Webinar: Equitable Access to Rare Diseases Therapies: Why We Must Think Differently To Ensure Broad Access for All
Wednesday, February 23, 2022 | 11 AM - 12 PM CST
About the webinar
Next year will be the 40th anniversary of the Orphan Drug Act, which established incentives to research and develop indications for rare diseases. Since that time, 1,039 “orphan indications” have been approved by the FDA for previously unaddressed medical conditions with 26 approved in just 2021 alone. Advancing technologies have enabled healthcare providers to more effectively diagnose rare conditions that impact 1 in 10 Americans - thanks largely to advocacy and legislative efforts to expand and fund newborn screening. The general public has also become aware of and increasingly familiar with rare diseases as new indications therapies have received marketing authorization from the FDA.
But it is not always easy for people living with rare diseases to be aware of and have access to these treatments, particularly for patients of color. Communities of color, who are impacted by alarming and stark health disparities, have diminishing levels of trust in our healthcare system. In an October 2020 poll, 7 of 10 Black Americans say they’re treated unfairly by the health care system, and 55% percent say they distrust it (1). Half of Black Americans (50%) report they have experienced health care discrimination; more than 2 in 5 Native Americans (42%) and one-third of Asian Americans (34%) and Hispanic Americans (33%) likewise report having experienced discrimination when seeking health care (2).
According to the Gallup National Health and Well Being Index, compared to whites, Hispanics are almost three times as likely to be uninsured, and African Americans are almost twice as likely to be uninsured In all age groups, Blacks were more likely than whites to report not being able to see a doctor in the past year because of cost (3)(4). Blacks have higher death rates than whites for 12 of the 15 leading causes of death in the United States, and almost all rare diseases, said Linda Goler Blount, president of the Black Women’s Health Imperative, a nonprofit that focuses on health equity in rare diseases (5)
It is also no surprise that rare diseases consume a disproportionate quantity of healthcare resources relative to their prevalence. In 2019, the economic cost of rare diseases in the United States reached $1 trillion, including $111 billion in non-medical and uncovered healthcare costs (6). The excess direct medical costs for patients with a rare disease are, on average, $26,800 more per patient annually than those for patients with chronic conditions (7). Individual medical costs for people with a rare disease are 3-5 times greater than for those who do not have a rare disease (8).
In this webinar, distinguished panelists representing diverse patient communities, healthcare providers, multifaceted manufacturers, and patient support providers, will address opportunities and challenges to equitable access to rare disease therapies. What gaps must be addressed? What approaches can be incorporated into the way rare disease therapies are developed and made commercially available? Given the high cost of many rare disease therapies, who have the onus for enabling solutions that are fair and far-reaching? The discussion promises to be both provocative and informative.
You are invited to submit questions for our panelists in advance of the webinar to info@chisite.org.
Joff Masukawa is President and founder of Diligentia Strategy, a life-sciences consultancy founded in 2015 that develops commercialization strategies for manufacturers of rare orphan and specialty drugs, cell and gene therapies, and other novel emerging medical innovations. Diligentia also offers federal and state government relations and public affairs strategy and services to facilitate client engagement with policymakers and government agencies in support of business objectives. Prior to founding Diligentia, Joff was Vice President and Global Head of Government Relations and Public Affairs at Shire, where he designed and led strategies to build the company’s brand, engagement and leadership with government representatives and agencies, patient advocacy groups, payors, industry associations and other key external stakeholders. Joff has held senior level leadership roles spanning commercial and corporate functions, including market access, government and public affairs, patient and provider services, and sales and marketing at Shire, Genzyme, Visible Genetics, Gentiva and Labcorp. He has developed and executed numerous commercial launch strategies for first-in-class products and is an expert at developing and aligning optimal legislative, regulatory and market access solutions to support business objectives. He began his career as a clinical sales representative for Merck. He serves on the boards of Phoenicis Therapeutics, Caregiver Action Network, and the Center for Healthcare Innovation. Joff is also an advisor to America’s Health Foundation and a member of the American Society of Gene and Cell Therapy and the Innovation and Value Initiative. A first-generation American, Joff is a passionate advocate of diversity and inclusion in life sciences. He regularly collaborates across the industry, NGOs, and his personal network to convene important conversations and forums to advance the cause of older adults, racial and ethnic minorities, and the LGTBQ+ community to help them achieve their full potential. He holds a BA in international studies from Johns Hopkins University and completed his graduate studies in international affairs at the Johns Hopkins School of International Studies and in strategic communications at Columbia University.
Distinguished Speakers
Ms. Sara Aswegan
Rare Disease Global Medical Affairs Head-Managed Access and Communications, UCB
With over 20 years in the pharma/biotech industry, Sara has extensive Global Rare Disease expertise spanning several therapeutic areas and functions (sales, training, marketing, management, alliances, business development, portfolio strategy, global leadership). Sara has extensive experience in the design and implementation of managed access programs/early access starting from her time with Shire Human Genetic Therapies, Ultragenyx and currently at UCB. She serves as an advisor and maintains a strong network among clinicians and patient advocates in the rare disease space and is active in developing the first framework and guidelines for charitable access through an initiative led by the Global Alliance for Rare Disease and PQMD.
Sara serves as the Head of Rare Disease Medical Communications and Access, Asset and Commercial Strategy at UCB, based in Switzerland. She most recently served as Senior Vice President, Commercial, Global Strategy for Ultragenyx Pharmaceuticals, based in Basel, Switzerland. Prior to that, Sara held multiple roles with advancing leadership responsibility with Shire (2007-2016), her most recent as Vice President, Product Strategy Lead, for both the GI and LSD-MPS II Franchises based in the US and also Switzerland. Prior to joining Shire, Sara worked at Astellas, Sanofi and Bayer, all based in the US. Sara holds a B.A. from the University of Nebraska-Lincoln and Executive Education from both Harvard School of Public Health and Columbia Business School.
