Dear Representative Syed:
On behalf of people living with cystic fibrosis in Illinois, the Cystic Fibrosis Foundation writes to provide comments on HB 4472. We appreciate the need to improve affordability of care for Illinoisans and address rising costs to ensure sustainability of the state’s health care system. However, the paramount goal must be preserving access to care and therapies for people living with a disease, and we caution that Prescription Drug Affordability Boards may be working towards two separate aims that require separate consideration and policy solutions: reducing drug costs for the state of Illinois and reducing drug costs for consumers. Policies that cap reimbursement for drugs may not ultimately impact what Illinoisans pay at the pharmacy counter and it is important that the state recognize this distinction as it proceeds with this legislation.
We also recommend additional provisions to HB 4472 outlined below to ensure the Health Care Availability and Access Board puts the needs of people living with a disease, including CF, at the center of the discussion when selecting drugs for affordability review, conducting affordability reviews, or considering upper payment limits (UPLs).
About Cystic Fibrosis & the Cystic Fibrosis Foundation
Cystic fibrosis is a life-shortening genetic disease that affects nearly 40,000 children and adults in the United States, including nearly 850 in Illinois. CF causes the body to produce thick, sticky mucus that clogs the lungs and digestive system, which can lead to lung damage, life-threatening infections, malnutrition, and other complications. Cystic fibrosis is both serious and progressive; lung damage caused by infection is often irreversible and can have a lasting impact on length and quality of life, resulting in extended hospitalizations, transplant, or premature death. As a complex, multi-system condition, CF requires targeted, specialized treatment and medications. There is no cure.
As the world’s leader in the search for a cure for CF and an organization dedicated to ensuring access to high-quality, specialized CF care, the Cystic Fibrosis Foundation supports the development of CF clinical practice guidelines and accredits more than 280 care programs nationally — including 17 in Illinois.
Upper Payment Limits
HB 4472 requires the Health Care Availability and Access Board to set an upper payment limit if the Board determines that the drug has led to or will lead to an affordability challenge. However, we ask that HB 4472 be amended to give the Board the option to determine whether setting an upper payment limit is appropriate or whether another course of action is more prudent. For example, the law that established Colorado’s Prescription Drug Affordability Board gives them the option to either set an UPL, make policy recommendations to the legislature to improve prescription drug affordability, or take no further action. An UPL may not always be the best solution to lower costs to consumers, depending on specific drug and market considerations. HB 4472 should provide the Board with the freedom to consider all economic and political levers to determine what would be most effective and appropriate to lower prices for consumers.
Stakeholder Engagement
All boards conducting affordability reviews on prescription drugs must have processes for stakeholders, especially people living with a disease who may be impacted by potential board action, to meaningfully engage and inform processes and decision-making. We urge you to amend HB 4472 to include a robust stakeholder engagement process through which people living with a disease, their caregivers, and the clinical providers who care for them can provide input on drug affordability reviews and proposals to set upper payment limits.
First, we ask that HB 4472 direct the Health Care Availability and Access Board to provide diverse opportunities for stakeholder involvement during the drug selection and review process and upper payment limit determinations to address time and technology limitations for some people. For example, we encourage the Board to provide multiple opportunities for involvement at a variety of times to accommodate adults living with a disease and adult caregivers that are working and unable to join a meeting during business hours. Avenues for public engagement can include online surveys, written comments, oral testimony, and focus groups. It is also crucial that people living with a disease and caregivers be involved in the development of survey and focus group questions. All of these processes should be conducted not only during drug selection and affordability reviews, but also if the Health Care Availability and Access Board begins any determinations of upper payment limits.
We also request that HB 4472 require the Health Care Availability and Access Board to consider input from clinicians when conducting affordability reviews. Clinicians who specialize in the treatment of certain diseases and conditions bring a critical perspective about the benefit of therapies and availability of alternative treatments, and the Health Care Availability and Access Board should be required to solicit their input. The Health Care Availability and Access Board should meet with clinicians at least once during the affordability review process to discuss the drug under review and their experience caring for people living with the condition treated by the drug.
Transparency of the Process
HB 4472 should require the Health Care Availability and Access Board to be transparent about their processes, methods, and utilization of value assessments. For example, any eligible drug list or dashboard developed by the Health Care Availability and Access Board—with the exception of proprietary information — should be visible to the public, and the Health Care Availability and Access Board should only have discussions about affordability reviews during public meetings. The Foundation also emphasizes the importance of explaining the process in a lay-friendly manner to ensure the public can understand the process and authentically engage with the Board.
