
Rare disease clinical trials: the urgent need for patients to be heard
Betsy Goodfellow | May 22, 2024 | Feature | |Â Â Alexion, Pharmafocus, Rare Diseases, clinical trials, featureÂ
Ahead of Clinical Trials Day, Soraya Bekkali, from Alexion, AstraZeneca Rare Disease, highlights the urgent need to elevate patients’ voices to accelerate rare disease innovation
Recent estimates show that the number of rare disease clinical trials has risen over recent years, with over 16,000 studies being conducted across the globe.1Â While we must celebrate this progress, there remains an urgent need to accelerate innovation, with no meaningful treatment options for 90% of the 10,000 known rare diseases.2Â Critical to clinical trial success is ensuring the voices of people living with rare diseases are amplified and heard.3,4
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