A small pharmaceutical sponsor of a proposed treatment for multi-drug resistant (MDR) HIV patients approached Westat to manage its Phase III trial. MDR HIV patients had extremely limited options for treatment, so the proposed drug was in high demand as an addition to viable HIV therapies. The sponsor’s needs were clear: devise creative solutions to rapidly enroll subjects; contribute to the design of a rigorous, Good Clinical Research Practice (GCP)-compliant study; and support the Biologics License Application (BLA) preparation for approval by the U.S. Food and Drug Administration (FDA) and the marketing application for approval by the European Medicines Agency (EMA).

Expediting the Study Start-up

Westat CLS staff promptly met with the sponsor to discuss plans for an expedited study and use of a central Institutional Review Board (IRB). Our experts advised that the central IRB would reduce both study start-up time and costs and allow our well-controlled study’s rapid progression. With the sponsor’s approval, we established the hub, identified sites having patients who met the study’s eligibility criteria, and gained FDA approval to assign orphan drug status to the treatment. Ensuring the staff’s peak performance was next on our agenda, and we designed and launched site trainings for clinical research associates (CRAs) via webcast and customized trainings for clinical site staff, and data and safety managers.  

Efficiently Managing a Complex Collaboration

With a seamless collaborative partnership across 31 sites and two labs, we ensured the study’s optimal efficiency. To guarantee a streamlined start-up, we held regular conference calls with the sponsor and labs to discuss timelines, tasks and assignments, and potential risks and resolutions. In collaboration with the sponsor, we developed a protocol training agenda and materials for the site staff investigator meeting. After study initiation, Westat CRAs maintained regular communications with study teams to answer protocol-related questions, provide consultation on enrollment strategies, and provide refresher training as needed. During these weekly meetings, our team flagged issues and reported on proposed and implemented solutions and their outcomes.   

Addressing Sluggish Subject Recruitment

Despite the efficiency of our operation, the effort was not without challenges. In particular, enrollment lagged at times. We devised creative solutions including offering site staff incentives that encouraged accelerated screenings and enrollment, as well as crafting and submitting information about the study to publications focused on HIV patients to expand our reach to patients in dire need of treatment. As other problems surfaced, our project manager and CRAs quickly resolved them to avoid delays. For example, during an isolated incident of missing lab results, we immediately requested a call with the lab to determine the delays, obtained their commitment to making the results available, and requested a corrective and preventive action plan to ensure the sites received future lab results quickly.   

Expediting Study Closure and BLA Submission

To expedite the study’s closure, we performed tasks in parallel to reduce the number of outstanding data discrepancies at the study’s conclusion. We conducted four quality assurance site audits; analyzed and integrated data for 153 adult subjects from the Phase II study and Phase III registered trial; and reported on the drug’s safety and efficacy in the clinical study report for the BLA submission to the FDA. The accelerated timeline required a high level of organization, attention to detail, quality control, and project management skills to ensure all tasks were performed with the highest quality and no errors were made.

Advancing the Treatment to Market

Our regulatory and trial management expertise; honest and reliable communication with the sponsor; staff continuity; innovation; and long-term relationships with vendors facilitated rapid advancement of the study drug to market. In March 2018 the FDA approved the sponsor’s treatment for U.S. patients; in September 2019 EMA authorized its distribution in the European Union.