Challenge

A rare disease company developing a new drug for a lysosomal storage disorder partnered with WEP Clinical to run an Expanded Access Program (EAP). The drug was already being provided under various investigator-sponsored EAPs. When this company opened their blinded clinical trial, additional EAP requests were being received and the company needed a trusted partner to manage the EAP requests. This program served many purposes: 1) transition existing compassionate use programs under a contract with company, 2) enroll new patients under an EAP for patients ineligible for the clinical trial and 3) transition patients from the clinical trial to an EAP for continued treatment. The purpose of the EAP expanded to include data monitoring of prior investigator-sponsored studies. The manufacturer decided that they’d then use the EAP data to support their NDA.

Solution

WEP Clinical and the manufacturer developed an EAP monitoring plan utilizing WEP Clinical’s policies and guidelines to include retrospective and prospective monitoring for the US EAP sites. Many CROs would charge high rates for such a large amount of monitoring services and would not be flexible to monitoring for an investigator-sponsored EAP versus a industry-sponsored clinical trial. WEP Clinical’s monitoring services enabled the company to quickly and efficiently monitor an EAP that had changed course multiple times, and WEP Clinical continued these services to support the EAP to ensured data validity and program compliance.