Continuing Access Post-Clinical Trial

Published by Idis, 2012

Situation

A mid-sized European biotechnology company had several products in development for CNS diseases. The phase III trial for a neurodegenerative genetic disorder drug with orphan designation had recently concluded. The drug represented a major advancement over existing therapies that were currently being used off-label with limited efficacy and significant side effects.

Access Challenge

As the phase III trial came to a close, the company sought to continue providing access to the drug for patients who had participated in the trial, prior to the drug being commercially available. Demand for the drug was expected from a wider patient population, but the company wanted to limit access to ex-trial patients.

Unfortunately, extending access for study participants via an open-label extension trial would have significant costs and providing such access would require internal resources that the company needed elsewhere.

Access Objectives

The main objective was to provide uninterrupted access to the drug following participation in a clinical trial using a more economical mechanism compared to an extension trial. The company wished to validate that a Managed Access Program would be a viable option for providing access in each market. They also needed to assure that there would be seamless transition from the trial setting to the Managed Access Program, and that this would be effectively communicated to sites.

How best to continue uninterrupted access to trial participants 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 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 Solution

Idis designed and implemented a Managed Access Program that enabled trial participants to continue use of the drug prior to its commercial availability, taking resource burden away from the company. Communication plans were developed to manage requests for non-clinical trial participants as well as plans to guide sites and physicians with patient transitions. Idis also provided necessary training to key stakeholders to ensure safe and timely access to the drug, and conducted due diligence in each market to assure compliance with local regulations.

MAP Results

Post-trial, pre-approval access was provided to 150 patients previously enrolled in the phase III trial, in eight European countries. More than 30 clinical investigators participated in the program.

MAP Benefits

For Patients

• Trial participants were able to continue use of a life-changing drug at the conclusion of the trial, and prior to commercial availability

For Physicians

• Physicians were provided a simplified access option to a new treatment post-clinical trial

For the Company

• The program enabled the company to continue to meet the needs of patients who did not have viable alternative treatment options, in a way more cost-efficient than an extension trial
• Management of the program by Idis allowed the company to focus internal resources on regulatory filings