Providing High-Cost Products to Patients ex-US/EU through a Post-Approval Named Patient Program

Condition

Life-threatening, hereditary disease

Drug Type

Approved

Where Approved

US and Europe

About Sponsor

The sponsor is a global biotechnology company developing promising new medicines for patients with serious rare diseases. The company has a portfolio of approved medicines as well as a pipeline of transformative medicines currently being studied in clinical trials.

Challenge

The company currently has a portfolio of US and European commercially approved, ground-breaking medications that are used to treat life-threatening hereditary conditions. While the company manages the commercial sales and strategy in these major markets, it wanted help making these products available on an unlicensed basis to patients in additional territories. The sponsor wanted to make the products available in as many countries as possible, so that no patient is left without treatment due to geography. To complicate the matter, these drugs are high priced treatments, so careful considerations had to be used when designing the program.

WEP Solution

The sponsor chose to partner with WEP Clinical to take advantage of our global distribution capabilities and regulatory expertise in the unlicensed medicines space. Our project management team worked with the company to develop an access strategy and distribution plan, using logistics and distribution partners in certain countries. We then compiled and completed all the regulatory documentation required to import the drugs on an unlicensed basis. We set a price for the products and worked with local agencies on the ground in each country to ensure HCPs and hospitals were aware of the company’s medicines. Under the terms of the agreement, WEP Clinical receives and holds the US-sourced packs at our EU warehousing facility to manage global requests from physicians. The sponsor has been so impressed with WEP Clinical’s service that it has extended our existing contract for another 3 years, through to 2024.

Outcomes:

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The program has been running for over 3.5 years

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3752 units of drug have been shipped

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20 hospitals currently in scope

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Patients in 16 countries Ex-US and Europe

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