Dear Chair Cavanagh and Members of the Committee on Finance, Insurance, and Consumer Protection:
On behalf of the people living with cystic fibrosis in Michigan, the Cystic Fibrosis Foundation writes to provide comments on SB 3. We appreciate the need to improve affordability of care for Michiganders 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 (PDAB) may be working towards two separate aims that require separate consideration and policy solutions: reducing drug costs for the state of Michigan and reducing drug costs for consumers. Policies that cap reimbursement for drugs may not ultimately impact what Michiganders 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 SB 3 as outlined below to ensure the PDAB 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 (UPL).
About Cystic Fibrosis & the Cystic Fibrosis Foundation
Cystic fibrosis is a progressive, genetic disease that affects the lungs, pancreas, and other organs. There are close to 40,000 children and adults living with cystic fibrosis in the United States, including more than 1,200 people in Michigan, and CF can affect people of every racial and ethnic group. 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 eight in Michigan.
Goals of the PDAB
We caution that SB 3 may be working towards two separate aims that require separate consideration and policy solutions: reducing drug costs for Michigan’s health care system and reducing drug costs for consumers. Policies that cap reimbursement for drugs may not ultimately impact what consumers pay at the pharmacy counter and it is important to recognize this distinction as lawmakers proceed with this legislation. Any ambiguity about whether the PDAB is reviewing excess costs for health care systems or for consumers can create confusion about how the Board should review drugs and appropriate policy remedies.
Due to the complexity of the U.S. health care system, there are many factors and entities involved in determining what patients pay for their drugs. For instance, while people with CF rely on expensive specialty drugs, their out-of-pocket costs for these medications are often more affordable because of manufacturer or non-profit copay assistance programs. Navigating the intricacies of health plans and assistance programs can be burdensome and time consuming but often means that people may be able to afford the cost-sharing for their most expensive therapies. Far too many people with CF still struggle to afford all of their care which includes an extensive treatment and care regimen — but their affordability challenges are not always driven by the cost of one specialty drug. We recognize that copay assistance programs can mask bigger cost and affordability issues; however, we share this information to highlight that affordability challenges for the system do not always align with affordability challenges for consumers. We ask that lawmakers clarify the definition of affordability challenge and the circumstances in which upper payment limits should be considered with these nuances in mind.
Stakeholder Engagement
All PDABs must have processes for stakeholders, especially people living with a disease who may be impacted by potential PDAB action, to meaningfully engage and inform processes and decision-making. We urge you to amend SB 3 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 SB 3 direct the PDAB to provide diverse opportunities for stakeholder involvement during drug selection, the 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 PDAB begins any determinations of upper payment limits.
We also request that SB 3 require the PDAB 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 PDABs should be required to solicit their input. The PDAB 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
SB 3 should require the PDAB to be transparent about their processes, methods, and utilization of value assessments. For example, any eligible drug list or dashboard developed by the PDAB — with the exception of proprietary information — should be visible to the public, and the PDAB 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 PDAB.
SB 3 should also require the PDAB 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 PDAB 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.
Drug Selection and Review Criteria
Orphan drug status
The CF Foundation urges the legislature 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 people in rare disease populations can create unique challenges for drug development and present different market considerations compared to other therapies. This limited market size for these kinds of drugs is a factor in determining the price of these therapies, and it is important to preserve financial incentives to bring more drug developers into this space as there are many rare diseases without any approved treatment. For instance, some CF treatments are indicated based on specific genetic variants and therefore even for a disease like CF with many approved treatments, there must still be incentives to continue investing in this space. The PDAB should consider orphan drug status alongside other existing factors already outlined in SB 3 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 PDAB must consider when selecting drugs for affordability review, conducting the affordability analysis, and 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 the availability of therapeutic alternatives as there are unique access concerns for drugs without alternatives.
Moreover, given that the PDAB would only have authority over state-regulated plans, the board would have limited leverage for drugs without therapeutic alternatives. In contrast to Medicare price negotiation — where manufacturers would be hard pressed to walk away from all Medicare beneficiaries — the PDAB is dealing with a relatively small market and a single source manufacturer may be willing to give up the state market rather than accept a UPL. UPLs may hold more promise for drugs with therapeutic equivalents. It is feasible that a state could achieve savings in this scenario while preserving access for patients.
Length of time on market
SB 3 should 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 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, SB 3 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, SB 3 should require that the PDAB 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. Michigan should take steps to ensure that there are no negative effects on health outcomes when implementing UPLs. This should include analysis of relevant service utilization pre- and post-UPL for relevant patient populations to identify any concerning changes.
Thank you for the opportunity to comment on SB 3. 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 Michiganders.