The view of experts on initiatives to be undertaken to promote equity in the access to orphan drugs and specialised care for rare diseases in Spain: A Delphi consensus

• Published in: Health policy (Amsterdam) 2018-06, Vol.122 (6), p.590-598
• Main Authors: Torrent-Farnell, J., Comellas, M., Poveda, J.L., Abaitua, I., Gutiérrez-Solana, L.G., Pérez-López, J., Cruz, J., Urcelay, J., Lizán, L.
• Format: Article
• Language: English
• Subjects: Access; Agreements; Clinical effectiveness; Clinical medicine; Clinical practice guidelines; Consensus; Delphi method; Delphi Technique; Disease; Drugs; Effectiveness; Etiology; Expert consensus; Experts; Focus Groups; Group research; Health administration; Health care policy; Health Equity; Health Plan Implementation - methods; Health services; Humans; Implementation; Interest groups; Knowledge; Literature reviews; Medical prognosis; Networks; Orphan Drug Production - economics; Orphan drugs; Patients; Public health; Questionnaires; Rare diseases; Rare Diseases - drug therapy; Rare Diseases - economics; Rare Diseases - epidemiology; Referrals; Spain; Spain - epidemiology; Surveys and Questionnaires; Teams
• ISSN: 0168-8510; 1872-6054
• DOI: 10.1016/j.healthpol.2018.03.002

•The most feasible actions were related to OD access, referral medical teams and RD registries.•Review of OD price and reimbursement based on clinical and epidemiological evidence were suggested.•A unified, usable, aetiology-based registry created to generate evidence was required.•Reference teams conceived to provide specialised care and define referral protocols were desired. To reach a consensus amongst experts on the most feasible actions to be undertaken to facilitate patient access to specialised care and orphan drugs (OD) in the public health sector in Spain. Two Delphi rounds were completed. The questionnaire was based on a literature review and 2 focus groups. Agreement was sought on the desire (D) and prognosis (P) for the implementation within the next 5 years, on a 5-point Likert scale. Consensus was reached when ≥75% participants chose agreement (1–2) or disagreement options (4–5). 82 experts on rare disease (RD) participated. Agreement on the D and P was reached in 66.07% statements: OD pricing review [absence of clinical effectiveness (D:85.37%; P:85.90%), target population increase (D:79.27%; P:91.03%)]; reference team definition of referral protocols and clinical practice guidelines (D: 97.56%; P: 89.74%); and a unified, usable, etiology-based registry (D:97.56%; P:84.62%). D and P assessment diverged in 32.14% items: creation of a specific funding system for OD (D: 97.56%; P: 60.25%); and a network of medical teams to coordinate the care of RD patients (D: 99%; P: 62%). The results have shown the need to promote dialogue between stakeholders, introduce European recommendation to national and regional Spanish policies and set up priorities and undertake actions to drive relevant changes in current medical practice in managing RD patients.