Not-for-Profit Supply of a Treatment to a Rare Disease Patient Population in the USA and Other ex-EU Countries
Condition
Rare inherited disorder
Drug Type
Approved
Where Approved
Europe
About The Advocacy Group
The United Leukodystrophy Foundation (ULF) is a non-profit, voluntary health organization dedicated to providing patients and their families with information about their disease and assistance in identifying sources of medical care, social services, and genetic counseling. The ULF works to establish a communication network among families, increase public awareness and act as an information source for health care providers, while promoting and supporting research into causes, treatments, and prevention of the leukodystrophies.
Challenge
We were contacted by the ULF as they needed help trying to find a suitable treatment option for their patients. There is currently no approved treatment available to patients in the USA, but the foundation had heard of a product being used by patients in Europe to successfully manage symptoms of the condition. The foundation wanted its patients to have the same opportunity to benefit from this product, however, the group did not know if they could import the product into the USA.
WEP Solution
WEP Clinical has extensive experience helping patients import medicines into the USA, that are approved in another country, through a process known as the Personal Importation Process. As such, WEP Clinical agreed to work with the advocacy group to support each patient’s request, working with the treating physicians to complete all the necessary importation documents. Our team then sourced the product from a supplier in the EU and managed the shipment, customs clearing, and delivery to the treating clinics. WEP Clinical now manages a not-for-profit distribution program for this product that provides access to patients across the US and other Ex-EU countries.
Outcomes:
The program has been running for over 5 years
771 units of drug have been shipped
17 families enrolled in the program
Patients in 5 countries across the world