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Drugs, Diseases, and the Managed Care Impact

Rare Disease Spotlight: Transthyretin Amyloidosis Cardiomyopathy (ATTR‐CM) Disease Burden, Unmet Needs, and Treatment Advances

(D3) Rare Disease Spotlight: Transthyretin Amyloidosis Cardiomyopathy (ATTR‐CM) Disease Burden, Unmet Needs, and Treatment Advances

Tuesday, October 15, 2024
3:30 PM - 4:45 PM PT
Location: 121
CPE
ACPE UAN:0233-0000-24-048-L01-P | Contact Hours: 1.25 | Activity Type: Knowledge-based
This activity is supported by an independent medical education grant from BridgeBio Pharma, Inc.

Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM) is a rare and progressive disease characterized by the misfolding of amyloid fibrils that aggregate and deposit locally in one specific area of the body or deposit systemically which affects many tissues and organs. The disease burden of ATTR-CM is considerable, given its progressive nature and symptoms of chronic heart failure and significant impact on patients' quality of life and survival. In the US, significant diagnostic gaps are evident among Black and Hispanic/Latinx communities with ATTR-CM. The estimated healthcare cost to treat all eligible ATTR-CM patients is $32.3 billion annually.

The faculty for this session will provide an in-depth knowledge of ATTR-CM disease burden, unmet care needs, patient value, timely clinical data on new and emerging therapies, and, more broadly, managed care approaches to optimize health outcomes.

Learning Objectives:

At the completion of this activity, participants should be able to:
  1. Define transthyretin amyloidosis cardiomyopathy, including clinical manifestation, screening diagnosis, and at‐risk populations.
  2. Explain the disease burden of ATTR‐CM from a patient perspective in relation to the quality of the lived experience and treatment value.
  3. Identify unmet needs in ATTR‐CM by evaluating new and emerging therapeutic options to distinguish clinical efficacy, safety, and dosing.
  4. Discuss managed care considerations to incorporate the patient value and access to innovative therapies into managed care decision‐making for improved health outcomes.