Founded in 1996, AOP Health has spent the past 30 years focused exclusively on developing therapies for rare and complex diseases. Dr Steinhart joined AOP Health in 2019 as Chief of Research & Development and was appointed CEO in 2023. With this long-term commitment in mind, Dr Steinhart began by sharing the origins of AOP Health.
Rare Diseases Cannot Wait: Why Innovation Must Move Faster
February 26th 2026
To mark Rare Disease Day 2026, AOP Health Co-CEO Dr Martin Steinhart shares his thoughts on the evolution of rare disease care, the challenges that remain, and why collaboration is essential to delivering innovation at the pace patients deserve.
Dr. Martin Steinhart
Innovation in rare diseases begins with a simple belief: no patient should be forgotten.
What inspired the founding of AOP Health 30 years ago?
Back in 1996, the pharmaceutical industry was focused on blockbuster drugs - no one was talking about rare diseases. We knew there were more than 6,000 different rare diseases impacting millions of people, and most had no available treatment.
This sparked the founding idea of AOP Health: to focus purely on forgotten and under-investigated diseases in search of solutions.
Why is rare disease drug development different from other therapeutic areas?
Rare diseases are defined by scarcity. When a disease affects fewer than one in 2,000 people, every aspect of drug development becomes harder.
On average, it can take up to 15 years to bring a therapeutic solution to the market, around 20% longer than in non-rare diseases.1
Our clinical trials involve very small patient populations, which is a challenge that is often compounded by limited awareness and delayed diagnoses. This makes generating robust data difficult. A trial for a rare disease may include only around 100 participants. If only a few patients are recruited each year, enrollment can take several years. Finding the right candidates at the right time can be like finding a needle in a haystack.
Has progress been made in rare disease innovation over the past three decades?
Over the past 30 years important progress has been made. The adoption of the Orphan Regulation by the European Parliament in 1999 marked a key turning point.2
Over the next 20 years we saw a twofold increase in the number of therapies for orphan diseases each year. Today, more than 200 novel orphan medicines have been approved since its introduction.3
While approvals for rare disease treatments have accelerated, these 200-plus therapies still account for only around one in ten medicines approved in the European Union.4
Critically, around 95% of rare diseases still do not have an approved treatment.5
For patients, progress has simply not come fast enough, and many are still searching for answers and support.
Dr. Martin Steinhart
Collaboration and partnership are essential in rare diseases, especially in early development and across the different stages of clinical development.
Why is collaboration so critical in rare disease research and development?
Through partnership with centres of excellence and patient advocacy groups, we can all work together to better understand the disease itself, improve diagnoses, and generate better data.
What do you see as the biggest barrier to faster progress in rare diseases?
The greatest barrier I see in rare disease drug development is not a lack of knowledge, but the way we run clinical trials.
While the world around us has continued to evolve, the way we conduct clinical trials has changed very little over the past 50 to 70 years. They were designed for large patient populations and when applied to rare diseases, where patient numbers are small and recruitment is inherently challenging, a one-size-fits-all approach inevitably leads to delays. If we want different outcomes in rare diseases, we must adopt tailored systems that reflect their reality.
How can innovation help accelerate rare disease drug development?
To accelerate drug development timelines, emerging technologies such as artificial intelligence and big data, offer real opportunities to rethink how clinical trials are designed and delivered.
We have already seen the EMA accepting digital ‘synthetic’ control arms in clinical trials for several years, where nearly one in five EMA-approved cancer drugs included synthetic controls.6
Patient candidate identification through electronic health records and registries has significant potential for radical optimisation. The same applies to the selection of endpoints based on real-world data. There is also considerable opportunity in the use of existing molecules for treatment, particularly in cases where the links are not immediately apparent. To me, this is the biggest opportunity before us.
By embracing such innovations, we have the potential to uncover new treatments for old diseases much more rapidly, provided we are ready to adapt and move faster.
What does Rare Disease Day mean to you personally?
Rare Disease Day is an opportunity to pause and reflect, but not to stand still.
It reminds us of what we’ve achieved since its inception in 2008 and inspires us with what once seemed impossible.
I will always be inspired by the rare disease community, standing up for others, often while suffering themselves.
Accelerating innovation is not optional, it’s a responsibility.
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. (aop-health.com)
References:
1 PhRMA. ICYMI: Orphan drug development brings unique challenges. Available at: phrma.org
Accessed February 2026
2 European Medicines Agency. Legal framework: orphan designation. Available at: ema.europa.eu
Accessed February 2026
3 Hahl, E., Kurko, T., Koskinen, H. et al. EMA approved orphan medicines since the implementation of the orphan legislation. Orphanet J Rare Dis 20, 266 (2025). Available at: doi.org
4 European Medicines Agency (2021) Available at: publicaties.vereniginginnovatievegeneesmiddelen.nl/C011_UK_WP.pdf
Accessed February 2026.
5 Global Genes. RARE Diseases Impact More People Than Cancer and AIDS Combined. Available at: globalgenes.org
6 Xiaomeng Wang, et al.. Current perspectives for external control arms in oncology clinical trials: Analysis of EMA approvals 2016–2021. Journal of Cancer Policy, 2023;35:1-6.
Photocredit Stock-Photo:
Photocredit Portrait: "AOP Health/David Bohmann"