Early access to medicines in Spain: a retrospective hospital study with a proposal for a regulatory change

BACKGROUND: Medicines awaiting price and reimbursement (P&R) decisions represent a delicate situation in the Spanish NHS. The interval between marketing authorisation (MA) and PR averages 616 days, leaving patients without standard access to EU-approved treatments for almost 2 years, despite their eventual availability. While early access before MA is regulated, no framework exists for the period between MA and PR. Responsibility for funding falls to hospitals or autonomous regions, with no harmonised criteria, leading to inequities in access. OBJECTIVES: To describe our access model to medicines in special situations (MSS), and to evaluate the clinical and regulatory implications of this procedure. METHODS: We conducted a 2-year retrospective review of MSS access requests. Our tertiary hospital uses a transparent pathway: clinicians submit a consultation to Clinical Pharmacology and Hospital Pharmacy; departments issue independent reports to Hospital Administration, which decides, considering individual justification, clinical need, early access, and treatment benefit. RESULTS: A total of 295 request were received, and 78 involved products pending PR decision. Of them, 73 (93.6%) were granted local access. Subsequently, PR status changed to regular access in 64.1% of these requests (50/78), ensuring early access for 62.8% of patients (49/78). Onco-haematological patients granted early access showed an overall disease control rate of 63%, with a median survival of 16 months. CONCLUSIONS: Until legislation regulates access to medicines awaiting reimbursement, or decision times are reduced, implementing robust local procedures is essential to guarantee fair and equitable MSS access, particularly for patients requiring treatment before national PR resolution.