OBJECTIVES: Advanced therapy medicinal products (ATMPs) are breakthrough interventions that are based on gene, cell, and tissue manipulation. They have great therapeutic potential, but are often associated with a high cost. Consequently, the need for new funding mechanisms is growing. The aim of this study was to identify key considerations and challenges in the HTA of ATMPs in Sweden.

METHODS: A review of the European Medicines Agency (EMA) website was conducted to identify ATMPs granted marketing authorisation. European public assessment reports from January 2009 to June 2022 were assessed. Identified drugs were used to guide a targeted review of the Swedish Dental and Pharmaceutical Benefits Agency (TLV) and New Therapies Council (NTc) websites. Documents pertaining to the basis for decisions were reviewed. Recommendations for reimbursement and national introduction were then mapped.

RESULTS: The EMA granted marketing authorisation to 15 ATMPs (17 indications). A TLV assessment was identified for most drugs (n=10). Additionally, three drugs were listed as planned health economic assessments. NTc recommendations were identified for 9 drugs, while four drugs had ongoing assessments. Positive recommendations were granted for 5 ATMPs. Most of the drugs were gene therapies administered in the inpatient setting. Key considerations for market access are difficult to discern. However, a price agreement and a rare disease prevalence appear to correspond to positive NTc recommendations.

CONCLUSIONS: The majority of drugs assessed by the NTc received a positive recommendation, which may reflect positively on the availability of ATMPs in Sweden. The high price of ATMPs may be a limiting factor to market access as evidenced by the connection between price negotiations and positive HTA outcomes. Alternative funding processes should be explored to increase the flexibility for ATMPs assessments and ensure patient access.