Prairie State Wire

The newly formed Advisory Council on Financing and Access to Sickle Cell Disease Treatment and Other High-Cost Drugs and Treatments has commenced its work to explore options and make recommendations for developing payment models and financing structures for novel treatments. This initiative aims to address the accessibility of high-cost drugs and therapies that are currently not readily available to those in need.

Earlier this year, Governor JB Pritzker signed Executive Order 2024-01 to establish the Advisory Council. The council's mission is to provide the Illinois Department of Healthcare and Family Services (HFS) with input as they develop new financing arrangements. These arrangements are intended to ensure equitable access to innovative treatments, ultimately improving health outcomes for Illinois residents.

The group's first meeting took place on June 25, and they will continue their sessions throughout 2024. They plan to deliver their recommendations to the Governor and the Illinois General Assembly by year-end.

"The pipeline for new and potentially transformative cell and gene therapies is expanding quickly, and Medicaid will be a major payor," HFS Director Elizabeth Whitehorn stated. "I am thrilled that the Advisory Council has begun its incredibly important work to develop solutions that will expand access to these new drugs and treatments, which often have costs that are insurmountable for most people who could benefit from them."

Under HFS leadership, the Advisory Council will conduct a comprehensive review of innovative approaches for establishing sustainable publicly funded payment models. The council will focus on value-and-outcome-based models while engaging with subject matter experts, providers, insurers, drug manufacturers, and Medicaid customers affected by these conditions.

The council will recommend financing models that could be implemented in Illinois or nationally with support from federal Centers for Medicare & Medicaid Services (CMS). They will also identify necessary state and federal policies along with legal and regulatory changes required for implementation.

At the federal level, the U.S. Department of Health & Human Services is launching a new Cell & Gene Therapy Access model starting January 2025. This model will negotiate outcomes-based agreements with drug manufacturers on behalf of participating states, focusing initially on Sickle Cell Disease treatment.

Sickle Cell Disease patients often face barriers such as geographic limitations in care access, high treatment costs, limited provider availability, and disproportionate effects on communities of color. In December, the FDA approved two gene therapies for Sickle Cell Disease treatment costing between $2 million-$3 million per therapy session. Lifetime management costs can range from $4 million-$6 million for severely impacted individuals.

Sickle Cell Disease affects over 5,000 residents in Illinois predominantly impacting Black Americans at a rate of one in every 365 births among this group. Over 40% of Illinoisans living with Sickle Cell Disease are covered by Medicaid; approximately 49% have severe forms of the disease.

Despite known causes since nearly 70 years ago, racial discrimination within healthcare systems has led to inadequate resource allocation towards research and treatment support. Consequently, there are limited healthcare providers specialized in managing Sickle Cell Disease comprehensively leading patients into further inequities due primarily due cost barriers exacerbating existing disparities affecting people of color more significantly.

Besides treatments targeting Sickle Cell Disease recent FDA approvals include drugs treating Duchenne muscular dystrophy metachromatic leukodystrophy Hemophilia A Hemophilia B Additional drugs await FDA review later this year

The Advisory Council comprises twenty members holding diverse backgrounds expertise Their names roles include:

Elizabeth Whitehorn serves Chair Director HFS appointed Gov Pritzker January following five years Assistant Deputy Governor Health Human Services Previously played leading role state COVID-19 response initiatives including testing vaccine efforts rental assistance childcare restoration programs She closely worked administration’s healthcare initiatives supporting legislation authorizing state-based exchange increased service access expanded reproductive healthcare children’s behavioral early childhood systems Before joining administration senior policy advisor Governor campaign focusing economic development criminal justice proposals

Alexandra Carpenter mother daughter diagnosed Spinal Muscular Atrophy type November genetic condition missing SMN gene causing muscle weakness affecting ability sit walk breathe swallow Without fatal before second birthday approved therapy Zolgensma purchase price infusion birth screening occurred treated after permanent damage diligent persistent pressure insurance company received ten days diagnosis As January states screen newborns allowing prior symptom development

Ifeanyi Beverly Chukwudozie public health administrator researcher dual master degrees advocacy experience patient rights particularly affected expertise equity chronic diseases perspective individual enriches advocacy efforts shared local regional national levels advisory boards community organizations grant-funded programs ensuring perspectives central discussions policies

Dr James LaBelle Associate Professor Pediatrics University Chicago Director Pediatric Stem Cellular Therapy Program Comer Children’s Hospital advancing clinical leadership evolving science oversight application economy meeting unmet needs children malignant non-malignant diseases translational science laboratory focuses manipulation immune system small molecules targeting BCL-2 family apoptotic proteins nanoparticle-based targeted peptide delivery diseased cells

