Pharmacists Can Increase Access to Care for Patients With ATTR-CM


Major barriers to care for patients with transthyretin amyloid cardiomyopathy (ATTR-CM) can include navigating therapeutics and the cost of medications.

Major barriers to access of care for patients with transthyretin amyloid cardiomyopathy (ATTR-CM) include the cost of medication for the available therapeutics and navigating therapeutics for the disease. Clinical pharmacists are an essential part of a multidisciplinary team to help patients understand and navigate the therapeutic landscape for ATTR-CM, according to a study published in the Journal of the American Heart Association.1

Cholesterol plaque in artery with Human heart anatomy | Image Credit: Rasi -

Rasi -

ATTR-CM is a potentially fatal disease affecting the heart when clumps of amyloid are deposited in the heart tissue, impacting its ability to properly function.2,3 According to the American Heart Association, ATTR-CM has 2 types: hereditary ATTR-CM, including a variant of the transthyretin gene, and wild-type ATTR-CM, without the transthyretin gene. ATTR-CM is often underdiagnosed in patients.2

Risk factors for hereditary ATTR-CM include having a family member who has the disease or heart failure (HF), being aged 50 and older, Black, and/or male. Risk factors for wild-type ATTR-CM include being aged 65 and older and/or male.3 Symptoms, which can begin anywhere from age 20 to 80 depending on the type of ATTR-CM, can vary and are subtle, often causing the disease to be misdiagnosed because the symptoms are similar to HF and hypertrophic cardiomyopathy; others may have no symptoms.3 The symptoms can include fatigue; leg, ankle, or abdominal swelling; arrhythmia; and shortness of breath with activity.2,3

Recently, research has shown that there is a significant population with ATTR-CM, which was previously thought to be a rare disease, according to the study authors.1 They added that ATTR-CM coexists in approximately 16% of those with aortic stenosis and 13% to 17% of those with HF with observed ejection fraction.1 In a study published by JAMA Cardiology, investigators found that ATTR-CM was present for a statistically significant number of individuals with HF with preserved ejection fraction, suggesting that an routine guideline-recommended evaluation can help increase the diagnosis of ATTR-CM.4

About The ATTR-ACT Trial

Trial Name: Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy ID: NCT01994889

Sponsor: Pfizer

Completion Date: February 2018

Currently, tafamidis (Vyndamax; Pfizer) is the only medication approved for ATTR-CM, including hereditary and wild type.1 In the ATTR-ACT trial, published in JAMA Cardiology, the study authors reported that tafamidis in the 80 mg dose lessened the decline of left ventricular (LV) systolic and diastolic function in a 30-month period for patients with ATTR-CM. The improvements in decline included LV stroke volume, LV global longitudinal strain, and septal and lateral early diastolic mitral annular velocity.5

Additionally, inotersen (Tegsedi; Sobi) and patisiran (Onpattro; Alnylam) are approved to improve symptoms of polyneuropathy in those with hereditary ATTR.1 In the review, the study authors reported that out of 72 individuals, 67% were Medicare beneficiaries. Out of the 67%, 51% received financial assistance from Medicare Part D that reduced the copay to $0. They also found that approximately one-third of patients on Medicare were able to afford the medications without any financial assistance.1

As part of the study, the authors explained the role of their clinical pharmacist, who specialized in cardiac amyloidosis. They were included in the comprehensive pharmacy team, which also included a medication assistance team and an on-site specialty pharmacy.1

The multidisciplinary team can review benefits and assess assistance programs for the patients.1 The pharmacist’s role is to ensure that enrollment in the assistance program and prior authorizations are correctly submitted. Clinical criteria in prior authorizations are critical for approval, where incorrect codes or missing documents could affect the approval of medication or assistance programs, causing a delay in treatment.1

Pharmacists can also discuss treatment with patients and explain how the medication benefits the overall regimen, while also overseeing the processes to ensure the best actions to acquire the prescribed treatment for patients.1 The pharmacist’s role also includes identifying financial assistance programs or alternative or concurrent therapies, including for other indications, according to the study authors of the review. They can also help patients understand and navigate specialty pharmacies and mail-order services. Refilling specialty medications requires enough time for proper delivery and adherence.1

However, the study investigators acknowledge that not all teams are able to employ a clinical pharmacists dedicated for this specific role, though they add that existing staff may be able to take on this role, such as medical assistants or coordinators.1 Social workers and case managers might also be a good choice, according to the investigators, but would require training on specialty medication and financial programs. Although incorporating a clinical pharmacist to help patients navigate the therapeutic space and financial burdens is not an easy task, it could be one that benefits patients profoundly, the study authors concluded.1


  1. Chen H, Chandrashekar P, Fischer K, Carlson D, et al. Navigating the Complex Web of Prescribing Amyloidosis Therapeutics: A Primer. J Am Heart Assoc. 2022;11(7):e023895. doi:10.1161/JAHA.121.023895
  2. American Heart Association. Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Updated December 8, 2021. Accessed November 21, 2023.
  3. Pfizer. Understanding This Rate Disease Called ATTR Amyloidosis. Accessed November 21, 2023.,heart%20failure%20and%20enlarged%20heart
  4. AbouEzzeddine OF, Davies DR, Scott CG, et al. Prevalence of Transthyretin Amyloid Cardiomyopathy in Heart Failure With Preserved Ejection Fraction. JAMA Cardiol. 2021;6(11):1267–1274. doi:10.1001/jamacardio.2021.3070
  5. Shah SJ, Fine N, Garcia-Pavia P, et al. Effect of Tafamidis on Cardiac Function in Patients With Transthyretin Amyloid Cardiomyopathy: A Post Hoc Analysis of the ATTR-ACT Randomized Clinical Trial. JAMA Cardiol. doi:10.1001/jamacardio.2023.4147
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