The biggest barriers to the treatment of tranthyretin amyloid cardiomyopathy (ATTR-CM) included a persistent lack of affordability, delayed diagnosis and underrepresented populations in clinical trials, according to a study recently published in Pharmacotherapy.
“Disease-modifying medications approved and in development to treat ATTR-CM offer hope for patients with this disease, but their lack of affordability is the biggest barrier to their use,” study authors said.
What is ATTR-CM?
Transthyretin amyloidosis cardiomyopathy (ATTR-CM) is a rare progressive disease of the heart muscle that leads to congestive heart failure. It occurs when the transthyretin protein produced by the liver is unstable. Symptoms include fatigue; shortness of breath; irregular heart rate or palpitations; swelling of the legs, ankles and stomach; brain fog; wheezing; and dizziness. It often goes underdiagnosed because of a lack of awareness and knowledge of the disease. There is currently no cure for ATTR-CM.
Annual acquisition costs for ATTR-CM modifying therapy vary between $60,000 and $100,000, the study said. Several financing and patient-support programs are available in the United States, but many patients find most programs difficult to navigate, with average yearly out-of-pocket costs surpassing $6,000, the study said.
Read more about ATTR-CM therapies
Researchers aimed to evaluate current data on disease-modifying therapies for ATTR-CM, including approved and experimental drugs. An extensive search for ATTR-CM clinical trials in several medical databases yielded information on several drugs, including tafamidis, diflunisal, acoramidis and tolcapone.
“As these and other potentially life-saving therapies are developed to treat ATTR-CM, future work should seek to improve the cost-effectiveness of these drugs to maximize patient access,” the authors said.
Most reports from past and ongoing clinical trials showed promising results from currently approved and investigational drugs, the study said. But the search highlighted several unmet needs and future challenges for ATTR-CM management. (The efficacy and safety of combined therapy with disease-modifying agents is yet to be determined.)
The increased effectiveness of disease-modifying therapy in early disease stages is a well-established fact. But nonspecific symptoms and lack of disease awareness lead many patients to experience significant diagnosis delays.
Furthermore, strategies such as community screening programs with biomarkers and advanced imaging assessment in high-risk patients have not shown enough results to be cost-effective.
The authors also observed that most ATTR-CM clinical trials lacked diversity. Women and patients who self-identified as Black appeared to be particularly underrepresented, with both groups representing less than 15% of the population in ATTR-CM clinical trials.
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