When testing potential new therapies in clinical trials, it is critical to include the broadest possible range of individuals to ensure these treatments have the best chance of improving outcomes for the all people who will ultimately receive the medicines. Diversity in clinical trials has been championed by leading health authorities and industry organizations, including the Food and Drug Administration (FDA) and Pharmaceutical Research and Manufacturers of America (PhRMA), which have released guidelines and principles calling for expanded health equity through greater clinical trial diversity.
At BioMarin, we create medicines for people with complex conditions around the world, and we are deeply committed to enrolling representative populations in our clinical trials. Our vision is to become a leader in promoting and practicing clinical trial diversity (CTD) in these conditions, with a mission to develop a blueprint for clinical trial diversity in the represented population that is far-reaching impactful, sustainable and measurable.
Our progress to date includes:
Going forward, we are particularly focused on advancing three major recommendations from the BioMarin CTD Working Group, which fall under BioMarin’s greater DEI Initiative:
The CTD working group has created strategies and activities to support the three recommendations noted above, including:
Moving forward, the study teams will collaborate with relevant functions to implement strategies and activities that are most appropriate for the stage of the programs. Progress against objectives will be evaluated by the CTD working group at least twice each year, with the goal of identifying lessons learned and best practices, and adjusting actions as needed.
Once the trials are of sufficient size, the representation and alignment of the trials to the populations at large will be assessed, with prospective demographic targets set as clinically feasible and appropriate for the complex conditions under study.