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Exagamglogene autotemcel (exa-cel, Casgevy^®) received conditional marketing authorization from the European Medicines Agency (EMA) on February 9, 2024, as the first CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)-based gene therapy. It is classified as an Advanced Therapy Medicinal Product (ATMP) and has orphan drug status for two indications. Exa-cel is approved for treating transfusion-dependent ß-thalassemia (TDT) and severe sickle cell disease (SCD) in patients aged twelve years or older who are eligible for hematopoietic stem cell transplantation but lack a human leukocyte antigen (HLA)-matched related donor.

The studies to date (two single-arm studies) show a significant improvement in symptoms in the majority of patients treated. In beta-thalassemia, 32 out of 35 people no longer needed blood transfusions after the therapy. In sickle cell disease, 29 out of 30 patients no longer had severe pain crises. These improvements lasted for at least one to two years in both diseases. The patients' quality of life was noticeably improved as a result. Long-term experience is not yet available.

The number of people living with diabetes is increasing globally, with approximately 90% having Type 2 Diabetes Mellitus (T2DM). The fundamental management of T2DM relies on maintaining a healthy lifestyle. This includes regular medical visits for monitoring, balanced diet management, regular exercise, and maintaining a healthy body weight. Telehealth has gained attention as a means of supporting the continuous self-management and treatment of people living with diabetes.

Healthcare systems worldwide face the challenge of optimising resource allocation and maximising health benefits through their funding decisions.

This report examines how healthcare systems approach resource allocation through incremental cost-effectiveness ration (ICER) thresholds. Through analysis of literature, policy documents, and health economic guidelines from various countries, the research explores theoretical foundations and practical applications of these thresholds. The findings reveal significant variations in threshold implementation across countries and show important relationships between thresholds and health system outcomes. The research also identifies key factors for implementation. This report offers insights into how threshold implementation can enhance healthcare system transparency and efficiency when adapted to national contexts.