HB 4472 should also require the Health Care Availability and Access Board to educate people living with a disease, providers, and other members of the public about its process and timeline for selecting drugs for affordability review, how the review will be conducted, and any potential outcomes from the review. The Health Care Availability and Access Board should be transparent to the public about how data and information collected, especially from people living with a disease, will be used in the decision-making process and with whom it will be shared as well.
Board and Stakeholder Council Composition
We appreciate that the Health Care Availability and Access Board is statutorily required to include diverse membership, including members with expertise on clinical practices as well as the systemic impacts of market interventions recommended by the Board. We request that the legislature also require that the Health Care Availability and Access Stakeholder Council include a variety of backgrounds. Specifically, HB 4472 includes a list of six areas of knowledge and requires each member of the Stakeholder Council to have knowledge in at least one of these areas. However, there is no requirement that each of these areas of expertise be represented on the Stakeholder Council. This language should be amended to specify that all six of these perspectives must be represented on the Health Care Availability and Access Stakeholder Council.
Drug Selection and Review Criteria
It is important that legislation establishing affordability review boards include criteria that the Board must consider when reviewing drugs and setting UPLs. Of the five states that have enacted laws to establish Prescription Drug Affordability Boards with the authority to set UPLs, all five include a set of criteria that the Board is statutorily required to consider when reviewing the affordability of a drug. We request that HB 4472 include review considerations and have provided examples of some criteria that are important for a Board to consider during any affordability reviews.
Orphan drug status
The CF Foundation urges the Illinois General Assembly to include orphan drug status as a criterion when the Health Care Availability and Access Board selects drugs for affordability review, conducts affordability analyses, and determines whether to set an upper payment limit. The small number of people in rare disease populations can create unique challenges for drug development and present different market considerations compared to other therapies. The Board should consider orphan drug status alongside other existing factors already outlined in HB 4472 to ensure a more comprehensive view of the treatment and access landscape for people living with a rare disease.
Availability of therapeutic alternatives
We urge you to include the availability of therapeutic alternatives as a criterion the Health Care Availability and Access Board must consider when selecting drugs for affordability review, conducting the affordability analysis, and determining whether to set an upper payment limit as well. In CF care, treatments are finite and therapeutic alternatives are often not available. For example, a class of drugs called CFTR modulators only works for individuals with certain genetic profiles; they are not interchangeable and there are currently no generics or therapeutic alternatives. The Health Care Availability and Access Board must consider the availability of therapeutic alternatives as there are unique access concerns for drugs without alternatives.
Length of time on market
HB 4472 should establish a minimum period of time that drugs must be on the market before they are eligible for review. While data from clinical trials is important for establishing safety and efficacy, it can take years to fully understand the benefits of a given drug. For instance, collection of real-world evidence is vital to understand how a drug impacts people living with a disease in a real-life setting. Such data also allows researchers to capture information on additional outcomes beyond those evaluated in a clinical trial, such as patient-reported outcomes related to quality of life, productivity, and well-being. For diseases with complex care regimens such as cystic fibrosis, it is also important to also give adequate time to study the impact of a new therapy on other aspects of care. These studies require ample time to assess changing existing care in response to new treatments. Collection of real-world evidence takes time as well and may not be available until a drug has been on the market for a number of years.
Moreover, in cystic fibrosis, the Food and Drug Administration initially approved CFTR modulators for people with certain genotypes ages 12 and up. As sponsors collect additional data, the labels have been expanded to include additional genotypes and younger age groups. As a progressive disease, understanding the impacts of CFTR modulators on younger populations is essential for a comprehensive affordability review as these treatments may delay or halt disease progression, thus impacting healthcare utilization, productivity, and the overall trajectory of cystic fibrosis. As such, HB 4472 should establish a minimum time that drugs must be on the market before they are eligible for an affordability review.
Lived experiences of people living with a disease
Cost-effectiveness methodologies cannot accurately measure value if they do not include data on the experiences, preferences, and outcomes reported by people living with a disease. To that end, HB 4472 should require that the Health Care Availability and Access Board seek out patient-reported data for affordability reviews and UPL determinations, including patient surveys, focus groups, presentations from patient-focused drug development meetings, and registry data. This is essential to complement data from clinical trials, claims data, and other sources and give a full picture of how a therapy works for people living with a disease.
Thank you for the opportunity to comment on HB 4472. The Cystic Fibrosis Foundation stands ready to serve as a resource as the legislature explores solutions to improve access to and affordability of care for Illinoisans.