Health and economic impact of the correct diagnosis of transthyretin cardiac amyloidosis in Spain

ABSTRACT

Objective: To estimate the health and economic impact of the reduction in mortality and cardiovascular hospitalizations, associated with correct diagnosis of cardiac transthyretin amyloidosis (ATTR-CM), from the Spanish National Health System (NHS) perspective.

Methods: A costs and effects analysis were performed (probabilistic Markov model) with time horizons between 1 and 15 years, comparing the correct diagnosis of ATTR-CM versus the non-diagnosis. Transition probabilities were obtained from the ATTR-ACT study (placebo arm) and from the literature. Costs and healthcare resources were obtained from Spanish sources (€ 2019) and from a panel of Spanish clinical experts.

Results: After 1, 5, 10 and 15 years, the diagnosis of ATTR-CM would generate a gain of 0.031 (95%CI 0.025; 0.038); 0.387 (95%CI 0.329; 0.435); 0.754 (95%CI 0.678; 0.781) and 0.944 (95%CI 0.905; 0.983) life years per patient, respectively, with savings of € 212 (95%CI € −632; 633), € 2,289 (95%CI € 2,250; 2,517), € 2,859 (95%CI € 2,584; 3,149) and € 2,906 (95%CI € 2,669; 3,450) per patient, respectively, versus the non-diagnosis.

Conclusions: Just by correctly diagnosing ATTR-CM, years of life would be gained, cardiovascular hospitalizations would be avoided, and savings would be generated for the NHS, compared to the non-diagnosis of the disease.

KEYWORDS:

• Amyloidosis
• cardiomyopathy
• transthyretin
• diagnosis

Article highlights

• ATTR-CM is an underdiagnosed disease with a clinical presentation similar to that of much more prevalent cardiovascular diseases, with serious consequences for patients, increasing the mortality and hospitalisations for cardiovascular causes.

• Based on this model, the diagnosis of ATTR-CM and resulting correct symptomatic treatment of the disease produce a significant gain in life-years and cost savings for the National Health System, specifically due to the hospitalisations for cardiovascular causes that would be avoided.

Declaration of interest

Carlos Rubio-Terrés and Darío Rubio-Rodríguez are employees of Health Value and have received consultancy payments from Pfizer in connection with the conduct of this study and the development of the manuscript. CP, PT and AL work at Pfizer SLU. CP and PT own shares in Pfizer SLU. PGP has received fees for lectures and/or advisory activity from Akcea, Alnylam, Eidos, Neuroimmune, and Pfizer. The PGP center has received research and/or training funding from Akcea, Alnylam, Eidos, Pfizer, and Prothena. ANR, FF and FJMS have received advisory fees from Pfizer. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewers disclosure

Peer reviewers on this manuscript have no relevant financial relationships or otherwise to disclose.

Additional information

Funding

This study has been promoted and financed by Pfizer, S.L.U.