OBJECTIVES: Vyndaqel® (tafamidis) 61 mg capsules were reimbursed in Ireland under the High Tech Arrangement effective 1 March 2022, for the treatment of wild-type (wt) or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Reimbursement is subject to a Health Service Executive (HSE)-Managed Access Protocol (MAP). This study provides an overview of applications for tafamidis (Vyndaqel®) reimbursement in the first year of the protocol.

METHODS: All applications submitted to the HSE-Medicines Management Programme (MMP) between 1 March 2022 and 28 February 2023 were reviewed. Data was compiled and analysed in Microsoft Excel™.

RESULTS: A total of 104 applications were received. The majority of applications (84.6%) were for males (n=88), with 15.4% applications for female patients (n=16). The average age of applicants was 76.4 years (range 48-89 years).

Of the 104 patients, 20 (19.2%) had a confirmed hereditary diagnosis (hATTR-CM), and 79 (76%) had a confirmed diagnosis of ATTR-CMwt. Genotype was not available for five patients (4.8%). The diagnosis of ATTR-CM was established by biopsy in 29 patients and by nuclear scintigraphy in 74 patients. Further information in relation to diagnosis was still outstanding for one patient.

Reimbursement was approved for 87.5% of applications received. Further information relating to 10 applications (9.6%) was still outstanding at the end of year one. Three applications (2.9%) were not approved as they did not meet the reimbursement criteria. Reimbursement approval was withdrawn for two patients, where the prescriber wished to switch the patient to alternative treatment (patisiran (Onpattro®)).

The number of applications received in year 1 is double what was estimated in the Health Technology Assessment (HTA) conducted by the National Centre for Pharmacoeconomics.

CONCLUSIONS: The managed access protocol ensures drug reimbursement is in line with the criteria agreed by the HSE. Data collected may serve to inform future HTA and reimbursement decisions.