Equitable Access to Rare Disease Therapies Workshop
Equitable Access to Rare Disease Therapies Workshop
October 22, 2019 | Executive Summary
Key Insight
“There is a concerningly large discrepancy in treatment costs between those with more common rare diseases and those with smaller patient populations, creating additional barriers to proper care.”
Introduction
On May 23, 2019, the Center for Healthcare Innovation (CHI) held an Equitable Access to Rare Disease Therapies Workshop which took place in Washington D.C.
The workshop focused on identifying solutions to the challenges preventing equitable access to rare disease therapies in the United States. This workshop brought together patients, families, patient advocacy groups, biopharmaceutical firms, providers, policymakers, and other NGOs to discuss how to achieve equitable access to care. The panels focused on the latest developments and prominent challenges in accessing novel rare disease treatments, as well as encouraging and accelerating innovation and utilization. In addition, we also discussed the current barriers to care, including delayed access to treatments and drug pricing.
The workshop featured a Distinguished Keynote Address by Dr. Marshall Summar, Chairman of the Board of National Organization for Rare Disorders. Panel discussions included industry, provider, and patient focuses to facilitate dialogue around the challenges and opportunities for each group.
This executive summary aims to:
Define the challenges for patients, providers, and other professionals in the rare disease community
Summarize the innovative ideas and potential solutions on innovation and access
Discuss best practices to better serve rare disease patients both in current and future state
The paradigm shift towards patient-centered care and increasing emphasis on rare diseases highlights the need for innovative treatments and technologies. This executive summary aims to serve as a guide of the ideas and insights regarding the challenges and calls to action in the rare disease community.
Key Statistics and Infographics
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Authors
Mounika Kata, MS
Senior Analyst at CHI
Joseph Gaspero
CEO and Co-Founder at CHI
Abigail Pirron
Analyst at CHI
Joseph Gaspero is the CEO and Co-Founder of CHI. He is a healthcare executive, strategist, and researcher. He co-founded CHI in 2009 to be an independent, objective, and interdisciplinary research and education institute for healthcare. Joseph leads CHI’s research and education initiatives focusing on including patient-driven healthcare, patient engagement, clinical trials, drug pricing, and other pressing healthcare issues. He sets and executes CHI’s strategy, devises marketing tactics, leads fundraising efforts, and manages CHI’s Management team. Joseph is passionate and committed to making healthcare and our world a better place. His leadership stems from a wide array of experiences, including founding and operating several non-profit and for-profit organizations, serving in the U.S. Air Force in support of 2 foreign wars, and deriving expertise from time spent in industries such as healthcare, financial services, and marketing. Joseph’s skills include strategy, management, entrepreneurship, healthcare, clinical trials, diversity & inclusion, life sciences, research, marketing, and finance. He has lived in six countries, traveled to over 30 more, and speaks 3 languages, all which help him view business strategy through the prism of a global, interconnected 21st century. Joseph has a B.S. in Finance from the University of Illinois at Chicago. When he’s not immersed in his work at CHI, he spends his time snowboarding backcountry, skydiving, mountain biking, volunteering, engaging in MMA, and rock climbing.