Open Source Drug Discovery for Malaria Meeting - Session 1, Part 2, Mary Moran

Published by MatTodd on 8 April 2012 - 3:48am



Part of the first session at the Open Source Drug Discovery for Malaria meeting at The University of Sydney, February 24th 2012. Speaker is Dr Mary Moran, Policy Cures.




1) Patents have big benefits but also big drawbacks. The monopoly a patent brings is useful for generating money for R&D investment. That companies see this as attractive has the knock-on that governments don’t have to pay for R&D, and so companies (rather than governments) take on the risk. The downside of the commercial/patent route is that control of the research agenda is set by the people paying for the research (companies and their shareholders). This means there is little incentive for collaboration.


2) (02:50) However, this patent situation doesn’t apply in NTD research since there is no market because there’s no profit margin; i.e. don’t have the benefits or drawbacks. IP in this area is a “ghost”: not a source of money or an incentive. Companies are more happy about giving IP away that is commercially worthless, and we are seeing this happening more and more in NTD research. e.g. WIPO has a database of available IP for NTDs. Patent pools are becoming more common. Screening in collaboration with companies is getting better/easier in the sense of being more collaborative and hence faster.


3) (05:50) Excluding NIH funding, about 40% of R&D in NTDs is collaborative via Product Development Partnerships (becoming the norm). Not open source but still collaborative on same IP, i.e. small teams, working internationally. e.g. GSK – Tres Cantos as the example (very relevant to this discussion).


4) (07:00) The bad thing about NTD R&D IP being worthless: it’s difficult to fund the work. So the question is can we capitalise on the fact that the patents are worthless? $3 Bn a year now put into making NTD products, all of which is raised outside the market. The money comes from governments ($2 Bn), philanthropists ($0.5 Bn, mainly Gates) and companies ($0.5 Bn). Half a billion of the total goes to malaria product development, and of that 200-300 million a year is invested in malaria drugs. That’s for development of new products (R&D funding) up to the point of registration. (Detailed information is available via Policy Cures G-Finder reports.)

Question from Stuart Ralph at 10 min concerns whether the money discussed is for discovery or late-stage development: [audio unclear]

There have been 300 new NTD products in the development pipeline since 2000, 20 registered, 23 in final stages of trials. Of those, 7 new malaria drugs were registered, 5 of which are “useful”.


5) (11:30) Generally there has been an important shift in mindset about the issue of social responsibility – that NTDs are a public problem arising from a market failure. Companies can contribute, but NTDs are not at heart their problem. Australia puts in less money to product development for NTDs, less than any other “investor” country. Stuart Ralph Question (audio slightly unclear) (12:50): What’s the distinction between basic R&D and product development for the investment numbers. Moran – no distinction, grouped together in this analysis (discovery through to registration). There has recently been a review of Australian funding of NTD R&D, with the recommendation being that e.g. AusAID needs to start funding this area. Typically money has come from overseas to fund NTD R&D in Australia.


6) (14:00) Not having patents gives some freedom: funders can control the whole process. The research is usually done in a not-for-profit way, but the funder takes over the risk. The great benefit: increased collaboration. But the cost savings are quite minor. Discovery is cheap, but has a significant failure rate. The open source impact would be to: i) reduce failure rates by ii) providing better ideas up front and faster through sharing data. Though this is good, it is not a big saving because the R&D is inherently quite cheap. Preclinical research is more expensive, but the open source impact here is less, since you at this stage one needs to share work (rather than data) which is harder and there is less to gained from parallelization, and the work needs to be done in any case. Clinical trials for NTDs (costing e.g. $15-20 M) have high failure rates (3 in 4 early on, then 1 in 4 later). Governments are particularly uneasy about failure rates in drug discovery. Can open source make this more efficient? So open source the easiest to apply in the NTD area because IP is not an issue, and needs to be done. But it also has the least additional benefit because people are already collaborating. But it’s a pilot for the big question for the future: application of open source to commercial areas, which is more difficult, but potentially more impactful. Companies know they can’t keep doing things as they currently are. Keeping the process open as long as possible would reduce cost and risk. NTD R&D is the testing ground for this.


[Questions 21-22 minutes have audio issues. One from Hazel Moir about percentage of funding going to Phase III. One from Stuart Ralph is unclear.]


Not publishing data is a problem in the field. Policy Cures releases their G-Finder info freely, and they receive a lot feedback on that. Efficient. Avoids duplication of effort.


7) (23:50) Question from Mat Todd: [problem with audio] The basic impact of open source in for-profit areas: with open release of data, and with many more eyeballs on the problem, people can spot problems with data earlier on, which helps to reduce time wasted.

Moran: agrees, that if you have more people looking at things, you will improve the work, particularly because there will be critical eyes. As she says “When you’re looking at your own baby it’s always beautiful”. Drug discovery is still fundamentally a risky field, even when the molecules look great late in the process. Early failure saves money.


8) (26:28) Question from Hazel Moir (remote) about US orphan drugs act. [Audio problem] Moran: Orphan drugs are not commercially interesting, so market monopolies are of no use. There is a possibility of reciprocal orphan drug arrangements – for e.g. malaria and TB. But the problem with NTDs, is that nobody in the developed world really has them.


9) (28:50) Question from Paul Willis [Audio Problem]. Moran: Infectious disease is easier to make drugs for because there’s an endpoint. Not so for other problems like high blood pressure. Prior to 2000 there was very little research into drugs for NTDs. So we don’t really know attrition rates yet for the most recent work in the area.


10) (31:40) Richard Jefferson Q: Why are we so obsessed with drugs? The US solved many of these diseases with public health interventions. Why aren’t we focussed on those? Moran: Not sure. Drugs are simple. People reach for tablets/vaccines rather than intervention. Jefferson: Are we doing this because we’re too comfortable with the process? Whereas we need to be honest about whether the research is going to give the desired outcome. Moran: one still feels the strong need to do something proactive when you’re doing the public health R&D. Drugs can do that. Jefferson: What’s the timeline of the two approaches? Say we have a hugely promising lead in the lab. What’s the timeline for that to become a viable treatment vs. an intervention timeline e.g. bednets. Moran: It doesn’t have to be an either/or debate. Todd: public health challenges are daunting, e.g. for schisto, where there is no equivalent of bednets because the parasite is in fresh water, so the availability of a drug allows us to do a lot in the meantime.