Dear Senator Cassidy:
On behalf of the nearly 40,000 children and adults living with cystic fibrosis, we write in response to Ranking Member Cassidy’s request for information on improving access to gene therapies. While people with cystic fibrosis do not currently have any gene therapies available to them, there are several gene therapies in the cystic fibrosis treatment pipeline, and we are grateful for the opportunity to help shape potential coverage and reimbursement policies. The CF community has experience with coverage and access for high-cost therapies known as CFTR modulators, which address the underlying cause of the disease; this experience may be instructive for anticipating potential coverage challenges with gene therapies. Recent experience has shown early signs that the cost of some prescription drugs is not sustainable, and we are seeing growing resistance from insurers to cover some specialty drugs — and people with CF are too often caught in the middle of this conflict between payers and manufacturers. Coverage of cell and gene therapies will require new, innovative models to address these dynamics and other related challenges in order to ensure that those who can benefit from therapies have access.
About cystic fibrosis & the Cystic Fibrosis Foundation
CF is a life-shortening genetic disease that causes the body to produce thick, sticky mucus that clogs the lungs and digestive system. This can lead to lung damage, life-threatening infections, malnutrition, and other complications. CF 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 invests in research and development of gene therapies for people with CF. Gene therapies for cystic fibrosis
Gene therapies for cystic fibrosis
There are currently multiple gene therapies in the CF treatment pipeline. Should any of these therapies become FDA-approved, we anticipate there will be significant benefits for eligible populations; however, we do not expect that the initial gene therapy treatments will be completely curative. First generation gene therapies for CF may not last the duration of a person’s life and some of the therapies may need to be re-dosed. Further, these therapies target the lungs and will not benefit other organs that are impacted by CF. People with CF will likely need to continue other aspects of their current treatment regimen to manage CF manifestations outside the lungs. Throughout this comment letter we highlight the challenges that people with CF are facing today — without available gene therapies — due to cost burden and insurance barriers to care. We are concerned that some of these trends may be exacerbated as even more expensive treatments come to market. It is imperative that payers and manufacturers come together to develop innovative payment models that incorporate the needs of people with complex multi-system diseases such as CF.
Cost burden and cystic fibrosis
CF has a high treatment burden and people with CF report high out-of-pocket costs for the care they require. Below we describe data on cost burden for people with CF, which illustrates ongoing barriers and challenges within the current coverage landscape. The experiences of people with CF in affording their necessary care provides insights on how gene therapies could impact individual cost and how payers may respond to new high-cost therapies.
Healthcare costs for people with CF
The vast majority (93%) of people with CF have health insurance for the entire year; however, having insurance does not protect many people with CF from experiencing significant cost burden: 74% of people with CF use at least one additional form of financial assistance – including nonprofit grants and manufacturer assistance programs – to afford their care. The financial resources people use depend in part on their unique situation as availability of some programs — such as state assistance programs — vary by location and assistance programs have different requirements based on income level and type of insurance coverage. For a significant majority of people with CF, the ability to afford the specialized care they need requires multiple types of insurance and/or assistance programs. The complexity of navigating these programs are burdensome and too often leave the person with CF shouldering significant financial burden. As cell and gene therapy payment models are developed, every effort should be made to simplify the process and minimize the financial and administrative burden for the patient.
Even with financial assistance, 44% of people with CF reported problems paying for at least one aspect of their CF care. These financial challenges can impact care-seeking behavior, with one out of every four people with CF reporting skipping medication doses, taking less medication than prescribed, or delaying filling a prescription due to cost.
Financial burden may not be consistently spread throughout the year: often there is a high-cost burden in the beginning of a new plan year with 53% of people with CF meeting their deductible in the first half of the year. These challenges affording care are not limited to those who are lower income. Financial assistance programs may not always be available to those above a certain income threshold, thus limiting their ability to alleviate cost burden caused by CF: 34% of people whose households earned between $120,000-$150,000 reported having financial problems caused by paying for their CF care. Living with a high-cost disease such as CF can put financial strain on families that is pervasive across income levels, and one should not assume financial need is limited to those with a low socioeconomic status.
