Addressing Health Inequity in Rare Disease Research

Apr 01, 2021

Guest Blog by Heather Dean, Vice President, Hematology Franchise Head

More than 30 million Americans are living with a rare disease – one in every 10 people – making rare disease not that rare. Unfortunately, for many, it will take several frustrating years for an accurate diagnosis. And for ethnic and racial minorities, this journey can be even more challenging.

On February 26, I had the privilege of participating in MassBio’s 2021 Rare Disease Day panel discussion on health inequity in rare disease research alongside esteemed biotechnology executives and nonprofit partners at CRISPR Therapeutics, Biogen, and Sickle Cell Community Consortium. The panel agreed on several critical approaches to improving health equity: address historical reasons for distrust, increase diversity during drug development as well as in clinical trials, and broaden education for communities most affected by rare disease.

Health inequity is not a new issue. However, the COVID-19 pandemic put a spotlight on these challenges as minority populations are getting sick at a disproportionate rate and are the most severely affected. The panel conversation highlighted the fact that the distrust we are seeing with the COVID-19 vaccine among minority groups runs deep throughout healthcare. The work being done now by health care organizations and advocacy groups to overcome vaccine hesitancy needs to be used as a model for how we approach diversity in rare disease conversations. It starts with acknowledging the history behind the distrust by minority groups. Recognizing these issues can be the first step toward build a trusting relationship with people with rare diseases.

Diversity in clinical trials has been a challenge for many years. The panel suggested that one way to overcome this challenge is to involve diverse patients with rare diseases in early phases of drug development. By gathering diverse groups of patients, we can better understand their daily lives, the challenges they face, and their barriers to care in order to discover connections or synergies in symptoms. Without the full participation of minorities with rare disease it is more difficult to advance innovation, particularly for those rare diseases, like Sickle Cell Disease for example, that disproportionately affect minority populations. Early, active involvement of diverse populations also helps to build trust and advocacy.

Finally, the panel agreed that education, for both the healthcare industry and people with rare diseases, is a critical tool that is often overlooked or underestimated. The healthcare industry needs to better understand the social determinants of health for minority patients – how the conditions where they are born, work and age affect their health —  as well as where they access their healthcare and disease information. By understanding these elements, healthcare organizations can provide better educational and community-based resources that will activate patients to seek care. Furthermore, organizations can create materials and messaging that helps people with rare diseases in underrepresented populations see and hear themselves reflected and feel that they are being heard.

To help overcome health inequities, the life sciences industry has a responsibility to engage with patients who truly represent the communities we serve – reflecting all races, genders, ethnicities and socioeconomic backgrounds — to address the apprehension they may have regarding trust in the healthcare industry and glean meaningful insights that drive education and innovation. Innovation that goes beyond “me too” therapies to address new areas or gaps in care.

As an industry, we need to use deeper patient insights to more effectively engage, educate and meet the underlying needs of diverse patients who count on us for their health and wellbeing. Local events such as MassBio’s Rare Disease Day celebration are important forums that allow us to come together to share and discuss the critical rare disease research being done and to more effectively engage with patients.

About the Author:

Heather Dean
Vice President, US Hematology Franchise Head
Takeda Pharmaceuticals USA, Inc.

Heather Dean is the Vice President and Hematology Franchise Head for Takeda’s US Commercial organization. She is responsible for leading the sales and marketing teams for Takeda’s rare hematology portfolio, which currently includes treatments for bleeding disorders such as Hemophilia A, Hemophilia B, and von Willebrand’s Disease. Heather has been with Takeda since 2004, and previously served as VP of Sales and Key Accounts for Takeda’s Gastrointestinal (GI) business unit.

Heather’s pharmaceutical experience is focused in the areas of marketing and sales leadership. She began her pharmaceutical career at Eli Lilly and Company in 1999 where she held various commercial positions, launching two diabetes products in sales and a CNS product in marketing. Heather joined Takeda to lead the pre-launch, launch and post-launch phases of the metabolic commercial program. She then went on to head the GI Marketing franchise, which included two primary care GI brands and one pre-launch global specialty GI brand. In 2014, Heather returned to sales as an Area Sales Vice President, leading half of the General Medicine commercial sales organization.

Heather holds a Bachelor of Science degree with honors from Northern Illinois University. In 2013, Heather graduated from the Takeda Leadership Institute global leadership development program (through INSEAD). In 2016, Heather was selected to attend the Takeda Global President’s Forum in Tokyo.

See all MassBio News