From treatments priced beyond the reach of the poor to a refusal to tackle some of the world's most urgent health threats, critics of “big pharma” have seldom had it so easy.
Of the $157 billion spent by the private sector on health research and development (R&D) in 2016, just $505 million was for neglected diseases – conditions such as malaria and tuberculosis which affect billions of the poorest people worldwide.
But what if there was another way of developing the medicines we need? A way that eschews market incentives that stop pharma companies from developing medicines for diseases of poverty and does away with the secrecy that shrouds drug development.
According to advocates of open source pharma, there is.
“The goal of the pharmaceutical industry is to maximise financial return on investments through sales, high prices and anything that can deliver a lot of profit ,” says Els Torreele, executive director of the Access Campaign at Médecins Sans Frontières. “This is obviously not the case for diseases of poor people or neglected populations.”
“Scientific progress necessitates building and learning from what others are doing,” she says. “Open source is a matter of going back to the common sense of how we can best make progress by not working in isolation and secrecy.”
Inspired by the open source movement in software, open drug discovery projects make their data and ideas available on the internet to anyone.
Matthew Todd, a professor of drug discovery at University College, London and one of the founders of the open source pharma movement believes that open source could potentially transform the way we find cures.
“You can work with the best people anywhere and you can bring in expertise that you didn’t even know existed,” he says. “We’ve seen it work really quickly. It can accelerate the science”.
Although a growing number of projects use the label, Prof Todd says only a handful are truly open source. This means that none of the ideas can be patented and anyone - including pharmaceutical companies – can contribute.
“It’s a bit like Wikipedia. Anybody can take anything we’ve done and do anything they want with it,” he says.
One such project is Open Source Malaria, which is currently working on two molecules to beat the disease. One of them, says Prof Todd, is “a small number of experiments” away from clinical trials.
Another, the Pandemic Response Box – an initiative from non-profit Medicines for Malaria Venture (MMV) launched earlier this year – contains 400 antiviral, antibacterial and antifungal molecules that could potentially work against pathogens such as the Zika and Ebola viruses. The boxes are shipped out free of charge to any researcher who requests one. The only catch is that any data from experiments on the molecules have to be publicly shared.
At the launch of the box, Dr Timothy Wells, chief scientific officer of MMV, said the concept was a follow up to what the organisation did with malaria.
"We built a box of malaria drugs, that had come out that weren't widely available. If it works like it did for malaria it will create a common set of compounds that everyone can test. It should further collaboration."
According to Dr Torreele, open source is one of several new ways of doing R&D for drugs that don’t turn a profit that have emerged in the last two decades. Among them are also publicly and philanthropically-funded partnerships such as the Drugs for Neglected Diseases Initiative (DNDi).
And the lack of market incentive doesn’t just apply to drugs for diseases that affect the poor. Despite a looming antimicrobial crisis, there’s a dearth of research into antibiotics - another medicine for which profit margins are low.
For Dr Torreele what open source and public and philanthropically-funded drug discovery have in common is they both challenge the idea that developing drugs has to be costly.
“DNDi and other [public-private partnerships] have shown that it’s possible to develop new drugs and new treatments that address people’s health needs at an affordable price and at a fraction of the cost of R&D claimed by pharmaceutical companies,” she says.
And it’s the openness that brings down costs, says Piero Olliaro, a professor at Oxford University and former head of implementation research at the World Health Organization’s special programme in tropical diseases.
“Whatever is done - if it’s in the public domain, if you fail everyone knows it failed and they won’t do it again,” says Prof Olliaro. “The current system is highly inefficient and the reason for some of that is the secrecy.”
It’s not clear how much it currently costs to develop a new drug and figures given by pharmaceutical companies include the cost of failure for drugs that don’t see the light of day. One study by Tufts University put the figure for coming up with a new drug at $2.6 billion, a figure Prof Olliaro disputes.
“Those figures are made up and they are made up to justify the high price of medical products,” he says.
Although there’s no precedent yet of a “born open” drug coming to market - most molecules are in the early stages of development - open source’s advocates say drugs developed their way would be far cheaper.
Funding still however, remains a stumbling block, according to Christine Ardal, a senior adviser at Norway’s Institute of Public Health who has done research into open source pharma.
“Open source is not the silver bullet that’s going to solve this problem. You still need to have a lot of funding,” says Dr Ardal. “For neglected tropical diseases the funding comes almost solely from the public or philanthropic sector, but it’s costly so you also want private investments in R&D but with open source there are concerns about intellectual property rights.”
One area where she says an open source approach could have a lot of potential is in cases where the science is not well understood.
“It might work well in some instances where there is little market or where there is just not enough knowledge and you need some sort of collaboration,” says Dr Ardal.
Although still a niche approach, should an open source molecule make it all the way to market, its proponents say it has the potential to transform the way that drugs are discovered.
“The potential is enormous,” says Prof Todd. “Once we get a precedent or investment in a proper pilot project we have the chance to develop a competing model for the pharma industry - it’s that kind of scale.”
Prof Olliaro is enthusiastic about the potential for open source pharma but says it will take more than good will to bring a new drug to market.
“You can’t expect that to happen by chance. You need to coordinate and mastermind it,” he says. “You have to find a way of channeling all the energy from all the people who might be interested in contributing into a coherent process with a purpose.”
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