Rare diseases: when patients drive the pharmaceutical industry forward

Who really assesses the performance of a pharmaceutical company? It is not just financial analysts or regulators, but increasingly the people who live with a rare disease every day and the organisations that represent them. It is from this perspective that the latest verdict on the sector comes: in the “Corporate Reputation of Pharma 2025/2026 – Rare-Diseases Edition”, PatientView’s independent survey which gathered the views of 502 patient organisations active in 63 countries – representing nearly 3 million people – Sobi has retained first place for the second consecutive year, out of 38 pharmaceutical and biotech companies assessed.

This result is complemented by two further achievements for the Swedish company, which specialises in rare diseases: first place in the edition dedicated to haemorrhagic disorders, and second place in the global ranking comparing all therapeutic areas. The highest scores were achieved in the areas of patient-centred care, relationships with patient organisations and services ‘beyond medication’.

“This is not just a ranking, but confirmation that our commitment is recognised by those who, every day, represent and support people living with a rare disease,” explains Carla Rapaccioli, Head of Community Engagement & Communication at Sobi Italia. For the company, advocacy is not a separate activity from the rest of the business: “It means working to ensure that the voices of people with rare diseases and their carers are heard and valued,” says Rapaccioli, “and it is an integral part of our sustainability strategy: creating value means generating a tangible and lasting impact for the communities we serve.”

The operational framework is the global “Unite4Rare” initiative, through which the company says it translates feedback from communities into concrete initiatives: in Italia, over the last 12–18 months, this has included campaigns such as Cl3Ar on kidney health, projects to actively engage people with immune thrombocytopenia (ITP), programmes developed with FedEmo on haemophilia, and the ‘Rare Means Care’ initiative for Rare Disease Day.

However, it is in the area of access, rather than that of recognition, that Rapaccioli identifies the outstanding issues for the Italian system. ‘One of the most significant issues remains early diagnosis: for many rare conditions, the diagnostic process can still be long and complex, with a significant impact on patients and carers,’ he observes. Added to this is ‘equity of access: it is essential that innovation translates into tangible opportunities for all those who need it, and the need to ensure continuity of care and coordination’ throughout the entire care pathway, between hospitals, the local community and support services.