Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group

Successful development of new treatments for rare diseases (RDs) and their sustainable patient access require overcoming a series of challenges related to research and health technology assessment (HTA). These impediments, which may be unique to RDs or also apply to common diseases but are particularly pertinent in RDs, are diverse and interrelated.

Source: Value in Health - April 11, 2018 Category: International Medicine & Public Health Authors: Sandra Nestler-Parr, Daria Korchagina, Mondher Toumi, Chris L. Pashos, Christopher Blanchette, Elizabeth Molsen, Thomas Morel, Steven Simoens, Zolt án Kaló, Ruediger Gatermann, William Redekop Source Type: research