Patients, industry leaders and legislators participate in celebration of power of hope and innovation in MA
When/Where: Monday, February 29th
Panel Discussion at 9 a.m. (State House, Great Hall)
Rare Disease Day Celebration at 11 a.m. (State House, Great Hall)
What: Hundreds of Massachusetts patients and patient advocates will come together to share stories of Living With Rare Disease and advocate for legislation capping patients’ co-payments as part of the annual Rare Disease Day celebration.
The last day of February has been designated as Rare Disease Day in Massachusetts and around the world to call attention to the public health issues associated with rare diseases, which affect one in 10 Americans.
9 a.m. The Patient Journey in Rare Disease Research & Development
This expert panel will discuss: • Optimizing and strengthening patient engagement models across the life sciences and healthcare industries.
• Patient engagement in policy to drive outcomes and ensure that patient needs are reflected in regulatory and policy decisions.
• FDA’s current initiatives on patient-focused drug development.
• Industry initiatives on patient-centric organizations, healthcare delivery models, clinical trial programs and increasing quality of care.
Speakers are: • Pat Furlong, Founding President & CEO, Parent Project Muscular Dystrophy (PPMD)
• Mihaela MacNair, PhD, MSc, MPSII Global Regulatory Team Lead, Shire
• Gregory S. Sawicki, MD, MPH, Director of the Cystic Fibrosis Center, Boston Children’s Hospital & Assistant Professor of Pediatrics, Harvard Medical School
• Pujita Vaidya, MPH, Operations Research Analyst, Office of Strategic Programs, FDA
• JC Gutierrez-Ramos, PhD, CEO, Synlogic (Moderator)
11 a.m. Celebration Ceremony
• Chris Anselmo, MBA student and Miyoshi Myopathy patient
• Tim Boyd, Associate Director of State Policy, National Organization for Rare Disorders (NORD)
• Katie Brandt, Caregiver & Volunteer Coordinator, Association for Frontotemporal Degeneration
• Rachel Mooney, daughter of a parent living with Posterior Cortical Atrophy (PCA)
• Karen Richards, high school student living with Ehlers-Danlos Syndrome (EDS)
• Tajus Suarmpudi, college student living with Duchenne muscular dystrophy
• Representative Joseph Wagner, Co-Chair of the Massachusetts Biotech Caucus
For more information or to RSVP, contact Jessica Roche at MassBio
jessica.roche@massbio.org or 617-674-5100.
