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Neglected Diseases

Neglected Diseases PLOS Medicine's Neglected Diseases section began with the launch of the journal in October 2004. Up until February 2008, the section focused on tropical infectious diseases, such as Buruli ulcer, trachoma, and hookworm. A list of the 21 articles published from October 2004 to February 2008 can be found in the Supporting Information section of our February 2008 Editorial. With the launch of PLOS Neglected Tropical Diseases in October 2007, the focus of the Neglected Diseases section of PLOS Medicine shifted from tropical diseases to other health problems that could be considered neglected and that have a significant global burden (such as reproductive and maternal health problems, mental illness in low- and middle-income countries, road traffic injuries, and health problems related to migration and conflict.). Read the February 2008 Editorial for information about the section.

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# A Breakthrough in R&D for Neglected Diseases: New Ways to Get the Drugs We Need

• Published: September 08, 2005
• DOI: 10.1371/journal.pmed.0020302
Corrections

25 Oct 2005: Moran M (2005) Correction: A Breakthrough in R&D for Neglected Diseases: New Ways to Get the Drugs We Need. PLoS Med 2(10): e376. doi: 10.1371/journal.pmed.0020376 | View correction

Research suggests that long-held beliefs on neglected-disease drug development activity are no longer accurate, and that these inaccurate beliefs have led—and are leading—to poorly designed and targeted government policies and incentives. On a more positive note, this research also highlights opportunities for better-targeted government policies that will more closely match the reality of neglected-disease drug development and the needs of public and industry groups.

### Current Perceptions

Current government policies to stimulate development of new drugs for neglected diseases are based on a set of shared understandings.

One of these understandings is that only 13 new drugs have been developed for neglected tropical diseases since 1975, with the main problem being that these diseases are simply non-commercial for companies to invest in [1]. Another is that, although public-private partnerships (PPPs) for drug development have started, they are problematic. (In this article, PPPs are defined as public-health-driven not-for-profit organisations that drive neglected-disease drug development in conjunction with industry groups.) Governments are uncertain which of the plethora of PPPs they should support, particularly as most are thought to be too young to judge their success. At the time this article went to press, the Initiative on Public-Private Partnerships for Health (http://www.ippph.org/) listed 92 PPPs in its database (this number includes all PPP activity, including the small number of organisations that make drugs, vaccines, and microbicides; and one-off partnerings such as donations and cut-price deals).

The general view is that PPPs are inexperienced in drug development, and may eat up public cash without delivering the tools we need, while the real experience and capability in drug development lies with multinational pharmaceutical companies, which must be brought back into the neglected-disease field if we are to achieve success. For example, the Commission for Africa recently stated that we need to increase neglected-disease R&D by “giving large pharmaceutical firms incentives to investigate the diseases that affect Africa, instead of focusing on the diseases of rich countries” [2].

##### Overall performance.

It is important to note that these outcomes are not evidence of the capacity of the individual players, but rather of the ability of different R&D approaches to deliver optimal outcomes. A company working in a partnership may be able to deliver a better outcome than the same company working alone, for a number of reasons. Companies working alone (as was generally the case under the pre-2000 model) tend to reduce the cost and risk of neglected-disease drug development by focussing on less-expensive, less-risky, “adaptive” R&D such as label extensions of veterinary drugs to humans, or new formulations of existing drugs, and/or by working slowly, as staff and funds are prioritised to more commercial programmes. Under the post-2000 partnering model, the same companies can still restrict costs and risks but in a far more productive way, focussing on discovery of breakthrough leads in the knowledge that others are available to help develop these and deliver them to patients.

### What Does This Mean for Government Policies?

There is a clear disjunct between the reality of neglected-disease activity and current government thinking, which is focussed on “commercialising R&D to bring big companies back into the field”. This thinking is built firmly on the beliefs outlined at the start of this report and is now significantly out of kilter with the industry neglected-disease drug landscape.

Two policy issues stand out. Firstly, there is an urgent need to support the new model of neglected-disease drug development, in particular the PPP approach, which is already generating new drugs, is highly cost-effective, appears to offer the highest health value, and is a crucial factor in continuing cost-effective industry involvement in neglected-disease R&D. On this point, we note—and welcome—the recent G8 commitment to “increasing direct investment … through such mechanisms as Public Private Partnerships … to encourage the development of … drugs for AIDS, malaria, TB and other neglected diseases” [7]. We look forward to seeing the shape of new policies and mechanisms to make this commitment concrete, and encourage policy-makers to ensure that these are designed to incentivise optimal practices within the PPP approach, and to do so in the most cost-effective manner. Simply handing over cash may not be the best way.

Secondly, we suggest that policy-makers review their approach to “commercialising” R&D in light of the information above. If big companies tell us that public “commercial” markets are not a catalysing factor in their decision to engage in neglected-disease R&D, then we need to listen carefully to them. Policies to stimulate new multinational company activity are one thing; policies that shift existing industry activity from a not-for-profit approach to a for-profit approach are quite another, and may do so at a potential cost of many billions of dollars across all neglected-disease products. Policy-makers may also want to consider whether commercial incentives should be preferentially targeted toward smaller companies that have a closer fit with commercial neglected-disease markets, and whether these new incentives should be tailored to encourage industry-alone R&D, or to encourage partnered models, which metrics suggest may deliver a better health outcome. The latter is particularly a concern given the relative inexperience of most Western pharmaceutical companies (and in particular small companies) in later-stage clinical development and implementation of tropical disease or TB drugs for use in rural Africa or South Asia, as opposed to their undoubted experience in developing drugs for large-scale US and European disease markets.

### Next Steps

The post-2000 renewal of neglected-disease R&D activity is good news for patients with neglected diseases, but it is only a beginning. We hope that this closer analysis will contribute to our store of information, and allow development of policies to encourage and improve these promising new trends in neglected-disease drug development.

### References

1. 1. Pecoul B, Chirac P, Trouiller P, Pinel J (1999) Access to essential drugs in poor countries: A lost battle? JAMA 281: 361–367.
2. 2. Commission for Africa (2005) Final report: “Our common interest”. March 2005. Available: http://www.commissionforafrica.org/engli​sh/report/thereport/english/11-03-05_cr_​report.pdf. Accessed 29 July 2005.
3. 3. DiMasi J, Hansen R, Grabowski H (2003) The price of innovation: New estimates of drug development costs. J Health Econ 22: 325–330.
4. 4. Shibuya K, Bernard C, Ezzati M, Mathers C (2005) Global burden of onchocerciasis in the year 2000: Summary of methods and data sources [draft]. Geneva: Epidemiology and Burden for Disease (EBD). Global Programme on Evidence for Health Policy (GPE), World Health Organization.
5. 5. Chitsulo L, Loverde P, Engels D (2004) Schistosomiasis. Nat Rev Microbiol 2: 12–13.
6. 6. PAREXEL International (2002) PAREXEL's Pharmaceutical R&D Statistical Sourcebook 2002/2003. Waltham (Massachusetts): PAREXEL International. 378 p.
7. 7. DFID (2005) Highlights of the G8 communiqué on Africa. Available: http://www.number-10.gov.uk/output/Page7​880.asp. Accessed 13 July 2005.

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