TRANSTHYRETIN AMYLOID CARDIOMYOPATHY (ATTR-CM) TREATMENT PREFERENCES AMONG PATIENTS AND CARDIOLOGISTS: FINDINGS FROM TWO ADVISORY BOARDS IN THE UNITED STATES
Author(s)
Joshua Mitchell, MD1, Joshua Coulter, MA2, Muriel Finkel, BA3, Janice Chung, MPH2, Kelly Haque, MD2, Sanjana Chandrasekar, MPH2, Kathleen Beusterien, MPH4, Gabriela Burgos, MPH4, Oliver Will, PhD4, Vanessa McWilliams, PharmD2, Leila G. Lackey, MHS, PhD2, Brett Hauber, PhD2, Alexandra Haddad Angulo, MD2;
1University of Washington Medicine, St. Louis, MO, USA, 2Pfizer, New York, NY, USA, 3Amyloidosis Support Groups Inc., Wood Dale, IL, USA, 4Oracle Life Sciences, Austin, TX, USA
1University of Washington Medicine, St. Louis, MO, USA, 2Pfizer, New York, NY, USA, 3Amyloidosis Support Groups Inc., Wood Dale, IL, USA, 4Oracle Life Sciences, Austin, TX, USA
OBJECTIVES: With the approval of novel ATTR-CM disease modifying therapies that have varying mechanisms of action and administration regimens, this research sought to understand patient and physician preferences for ATTR-CM treatments and outcomes.
METHODS: Two separate virtual advisory board meetings were held with patients with ATTR-CM and with cardiologists. Prior to the meetings, participants completed an online survey to support the discussions held during the meetings. The survey included three best-worst scaling exercises to elicit the relative importance of benefits, side effects, and dosing regimens, and a point allocation exercise about access challenges.
RESULTS: Nine patients (mean age, 70 years) and 10 cardiologists participated. Both patients and cardiologists placed high importance on reducing mortality. Having test results and imaging that confirmed their disease wasn’t progressing was highly important for patients. In contrast, cardiologists considered imaging and testing less important, stating that findings may not correlate with disease status. For cardiologists, reducing the number of urgent encounters or the need for a heart transplant was highly important. Assuming equal efficacy, both patients and physicians preferred more convenient regimens (taken at home, less frequent, and/or fewer pills); both groups indicated that these attributes may translate into higher adherence. Key challenges to treatment access include low awareness of diagnostic testing procedures among non-specialists, resulting in diagnostic delays, as well as difficulties obtaining insurance coverage for treatment. Patients reported distress as they often need to serve as their own healthcare advocates because some healthcare providers lack knowledge about ATTR-CM.
CONCLUSIONS: Because treatment priorities differ between ATTR-CM patients and cardiologists, promoting shared decision making can help ensure patient perspectives are considered in treatment decisions and potentially improve treatment adherence. Educating non-specialists about ATTR-CM testing and criteria for referrals may reduce delays in diagnosis and treatment initiation. Together, these treatment considerations may improve ATTR-CM patient care and outcomes.
METHODS: Two separate virtual advisory board meetings were held with patients with ATTR-CM and with cardiologists. Prior to the meetings, participants completed an online survey to support the discussions held during the meetings. The survey included three best-worst scaling exercises to elicit the relative importance of benefits, side effects, and dosing regimens, and a point allocation exercise about access challenges.
RESULTS: Nine patients (mean age, 70 years) and 10 cardiologists participated. Both patients and cardiologists placed high importance on reducing mortality. Having test results and imaging that confirmed their disease wasn’t progressing was highly important for patients. In contrast, cardiologists considered imaging and testing less important, stating that findings may not correlate with disease status. For cardiologists, reducing the number of urgent encounters or the need for a heart transplant was highly important. Assuming equal efficacy, both patients and physicians preferred more convenient regimens (taken at home, less frequent, and/or fewer pills); both groups indicated that these attributes may translate into higher adherence. Key challenges to treatment access include low awareness of diagnostic testing procedures among non-specialists, resulting in diagnostic delays, as well as difficulties obtaining insurance coverage for treatment. Patients reported distress as they often need to serve as their own healthcare advocates because some healthcare providers lack knowledge about ATTR-CM.
CONCLUSIONS: Because treatment priorities differ between ATTR-CM patients and cardiologists, promoting shared decision making can help ensure patient perspectives are considered in treatment decisions and potentially improve treatment adherence. Educating non-specialists about ATTR-CM testing and criteria for referrals may reduce delays in diagnosis and treatment initiation. Together, these treatment considerations may improve ATTR-CM patient care and outcomes.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR26
Topic
Patient-Centered Research
Topic Subcategory
Patient Engagement
Disease
SDC: Rare & Orphan Diseases, STA: Multiple/Other Specialized Treatments