This International Mastocytosis and Mast Cell Diseases Awareness Day, we reflect on the strength and resilience of the mast cell disease community, including people living with systemic mastocytosis (SM). Our vision is a future in which individuals living with SM can pursue their life goals unimpeded, supported by innovative treatments that allow them to thrive. Though with rare diseases like SM, each person’s journey and needs are unique.
In collaboration with the mast cell disease community, Blueprint Medicines has pioneered research across the spectrum of SM for more than a decade. We know that advancing science is just one piece of the puzzle; through tailored patient support and education, collaborations within the community, and bringing forward patient stories, we strengthen our understanding and impact for people affected by SM and other mast cell diseases.
At Blueprint Medicines, we do more than deliver innovative medicines. We strive to be partners to the patient communities we serve through a range of initiatives. For example:
- We analyze SM datasets from electronic health records, patient registries and patient-reported outcome measures to gain comprehensive insights into patient experiences across diverse populations; this evidence helps ensure that our actions are guided by real patient needs and experiences. For example, we conducted two global patient- and HCP-reported outcomes studies TouchStone (U.S.) and PRISM (Europe) to understand the real-world burden of SM based on patient and healthcare provider perspectives.
- We develop educational resources to meet the diverse needs of patients and empower them with disease-related knowledge, such as ItsSMthing and Navigating SM.
- We initiate targeted efforts to address the unique healthcare needs and challenges of marginalized and underrepresented groups. Through culturally sensitive care programs, health equity assessments and community engagement strategies, we work to reduce health disparities and promote equity in healthcare access and outcomes. We’re committed to removing barriers to participation in our clinical trials by maintaining a travel and reimbursement policy to help alleviate financial and socioeconomic burdens, providing translation of study materials, and partnering with patient advocacy groups to expand disease state awareness and trial participation across diverse and representative populations.
- We host educational programs both in person and virtually that keep in mind the unique needs of people living with SM, attend regional SM conferences, and join advocacy group events to meet with patients and caregivers, including The Mast Cell Disease Society’s MastCellCon earlier this year.
Beyond engaging with the patient community, we seek out partnerships with renowned healthcare institutions and universities to advance research and treatment for mast cell diseases. We collaborated with the University of Pennsylvania to develop and publish an algorithm that applies machine learning techniques to pinpoint potential SM cases using electronic medical records, with the goal of shortening the time to diagnosis.
Our commitment to patients expands beyond partnering with the external community and is engrained in our company culture, enriching our daily work and sense of purpose. Storytelling has always been an integral part of our culture and how we learn, grow and connect with one another. Throughout the year, we provide opportunities for our Blue Crew and beyond to hear directly from patients and learn what it means to live with a mast cell disease.
Most recently during our annual Core Values Week – a celebration of how our five core values guide our work – we heard inspiring stories from women living with indolent SM (ISM) about their journey with the disease. One of these women also participated in the first year of Blueprint’s Colors of SM: Expressions of Life with Systemic Mastocytosis program. This initiative, developed in collaboration with the patient organization Twist Out Cancer, highlights the personal stories of those impacted by SM through the transformative power of creative arts, with the goal of fostering a deeper understanding of the experience, aspirations and hopes of those with the condition. These efforts bring the impacts of our work to life and further inspire us to make a meaningful difference in the lives of people around the world.
Today, we’re looking to the future as we build on our SM expertise to advance research, accelerate treatment innovation and work to improve outcomes for more people living with mast cell diseases. Below, hear directly from individuals living with SM about their experience with this disease and their hopes for the future.