“For us at AOP Health, data is much more than numbers—it is the key to making rare diseases and their impact on patients and on our healthcare system visible,” says Dr. Martin Steinhart, CEO of AOP Health. The global pharmaceutical company is specialized in research and development of therapies for rare diseases and in intensive care.
Dr. Martin Steinhart, CEO of AOP Health
We are at a turning point where big data and advanced analytics allow us to uncover hidden patterns, generate deeper insights, and shape better treatments. The more robust our data, the better we can serve patients and healthcare systems alike.
Harnessing Data to Assess Therapy Value
Rare diseases often come with limited treatment options and gaps in medical knowledge. Prof. Tanja Stamm, Institute of Outcomes Research, Medical University of Vienna, emphasizes the crucial role of data in evaluating therapies:
Prof. Tanja Stamm, Institute of Outcomes Research, Medical University of Vienna
Data makes the invisible visible by capturing the real-world impact of treatments while patient-reported outcomes and preference measures allow us to understand what truly matters to patients. It is essential to involve all stakeholders—especially patients—early on to ensure we are measuring the right values.
However, data privacy requirements create challenges, particularly for rare diseases where small patient populations make anonymization complex. “While data protection is crucial, access to meaningful data is equally important for understanding diseases and assessing the effectiveness of therapies,” adds Prof. Stamm.
With Them, Not About Them: Engaging Patients from the Start
Beyond data, patient engagement is a fundamental aspect of effective drug development. Dr. Rainer Riedl, Co-Founder and board member of DEBRA International as well as chairman of DEBRA Austria and Pro Rare Austria, emphasizes the need for early and meaningful patient involvement:
Dr. Rainer Riedl, Co-Founder and board member of DEBRA International
Many rare disease patients are highly knowledgeable about their condition and the latest research. They should not just be talked about but have a seat at the table - especially when it comes to setting research priorities. If a drug doesn’t address the patient’s real concerns, it will not succeed.
Study designs should also reflect patient needs, ensuring accessibility and feasibility. “Patients should be able to participate in clinical trials without undue burden,” he adds. “That means considering travel distances, visit frequencies, and other daily-life factors.
Driving Rare Disease Innovation Forward
AOP Health remains committed to leveraging data, patient engagement, and scientific innovation to improve outcomes for rare disease patients. On Rare Disease Day, the company calls for stronger collaboration between researchers, policymakers, and patient organizations to ensure rare diseases receive the attention they deserve.
Giving Rare Disease Patients a Voice: New Podcast-Series
Rare diseases are not only a medical challenge but also a deeply personal journey for those affected. To bring visibility to their experiences, AOP Health has launched a new German-language podcast series, “Ab jetzt ist alles anders? Leben nach und mit einer schwerwiegenden Diagnose.” (“Will everything be different now? Life after and with a serious diagnosis.”)
The podcast takes an empathetic look at the realities of patients, addressing concerns that often remain hidden. By sharing personal stories, it aims to offer support, raise awareness, and foster a stronger sense of community among rare disease patients, their families, and caregivers.
Link to Podcast: https://alles-anders.podigee.io/
About AOP Health
AOP Health is a global enterprise group with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH ("AOP Health") is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions to address unmet medical needs, particularly in the fields of rare diseases and intensive care medicine. The group has established itself internationally as a pioneer in integrated therapy solutions and operates worldwide through subsidiaries, representations, and a strong network of partners. With the claim "Needs. Science. Trust." the AOP Health Group emphasizes its commitment to research and development, as well as the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company’s actions.
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