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Canepa et al. Vessel Plus 2022;6:30 https://dx.doi.org/10.20517/2574-1209.2021.106 Page 9 of 13

There has been considerable debate regarding the costs of treatments for ATTR-CA, and particularly of
[79]
tafamidis [76-78] , recently labeled as “the most expensive cardiac medication in history” . In the words of
experts, “unreasonable pricing decisions have been made for this pharmaceutical, considerably above
[76]
typically accepted cost-effectiveness thresholds” . Cost-effectiveness analysis have shown that at a current
list price of $225,000 annually, tafamidis would require an estimated 92.6% price reduction to meet
commonly accepted cost-effectiveness standards of $100,000 per quality-adjusted life year . This pricing
[76]
issue also raises problems of access to treatment and its affordability, both from a patient’s perspective
(especially in countries with mandatory health insurance coverage and direct participation to medical
expenses) and health authorities’ perspective (US pharmaceutical spending, once starting tafamidis in all
[78]
eligible patients, is estimated to increase up to 10%) .

Comorbidity burden, frailty, and life expectancy of newly diagnosed ATTR-CA patients, together with cost-
effectiveness of new ATTR-CA treatments such as tafamidis, should be the objects of future specific studies,
before the widespread approval of other pricy medications for ATTR-CA on the market. Physicians should
carefully and openly discuss with each patient the expected prognostic benefit of starting these new
treatments, taking into account the whole context in which the diagnosis of ATTR-CA has been made. In a
moment in which drug industries are fostering their products in the market and media are fueling false
expectations in the public, the discussion with our patients should always be straight and fair. The
continuous consultation with CA referral centers may allow a better understanding of these issues and
selection of candidates with greater chance of benefits from these new-targeted treatments.

CONCLUSIONS
ATTR-CA is rapidly becoming the most prevalent form of CA, and screening of conditions considered at
risk of CA has revealed that the prevalence of ATTR-CA might be higher than initially thought. At least one
out of ten patients with HFpEF, AS, or HCM diagnosed later in life might have an overlooked ATTR-CA,
and nowadays the diagnosis is made mostly at later stages of the disease. The therapeutic and prognostic
implications of this finding appear limited, and screening at earlier stages is warranted. Nonetheless,
challenges exist in systematically optimizing the diagnostic accuracy of the screening process, which should
always exclude a plasma cell dyscrasia first and then use bone scintigraphy with tomographic imaging
followed by genetic testing. Earlier and more comprehensive screening programs will likely result in
increasing the number of ATTR-CA patients who will be candidates to newly available disease-specific
treatments with a favorable cost-benefit balance.

DECLARATIONS
Authors’ contributions
Wrote the first draft: Canepa M, Vianello PF
Revised it thoroughly before submission: Canepa M, Vianello PF, Porcari A, Merlo M, Scarpa M
All authors contributed equally to the design of this manuscript.

Availability of data and materials
Not applicable.

Financial support and sponsorship
None.

Conflicts of interest
Dr. Canepa received speaker and advisor fees from Akcea Therapeutics, Menarini, Novartis, Pfizer, Sanofi e
Sanofi Genzyme, Vifor Pharma, and two investigator-initiated grants from Pfizer. Dr. Merlo received an