Dr Ruchika Goel Professor Internal Medicine Hematology Oncology Simmons Cancer Institute SIU School Adjunct Division Transfusion Medicine Johns Hopkins Senior Medical Director Vitalant nonprofit biotherapies organization hospitals safe blood supply network donation centers nationwide host blood drives annually special services patients hospitals across country

George Kitchens co-CEO Artia Solutions founded consultation pharmaceutical manufacturers Medicaid environment previously Vice President Business Development Provider Synergies managing rebate programs served Pharmacy Bureau Chief Florida held positions Eckerd Corporation Regional Operations Manager Managed Care Development member Academy Pharmacy Florida Association Rho Chi Honor Society

Susan Stuard Director State Technical Assistance Center Evidence-Based Policy provides leadership support projects helping evaluate facilitating meetings stakeholder input processes developing analyzing frameworks translating evidence outcomes creating strategies decision-tools enable change led THINC convening organization established criteria enhance quality value Hudson Valley technology policy New York-Presbyterian Board Directors National Committee Quality Assurance NCQA

Nathan Schaefer Senior Vice President Public Policy Access National Bleeding Disorders Foundation NBDF formerly Hemophilia senior Nonprofit Executive progressive leadership political advocacy supporting disorders LGBTQ HIV-affected communities Chicago Washington dedicated finding cures inheritable bleeding addressing preventing complications research education advocacy Steve Sproat Pharmacy Director Aetna Better overseeing implementation expansion benefit large Managed Care Prior Regional Specialty Prime Therapeutics multiple roles Caremark including Vice Product Management

Brian Smolich Vice President Quality Managed Operations Health Alliance Medical Plans leads team Pharmacists Coordinators providing expertise patients better understand medications maximize troubleshoot prescription-related issues held operational roles Department Prescription Benefit Manager oversight Prior Authorization Policy engaging providers members quality initiatives retail pharmacist Walgreens Champaign market

Melissa Creary Assistant Professor Department Health Management Policy School Advisor Equity American Thrombosis Hemostasis Network ATHN Associate Anti-Racism Michigan Social Interventions Eliminate Disparities MSHIELD Michigan Medicine PhD Interdisciplinary Studies focusing History Culture Graduate Institute Liberal Arts Masters Emory nine-year career Centers Control Prevention Division Blood Disorders helped create lead national program data collection agency

Rachel Sachs Professor Law Washington St Louis analyzes problems innovation technologies recently served Biden-Harris Administration Senior Advisor Office Counsel CMS Division joining Academic Fellow Petrie-Flom Center Biotechnology Bioethics Lecturer Harvard clerked Honorable Richard Posner United States Court Appeals Seventh Circuit

Dr Anirban Basu professor Economics Stergachis Family Endowed Director CHOICE Institute University Washington joint appointments Departments Systems Population Research Associate National Bureau elected Fellow Statistical Association understanding economic generating causal evidence potential discrimination machine learning algorithms co-led NHLBI Cure Consortium Impact Analysis served Panel Cost-effectiveness Analysis Editorial Advisory Board Value Journal

Rena Conti associate professor Markets Public Questrom Business Boston co-Director Technology Initiative faculty University between holds PhD Harvard Graduate Arts Sciences policy internationally recognized expert fields biopharmaceutical industry Special Government Advisor CMS held additional advisory roles FDA

TaLana Hughes Executive Director SCDAI started intern completing last Northern obtained Bachelor Degree graduating became Case Manager Educator Newborn Screening educating counseling parents infants positive Trait worked SCDAI years obtained Master becoming director mother daughter son Trait

Dr Radhika Peddinti Associate Pediatrics/Pediatric Hematology/Oncology evidence-based approach clinical care La Rabida largest pediatric centers closely Adult Program transition standardizing pain emergency inpatient settings

Mark Trusheim Strategic NEWDIGS Tufts Medical co-leads Financing reimbursement Cures FoCUS Project focuses economics biomedical innovation precision patient pathways platform trials biosimilars digital advances appointments MIT Sloan Applied Economics Special Employee Commissioner President Consulting career spanned policy Massachusetts Biotechnology diagnostics founder Cantata Labs genomics Cereon Genomics eHealth Monsanto marketing Pharmaceuticals big data Kenan Systems

Joan Ehrhardt joined IDPH Newborn Counselor fall prior clinical reproductive pediatric genetics current role helps manage Programs Grant Programs including screening follow improved individuals Follow development implementation Prevention Care Grant Program

Theodore Tapas Budget Analyst Governor's Office Management working state agencies past three graduate Mount Mercy double major Finance minor lives Springfield accomplished background servant enjoys finding ways back empowering others Adam Flores Senior Insurance DOI helps analysis variety legislative policy roles State City alumnus Washington Harris School next meeting July

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