A retrospective observational analysis of the realworld care pathway of people with hereditary transthyretin amyloidosis with polyneuropathy in Italy

Study identification

• EU PAS number:
  EUPAS1000000621
• Study ID:
  1000000621
• Official title and acronym:
  A retrospective observational analysis of the realworld care pathway of people with hereditary transthyretin amyloidosis with polyneuropathy in Italy
• DARWIN EU® study:
  No
• Study countries:
  Italy
• Study description:
  Background: This retrospective observational study described the epidemiology and the burden on the; Italian healthcare service (SSN) of patients with polyneuropathy (PN) associated with hereditary transthyretin; amyloidosis (ATTRv). ; Research design and methods: ; From the Fondazione ReS (Ricerca e Salute) administrative healthcare database (~5.5 million inhabitants in 2021), patients were identified as having ATTRv-PN in 2021 if they had received treatments for ATTRv-PN under SSN reimbursement (i.e. tafamidis, patisiran, or inotersen); from 1 January 2014 to 31 December 2021 (index date in 2021). Demographics and comorbidities at the; baseline, healthcare resource consumption, and related direct costs reimbursed by the SSN throughout; the one-year follow-up were described. ; Results: In 2021, 36 patients with ATTRv-PN (prevalence: 7.4/1,000,000) were identified (males were 83.3%; patients with ≥2 comorbidities were 61.1%; the mean age was 73 ± 8 years). During follow-up, of patients, 91.7% received drugs for ATTRv-PN; >50% received antiepileptics and acid suppressants; 22.2% were admitted to overnight hospitalizations; 30.6% accessed the emergency department; 97.2% received local outpatient specialist care. ; Th e per patient mean annual cost was € 122,017; drugs forATTRv-PN accounted for 94.7% of the total expenditure. Conclusions: This study of real-world patients with ATTRv-PN showed a high rate of comorbidities, and substantial direct healthcare and economic burdens on the SSN.
• Study status:
  Finalised

Research institutions and networks

Institutions

Fondazione ReS (Ricerca e Salute), CINECA partner

Italy

Study timelines

Date when funding contract was signed

Actual:15/03/2024

Study start date

Actual:15/04/2024

Date of final study report

Actual:15/07/2024

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

This paper was funded by AstraZeneca SpA.

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable