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U of Toronto researcher creates new model for pharma company in bid to make drugs affordable for patients with rare diseases


By Jesse Schwartz
Published: November 8th, 2017

A researcher from the University of Toronto (UT) is launching his own pharmaceutical company using a novel business strategy that he hopes will lead to personalized therapeutics that patients with rare diseases can actually afford.

Aled Edwards’ new company M4K Pharma (Meds for Kids) is capitalizing on the rise of advanced genomics research, which has allowed for many illnesses to be splintered into smaller categories of disease, thereby shrinking the groups of people that fall under a specific illness and giving rise to personalized medical approaches.

Unfortunately, however, with the average cost of developing a single new drug at $2.6 billion, pharmaceutical companies are unlikely to focus on treating maladies that only affect a few thousand patients each, unless they charge enormous sums for the medications.

 “This is currently true for hundreds of diseases,” says Edwards. “It’s only going to get worse. Do the math — there’s probably a hundred kinds of autism, and each one might need a different treatment. There’s no way it will work with the current system. We need to reinvent how the business model works.”

Alongside his business partner Owen Roberts, Edwards launched M4K, an open source drug developer that seeks cures for diseases that major pharma companies have deemed too rare and too risky for drug development. Edwards’ strategy is to gather, organize and accelerate open source research that is focused on rare diseases and funded through public and philanthropic resources around the world.

M4K will also encourage researchers to publish and share their discoveries rather than patent them, and it will protect its marketing rights only through the test data exclusivity provided by regulators. Any profits will be funneled back to the company’s parent, Agora Open Science Trust, which will reinvest the funds in further research.

 “A key part of the strategy is eliminating the idea that the people involved need to personally benefit from this,” says Edwards. This is especially relevant to the researchers, who are submitting their work with the understanding that it would primarily benefit patients.

Edwards has prior experience with open source concepts, namely through the research and development company he cofounded, SGC, which has raised about $200 million in private sector investment — proving that charitable concepts can actually be good business.

 “We’re not doing it necessarily for peace and love purposes, although that’s definitely a consequence,” says Edwards. “We think it’s the most cost effective way to reduce the risk.”

Source: U of T News

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