Further, there is not one single therapy or aspect of care that is the cause of the financial burden in CF; it is caused by the totality of the healthcare requirements as well as their indirect costs such as transportation, time off work, caregiver burden, and many others. When considering the benefits and potential coverage models for a new therapy, such as a cell or gene therapy, it is important to recognize:
- The potential role of that therapy in reducing this healthcare burden overall, including but not limited to a reduction in other standard of care treatments and reductions in healthcare utilization such as hospitalizations or care visits.
- That even with reductions in other aspects of care, people with CF are subject to a costly and burdensome disease and today rely on a myriad of resources to afford their care. The ability for people with CF to bear the burden of a high-cost cell or gene therapy depends on many factors including the coverage model for their care and the financial resources available to them.
Unaffordable costs and other financial challenges that force people to skip or delay needed care fail to ensure optimal outcomes. As payment models for genetic therapies are designed, they must include all components of the required treatments and support services necessary to ensure the best outcomes.
Indirect costs due to CF and social risk factors
Living with CF can impact a wide range of economic outcomes beyond just direct medical costs. For example, 46% of people with CF who report working less than full-time identified as doing so due to health reasons. Further, 59% of adults with CF or caregivers of people with CF state that the availability or quality of health insurance has impacted their decision about whether to take a job, change jobs, or accept a raise. Educational and employment attainment are just two of the long-term outcomes that can be impacted by new therapies at both an individual and societal level. Value assessments for cell and gene therapies must consider the variety of impacted economic outcomes when evaluating the potential benefit of a treatment.
Further, some gene therapies may be administered in a way that requires more intensive monitoring and these therapies should not only be limited to those with extensive financial and social resources. In CF today, there are disparities for people with CF with advance lung disease, those who live farther from a transplant center, are low socioeconomic status, and those without a social support network are less likely to be referred for and receive a transplant. Lack of transplant access disproportionately impacts people of color and exacerbates existing health disparities. Gene therapy coverage and payment models must not exclude those experiencing social risk factors and financial burden and instead should incorporate supports into the model itself to ensure equitable access to these treatments for all those who can benefit.
Insurance and access for people with cystic fibrosis
Ninety percent of people with CF are eligible for treatments known as CFTR modulators, which address the underlying cause of the disease and have provided unprecedented clinical benefits for people who are eligible to take them. However, due to the cost of these therapies, people with CF have faced increasing challenges with their insurance coverage, often resulting in significant administrative and financial burdens and sometimes delays in accessing their medications. Below we identify several barriers to access currently experienced by those with CF and we anticipate these trends will continue to escalate as more expensive therapies come to market.
Utilization management
The CF Foundation recognizes the role that utilization management can play in lowering health care costs; however, people with CF are often subject to lengthy and inappropriate utilization management requirements, resulting in significant time and financial burden. In CF care, treatments are finite and therapeutic alternatives are often not available. For example, CFTR modulators only works for individuals with certain genetic profiles; they are not interchangeable and there are currently no generics or therapeutic alternatives. As such, utilization management such as step therapy is often not clinically appropriate and may require people who are stable on an established medication to experience a health decline before being able to continue accessing their treatments.
Further, these utilization management techniques vary across plans and can change year-to-year within the same plan. People with CF are frequently subject to recurring and ever-changing prior and re-authorizations requirements; repeatedly doing this for multiple chronic medications creates significant burden that requires high health insurance literacy and time to devote to working through any inappropriate coverage denials to avoid a gap in treatment access. As discussed below, CF care teams routinely support people with CF in navigating the insurance approval process, but this does not entirely shield the patient or caregiver. Anecdotally, members of the CF community have shared that the time they spend navigating insurance is akin to “a part time job.”
Some insurers are particularly problematic with their coverage criteria for high-cost medications — such as CFTR modulators — and implement clinically inappropriate coverage criterion related to restrict access and minimize their costs. For example, we have seen payers restrict access to CFTR modulators for those with a lung function within a certain range. This criterion requires those who are healthy and could have their lung function stabilized by modulators to experience a health decline and excludes those with advanced lung disease, of which there is ample evidence supporting the clinical benefit of modulator initiation.
We recognize that payers put these utilization management policies in place in response to rising costs. Nonetheless, in CF, the ultimate result is often added administrative barriers that delay access to evidence-based care for a genetic disease. When considering coverage models for gene therapies, there must be a balance between policies to manage overall health care costs and actions that results in inappropriate delays or restrictions to treatment.
