The Fight for Access: Patients with Rare Liver Disease in Spain Face Growing Challenges

In recognition of Rare Disease Week, The Educated Patient continues its four-part series amplifying the voices of those affected by rare liver diseases. This year, the Global Liver Institute (GLI)’s Rare and Rural campaign highlights the lived experiences of individuals managing rare liver conditions in rural settings, shedding light on their unique challenges and needs.

Spain’s rural communities face significant challenges when it comes to accessing care for rare diseases, particularly rare liver diseases. Many primary care doctors in these areas lack information on these conditions, making early diagnosis and appropriate referrals difficult. When patients need specialized tests and treatments, the journey to hospitals is long and demanding, further complicating access to care.

In an interview with The Educated Patient, Gema Iribar, a patient with primary biliary cholangitis (PBC) who is closely involved with multiple advocacy groups, including Albi España, explains that patient-led advocacy groups play a critical role in bridging these gaps.

One such association is actively working with doctors, hospitals, nurses and educational programs to improve awareness and support for patients. Beyond medical care, Iribar says living with a chronic illness fosters a more engaged and empathetic approach, as patients become more proactive about their treatment and condition management.

For patients in rural communities, the needs are even greater due to isolation. To address this, Albi España provides comprehensive support, including education, emotional assistance and peer connections. A dedicated support group allows members to share experiences and offer encouragement. Additionally, a helpline is available at all times to ensure that patients can reach out whenever they need help.

Adding to these challenges, Spain is now facing a crisis regarding the availability of obeticholic acid, a treatment for PBC. Since July, this medication has lost authorization in the European Union. Currently, it is only available to patients who were already taking it or in special cases under the supervision of a specialist. This leaves patients with PBC with limited or no second-line therapy, leading to immense uncertainty.

Despite this, Iribar believes there is hope on the horizon. New medications are under review by the European Medicines Agency (EMA) and several promising drugs are in clinical trials. While these developments offer optimism for the future, she emphasizes that urgent solutions are needed now to support patients who currently have no viable treatment options.