At BioMarin, we develop medicines to treat people with complex conditions around the world, and we are deeply committed to enrolling representative populations in our clinical trials. We are dedicated to building a blueprint for clinical trial diversity (CTD) that is far-reaching, impactful, sustainable and measurable.

Designing clinical trials for rare and complex conditions poses unique challenges, such as lower prevalence, disease heterogeneity and limited information on disease pathology that can hinder efforts to build a diverse clinical trial. For these reasons, we believe it is critically important to take a holistic approach when incorporating CTD elements into rare disease clinical trials.

Our progress to date includes:

• Establishing a CTD Working Group, comprised of cross-functional leaders in clinical trial design and conduct
• Completing a comprehensive baseline assessment by the CTD Working Group, identifying strengths and areas for improving diversity in BioMarin clinical trials to date, along with recommending strategies to enhance representation in future trials

Strategies: Increasing Clinical Trial Diversity

Going forward, we are particularly focused on advancing three major recommendations from the CTD Working Group, which are part of our broader diversity, equity and inclusion (DEI) initiatives:

• Partnering with patient advocacy groups with the goal of increasing representation of underserved communities in our clinical trials
• Collaborating with clinical trial sites on increasing representation of diverse populations in our clinical trials
• Expanding educational efforts to reach and address needs of individuals from diverse and underserved communities, including people from various socioeconomic backgrounds

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

• Developing and publishing pamphlets in 17 languages to educate prospective participants about gene therapy
• Developing and publishing patient pamphlets in 17 languages to educate prospective participants about gene therapy, and to highlight how these studies differ from other hemophilia studies
• Reducing barriers to participation in our clinical trials by offering virtual visits, home health nursing, mobile tools (eDiaries), and patient stipends
• Conducting an analysis of historical and current real-world data to understand disease demographics and geography, which will further support site selection and enrollment targets for underrepresented populations
• Offering unconscious bias training to clinical/investigative sites
• Securing a supplier to provide unconscious bias training to 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, study teams will collaborate with relevant functions to implement strategies and activities that are most appropriate for the stage of each program. Progress against objectives will be evaluated by the CTD Working Group bi-annually, with the goal of identifying best practices, lessons learned, and adjusting actions as needed.

Once the clinical trials are of sufficient size, the representation and alignment to the populations at large will be assessed, with prospective demographic targets set as clinically feasible and appropriate for the complex conditions under study.

Learn more about our approach to clinical trials