UTAH RARE DISEASE ADVISORY COUNCIL

February 16, 2023

Dear Senators and Representatives,

In 2020, the Utah legislature established the Rare Disease Advisory Council (RDAC) (Utah Code Annotated Section 26-1-41) to advise the state legislature and state agencies on improving access to treatment and services provided to individuals with a rare disease; and identify best practices to improve the care and treatment of individuals in the state with a rare disease. The RDAC is housed as an independent advisory body under the Utah Department of Health and Human Services. Members include researchers and physicians, rare disease patients, caregivers, and representatives from rare disease organizations.

Rare diseases are present across a broad spectrum of medical conditions, from immune deficiencies and genetic disorders to cancers and more. An estimated one in ten Utahns, or an estimated 350,000 women, men and children in our state are suffering from a rare disease.

As part of the RDAC’s charge to advise the legislature, we provide you with the attached policy guide which covers the following topics:
• Access to Genetic Testing
• Ban on Co-Pay Accumulator Programs
• COVID-19: Public Health Guidelines, Access to Vaccines and Treatments
• Gene Therapy
• Pharmacy Benefit Managers
The policy guide will be updated as needed to address additional issues affecting the rare disease community.

We are grateful to the legislature for providing us this opportunity, through the RDAC, to help lawmakers and other stakeholders better understand and meet the challenges and needs of this constituency.
For more information, please contact RDAC Chair, Dr. Lorenzo Botto, at lorenzo.botto@hsc.utah.edu, or
RDAC Vice Chair, Gina Zanik, at ginaszajnuk@gmail.com.

Sincerely,

Lorenzo D. Botto, MD, FACMG
Chair, RDAC
Professor of Pediatrics
Director, Penelope Undiagnosed and Rare
Disease Program
PI, Intermountain West Clinical Site of the
Undiagnosed Disease Network
Division of Medical Genetics, Department of
Pediatrics, University of Utah

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Gina Szajnuk
Vice Chair, RDAC
Executive Director, Rare and Undiagnosed
Network (RUN)

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UTAH RARE DISEASE POLICY GUIDE

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