title: A pilot study of multicriteria decision analysis for valuing orphan medicines.
abstract: OBJECTIVE
  To pilot the use of multicriteria decision analysis to establish and apply a framework of weighted attributes to value orphan medicinal products.
  
  
  METHODS
  Literature searches on the natural history and burden of 40 rare diseases and of how payers assess treatment value and three workshops with, respectively, GlaxoSmithKline managers working on orphan medicinal products, European Union clinical and health economics experts, and representatives of rare diseases patient groups in the European Union.
  
  
  RESULTS
  Eight nonmonetary attributes were identified and weights agreed: four concern the disease being treated and four the treatment itself. About half of the weight went to attributes of the disease treated and half to attributes of the treatment. Patient group representatives gave greater weight than did the experts to patients' and carers' quality of daily life.
  
  
  CONCLUSIONS
  The multicriteria decision analysis approach piloted works and could be developed for use by payers and health technology assessment bodies.
authors: Office of Health Economics, London, UK. Electronic address: jsussex@ohe.org.
authors: Jon
authors: J
authors: Sussex
authors: Pierrick
authors: P
authors: Rollet
authors: Martina
authors: M
authors: Garau
authors: Claude
authors: C
authors: Schmitt
authors: Alastair
authors: A
authors: Kent
authors: Adam
authors: A
authors: Hutchings
mesh_terms: D017028:Caregivers / Q000523:psychology; D017281:Cost of Illness; D003365:Costs and Cost Analysis; D003661:Decision Support Techniques; D005060:Europe; D006801:Humans; D009965:Orphan Drug Production / Q000191:economics*; D010361:Patients / Q000523:psychology; D010865:Pilot Projects; D011788:Quality of Life; D013673:Technology Assessment, Biomedical / Q000379:methods*
keywords: HTA bodies; methods; orphan drugs; payers; rare diseases