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Rare Disease Campaign 2020

Drug repurposing is bringing hope to rare diseases

Dr Rick Thompson

CEO, Findacure

“A cheap and widely available drug can help save the lives of patients seriously ill with coronavirus.”

This was the headline of a recent BBC news story that proclaimed dexamethasone as the first proven, life-saving drug in the race to treat coronavirus. In the world of rare disease treatments, ‘cheap’ and ‘widely available’ aren’t terms often heard, so how was this achieved for COVID-19?

Finding a new treatment, quickly

The answer is recycling, or more accurately, repurposing. Dexamethasone has been used since the early 1960’s to treat conditions as common as asthma and arthritis. Scientists investigating COVID-19 have now shown that this 60-year-old drug can significantly reduce the risk of death in patients most severely affected by COVID-19. Dexamethasone’s age and breadth of use means that it is cheap, and widely available, to patients globally.

While it is no miracle cure, dexamethasone is a frontline treatment that will save lives and give clinicians another option in the fight against COVID-19: all of which was achieved in a matter of months.

Drug repurposing – a great option for challenging conditions

This example perfectly captures the value of drug repurposing: rapid research; accessible, well-understood drugs; low-cost treatments; and the ability to deliver a step change in treatment. When attempting to quickly develop a treatment for a poorly understood illness, for patients with a high unmet need, repurposing offers a fantastic route. This is the scenario for an emerging infection like COVID-19 and for the hundreds of millions of rare disease patients around the globe.

If this is the case, why aren’t low-cost, widely-available, repurposed treatments for rare diseases the norm?

Urgency drives change

The fundamental difference – between rare diseases and unknown, emerging infections – is urgency. The pace of COVID-19’s spread, and the imminent risk posed to the global population, created the sense of overwhelming urgency that was needed to fast-track treatments through drug development. By contrast, most rare diseases are genetic, spread on a generational timescale and are not infectious.

The threat of COVID-19 has led to unprecedented levels of research investment, scientific and international collaboration, and the rapid delivery of potential treatments into the clinic. Rare diseases remain something of a policy afterthought.

Repurposing is a growing phenomenon in rare diseases, with projects throughout the development pipeline being led by pharma, clinicians, and even patient organisations. Whether it be the repurposing of everolimus for tuberous sclerosis, nitisinone for alkaptonuria, or tamoxifen for Duchenne muscular dystrophy, the research is happening.

What is holding the research back, is that the urgency to develop rare disease treatments is rarely felt beyond the confines of our community.

Rare unity can deliver future change

COVID-19 has made the public aware of the true potential of drug repurposing; and has shown what can be achieved when a sense of urgency drives all players in the drug development process. Sadly, for too long, the urgency felt by patients, parents and carers living with some of the world’s rarest conditions has not been translated into a drive for new treatments. By acting as a unified rare disease community, to truly harness that urgency, we must urge decision makers to learn from the example set by COVID-19. In this way, we can help deliver new funding, incentives and pathways for rare repurposing, and deliver equitable and affordable healthcare for all.


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