Dear Honorable Members of the Banking, Commerce and Insurance Committee:
On behalf of the people living with cystic fibrosis in Nebraska, the Cystic Fibrosis Foundation writes to provide comments on LB 833. We appreciate the need to improve affordability of care for Nebraskans 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 Nebraska and reducing drug costs for consumers. Policies that cap reimbursement for drugs may not ultimately impact what Nebraskans 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 LB 833 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 nearly 300 people in Nebraska, 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 one in Nebraska.
Stakeholder Engagement
All PDABs should have robust processes for stakeholders to meaningfully engage and provide feedback on the Board’s actions and procedures. The Foundation appreciates that LB 833 includes a stakeholder process through which people living with a disease, their caregivers, and the clinical providers who care for them can provide input on the PDAB’s selection and review of prescription drugs. We recommend the following additions to the stakeholder engagement process to ensure that it is as accessible and transparent as possible and that it centers stakeholder voices throughout the process.
First, we ask that LB 833 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 LB 833 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
LB 833 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.
LB 833 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.
Board Composition
PDABs should be statutorily required to include diverse membership, including members with expertise on the systemic impacts of market interventions recommended by the Board. For example, a health economist would help the Board understand and consider the potential implications of setting a UPL on the health care system overall, as well as on coverage and access for people who take the prescription drug. We appreciate that LB 833 requires members to have “an advanced degree and experience or expertise in health care economics or clinical medicine.” However, we request that the committee amend this language to specify that all three of these perspectives must be represented on the PDAB.
Drug Selection and Review Criteria
Length of time on market
LB 833 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, LB 833 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, LB 833 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.
Thank you for the opportunity to comment on LB 833. 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 Nebraskans.