OBJECTIVES:

The objective of the analysis is to review patient`s persistence to treatment in one example indication Phenylketonuria (PKU), an inborn rare disease.

METHODS:

Analyze the treatment persistence of patients treated with Sapropterin , indicated for PKU, using German IMS^® LRx longitudinal prescription database, which collects information from centers nationwide that process prescription data related to patients with statutory health insurance, and contains information on approximately 82% of all prescriptions reimbursed in Germany. The timeframe for the persistence analysis is January 2016 to December 2019. Considering all of patients how started their therapy before December 2018 with an allowed treatment gap of 120 days, measured 3, 6 and 12 months after treatment start.

Additionally number of patients in the PKU patient support program as of March 2020 were reviewed.

RESULTS:

245 patients treated with Sapropterin can be analysed, 11 were included in the PSP.

Children aged 0-4 count for 32% of patients, while being reflected in the PSP with 36% with a persistence of 62%, 37% and 15%.

Children aged 5-12 count for 25% of patients, while being reflected in the PSP with 36% with a persistence of 40%, 26% and 16%.

Children aged 13-18 count for 6% of patients, while being reflected in the PSP with 0% with a persistence of 38%, 25% and 25%.

Patients above 18 count for 36% of patients, while being reflected in the PSP with 27% with a persistence of 24%, 21% and 6%.

CONCLUSIONS:

Most patients on the drug are between 0-4 years old and this is the most persistent age group. Adults are clearly the least persistent patients, reflecting the necessity for support, particularly when young adults take charge of their own therapy. High drop outs in persistence in the first six month of treatment indicate need for support at therapy start.