OBJECTIVES: To identify evidence gaps in the literature for the burden of illness and treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) to support the launch of efgartigimod to treat this rare disease.
METHODS: A literature review using a structured search strategy was conducted from 1 July 2011 to 12 October 2021 in PubMed, Embase, and Cochrane regarding disease description, epidemiology, humanistic and economic burden, treatment guidelines and patterns. Online desktop searches were also conducted for health technology assessments, competitor labels, and ongoing clinical trials.
RESULTS: Epidemiology data are limited, and results vary depending on the diagnostic criteria used in each study. In lieu of a biomarker for diagnosis, easy-to-use, unified, diagnostic criteria are needed. There is a lack of studies that measure the level and duration of disability among the various CIDP phenotypes, including after therapy. No longitudinal studies that describe the natural history of CIDP were identified. A valid, reliable, and responsive composite measure that addresses all aspects of impairment faced by patients with CIDP is needed. Although health-related quality of life is significantly impacted in patients with CIDP, few studies have estimated it, and most of them used generic instruments. Data on the economic burden of CIDP is only available from a few studies in the United States, England, and Germany. Evidence-based CIDP treatments are limited to intravenous immunoglobulins, subcutaneous immunoglobulins, corticosteroids, and plasma exchange. Studies suggest intravenous immunoglobulins provide faster response rates than corticosteroids, but corticosteroids have longer-term remission rates than intravenous immunoglobulins; however, comparative data on remission rates are not available, and some patients require treatment for as long as 30 years.
CONCLUSIONS: There are many gaps in the literature to support the launch of a new product in CIDP, including epidemiology, humanistic and economic burden studies, and a clear definition of treatment response.
