand, while focusing internal resources on the US launch
- Management of the program by Idis ensured that the appropriate patients were receiving the drug in a controlled fashion, and physicians were receiving the necessary education about the drug