High cost of cardiac amyloidosis drugs may limit access for low-income patients
Before new drug treatments for transthyretin amyloid cardiomyopathy (ATTR-CM) became available in recent years, cardiologists had limited treatment options. As a result, discussions at industry conferences specifically focused on ATTR-CM were few and far between.
However, there has been burgeoning interest in ATTR-CM since those drugs did gain approval. In fact, awareness has increased so much that more patients are being identified today than anyone thought would ever possible.
On the other hand, the high cost of of these drugs—tafamidis, acoramidis and vutrisiran—may keep many of those patients from even receiving treatment. This was the subject of an editor's page article in the Aug. 4 issue of the Journal of the American College of Cardiology (JACC).[1] Editor-in-Chief Harlan M. Krumholz, MD, SM, a cardiologist with Yale School of Medicine, and co-authors Michelle M. Kittleson, MD, PhD, Smidt Heart Institute, Cedars-Sinai Medical Center, and Kushal T. Kadakia, MD, MSC, Massachusetts General Hospital, made the case that pricing policy reforms are needed to ensure equal access to these drugs.
"The high price complicates access and, for some people, precludes it entirely. We want companies to have strong incentives—and good returns—when they produce drugs or devices that have a big impact on people’s lives. But as the Institute for Clinical and Economic Review (ICER) has noted, even for therapies with immense value, prices can far exceed what might be justified by their benefit. The net result is that our health system incurs significant costs when these patients are treated, and we have not yet solved how to ensure broad, equitable access," Krumholz explained to Cardiovascular Business.
The researchers said U.S. list prices of these therapies range from $244,000 (acoramidis) to nearly $500,000 (vutrisiran) per year. They noted that tafamidis, at $268,000 annually, was called the most expensive cardiovascular drug ever approved by the FDA when its pricing was announced. Even with discounts available, the costs are very high.
Additionally, the drugs are to be taken throughout a patient's lifetime. The authors estimated that a 75-year-old patient diagnosed with ATTR-CM treated with these drugs over five to 10 years could exceed $1 million in costs. For younger patients with hereditary ATTR, they said the lifetime cost would be far higher.
In 2024, Medicare reforms capped the annual out-of-pocket costs for patients to $3,300, and dropped it further to $2,000 in 2025. But the authors said even these amounts can be unaffordable for patients on fixed incomes. For the uninsured, they said these costs are insurmountable. There are also growing concerns that the sheer costs of ATTM-CM drugs along will have a massive impact on Medicare spending. In 2021, total Medicare spending on tafamidis exceeded $1.2 billion, the authors said. Krumholz said the numbers of patients being diagnosed with ATTM-CM is rising as awareness continues to grow and there has been a focus on better imaging techniques for the disease.
"Medicare’s exposure will grow as more patients are diagnosed and treated. Sustainability will depend on policy changes, better targeting of therapies to those most likely to benefit, and shifting resources away from low-value care toward high-value innovation," Krumholz explained.
ICER concluded that tafamidis would need to cost 85% to 95% less, between $13,600 and $39,000 per year, to align with its clinical value. And the authors said this price-value disconnect is not unique to the United States or to these particular drugs and adds to the growing global crisis of access to care.
"It’s good news when breakthroughs enable us to identify and treat more patients—we should celebrate that progress. But it also forces us to ask: are we spending in the right places? Our challenge is to find ways to afford these innovations while reducing or eliminating low-value care. We need new payment approaches that match the value provided, especially when the patient population is larger than expected," Krumholz explained.
He said changes are needed to ensure ATTM-CM drugs and other high-cost breakthrough medications can be made available to treat all patients, rather than helping to accelerate the siding gap in health disparities between the "haves" and the "have-nots."
"We cannot accept a system where only those with means can benefit from life-changing drugs. We are a wealthy nation that spends trillions on healthcare. We must ensure that income and wealth do not determine access, and that requires new solutions in financing and coverage," Krumholz said.
If pricing reforms are not made, it is likely that many patients will go without these new drugs until they eventually go generic in the future. Krumholz said he already sees this with patients in his clinic who would clearly benefit from newer drugs on the market like GLP-1 receptor agonists, if they could afford them.
The authors offered an outline for a path forward that could dramatically improve access. This includes expanding tiered pricing for low- and middle-income countries, broadening compassionate use programs, and supporting diagnostic capacity in underserved regions where ATTR-CM is likely to go unrecognized. They advocated for medium-term strategies that focus on value-based pricing agreements that link costs to clinical outcomes. Longer-term solutions, they said, will require sustained investment in research to optimize treatment duration, identify stopping criteria and develop more cost-effective therapeutic approaches. This includes the need for more studies on when to initiate therapy, how to monitor its effectiveness and whether combination approaches offer a sufficient added benefit to justify the additional costs.
"This will require a partnership between industry, government, clinicians and health systems. All of us must be motivated to ensure patients get the best care, and that means working together on sustainable models," Krumholz said.
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Dave Fornell has covered healthcare for more than 17 years, with a focus in cardiology and radiology. Fornell is a 5-time winner of a Jesse H. Neal Award, the most prestigious editorial honors in the field of specialized journalism. The wins included best technical content, best use of social media and best COVID-19 coverage. Fornell was also a three-time Neal finalist for best range of work by a single author. He produces more than 100 editorial videos each year, most of them interviews with key opinion leaders in medicine. He also writes technical articles, covers key trends, conducts video hospital site visits, and is very involved with social media. E-mail: [email protected]