Intended for healthcare professionals

Letters Orphan drugs

Relating price determination to disease prevalence

BMJ 2010; 341 doi: (Published 24 August 2010) Cite this as: BMJ 2010;341:c4615
  1. Andrea Messori, coordinator1,
  2. Americo Cicchetti, professor2,
  3. Luigi Patregani, head3
  1. 1Laboratory of Pharmacoeconomics, ESTAV Centro, Health Service of Regione Toscana, 59100 Prato, Italy
  2. 2School of Economics, “Sacro Cuore” Catholic University, c/o Policlinico Gemelli, Roma, Italy
  3. 3Pharmaceutical Service, Health Service of Regione Marche, Ancona, Italy
  1. andreamessori{at}

    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 …

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