Using expanded access to treat patients that do not qualify for the clinical trial
Condition
Rare progressive genetic disorder
Drug Type
Investigational
Trial Phase
Phase 2
About Sponsor
The Sponsor is a specialty pharmaceutical company focused on providing medicines to address unmet patient needs. Based in the US and Europe, the company develops drugs to treat a variety of health conditions in the rare disease space.
Challenge
The Sponsor company chose to set very strict eligibility criteria for its phase 2 clinical trial. As a result, there were patients who wanted to access the company’s investigational drug but found they could not be enrolled into the study. Because there are no approved treatments for this condition and very few investigational options available, these patients were contacting the company and requesting access to the drug outside the clinical trial.
The company wanted to find a way of dealing with these unsolicited requests.
WEP Solution
The Sponsor partnered with WEP Clinical to set-up an EAP. Providing access through an EAP meant the sponsor could set less strict inclusion/ exclusion criteria. This allowed the company to offer a potential treatment option for patients excluded from the clinical trial, whilst still controlling patient access and reserving the right to limit the number of patients receiving treatment through the program.
Outcomes:
The program has been running for over 5 years
4,500 units of drug have been shipped
93 individual patients have been treated
Patients in 14 countries* across 4 continents
*Countries in scope: Australia, Canada, China, Costa Rica, France, Germany, India, Italy Mexico, New Zealand, Switzerland, Turkey, United Kingdom, United States
During the lifecycle of the EAP, the original sponsor company was acquired twice over by larger organizations. After each acquisition, the new sponsor was impressed with WEP Clinical’s best-in-class service and chose to continue to trust WEP Clinical with the management of the program. In addition, we have been awarded new programs off the back of our continuous efforts to put the sponsors, their sites and their patients first.