Dear Members of the Colorado Prescription Drug Affordability Board:
On behalf of the people living with cystic fibrosis (CF) in Colorado, the Cystic Fibrosis Foundation writes to provide comments on the Colorado Prescription Drug Affordability Board’s (PDAB) rulemaking to revise Rule 3: Affordability Reviews. As one of the first patient communities to participate in a PDAB affordability review, we value the ongoing dialogue between the CO PDAB, the CF Foundation, and our community, and appreciate the continued opportunities to provide feedback on the PDAB’s processes.
We recognize the need to improve affordability of care for Coloradans and address rising costs to ensure sustainability of the state’s health care system, but the paramount goal must be preserving access to care and therapies for people living with a disease. We recommend additional provisions to Rule 3 as outlined below to help clarify the objectives and methodology for affordability reviews, enhance transparency of PDAB processes, and expand relevant data inputs and criteria for drug selection.
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 over 710 people in Colorado, 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 two in Colorado.
Goals of the PDAB
We appreciate that the Colorado PDAB has further clarified how the Board should consider “unaffordable for Colorado consumers.” However, we caution that the proposed definitions include two separate aims that require separate consideration and policy solutions: reducing drug costs for all Colorado consumers and reducing drug costs for patients who take particular medications. There are important differences between evaluating affordability for patients who take certain medications and affordability for the entire health care system and all consumers. These different aims require different methodologies and data sources and without additional direction about how to assess the different definitions of unaffordability, these reviews run the risk of being highly subjective.
For instance, the PDAB may define “unaffordable for Colorado consumers” to mean “Colorado consumers using the prescription drug” and/or “Colorado consumers whose taxes fund health care costs for public employees, including employees of the state, county, and local governments, school districts, and institutions of higher education, and to public retirees whose health care costs are funded by public programs.” If using the former definition, the Colorado PDAB would need to assess prior authorization requirements, formulary placement and its impact on access, and other data points that they have outlined on their list of items for consideration. If using the latter, these data are not relevant and entirely different items for consideration would be required to conduct a comprehensive affordability evaluation. As such, the methodology and data sources, and potentially the conclusion reached, could vary based on how the population the PDAB is choosing to define “consumer.”
Additionally, we request the inclusion of a robust policy review process that encourages the PDAB to consider policy solutions other than a UPL to address the drivers and market failures causing a drug to be unaffordable. A UPL may not always be the best solution to lower costs for consumers, depending on specific drug and market considerations. For instance, recent analyses of the Medicare drug price negotiation program found that payers may move negotiated drugs to higher formulary tiers, resulting in increased costs for patients. The PDAB should have the freedom to consider all economic and political levers to determine what would be most effective and appropriate to lower prices for consumers, and we would like to see Rule 3 provide this flexibility.
Transparency of the Process
Rule 3 should require the PDAB to be transparent about their processes and methodology, and the Foundation emphasizes the importance of explaining these processes in a lay friendly manner to ensure the public can understand the process and authentically engage with the PDAB. Rule 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. When the Colorado PDAB conducted its affordability review of elexacaftor/tezacaftor/ivacaftor (Trikafta), many patients were confused about the process and afraid of losing access to this lifesaving medication and did not feel the PDAB adequately addressed their concerns during the process. We appreciate that the Colorado PDAB made an effort to educate the public about its processes during its previous reviews, but would like to see this requirement laid out explicitly in Rule 3 to ensure that stakeholders are appropriately educated during the process and their concerns are appropriately addressed.
Drug Selection and Review Criteria
Orphan drug status
The CF Foundation is encouraged to see that, as required by SB 24-203, Rule 3 directs the PDAB to consider orphan drug status during the drug selection process. 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.
While we appreciate that SB 24-203 requires patient input for orphan drugs during the drug selection process, we recommend that Rule 3 expand these provisions to apply to all drugs — not just orphan therapies — and require patient input at every stage of an affordability review including drug selection, affordability review, and determining whether to set a UPL. The PDAB should have robust processes for stakeholders to meaningfully engage and provide feedback on the Board’s actions and procedures at all stages. During the PDAB’s review of Trikafta, we witnessed up-close how important patient input is during affordability reviews, and the process in Colorado showed room for improvement. One lesson learned was that patient feedback is critical throughout the entire review process and patients must have a seat at the table from the start.
Rule 3 should also require the PDAB to consider input from clinicians who specialize in the treatment of certain diseases and conditions when selecting drugs and determining whether to set UPLs. The expertise of clinicians during the Colorado PDAB’s recent review of Trikafta brought a critical perspective about the benefit of therapies and availability of alternative treatments. The PDAB is already required to meet with clinicians during the affordability review to discuss the drug under review and their experience caring for people living with the condition treated by the drug, and we recommend expanding this requirement.
Length of time on market
Rule 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 — which may not be available until a drug has been on the market for several years — 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, which can take time to do safely and comprehensively.
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, Rule 3 should establish a minimum time that drugs must be on the market before they are eligible for an affordability review.
Availability of therapeutic alternatives
The Foundation appreciates the inclusion of patient input on the availability of therapeutic alternatives as a criterion the PDAB must consider when conducting an affordability review. 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. Patient perspectives on therapeutic alternatives are an important perspective as the Board conducts these reviews.
Thank you for the opportunity to comment on the PDAB’s rulemaking to revise Rule 3: Affordability Reviews. 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 Coloradans.