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Sunday, July 24 2016 @ 03:22 PM CDT

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What is Open Source Pharma (and why should you care)?

Monday Witness

I’m writing this over the north Atlantic as I return from most of a week of very compelling meetings at a castle in Germany. Nominally, the subject of the discussion was something generally referred to as “open source pharma” (OSP). But more particularly, the meeting was about working towards saving the millions of lives a year that are lost either to so called “neglected diseases,” or because those stricken cannot possibly afford the price of the drugs that could provide a cure. Even though the actual cost of manufacturing the drugs they so desperately need may be only pennies a pill.

What’s a neglected disease? It’s one that as much as half the world’s population is at risk of dying from, but which nevertheless doesn’t present an attractive profit target for any of the major pharmaceutical companies. Why? Because once again, those at risk can’t pay as much as patients in the developed world, and those in the developed world rarely get the disease. Diseases like malaria and drug resistant tuberculosis, which together claim millions of lives a year in developing countries. Open Source Pharma holds the promise of addressing these appalling realities, and it’s therefore imperative that it take hold in the marketplace.
 
While the name doesn’t fully explain what OSP is, it certainly suggests what it’s all about. But before we turn to a detailed definition, let’s look at what the alternative looks like. Today, pharmaceutical development is the very epitome of a proprietary business. Research results are closely guarded, developments are immediately patented, and ingenious strategies are employed to extend that patent protection for sometimes decades after the original drug should have become generic. Yet at the same time, the great majority of actual innovation goes on in universities rather than at the big pharma’s, often funded by government and non-profit grants. When innovative new startups do pop up, they are soon purchased by the big pharmas, thereby reducing the diversity of innovation.
 
A major reason that this system has evolved is because of the enormous cost of clinical trials and the low success rate of promising drugs once they are entered into that process. The result is that the big pharmas are constantly in search of the next blockbuster drug, and loath to lose their monopoly control over one until all options have been exhausted.
 
At the same time, there are many hundreds of promising discoveries that never go anywhere. Perhaps the discoverer is a startup that is acquired before it gets to clinical trials and the acquirer has no interest in seeing it through, or a big pharma’s strategy changes, or there’s a staff reorganization and the project is killed in the process. When this happens, the discoveries go on the shelf, usually never to be touched again.
 
There are also many hundreds of existing drugs that can be highly efficacious for other disease conditions. Doctors are frequently surprised when a patient makes a miraculous recovery, with the only identifiable change in their regimen being a prescription for an unrelated drug for a different malady. Most of this anecdotal evidence ends up going nowhere, because there is no easy way for overworked physicians to post and aggregate such possibly random, but occasionally very significant observations. The possibilities here are enormous, because so many of these drugs are already generic, and they have already been approved by the appropriate authorities. Such “off label” uses of very inexpensive, repurposed drugs can be immediate, and lifesaving.
 
Proprietary practices have other pernicious effects as well. Multiple pharmas may be exploring the same drug possibilities at the same time. Worse, one company may have already learned that the line of inquiry is a dead end, either because the animal trials did not replicate in humans, or because of toxicity. Similarly, where research is not published until the patents have been filed and the drug introduced, a decade or more can pass before other researchers can benefit from and build upon it.
 
This should begin to provide a clue as to what OSP is, and why it matters. What if, for example, all of the results of research were added into a common, publicly available database upon discovery? Unproductive pathways and toxic molecules could be avoided, saving enormous amounts of time and funding. And new fundamental discoveries could immediately become the foundation of additional research and promising new drug candidates. Proprietary pharmas would risk little by sharing failures and toxicities, and would realize significant costs savings as a result.
 
So the first fundamental concept behind OSP, as the name suggests, is transparency of data and results, at the earliest opportunity. And especially so, where the data was derived and the discoveries made using public funds.
 
But the concept can also be taken farther, extending to roughly incorporate the methodology of open source development. Counting in the costs of failures as well as successes, major pharmas estimate that it takes about $1.5 billion dollars to bring a new drug to market. At each step of the way, highly paid professionals and facilities are employed.
 
But imagine, if you will, if we were to map out the entire drug development process from initial theory through research, clinical trials and regulatory approval (and there are many, many sub-steps along the way). After we’ve produced our detailed work flow model, we can then spec out the type of IT platform needed to support that end to end process, and also the type of database needed to hold and share all the resulting data.
 
Of course the software tools we will want to use will insure that all of the data from one end to the other is in compatible formats to maximize efficiency in developing that drug, and also to permit maximum universal use of the resulting database by other researchers. The software tools comprising the resulting framework will be best of breed open source tools (many of which already exist), and to the extent that there are gaps, existing tools can be optimized, or new ones created.
 
