Answers are grounded in this paper and cite their sources.
For orphan drugs, the yearly cost per patient is inversely related to the prevalence of the disease.1 2 3 4 5 Although this relation is widely recognised qualitatively, to our knowledge, no attempt has been made to define it mathematically. We examined the decisions made for non-cancer orphan drugs approved in Italy over the past years and developed the …
No takes yet. Share an insight, caveat, or question.
Messori et al. (2010) studied this question.
Synapse has enriched 5 related papers. Consider them for comparative context: