A study published in Anales de Pediatría, the official journal of the Spanish Association of Paediatrics (AEP), has found that only 15 of the 67 orphan medicines authorised for paediatric use in Spain — 22.4% — are intended solely for children. The remaining drugs also cover adult patients. The research, reported by ConSalud, examines the situation as of 31 December 2025.
The 67 medicines with a paediatric indication represent about half of all orphan drugs available in the country. According to ConSalud, the findings point to a persistent gap in treatment options for children with rare diseases, a group of conditions that often begin early in life.
Why it matters
Rare diseases affect roughly three million people in Spain, and children carry a disproportionate share of the burden. Between 5,000 and 8,000 such conditions are estimated to exist worldwide, and around 75% first appear during childhood. An estimated 60% of cases become evident before a child turns two, according to the figures cited in the reporting.
Diagnosis adds another obstacle. In Spain, patients wait an average of six years for a diagnosis, a period during which many receive no appropriate treatment or see their condition worsen.
Orphan drugs are central to managing these low-prevalence conditions, which struggle to attract the investment needed to develop treatments. The study suggests that having a drug authorised does not guarantee that every young patient can obtain it.
Authorisation does not equal access
Of the 67 medicines with a paediatric indication, 49 (73.1%) were on the market in Spain, and 11 of those were for paediatric use only. Forty-six had funding through the National Health System (SNS), but coverage was often partial: 29 of the financed drugs — 63% — carried restrictions tied to their approved indications.
The study also recorded differences in how the medicines were authorised. Eight were advanced therapies, five held conditional authorisations and 13 were approved under exceptional circumstances. Nine had received the European Medicines Agency's PRIME designation, a scheme designed to support development of drugs aimed at unmet medical needs. According to ConSalud, these regulatory routes partly reflect the difficulty of gathering sufficient clinical evidence when studying diseases that affect small numbers of patients.
Availability also varies within the paediatric population itself. Fourteen medicines (28.6%) were authorised for all ages, while 45 (91.8%) listed patients over 12 in their indications. The distribution shows that treatment options are not uniform across childhood and adolescence — a concern for conditions that can appear in the earliest years.
Where the treatments concentrate
Therapeutically, drugs acting on the digestive tract and metabolism were the largest group, at 30.6% of the paediatric medicines on the market. Antineoplastic and immunomodulating agents followed at 20.4%. Biologics accounted for 42.9% of marketed products.
Intravenous administration was the most common route (38.8%), ahead of oral (34.7%), and all required a prescription. Hospital use applied to 75.5% of the medicines.
The researchers warn that limited availability has direct consequences for care. They estimate that treatment exists for only about 5% of rare diseases. For the rest, clinicians turn to alternatives such as off-label prescribing, investigational treatments through compassionate use, foreign medicines or compounded formulations. The last are prepared in hospital pharmacy services and allow doses to be adjusted from adult products or dosage forms to be developed specifically for children.
The study's authors frame paediatric orphan medicines as an unresolved area of Spanish pharmaceutical policy. What to watch is whether regulators and the health system move to widen funded indications and close the age gaps the analysis identifies.
