Impact of the COVID-19 pandemic on access to the cerliponase alfa managed access agreement in England for CLN2 treatment

Table 1 Criteria for entering and staying in the managed access agreement (MAA) [9]

Eligibility criteria	Confirmed diagnosis of CLN2 disease CLN2 Rating Scale ML score ≥ 2 [10] No other serious life-limiting conditions Patient/carer willing to sign MAA patient agreement Willing to undergo implantation of intracerebroventricular access device
Requirements to stay on treatment	Attend infusion appointments every 2 weeks Attend clinic assessment every 6 months Complete PRO questionnaires every 6 months^a Assessments show benefit with cerliponase alfa^b
Reasons for stopping treatment	> 2 infusions missed in any 14-month period^c < 2 hospital assessments^d or PRO questionnaires completed in any 14-month period Child is not benefiting from treatment Caregiver wishes treatment to stop Medical reasons

^aPatient-reported outcome (PRO) questionnaires: Pediatric Quality of Life Questionnaire (Peds QL™), CLN2 Quality Of Life Assessment (CLN2Qol), EuroQol 5-dimension, 5-level questionnaire (EQ-5D-5L)
^bEvidence of benefit based on hospital assessments and PROs
^cExcluding medical reasons or public health emergency
^dHospital assessments: mobility, cardiac function and speech/language skills assessed every 6 months; eye structure and function, brain structure and function and child development (knowledge, comprehension, social–emotional skill, behaviour) every 12 months; during the restrictions instigated during the COVID-19 pandemic, assessments were deferred or conducted by telephone/video

ISSN: 1750-1172