Coverage and cost
People with CF are facing growing cost burdens related to their treatment, demonstrating payers growing resistance to covering specialty drugs and that coverage of a therapy does not necessarily guarantee access. When a person is ultimately able to navigate their health plan’s utilization management requirements and gain approval of their necessary medications, they are routinely subject to high cost-sharing. Brand-name specialty drugs are often placed at the highest tier of benefit design, thus putting financial strain on people with CF, who require multiple specialty drugs and have no lower cost alternatives available.
In addition to the direct financial harm, patients going without medication can be detrimental to their overall health. Studies indicate gaps in CF medication adherence are associated with higher respiratory exacerbation, increased hospitalizations, longer hospital stays, increased number of pulmonary exacerbations requiring intravenous antibiotics, and lower baseline lung function. These secondary and tertiary effects cost patients and the health care system thousands of dollars, as well as cause irreparable harm to a patient’s health.
In addition, health plans are increasingly implementing accumulator programs — which prevent third-party payments from counting towards deductibles and out-of-pocket limits and therefore increasing out-of-pocket costs for patients. Many people with CF rely on third-party financial assistance to cover some of the costs associated with their care, as CF is an expensive disease. The CF Foundation recognizes that copay assistance programs mask bigger cost and affordability issues; however, cost containment strategies like accumulator programs that further burden people with CF are unacceptable.
Coverage models for gene therapies should utilize analysis of effective tools to foster and support utilization and adherence to evidence-based care regimens and exclude cost-sharing programs that burden people who can benefit from these therapies and can result in skipping or delaying vital aspects of the treatment protocol and supportive care.
Supply chain intermediaries
Another example of payers growing resistance to covering high-cost therapies is the proliferation of supply chain intermediaries involved in insurance coverage for people with CF, creating confusion and administrative barriers when people try to access treatment. There is a lack of transparency on the role of PBMs, insurers, and subcontracted third-party entities in coverage and cost-sharing decisions, especially in the self-funded insurance market. This causes confusion on the appropriate point of contact for appealing coverage decisions, increasing administrative burden on both patients and their care teams, and causing gaps in access to important therapies. New coverage tactics emerge frequently, requiring patients and care teams to consistently learn and adapt to new, opaque, and confusing policies.
Third-party entities such as maximizers — many of which are owned by PBMs — and alternative funding programs add complexity to an already opaque system. These entities exist to lower costs to the payer. Maximizers often outsource a patient’s drug coverage to a third-party entity that sets the patients’ cost-sharing at a level to maximize use of manufacturer copay assistance. Alternative funding programs (AFPs) also rely on third-party entities that seek to enroll patients in manufacturer patient assistance programs that provide free drugs, which are usually intended for people without insurance. AFPs cause consumers to experience financial losses and lose access to critical medications while they navigate these programs. When a patient is forced to enroll in a third-party program, any financial assistance the patient receives will not be counted towards meeting their deductible or out-of-pocket limit, increasing the cost burden for the patient. These programs illustrate the current coverage landscape for patients taking high-cost therapies and challenges that may worsen as more costly treatments come to market.
Role of CF care teams
Given the individualized nature of cystic fibrosis, CF clinicians, in consultation with their patients, are best positioned to determine which treatments are best for each individual and the right time to initiate those treatments; this remains true for any future FDA-approved gene therapies. It is imperative that a therapy is FDA-approved and prescribed through shared-decision-making, people with CF can access it and that there is no delay due to ongoing coverage or payment model negotiations.
Further, access and insurance challenges are not solely the burden of the person living with CF or their caregiver. CF care teams are often involved in assisting their patients with obtaining access to therapies and are regularly intermediaries in communicating with insurers, PBMs, other third-parties, and manufacturers. Some CF care teams report employing part-time personnel in the first quarter of the year solely to assist in the prior authorization process and to navigate the lack of transparency caused by each party in the system. In 2023, care team pharmacists reported spending an estimated one-hour per each individual CFTR modulator access issue.
Role of the government in gene therapy coverage and reimbursement
Given the increasing pace of approvals for gene therapies, we believe there is an urgent need for government attention to coverage and reimbursement for these treatments. The high upfront costs, limitations of clinical trial data, and patient churn across insurance markets all present significant challenges for payers when covering these therapies, which create concerns about patient access. The federal government is uniquely positioned to address some of these issues, particularly around the financial concerns and patient churn.
