Devising funding strategies to develop drugs for the treatment of rare diseases remains a challenge as patient groups are often too small
It is estimated there are more than 7,000 identified rare diseases, yet only around 400 have licensed treatments.
A rare disease is defined as affecting less than 200,000 people, but in some cases it could be as few as one or two families. Therefore, due to the smaller end-market, traditional drug-discovery financing models are often inadequate.
While legislation in both the United States and European Union, known as orphan drug designation, incentivises development for rare “orphan diseases” by providing a guaranteed market, it is far from a panacea.
To accelerate the discovery of affordable new therapies for those living with such diseases – around 30 million people in the EU alone, according to the European Medicines Agency – new, innovative thinking and funding models are required.
One burgeoning area of activity is the repurposing of existing or in-development drugs. This means running trials and testing drugs already available on the market or taking therapies that proved ineffective in their original trials, but whose toxicity and safety record are known, and retrialling them to test their effectiveness in specific rare diseases.
Tim Hoctor, vice president of professional services at Elsevier, a global information analytics firm, says the business model to repurpose drugs is strong and compelling, particularly as ongoing research through genomics and other studies have made it easier to test for and understand the molecular detail of rare conditions.
“The more we know about rare diseases in the general population, the more we can potentially target a therapy that has already been developed, and is known to be safe, and bring it to market relatively quickly and cheaply for another disease,” he explains.
This article was also published in The Times.
Heidi Vella 