The work flow model will also specify exactly what number of individuals, with what skills, are needed at each step along the way. It will also identify the points at which crowd sourcing of knowledge from appropriate experts would be helpful, as well as what types of funding might be appropriate and available at which junctures. Importantly, it can also be designed in such a way as to dramatically drive down the costs at every stage, from beginning to end.
 
The completed model can then be exposed to the database of interested professionals that have registered as being willing to provide volunteer efforts. Their rewards will be personal, but also professional, as they will receive credit for discoveries, as well as the ability to publish their results.
 
While so extensive an ecosystem is a long way from being fully realized, neither are we just at the starting line. Open Source Malaria is just one example of a project that is already making meaningful progress on attacking neglected diseases by expanding on open source software development techniques.
 
The result of creating this development model, with the associated tools and professional resources, would lay the foundation from which an alternative drug development ecosystem can rise. Once it exists, additional resources can be added. Imagine, for example, a non-profit entity that would assemble and maintain the IT infrastructure and databases needed to support the entire end to end process, and make it available free of charge, provide training as and when needed. Another might provide a master insurance policy to make the clinical trials possible, as well as pull together ethical panels for hospitals in emerging countries that do not have the resources to fulfill that function internally.
 
The likelihood of funding new projects would increase as well, as the costs of achieving real results would drop. One could imagine a billion dollar fund, made up of equal contributions from governments, foundations and even big pharmas, the latter benefiting from access to the data and selected rights in the discoveries.
 
The results of such a model would be profound. A non-profit brought a generic drug back to market to treat a neglected disease, and expended only $26 million to do so – orders of magnitude less than the usual cost for a new drug. That’s a number that is within the reach of private foundations to provide.
 
While this is the briefest of overviews, it should make obvious that something similar to the distributed methods used for open source software can also be used to change the way that pharmaceutical development is carried out. There are, of course, significant differences from open source software, since expensive equipment is required, and sometimes teams will need to work in the same physical lab. But these are details. After all, the Manhattan Project to develop the atomic bomb occurred at breakneck speed in scores of widely separated research facilities. And that was long before the Internet existed.
 
But there is one dimension to OSP that is very different from the creation of software. In the years after the drugs were developed that changed HIV/Aids from a death sentence to a treatable chronic condition, more than 10 million people died of the disease in Africa alone, largely because the drugs cost $15,000 per year per patient. And yet within only a few years generic drug manufacturers were able to make a full course of treatment available for $350 per person per year, and then for under $100. But the people continued to die, ten million of them, because the big pharmas that held the patents would neither drop the price, nor permit the generic drug manufacturers to distribute the medications.
 
Could this happen again? The only intervening change has been the adoption of an international treaty that binds countries even more tightly to respect the inviolability of drug patents than before.
 
It was a rewarding and humbling experience to take part in this meeting, along with about 30 other people, most of whom were prominent researchers from Europe, Asia and Africa and leaders from organizations like the Tata Trusts in India, the Foundation for Neglected Disease Research, Médecins Sans Frontières,  the European and Developing Countries Clinical Trials Partnership, the government of India, the Centre de Recherches Interdisciplinaire, Open Source Drug Discovery, and the Open Societies Foundation.
 
It has been observed that when one person dies, it’s a tragedy, but that when a million do, it’s a statistic. Perhaps the best way to bring the reality of non-existent and over-priced medications back into focus is to quote from an essay written by James Arinaitwe, one of the members of Open Source Pharma, the (so far) loosely affiliated group of individuals and organizations that have now met for the second year to further this cause:
 
I grew up in rural Western Uganda, where two of my siblings succumbed to measles before their fifth birthday and my father to HIV/AIDS before I turned 10. I often wondered why so many preventable and treatable diseases were still killing the world's poorest and most vulnerable people. Could it be possible that big pharmaceutical companies and big, bureaucracy-laden governments were so vertically aligned in their approach to bringing life-saving medicines to market, that they rarely saw any reason to find solidarity with the communities that would eventually benefit from their inventions and policies?

My father and my baby sisters did not die because the vaccines for measles or the antiretroviral drugs for HIV were not available. They died, in large part, because life-saving vaccines and medicines were priced beyond our reach.
 
I hope to become more involved in furthering the cause of OSP, and as I do, I’ll continue to write about it, beginning with more detail regarding the underlying concepts. If you’d like to learn more, visit the Open Source Pharma site here. 
 
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