Some markets are particularly concerning — including self-funded plans and Medicaid programs — as these payers face even greater challenges paying for these therapies and the federal government should pay particular attention to those markets. In addition to Ranking Member Cassidy’s focus on these issues, we appreciate the Center for Medicare and Medicaid Innovation’s Cell and Gene Therapy Access Model and see great potential in supporting states as they grapple with the cost of these therapies and administrative challenges of alternative payment models. We think there is great value in exploring alternative payment arrangements for these therapies, such as a subscription model which has been used to provide access to gene therapies in other countries. The government should assess a range of financing arrangements as different markets and different therapies may lend themselves to different payment models.
Essential Health Benefits (EHBs)
Regarding EHBs, we have already seen how this standard is falling short for people with CF and need to be updated to reflect the pace of improvements in CF treatments. Under current law, the prescription drug benefit requirements are not clear. According to a recent Kaiser Family Foundation study, 65% of covered workers are in a plan that is self-funded and as fully insured risk pools, they are incentivized to adjust their benefits when even one enrollee has a high-cost condition. Many offer a prescription drug benefit, which is subject to Affordable Care Act’s (ACA) regulations of EHBs. Employer health plans and ACA Marketplace plans have begun circumventing prescription drug benefit requirements alleging they are only required to cover the minimum number of drugs in the state plan, and everything in addition to the minimum number of medications in the state plan is considered “non-essential.” Issuers claim non-essential drugs are still considered covered by the plan, but they are not subject to the ACA EHB requirements and therefore can be removed from the ACA’s out-of-pocket maximums requirement. This practice places a significant administrative and cost burden on consumers who use high-cost, specialty prescription drugs, like people with CF.
In addition, the current EHB standards governing prescription drugs require plans to cover the greater of one drug per U.S. Pharmacopeia (USP) Medicare Model Guidelines (MMG) class and category or the number of such drugs included in the state’s benchmark plan. This standard has not been updated since the EHB rules came into effect in 2014 and has proven to be inadequate for people with CF and the scientific advancements in CF care over the last 10 years. The Department of Health and Human Services has proposed transitioning from the USP MMG to the USP Drug Classification (DC) system. For purposes of the EHB framework and the consumers it is intended to serve, the USP DC is superior to USP MMG; the DC system includes additional drug classes needed by the broader patient population served by EHB and is updated more frequently. However, there are still improvements that could be made to the USP DC system to better reflect patients and their needs.
For instance, the USP DC classification system includes all therapies specifically indicated for CF in one category and class, even though there are different types of CF medications and formularies need to reflect those differences in order for people with CF to receive appropriate treatment. One type of therapy available for 90 percent of people with CF is CFTR modulators, which directly target defects in the processing, trafficking, and function of the CFTR protein. These therapies are distinct from other CF treatments which treat the symptoms of CF by addressing mucus clearance and bacterial infections, for example. By including all CF treatments in the same category and class, the USP DC system is not reflective of the complexities of therapies currently available on the market and in the CF development pipeline.
We recommend adoption of an annual process, specific to EHB regulation, that reviews the DC system to ensure it remains updated for patients’ evolving needs and to keep pace with development of new therapies. Such a review should include feedback from patients, practitioners, and evaluation of disease specific clinical guidelines.
Defining ultra-rare diseases
When evaluating whether a disease or disorder is “ultra-rare” it is important to consider variations within a disease. Cystic fibrosis is broken into subpopulations based on CFTR variant or mutational class. These distinctions determine eligibility for and effectiveness of small-molecule therapeutics, such as the currently available CFTR modulators. Therefore, while CF as a whole may impact nearly 40,000 people in the United States today, that population is broken into subpopulations that have varying eligibility for existing therapies. Lawmakers should consider the eligibility population within a certain disease when evaluating for ultra-rare disease status.
Thank you for the opportunity to provide information on the experiences of people with CF as they navigate coverage and access. The experience of this community is instructive as policymakers consider future challenges with coverage and reimbursement of cell and gene therapy, which will require new, innovative payment models to ensure access for those who need it. The Cystic Fibrosis Foundation stands ready to serve as a resource as the Ranking Member explores solutions to improve access to and affordability of gene therapies.