Open drug discovery methods explored

TDR news item
26 September 2013

New ways of developing affordable medicines were the subject of a meeting held recently at TDR. Experts from a variety of fields, including law, pharmaceutical development, ethics and national medical delivery, came together to explore how drug discovery can be done in an open and collaborative manner. Today, the internet can provide more access to data and introduce efficiencies in drug screening, discovery and development. However, policies and processes need to be developed to allow ways to share the data and use the research results.

"What does open really mean?"

Dr Matthew Todd, University of Sydney

TDR Director John Reeder told the participants that this more open approach to research is an area of growing interest, and is being discussed by Member States at the World Health Organization. This 19 September meeting was set up as a satellite event at the end of the Open Knowledge Conference held in Geneva. The convener and chair was Dr Matthew Todd of the University of Sydney and the event was supported by TDR and Medicines for Malaria Venture.

A panel with a wide range of expertise provided an overview of their work in exploring new approaches. These ranged from academia (Oxford University and the Structural Genomics Consortium) through non-governmental organizations (Drugs for Neglected Diseases initiative, DNDi; Medicines for Malaria Venture, MMV; TDR; the World Intellectual Property Organization) and industry (GlaxoSmithKline). Drug discovery and development is a complex field requiring consideration of the economics, law and policy beyond the science. The panel presentations were used to initiate a deeper discussion on new drug discovery approaches that could be set up and used, particularly for health problems where there is a low economic return for the developers.

The question at the forefront of the meeting was, "What does open mean?" Robert Terry, Knowledge Manager for TDR, set out the importance of not only providing free access but also how reuse of the data is managed, since decisions early on in a long drug discovery campaign impact the economic model for how a compound is developed into a drug. Dr Todd described the scope of approaches and reviewed his model of open source, where a research problem is posed openly on the web and other researchers are invited to contribute to solving that problem. All the collaborative work is shared and no intellectual property or patents are taken out. This approach using a network of researchers, some of whom may never have worked together before, is under test in the Open Source Malaria project. MMV, one of the funders of that project, is engaged in other open efforts to stimulate discovery that include the Malaria Box, a physical collection of new anti-malarials that is being shared with researchers around the world. DNDi is also working with researchers to promote a more open and collaborative approach to the most neglected of diseases, and described the development process of the anti-malarial compounds artesunate-amodiaquine and artesunate-mefloquine that are patent-free and licensed to companies for deployment.

The question of whether patents were necessary and whether they promote innovation was keenly debated, but not resolved. Tom Bombelles described WIPO's Re:Search initiative that makes intellectual property for pharmaceutical compounds available to researchers through the use of royalty-free licenses, and most importantly, the data and know-how to utilize those compounds via an online platform that they see as encouraging open innovation. Chas Bountra of Oxford University presented an alternative approach with examples in the kinase field that encourage research in underdeveloped target areas. The Structural Genomics Consortium's public deposition of data stimulated a great deal of downstream research activity, particularly where the clinical efficacy of compounds could be demonstrated and commercial interest focused. There was particular debate on where public or private funds should (and could) be used to mitigate the risk in research, such as in covering the costs of failure. While open source drug discovery may introduce efficiencies into upstream research and speed discovery, it remains less certain how utilizing open innovation per se would improve the funding situation and development of a product where the final market is poor.

Many of the issues raised in this session can guide future debate on how to drive innovation in the discovery of tomorrow's medicines. The meeting was recorded and will be placed online shortly to help stimulate further input. As Dr Todd summarized, further discussions will be needed to determine how best this kind of research can be carried out, and this session was a very useful start.

For more information, please contact

Dr Piero Olliaro