Extent of R&D Spending on Neglected Diseases

♠ Posted by Emmanuel in , at 8/05/2009 09:22:00 AM
I have, ah, neglected to mention this interesting study on funding for neglected diseases which is the most informative one I've found to date on the topic. The Gates Foundation commissioned this study with the self-explanatory title of "Neglected Disease Research and Development: How Much Are We Really Spending?" It was run in conjunction with the creation of G-FINDER, arguably the most comprehensive database on neglected disease R&D. The way I like introducing this topic to those unfamiliar with it is by contrasting big pharmaceutical firms traditionally spending massive sums on often cosmetic wants in the West--think of your Viagra and Botox. At the same time, life-threatening neglected diseases in LDCs lack for attention since these markets are not viewed as being profitable enough. While I dispute this notion, I must also admit that there have been limited examples of firms operating profitably with a longstanding or even exclusive focus on drugs for neglected diseases.

Encouragingly, the authors find that spending by some Big Pharma firms on drugs for neglected diseases is approaching that by official funders. While I remain convinced that it is more likely that firms in the developing world will provide their own affordable solutions to neglected diseases than a wholesale change in attitude by Western pharmaceutical giants, these results may yet prove me wrong. And, of course, why wouldn't I want to be wrong?

The article in its entirety is available either in HTML or PDF form from the well-respected online journal PLoS Medicine. What I excerpt here are the concluding observations:
We therefore restrict ourselves to a few observations. The first is that research funding is highly concentrated and has little correlation with burden of disease, as measured by DALYs [disability-adjusted life years].

For instance, HIV, TB, and malaria accounted for 125 million DALYs in low- and middle-income countries in 2004 and received nearly 80% of total funding; while pneumonia and the diarrhoeal illnesses accounted for 165 million DALYs in these countries in the same year but received less than 6% of total funding. Likewise, helminth infections received less than half the funding of kinetoplastid diseases although their disease burden was three times higher (12 million DALYs in 2004 compared to 4 million DALYs for the kinetoplastid diseases); while dengue had a disease burden 20 times lower than helminth infections at 600,000 DALYs in 2004 but received nearly twice as much funding.

This observation should not be construed as a statement or belief that funding levels can or should be based only on disease DALYs. DALYs are the most widely accepted quantitative indicator of burden of disease, but remain an imperfect measure. For some diseases, such as Buruli ulcer, reliable DALYs are virtually nonexistent; correct estimates can be difficult to obtain in regions with weak data; and DALYs do not reflect the impact of co-morbidities, such as HIV and TB. The DALY comparison is noted here chiefly to give a sense of the differences in scale of funding for different diseases.

It is important to note, however, that funding levels reflect many factors beyond DALYs. The current state of science for a given disease plays a role, since many would-be investors in product development look at feasibility as well as need. Conversely, other investors may specifically choose to invest in areas where science is lacking, believing their best contribution will be funding a breakthrough discovery to unlock the path to new products.

Funders may also look at the state of existing therapies, preferring to focus on areas where no safe effective treatments exist (for example, some kinetoplastid diseases) to areas where treatments exist even though they may be limited or sub-optimal (for example, some helminth infections). Funders may also want to focus on diseases with a higher relative or absolute mortality, such as dengue or meningitis, rather than diseases with lower relative mortality but a higher DALY burden.

The type of R&D needed in a given disease area is also a crucial factor in interpreting the data, since R&D costs vary dramatically depending on the kind of product being developed and how far down the development pathway that product is. Vaccines are far more expensive to develop than drugs, and drugs than diagnostics. Clinical development is also far more expensive than pre-clinical or discovery research. For instance, out-of-pocket development cost of a diagnostic test can be in the low tens of millions; while clinical development cost of a novel fixed-dose combination anti-malarial is estimated at US$30 million, and of a malaria vaccine at just over US$100 million. Thus, far more investment would be needed to develop a meningitis vaccine than a TB diagnostic, irrespective of the lower burden of disease for meningitis or a more advanced state of meningitis vaccine technology.

Despite this, the concentration of funding on AIDS, TB, and malaria nevertheless suggests that investment decisions are not only influenced by scientific or epidemiological considerations, but may also be influenced by factors such as the presence of PDPs or civil society groups with active advocacy, fundraising, and investment activities; by funder perceptions or preferences; or by the presence of policy frameworks and funding mechanisms that prioritise specific diseases.

The predominance of research into new products for HIV/AIDS, malaria, and TB is understandable—and the generosity of funding in these areas is a credit to donors—however, other high-burden, high-mortality diseases remain badly under-funded: pneumonia and the diarrhoeal illnesses stand out in this regard. The tendency to focus on drugs and vaccines over diagnostics, platform technologies, and developing-country-specific products is also of concern, particularly as these latter categories can represent a highly effective use of resources since any advance will be applicable to many neglected diseases and products.

A second observation relates to the concentration of funding. The participation of so many organisations and countries in development of new neglected disease products is a remarkable and welcome change from past decades of inertia and neglect. It is clear, however, that these efforts are not evenly distributed, with several major OECD governments missing in action from the top 10, top 20, or even the top 50 funders of R&D for neglected diseases. It is also remarkable that investment by some private firms is now rivalling or exceeding spending by many public organisations, and indeed many G7 and OECD countries. While we commend these companies and philanthropists, their efforts are meant to support, not replace, those of wealthy governments around the world.

A broadening of funding efforts so that all who are able to contribute do so, and all diseases receive the attention they deserve, would lead to a dramatic positive impact on the health of developing country patients afflicted with these diseases. We very much hope that the information presented in the G-FINDER report will contribute to this process.
I definitely think this fine article deserves a wider audience.