VIENNA -- Between 27 and 36 million[1] people in Europe are living with a rare disease, yet only around six percent currently have access to an approved treatment[2]. Progress in diagnosis and treatment is often slowed by knowledge gaps, including the lack of clear, reliable, and patient-friendly information needed to support informed decision-making. Drawing on 30 years of experience in researching and developing therapies for rare diseases, AOP Health underscores the importance of collaboration across the healthcare community. On Rare Disease Day 2026, the company joins healthcare professionals, researchers and patient representatives in highlighting the need for closer cooperation to generate, share and better use scarce data to improve care for people living with rare diseases.

“Working in rare diseases since 1996 has shown us that innovation is never a solo effort,” emphasized Melissa Fellner, Vice President Global Therapeutic Areas at AOP Health. “It requires continuous investment in data, open collaboration and information that reaches patients in a form they can use.”

Consistent with this approach, AOP Health is conducting five clinical studies in the field of rare diseases. The company collaborates with researchers at numerous renowned international universities, as well as 58 patient organizations, most of which focus on rare diseases. These partnerships aim to leverage synergies and to strengthen the evidence base.

Collaboration is key

Prof. Dr. med. Haifa Kathrin Al-Ali, a professor of internal medicine at Halle (Saale) University Hospital and Director of the Krukenberg Cancer Center Halle, shares AOP Health’s perspectives. “Without informed patients, it is difficult to generate evidence that truly represents daily clinical reality,” she said. “Close collaboration and an exchange on equal footing are therefore essential, especially in rare diseases.”

Thus, cooperation between physicians, patients and the pharmaceutical industry plays a crucial role in advancing research.

New EU regulations require informed patients

Furthermore, patient involvement is becoming increasingly central to European evaluation and decision-making processes. New frameworks, such as joint clinical assessments (JCAs), and structured evaluation approaches, such as PICO (which defines populations, comparators, and patient-relevant outcomes), explicitly rely on evidence that reflects real-world needs and experiences. Therefore, robust, patient-relevant data and informed patient perspectives are becoming increasingly critical.

However, meaningful patient participation in these processes requires access to appropriate knowledge and competencies. Eva Otter, Vice President of PHA Europe, a patient advocacy group representing people living with pulmonary hypertension globally emphasizes, “To meet European requirements and participate effectively in evaluation processes, we need access to reliable, evidence-based information presented in a language we can understand. Patient-friendly expert information is therefore not an add-on, but a prerequisite for informed participation and credible assessments.”

Rare Disease Day 2026: Extending access to patient-centred information