Rare Disease Day 2026
On Rare Disease Day, global attention turns to the millions of people living with rare conditions. While each individual disease affects only a small number of patients, rare diseases collectively impact more than 300 million people worldwide.
What Are Rare Diseases?
In the European Union, a disease is defined as rare when it affects no more than 1 in 2,000 people. Many rare diseases are chronic, severe, and often life-threatening. A significant number manifest early in life and require long-term medical care.
Due to small patient populations, research and drug development for rare diseases have historically faced economic and scientific challenges. This is also reflected in the term “orphan diseases,” describing conditions that for many years lacked sufficient commercial incentive for therapeutic development.
Although regulatory frameworks such as orphan drug designation programs have improved development incentives, manufacturing and clinical supply of these therapies remain highly specialized and demanding.
Unique Challenges in Rare Disease Drug Development
The development of new active pharmaceutical ingredients (APIs) and New Chemical Entities (NCEs) for rare diseases differs significantly from large-volume pharmaceutical production. Key challenges include:
- Small and well-defined patient populations
- Limited GMP batch sizes
- Complex and innovative molecular structures
- High regulatory and documentation requirements
- Tight clinical development timelines
These factors require flexible production capabilities, strong analytical expertise, and close collaboration between sponsor and manufacturing partner.
ChemCon as a Specialized CDMO Partner
For more than 25 years, ChemCon has supported pharmaceutical and biotechnology companies in the development and GMP-compliant manufacture of high-purity APIs and NCEs, from preclinical stages through clinical development.
Our core strengths particularly relevant for rare disease programs include:
- Manufacturing of small to mid-scale GMP batches
- Dedicated equipment to ensure highest purity and prevent cross-contamination
- FDA-inspected infrastructure and strong quality systems
- Comprehensive analytical and regulatory support
As a specialized CDMO based in Germany, ChemCon combines scientific expertise, regulatory reliability, and operational flexibility, critical elements for advancing innovative therapies for small patient populations.
Enabling Innovation Where It Is Needed Most
Rare diseases may affect only a few individuals at a time, but the unmet medical need is often significant. Supporting the development of innovative therapies for these patients requires precision chemistry, high quality standards, and committed collaboration.
On Rare Disease Day, ChemCon reaffirms its commitment to enabling progress in rare disease drug development, providing reliable GMP manufacturing solutions for therapies that can make a meaningful difference.
