For Immediate Release – January 9, 2025
Agency Issues First Report on Sickle Cell Gene and Cell Therapy Treatments
Advisory Council recommendations focus on Medicaid payment models for novel Sickle Cell Disease treatments and other high-cost drugs and therapies
CHICAGO — As required by Executive Order 2024-01, the Illinois Department of Healthcare and Family Services (HFS) submitted to the Governor and the General Assembly its first report from the Advisory Council on Financing and Access to Sickle Cell Disease Treatment and other High-Cost Drugs and Treatment. The report evaluates the success and challenges of various value and outcome-based payment approaches implemented in other states and includes recommendations for financing and increasing equitable access for these drugs in Illinois. In fulfillment of the Executive Order, the advisory council met six times over the past six months, culminating in the production of this report. The full report can be found here.
“When I signed the Executive Order to create this council, it was with the clear goal of making life-changing gene and cell therapy treatments affordable and available to every Illinoisan,” said Governor JB Pritzker. “These recommendations are a critical step forward in that effort, and Illinois remains committed to advancing this work to make transformative care more accessible.”
“Ultimately these recommendations are centered around the goal of promoting equitable access to healthcare for all Illinoisans,” said Acting HFS Director Elizabeth M. Whitehorn. “The state will continue to collaborate with other states, federal policy makers and stakeholders to build on these recommendations and ensure that Medicaid patients in Illinois are able to access the best care no matter their backgrounds.”
The healthcare landscape is rapidly evolving with the increasing availability of advanced treatments for serious and often life-threatening diseases. In the next decade many new cell and gene therapies are expected to become approved and available for use. Many existing and expected cell and gene therapies will target children and young adults, who are disproportionately insured by state Medicaid programs. These therapies can often have high prices, often exceeding one million dollars per patient.
“A new age in medicine is upon us. The ability to address debilitating diseases at their root cause has the potential to change patient’s lives in ways that were previously unimaginable,” said Dr. James LaBelle, Director of the Pediatric Stem Cell and Cellular Therapy Program at the University of Chicago. “I hope that access to these therapies will be dictated only on biology and not by lack of accessibility or cost. I commend the Governor and HFS for being at the forefront of addressing how to bring current and future transformative high-cost drugs, including gene therapies, to those in need throughout the entire state.”
In addition, many patients battling rare diseases face significant challenges to accessing available care even when a break-through treatment exists. Specialized provider shortages, a lack of geographically proximate care, and other financial and logistical barriers all stand as potential impediments to patient access to current standard of care practices. The recommendations in the report also aim to address these challenges.
“As a caregiver representative and oftentimes spokesperson for the Sickle Cell community, the creation of the Advisory Council was definitely a step in the right direction. Convening key stakeholder experts together in order to combat the many challenges, barriers and access to care that the rare disease community continues to face gave us a much-needed platform,” said TaLana Hughes, MPH Caregiver and Executive Director of the Sickle Cell Disease Association of Illinois. “As a council faced with the task of creating recommendations focused on the development of payment models and financing structures with a very tight timeline, we dove in. Many very thorough, healthy debate meetings led us to what we believe supports equitable access while contributing to a much larger national effort to formulate solutions.”
The report’s recommendations are grouped into three categories and include the following recommendations:
Recommendations for HFS on Financing CGT and Other High-Cost Therapies include:
- Ensuring that cell and gene therapies (CGTs) and other high-cost drugs are considered “covered outpatient drugs” to entitle the state to rebates and discounts on their use.
- Evaluate the federal Cell and Gene Therapies (CGT) Access model and determine whether participation may improve access benefits and cost reductions.
- Exploring all discounting and rebating strategies to help mitigate any financial burdens that may come with coverage for these drugs and therapies.
Recommendations for HFS on Ensuring Equitable Access to CGT and Other High-Cost Therapies include:
- Consider stratifying managed care customers with Sickle cell disease and other rare disease as high-risk early in diagnosis to enable timely access to specialized care coordination.
- Development of disease-specific performance metrics for managed care organizations (MCOs) to monitor and enhance the quality-of-care coordination and ensure timely access to high quality care and support for those impacted by these diseases.
- Explore the use of financial and nonfinancial incentives and supports to encourage specialist availability and patient access for rural populations.
Additional Considerations that Can Impact Financing of and Access to CGTs include:
- Strive to promote improved access to care at the provider-level for patients with rare diseases seeking complex therapies by supporting the expansion of complex care coordination and health IT infrastructure.
- Consider evaluating available financial risk mitigation strategies, such as reinsurance, annuity models, and multi-payer risk pools, and monitor the implementation of financial risk mitigation strategies in other state Medicaid programs.
- Consider expanding HFS staff to include clinical and pharmaceutical experts and seek consulting support to implement the above recommendations, including to support enhanced tracking, reporting, and negotiations for CGTs and other high-cost drugs.
Illinois also submitted a letter of intent to participate in a new Cell and Gene Therapy (CGT) Access Model that the federal Centers for Medicare & Medicaid Services (CMS) Innovation Center is launching. The initial phase of the federal CGT Access Model will focus on sickle cell disease. Federal CMS completed its negotiations of outcome-based agreements with both existing manufacturers of sickle cell disease CGTs in November. The agreements include details on up-front supplemental drug rebates and outcomes-based metrics for any state that chooses to participate.
