Michele Manion
President and Executive Director, Primary Ciliary Dyskinesia Foundation, Patient Advocacy Partner, Rare Bronchiectatic Diseases Consortium (RBDC)
Clinical trials may help in the creation of rare disease treatments by providing specific insight into a patient rather than approximations based on unrelated illnesses.
Through careful, evidence-based research, trials help answer essential questions: What causes a disease? How does it progress over time? Who is most at risk? And which treatments are safest and most effective?
The wider impact of clinical trials for rare diseases
The knowledge gained from clinical trials guides how diseases are treated and managed. When patients choose to take part, they play a direct role in advancing understanding of their condition and helping shape better care — not just for themselves, but for others who will be diagnosed in the future.
For people with rare diseases, clinical trials are especially important. Because these conditions affect relatively few people, they are often poorly understood, and it can be difficult to enroll enough participants to conduct research. As a result, treatments are frequently “borrowed” from more common diseases, even when there is little evidence they will help — and in some cases, they may do harm.
People with rare diseases deserve treatments designed for their condition, not approximations based on unrelated illnesses. Progress depends on recognising both the challenges of rare disease research and the vital role each patient plays. Every participant matters.
..treatments are frequently “borrowed” from more common diseases,
even when there is little evidence they will help — and in some cases, they may do harm.
How to learn more about clinical trials
Those interested in learning about clinical trials for a rare disease can start by:
- Talking with their doctor or specialist
- Following patient organisations and advocacy groups online
- Searching government databases, such as ClinicalTrials.gov
Clinical trials are not just research studies — they are a path toward better, more precise care for people living with rare diseases.
Learn more about clinical trials from the Rare Diseases Clinical Research Network (RDCRN).
