Emily Back

• Health Economics and Econometrics
• Senior Health Economics Manager at DHT.health

Aims: Sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and transfusion-dependent b-thalassemia (TDT) are serious conditions that often require caregiver support. This study aimed to describe the humanistic burden associated with caring for individuals with SCD with recurrent VOCs and TDT. Methods: Semi-structured, virtual inte...

Achondroplasia is a rare genetic disorder leading to disproportionate short stature impacting quality of life. People with these inherited and long-term health conditions may adapt to their given health state ( ie,. The “disability paradox”). Our study aims to develop an achondroplasia specific value set for the EQ-5D-5L to determine whether existi...

Sickle cell disease (SCD) is an inherited, progressively debilitating blood disorder. Emerging gene therapies (GTx) may lead to a complete remission, the benefits of such can only be realized if GTx is affordable and accessible in the low-and middle-income countries (LMIC) with the greatest SCD burden. To estimate the health impacts and country-spe...

Background Haemophilia A is a bleeding disorder caused by deficiency of coagulation factor VIII (FVIII) which leads to severe and repeated bleedings. There is a need to understand the optimal treatment pathway for FVIII inhibitors with the use of immune tolerance induction (ITI) and the role of haemostatic ‘bypassing’ agents (BPA) on-demand (OD) or...

To quantify the preferences of people with moderate and severe haemophilia (A and B) in the UK for different treatment attributes when considering gene therapy.

OBJECTIVES: Epidermolysis Bullosa (EB) is a group of rare, severe genetic skin conditions. Understanding the health related quality of life (HRQoL) impact of EB is essential in demonstrating the value of new treatments. The study elicited HRQoL from EB patients and caregivers using validated generic and disease specific instruments, and explored t...