Dr. Mauvareen Beverley, MD
Advisor, Westchester Sickle Cell Outreach
Patient Engagement and Cultural Competence Specialist
Dr. Mauvareen Beverley an executive-level physician, with 20 years’ experience advocating for improving the patient engagement and cultural competence for all populations, especially the geriatric, immigrant, and African American communities. As AVP, Physician Advisor for NYC Health + Hospitals, she sponsored the first Conference on Improving the Health of the Elderly Black Population. She implemented the concept of “The Bridge Team” whose role was to bridge the gap in care for the most complicated and vulnerable population and as a result improve care and health outcomes. As Deputy Executive Director of Kings County Hospital, her team decreased Congestive Heart Failure readmission from 30% to 18.7% in less than 2 years.
Dr. Beverley is a Fellow at the New York Academy of Medicine (NYAM) and her Abstract “Health Disparities and Epidemics: Perception vs. Reality was selected for presentation at NYAM 12th Annual History of Medicine and Public Health Night.
Dr. Beverley is also a Member of American Medical Association (AMA) and Medical Society, State of NY (MSSNY), collaborated with Westchester County Medical Society, Westchester Academy of Medicine and Putnam County Medical Society in developing Patient Engagement and Cultural Competence Training Program with CME credits. Dr. Beverley served as faculty of an education session for ACHE’s Thomas C. Dolan Executive Diversity Program. Her Poster on Solutions to Health Disparities: The Common Thread, The Human Experience was accepted and will be presented at the AHA Conference in Chicago, March 2022.
Dr. Beverley received her bachelor’s from Boston University and MD from University of Buffalo School of Medicine. She completed her internship and residency in Internal Medicine at Harlem Hospital-NY Columbia Presbyterian.
She is President of Mauvareen Beverley, MD. PLLC, Patient Engagement and Cultural Competence Specialist.
Ms. Jennifer Bright, MPA
Executive Director, Innovation and Value Initiative (IVI)
Jennifer L. Bright, MPA is Executive Director of the Innovation and Value Initiative (IVI), an independent, nonprofit research organization focused on improving patient-centered methods and practice in value assessment. IVI has partnered with rare disease community entities on patient-focused drug development initiatives and is exploring open-source model development in rare disease. Ms. Bright is also President of Momentum Health Strategies, a consultancy through which she offers clients her extensive expertise in health policy, advocacy and patient engagement, business strategy, and organizational development and leadership. Ms. Bright is Board Chair-Elect of Mental Health America and an editor for the American Journal of Accountable Care. She resides in Alexandria, Virginia with her husband and three children.
Mr. Dan Donovan
Founder & CEO, rareLife Solutions
Dan’s career in rare disease began in 2011 when he was appointed to the Board of Directors and later as Chief Business Officer of Cancer Prevention Pharmaceuticals (CPP). Having observed the fractured rare disease ecosystem combined with the always passionate stakeholders, Dan was inspired to establish rareLife solutions, inc., and create onevoiceTM, a cloud-based educational community-building software platform.
Prior to rareLife and CPP, Dan founded Envision Pharma in 2001. Dan was the visionary behind the creation and development of medical publications software, Datavision. Dan began his career at Pfizer serving in a variety of positions from sales to market research and marketing in the US and international marketplace. During his time at Pfizer, he played a pivotal role in the development and commercialization of some of the pharmaceutical industry's most successful product launches.
Dan earned a Bachelor of Science degree in Finance at Lehigh University.
Endnotes
1. Hostetter, Martha, and Sarah Klein. Understanding and Ameliorating Medical Mistrust among Black Americans. Commonwealth Fund, 2021. Print.
2. "2021 Alzheimer's Disease Facts and Figures." Alzheimer's & dementia 17.3 (2021): 327-406. MEDLINE. Web.
3. Austin Frakt. "Bad Medicine: The Harm that Comes from Racism." New York Times (Online), Jan 13, 2020, Research Library Prep. Web. <https://www.nytimes.com/2020/01/13/upshot/bad-medicine-the-harm-that-comes-from-racism.html>.
4. Nania, Rachel. "Covid Hospitalizations, Deaths Higher for Minorities." AARP. June 3, 2020. Web. <https://www.aarp.org/health/conditions-treatments/info-2020/covid-burden-minorities.html.>.
5. Clark, Sydney. "Diversity Elusive in Rare Disease Research." National Press Foundation. December 30, 2021. Web. <https://nationalpress.org/topic/diversity-elusive-in-rare-disease-research/>.
6. Garrison, Sheldon, et al. "The Economic Burden of Rare Diseases: Quantifying the Sizeable Collective Burden and Offering Solutions." HealthAffairs. February 1, 2022. Web. <https://www.healthaffairs.org/do/10.1377/forefront.20220128.987667>.
7. Waddill, Kelsey. "Rare Disease Healthcare Spending Tops Costly Chronic Diseases." HealthPayerIntelligence. March 1, 2021. Web. <https://healthpayerintelligence.com/news/rare-disease-healthcare-spending-tops-costly-chronic-diseases>.
8. Tisdale, Ainslie, et al. "The IDeaS Initiative: Pilot Study to Assess the Impact of Rare Diseases on Patients and Healthcare Systems." Orphanet journal of rare diseases 16.1 (2021): 1-429. CrossRef. Web.re
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