Dear Chair Carter and Honorable Members of the Committee on Insurance and Financial Services:
On behalf of people living with cystic fibrosis in Michigan, the Cystic Fibrosis Foundation writes to provide comments on SB 483: the Prescription Drug Cost and Affordability Review Act. We appreciate the need to improve affordability of care for Michiganders and address the rising cost of prescription drugs to ensure sustainability of the state’s health care system. However, as this committee considers the creation of a prescription drug affordability board (PDAB) and the potential use of upper payment limits, the paramount goal must be preserving access to care for patients. We recommend additional provisions to SB 483, outlined below, to ensure that the PDAB centers the needs of patients, including those living with CF, when selecting drugs for affordability review or considering upper payment limits.
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 over 1,180 in Michigan. 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 130 care centers nationally — including 5 in Michigan.
Stakeholder engagement process
We urge you to amend SB 483 to include a robust stakeholder process through which Michigan patients, their caregivers, and the clinical providers who care for them can provide input on drug affordability reviews and proposals to set upper payment limits. Currently, this bill does not mandate that the PDAB solicit and consider input from patients, caregivers, and medical experts outside of those appointed to the Prescription Drug Affordability Stakeholder Council.
It is essential that patients, caregivers, and clinical providers who are experts in treating the diseases impacted such reviews can participate in this process as early as possible and that their feedback is heard throughout the entire affordability review. We ask that the PDAB provide diverse opportunities for patient involvement and recognize time and technology limitations that community members may see as barriers to engagement. For example, we encourage the Board to provide multiple opportunities for involvement at a variety of times to accommodate adults and 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 stakeholder meetings. Additionally, the PDAB must be transparent to the public about how data and information collected, especially from patients, will be used in the decision-making process and with whom it will be shared.
Affordability review criteria
Orphan drug status
The CF Foundation urges you to include orphan drug status as a criterion when the PDAB selects drugs for affordability review, conducts affordability analyses, and determines whether to set an upper payment limit. The small number of patients in rare disease populations can create unique challenges for drug development and present different market considerations compared to other therapies. The PDAB should consider orphan drug status alongside other existing factors already outlined in SB 483 to ensure a more comprehensive view of the treatment and access landscape for patients.
Availability of therapeutic alternatives
We urge you to include the availability of therapeutic alternatives to a given drug in the list of criteria that the PDAB must consider. SB 483 includes language referencing the Board’s obligation to compare prices, rebates, and other associated costs between drugs and their therapeutic alternatives. However, it does not establish the availability of therapeutic alternatives as a criterion when selecting drugs for affordability review, conducting the affordability analysis, or determining whether to set an upper payment limit. 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 PDAB must consider whether therapeutic alternatives have been approved and are available to a patient population when selecting drugs for affordability review, conducting affordability reviews, or setting an upper payment limit.
Length of time on market
It is important the legislature establish a minimum period of time that drugs must be on the market before they are eligible for PDAB 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 patients 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 U.S. 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. For these additional populations, it will take time to understand the full benefit of CFTR modulators on life-expectancy, as one example. As such, SB 483 should establish a minimum time that drugs must be on the market before they are eligible for an affordability review.
Thank you for the opportunity to comment on SB 483. The Cystic Fibrosis Foundation stands ready to serve as a resource as the legislature explores solutions to improve access to care for Michiganders.