Orphan Drug Shortage in The United States: A Long Lasting Public Health Issue Calling for Effective Policies

Abstract

Objectives: To characterize unmet medical needs among adults with a history of moderate-to-severe atopic dermatitis (AD) in the United Kingdom (UK), Germany, and France. MethOds: Data were from the 2014 Adelphi AD Disease-Specific Programme, a cross-sectional survey of physicians from UK (n= 136), Germany (n= 134), France (n= 137) and their patients with history of moderate-to-severe AD (UK, n= 666; Germany, n= 649; France, n= 661). Each physician completed a Patient Record Form for up to 5 patients on demographic/disease characteristics, treatment, and physician-perceived current AD severity. AD was classified as controlled or uncontrolled, with uncontrolled defined by either currently flaring AD; deteriorating/changeable AD; or physician dissatisfaction with current control. Patients voluntarily completed a questionnaire including the Dermatology Life Quality Index (DLQI) scale. Descriptive statistics characterized the populations. Results: Patient demographics were similar across countries. Disease onset was predominantly during adolescence. Current severity was mainly moderate (54%-65%); 10% (Germany) and 16% (France, UK) were severe. Substantial proportions of patients in each country were uncontrolled (54%-59%) even though 23%-39% of uncontrolled patients were currently receiving any systemic immunosuppressant or phototherapy including cyclosporine (6%-10%). Uncontrolled AD was high regardless of current severity: 85%-88% of severe patients were uncontrolled, as were 56% (France) to 70% (UK) of moderate patients. Quality-of-life was worse among uncontrolled patients, and in particular among patients treated with immunosuppressants or phototherapy in the past 12 months: 68%, 40%, and 40% of uncontrolled patients previously on immunosuppressants or phototherapy in UK, Germany, and France, respectively, had DLQI > 10 (threshold for very large effect on patient's life). cOnclusiOns: Adults with history of moderate-to-severe AD have poor disease control even when treated with systemic agents. A substantial proportion of uncontrolled patients previously treated with immunosuppressants or phototherapy report a very large effect of AD on their quality-of-life. These results demonstrate high unmet medical needs associated with AD.