Providing Pre-Approval Access Globally

Published by Idis, 2012

Situation

A multinational pharmaceutical company had a metastatic, end-stage oncology drug with orphan designation in development. The drug represented a major advancement over existing treatments but was not yet approved. As the company sought approval, they were receiving requests for access to the drug from physicians and patients around the world based on published trial data.

Access Challenge

The company wanted to provide access but lacked pre-approval regulatory expertise and resources to address the expected global demand. Other key internal resources were primarily focused on the EU approval and commercialization. Additionally, the drug was not appropriate for all sub-groups of patients based on clinical trial data.

Access Objectives

The company wished to provide access with a centrally coordinated and consistent approach, but needed to take the burden away from internal resources focused on EU approval and launch. They wanted to facilitate access to the most appropriate types of patients where clinical benefit was expected. The company was keen to develop relationships with key opinion leaders and other clinicians who did not participate in the clinical trials. They also wanted to assure the best possible experience for physicians and their patients via provision of educational materials on appropriate use of the medicine. Finally, they required seamless transition from pre-approval to commercial supply.

How best to meet patient demand globally without placing additional burden on internal teams?

Managed Access Programs: Enabling Access Across the Product Lifecycle

Patient access to medicines across the lifecycle from Phase I testing through to discontinuation follows atypical 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 life cycle, 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 Solution

Idis designed and implemented a customized Managed Access Program which provided a central point of contact to coordinate regulatory effort to ensure global regulatory compliance. Idis screened patients prior to initiation of treatment to make sure only the proper subgroup received access. Idis also provided company-supplied medical information to support the educational objective. Communication plans were implemented within each market regarding timing and planning for transition from pre-approval to commercial supply. With the help of Idis, the company successfully facilitated seamless and efficient transition to launch supplies.

MAP Results

Pre- approval access was provided to over 3,800 patients in 36 countries throughout Europe, the Middle East, South America and Africa. More than 1,700 physicians participated in the program.

MAP Benefits

For Patients

• Patients gained access to life-saving therapy prior to approval in their country

For Physicians

• Prior to commercial availability in their country, physicians had a new treatment option for their patients

For the Company

• The program enabled the company to respond to patient demand, while focusing internal resources on approval and launch
• Idis ensured that the most appropriate patients gained access
• The MAP helped to generate a positive experience among key opinion leaders and clinicians who had not participated in the clinical trials