Rennie McCarthy, Chief Executive Officer at Stealth Biotherapeutics, discusses clinical trial development challenges for ultra-rare diseases.
Stealth Biotherapeutics is developing elamipretide, a mitochondrial protective agent, as a possible treatment option for mitochondrial myopathy and Barth syndrome – two ultra-rare diseases. However, clinical trial development comes with its own challenges.
The limited patient population makes it difficult to power clinical trials properly that appease both the regulatory bodies and payors. As an example, Ms. McCarthy notes that one clinical trial may often deplete most of the eligible patient population. That can dramatically impede drug development. What is needed, according to Ms. McCarthy, is more guidance from regulatory bodies like the Food and Drug Administration (FDA), that allow for less conventional methods to monitor efficacy, such as real-world data sets, natural history studies, and biomarkers.
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