Open Source Drug Discovery

February 28, 2018
Aled Edwards and Aidan Hollis

A recent study claims that almost 1 million Canadians give up food and heat to afford prescriptions. This is just a harbinger of things to come because the current business model that drives the discovery of innovative pharmaceutical treatments requires higher and higher prices. As one example, Luxturna, a new treatment for an eye disease, is priced at a staggering $1 million per course of treatment.

The problem is that industry’s investments in discovering medicines has generated dwindling returns for decades and will soon be unprofitable altogether. And as a result, pharma invents too few innovative new medicines, and for these has no choice but to charge prices that our health systems can’t sustain. We urgently need to explore new ways of supporting innovation that won’t bankrupt us.

The current pharma model relies on patents and the resulting government-granted monopolies to encourage investment in risky research, as well as competition. But this very system also encourages secrecy. And since most projects fail, competition leads to redundancy and inefficiency. High pricing results.

A new pharma research model based on “open science” offers the possibility of greatly increased efficiency. It would share the risk of research, and reward companies that commit to affordable pricing. But it would also be disruptive, and would require a complete re-think of the incentives, the reward structure and the investment model of drug discovery.

And by sharing the results, everyone would benefit - from the researcher in a university or pharma lab to the critically ill patient hoping to join a clinical trial for a new treatment.

Once, open source computer code was a radical notion in software development. Thirty years ago when IBM was king, open source was considered by many to have no future. But in reality, the explosion in open source code has driven the continuous innovation and disruption that is the digital revolution – and there’s no turning back.

It’s now time for a similar open-source pharmaceutical revolution. And we believe that Canada is uniquely positioned to lead this radical shift.

In fact, we’ve already started. For example, the Toronto-based Structural Genomics Consortium, which does not file for patents as a core principle and makes its research openly and rapidly available for the benefit of everyone, has been able to secure hundreds of millions of dollars of investment from both private and public sectors. And DNDi in Geneva has attracted private funding that led to the development of medicines for sleeping sickness and other diseases – despite their commitment to affordable pricing.

Last December, researchers and business leaders in Toronto, Montreal, and Europe formed M4K Pharma, the world’s first open-science drug discovery company. The company is focusing initially on a fatal form of childhood brain cancer so rare that traditional companies will not tackle it because “the financial numbers don’t work”.

M4K has demonstrated that there’s a clear appetite to support drug discovery in the public realm. In return for a commitment to share its test results freely and rapidly, M4K has already secured several million dollars from government, industry, charities, and individual philanthropists.

So while governments wring their hands over what to do about high drug pricing, this Canadian company is moving forward with an innovative open pharmaceutical research model that will deliver affordable medicines and create high-quality jobs.

At its core, open science means more public investment. In return, companies must commit to make scientific knowledge and materials rapidly available to everyone without restriction on use. This will result in smarter competition, and more affordable treatments.

In our opinion, this model creates a more appropriate bargain between the public and private sectors. We believe our children will look back in amazement that society once allowed commercial interests to dictate something as crucial as the discovery and pricing of new medicines.

The stakes are high. The more we learn about the genetics of disease, the more we realize their complexity: we’re learning, for example, that even Alzheimer’s disease, well-studied and incredibly common, is actually made up of a great many rare sub-diseases, personalized to each patient’s unique genetics. Ideally, physicians would prescribe custom treatments for custom diseases. But under the current business model, society simply cannot afford to pay for the discovery of these personalized medicines.

There is a tremendous opportunity for Canada to transform how medicines are invented. We believe that the federal and provincial governments should provide more support for any pharmaceutical company that carries out its research in Canada and agrees to freely and rapidly share all its scientific and clinical results.

Aled Edwards is CEO of the Structural Genomics Consortium, Chair of M4K Pharma, and Professor of Molecular Genetics and Medical Biophysics at the University of Toronto. Aidan Hollis is President of Incentives for Global Health and Professor of Economics at the University of Calgary.