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How can we care for underserved children affected by life-threatening rare diseases worldwide?

by Ashoka | ashoka | Ashoka UK
Thursday, 14 July 2016 09:15 GMT

* Any views expressed in this article are those of the author and not of Thomson Reuters Foundation.

While working at Merck Serono in the R&D knowledge management department, Caroline Kant discovered many drugs sitting on the company’s shelves, waiting to be researched on and which could become part of a much needed solution to treat rare diseases. As a mother of a child affected by an unidentified rare disease herself, Caroline decided to devote her professional life to giving a chance to these unexplored treatments that could be life-changing to so many other children affected by underserved diseases worldwide.

Her basic premise was the belief that regardless of whether a disease affected 2,000 people or 20 million, every patient should have an equal right to medical treatment. It is with this aim that in 2013 she founded the EspeRare Foundation, a non-profit organization based in Geneva.

 

 “I dream of a world where there is an accessible treatment for every single known disease, no matter how widespread or how rare.” Caroline Kant, Founder of the EspeRare Foundation, Ashoka Fellow since 2014 and Swiss CFE woman entrepreneur of 2015

 

Taking these diseases collectively, it is clear that they are a major healthcare burden: they affect 250 million people worldwide. Of the 7000 classified rare diseases, half affect children and only 5% have an approved treatment today. It has never been a priority for pharmaceutical companies to develop treatments for such small markets. The combination of scientific and funding challenges in those diseases implies that medical research is scarce and little treatments ultimately reach patients.

 

With the EspeRare Foundation, Caroline has implemented a novel business model that reduces the risk, cost, and time required to develop treatments, ultimately bringing unexplored, yet existing, investigational drugs to these small populations of patients at an affordable cost. With this model, she is accelerating the cost-effective development of new medicines based on an open research approach that engages the patient community, the pharmaceutical companies and medical centers of excellence. This model is complementary to the traditional commercial high-innovation R&D model and makes it possible to increase accessibility to treatments while supporting the sustainability of healthcare.

 

According to Caroline: “The idea started with the discovery within Merck Serono’s dormant portfolio of a gold nugget, Rimeporide, which had been developed for cardiac failure and that EspeRare is now repositioning for children affected by a deadly condition called Duchenne Muscular Dystrophy.”

 

EspeRare applies a proprietary bioinformatics methodology to identify therapeutic “gold nuggets”. It then works with Merck Serono and other pharmaceutical companies to gain development rights to these investigational drugs sitting on their shelves. This puts the foundation in a position to give a chance to these opportunities making them attractive to commercial partners once they have reached human proof of concept. At that point, pharmaceutical partners acquire these opportunities for late stage development and commercialization in exchange of access to medicine commitments and financial returns that EspeRare can then re-invests into addressing other rare diseases.

 

Today, EspeRare has achieved to prove its model by repositioning Rimeporide in Duchenne Muscular Dystrophy, validating the therapeutic potential of this drug. If late stage clinical testing confirms the therapeutic benefits, Rimeporide could reach the market by 2020, finally offering a treatment to the 50’000 children suffering from that disease worldwide. In parallel, the foundation is developing 3 other treatments for other deadly conditions. In particular, it is currently putting back on the market a therapeutic intervention that has already proven its medical benefits for newborns with severe cardiac defects. By revamping the distribution of this medical device in Europe and philanthropically in Africa, the foundation is set to save the life of infants with rare heart conditions in a year’s time.

 

Caroline’s venture philanthropic model is currently growing and seeking external funding to finance rapid scale-up of additional programs. In line with it model, reinvestment of research and development profits should enable EspeRare to achieve financial self-sustainability within ten years, allowing to expand health impact even more broadly to these vulnerable patients.

 

When replicated and scaled, Caroline’s model could be the solutions to provide affordable drugs for a significant proportion of the 1 in 10 people worldwide suffering from a rare disease.

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Caroline Kant is an Ashoka Fellow and an Ambassador for the UBS Social Innovators Programme, run in partnership with Ashoka – a global search and support package for social entrepreneurs looking to scale their impact through social business and social investment.  To apply to the UBS Social Innovators Programme, click here or follow the conversation online by searching #UBSInnovate on Twitter.

This article was first published on Virgin.com on 11th July 2016.

 

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