PATIENT-CENTRIC APPROACHES IN HEALTH TECHNOLOGY ASSESSMENT FOR ATYPICAL HEMOLYTIC UREMIC SYNDROME- CHALLENGES AND INNOVATIONS

Atypical Hemolytic Uremic Syndrome (aHUS) is a rare, life-threatening condition which is significantly heterogeneous, further burdening patients. Health Technology Assessment (HTA) is important for assessing access to new and innovative therapies for patients with aHUS, but patients are not always involved in the HTA process and, when involved, are frequently tokenized. In this review, we present the extent and involvement of patients in aHUS HTA. We outline systemic limitations including limited involvement of patients, poor transparency, limited resources, and inconsistent data collection methods. We demonstrate the necessity of patients' involvement and perspectives to appropriately capture lived experiences, unmet need, informing outcomes using patient reported outcomes (PROs), and understanding relative impacts on societal level. We suggest practical solutions such as improvements in transparency, advocacy group support, striving to have representation from diverse groups, integrating and developing patient registries and understanding the gaps in culture and language. To expand upon patient engagement, we consider the innovative possibilities of organ-on-chip technologies. Hence creating and enabling personalized modelling of diseases and testing of patient-specific treatments and therapies. This is a new opportunity for patients to engage in research and HTA. We present a case study and suggested the next steps and ultimately advocate for a patient-centered approach to HTAs.

Keywords: Atypical Hemolytic Uremic Syndrome, Health Technology Assessment, Patient Engagement, Organ-on-Chip, Patient-Reported Outcomes, Rare Diseases.