Orphan drugs: are they worth their extra costs?

Results of a new study suggest that whilst orphan drugs confer greater average health gains for patients compared with non-orphan drugs, they also tend to be less cost effective. Results of a new study, led by James Chambers at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center (MA, USA), suggest that whilst orphan drugs confer greater mean health gains for patients compared with non-orphan drugs, they also tend to have less-favorable cost—effectiveness ratios. The findings are published in the Journal of General Internal Medicine. Orphan drugs — drugs indicated for rare diseases —...