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When testing potential new therapies in clinical trials, it is critical to include the broadest possible range of individuals, in order to ensure these treatments have the best chance of improving outcomes for the various people who will ultimately receive them. 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 pioneering treatments for people with genetic diseases 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 rare genetic diseases, 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:

  • Established a CTD Working Group, comprised of cross-functional leaders in clinical trial design and conduct
  • Completed a comprehensive Baseline Assessment by the CTD Working Group, identifying strengths and areas for improving diversity in BioMarin clinical trials to date, along with recommended strategies to enhance clinical trial diversity in future trials

Strategies: Increasing Clinical Trial Diversity

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:

  • Partner with patient advocacy groups on the goal of increasing representation of underserved communities in our clinical trials
  • Partner with investigative sites on the goal of increasing representation of diverse populations in our clinical trials
  • Expand educational efforts to reach and address needs of diverse and underserved communities

The CTD working group has created strategies and activities to support the three recommendations noted above.

  • Developed and published patient pamphlets in 17 languages to educate prospective study patients about gene therapy, and to highlight how these studies differ from other hemophilia studies
  • Reduced barriers to participation in our clinical trials by offering virtual visits, home health nursing, mobile tools (eDiaries), and patient stipends
  • Conducted a deep dive analysis on historical and current real-world data to understand disease demographics and geography, which will further support site selection and enrollment targets for minority populations
  • Secured a supplier to provide unconscious bias training to our clinical/investigative sites, with a goal of launching this training in Q4 2022
  • Amending our criteria for clinical trial sites to accommodate sites that will help us to recruit trial participants from underrepresented communities

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 rare genetic diseases under study.



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