How Many Billions Is Enough? Prioritizing Profits Over Patients With Cystic Fibrosis

To the editor:

CFTR modulators have drastically improved outcomes for many people with cystic fibrosis (CF), including improved lung function and increased life expectancy by targeting the defective CFTR protein^1,2. As a result, many more people with CF are now fulfilling key life milestones, including pursuing education, careers, and parenthood³. We applaud the research and development efforts of Vertex Pharmaceuticals, the sole manufacturer of CFTR modulators. Vertex has been heralded as a shining star of pharmaceutical companies for personalized medicine and rare disease and has a stated commitment to the CF community. However, we are appalled at how Vertex is achieving financial success on the backs of vulnerable populations.

Only a small percentage of people with CF worldwide have access to these disease-altering CFTR modulators due to Vertex not allowing the development of generic forms or lowering pricing for lower-income or middle-income countries (LMICs), as done by other pharmaceutical companies for HIV, Hepatitis C, and Covid-19 medications⁴. Due to Vertex’s refusal to assist LMICs and Vertex holding the patent for elexacaftor/tezacaftor/ivacaftor until 2037, LMICs will not have generic ETI available for 14 more years. An entire generation of people with CF in LMICs may therefore miss out on the profound benefit of ETI. In many of their corporate actions, Vertex’s sole focus is greed and profit without regard for how their policies impact the lives of people with CF.

In the United States, recent decisions by Vertex are also endangering access to CFTR modulators among people who already have been receiving treatment. In October 2022, Vertex announced a plan to drastically decrease its copay assistance coupon program for CFTR modulators. Copay assistance allows many people to afford CFTR modulators, particularly those with high-deductible health insurance, a growing segment of our population. Until now, Vertex’s program has provided families $8950 per fill, up to 17 fills annually, which allows Vertex to make about $14,000 per fill in profit from billing the medical insurance company for CFTR modulators patients couldn’t otherwise afford. Vertex is reducing the copay assistance program to only a maximum of $20,000 per year with only $3,500 per fill, far below what many pharmacy plans charge as copayment. For families with health insurance plans that have adopted copay accumulators, $20,000 would only allow for about five medication refills per year, shifting the remaining exorbitant copays to the patient. Health insurance plans with copay accumulators take the maximum value of manufacturer copay assistance as quickly as possible without applying that amount to the deductible or out-of-pocket maximum. The patient is then responsible for paying the entire deductible and the remaining cost of the fills.

CFTR modulators are some of the most expensive drugs on the market, priced by Vertex at over $300,000 a year or over $25,000 a month. This has resulted in Vertex reporting $8,900,000,000 in profit in the past year⁵. This cost is unjustifiably high. CFTR modulators are overpriced compared to quality life years gained⁶ and the estimated cost of producing is 90% less than the list price⁷. The fact that Vertex is drastically decreasing its copay assistance program while earning these jaw-dropping profits truly exposes its corporate greed.

As CF clinicians, we are watching as the livelihood of people with CF is increasingly caught in a battle for record-breaking profits between Vertex and many health insurance companies. Vertex claims that their policy changes are a direct result of actions by insurance providers. Many insurance companies have implemented copayment accumulator programs. Such health plans will not allow manufacturer copay assistance coupons to apply to annual deductibles or out-of-pocket maximum. This benefits insurers by increasing profits while offsetting costs to patients. Copayment accumulators were meant to incentivize lower-cost generic medications. However, no generic forms of CFTR modulators are available due to Vertex policies.

People cannot pay thousands of dollars monthly, even for life-altering medications; the choice is paying for medication or paying for food and housing. People with CF already have high out-of-pocket expenses annually (median $8,244), which is about a quarter of the median per capita income in the United States⁸. There is no guaranteed coverage if people cannot afford this astronomical out-of-pocket cost. Changing the copay assistance program is akin to denying access to CFTR modulators and will lead to the deterioration of health in people with CF.

We, as healthcare providers for people with CF, will not stand by as corporate greed puts people with CF at risk of losing access to CFTR modulators. We call on Vertex to increase the copay assistance amount to ensure patients’ access to CFTR medications for an entire year ($3500/fill, 12 fills yearly). We call on insurance companies to end copay accumulator programs. We call on Congress to pass The Help Ensure Lower Patient (HELP) Copays Act (HR 5801) to ban copay accumulators. We call on Vertex to reduce the price of CFTR modulators to reflect the value given in the 2020 Institute for Clinical and Economic Review (ICER) report.

Vertex does not have to drastically decrease copayment assistance, yet they choose to. Insurance companies do not have to include copayment accumulators, yet they choose to. Vertex does not have to charge over $300,000 annually for its drugs, yet they choose to. Vertex and insurance companies choose to put their record-breaking billions of dollars in profits over the lives of people with CF, and they must be held to account for policies that threaten to bankrupt the very people they claim to want to help live longer lives with CF.

Conclusion:

CFTR modulators have transformed CF but are some of the most expensive medications on the market and are over-priced compared to the value given. The high price of CFTR modulators leaves people with CF dependent on Vertex’s copay assistance programs. Despite record-breaking profits, Vertex is drastically decreasing its copayment assistance programs, so people with CF will have to pay thousands of dollars monthly for CFTR modulators or discontinue therapy. Vertex continues to put profits above the lives of people with CF.

Copayment Assistance Programs’ Cost and Profits

	Copay Assistance Programs	Maximum Annual Assistance	Vertex Profit Minimum per Patient Annually (List price ~$300,000)
Vertex’s Former Copay Assistance Program	$8,950 x 17 fills	$152,150	~ $147,850
Vertex’s New Copay Assistance Program	$3,500/fill x 5 fills $20,000 maximum	$20,000	~ $280,000 if 12 fills/year ~ $105,000 if 5 fills/year
Our Suggested Copay Assistance Program	$3,500/fill x 13 fills	$45,500	~ $254,500

-The list price for elexacaftor/texacaftor/ivacaftor (ETI) is over $300,000 annually in the United States. The estimated profit for both Vertex’s new program assumes patients can afford to pay all out-of-pocket expenses, which is not realistic for many patients. If patients only can afford the 5 fills per year before discontinuing ETI with Vertex’s new copay assistance program, Vertex’s profit would decrease to only $105,000 per patient annually