EXPERTS CONSENSUS ON THE FUTURE OF RARE DISEASES CARE AND ORPHAN DRUGS ACCESS IN SPAIN- A DELPHI STUDY

Author(s)

Paz S1, Torrent J2, Poveda JL3, Perez J4, Moreno JL5, Martin A5, Gonzalez L6, Cruz J7, Comellas M1, Abaitua I8, Urcelay J5
1Outcomes 10, Universitat Jaume I, Castellon, Spain, 2Committee for Orphan Medicinal Products (COMP), London, UK, 3Hospital Universitario La Fe, Valencia, Spain, 4Hospital Vall d'Hebron, Barcelona, Spain, 5Shire, Madrid, Spain, 6H. U. Infantil Niño Jesús, Madrid, Spain, 7FEDER, Madrid, Spain, 8Instituto de Investigación de Enfermedades Raras, Madrid, Spain

OBJECTIVES: This study aims to determine the level of consensus that exits amongst experts on the most likely actions to be implemented to enable equal access to Orphan Drugs (OD) and specialized care to Rare Disease (RD)  patients’ in the public health sector in Spain. METHODS: Two-round Delphi survey; RD experts identified by the study scientific committee. The questionnaire [32 statements related to OD price and reimbursement (n=5); access to OD and specialized health care (n=19); RD registry (n=7); care model for RD (n=1)] was based on a literature review and 2 focus groups. Agreement was sought on the desire (D) and prognosis (P) for each statement to occur over the next 5 year. Consensus was reached when 75% participants, or more, choose the totally or partially agreed options (agreement), or the totally or partially disagreed alternatives (disagreement). Descriptive statistics were applied. RESULTS:

Conference/Value in Health Info

2015-11, ISPOR Europe 2015, Milan, Italy

Value in Health, Vol. 18, No. 7 (November 2015)

Code

PSY116

Topic

Health Policy & Regulatory

Topic Subcategory

Health Disparities & Equity

Disease

Rare and Orphan Diseases

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