Neglected Diseases: Research and Development Landscape

Dr Belén Pedrique Epidemiologist Drugs for Neglected Diseases initiative DNDi, 15 Chemin Louis Dunant 1202 Geneva, Switzerland Interview with :
Dr Belén Pedrique
Drugs for Neglected Diseases initiative
DNDi, 15 Chemin Louis Dunant
1202 Geneva, Switzerland : What are the main findings of the study?

Dr. Pedrique: Of the 850 new drugs and vaccines approved for all diseases in 2000-2011, 4% (37) were for neglected diseases, defined broadly as those prevalent primarily in poor countries: malaria, tuberculosis, 17 neglected tropical diseases (NTDs) as defined by the World Health Organization (WHO), 11 diarrheal diseases, and 19 other diseases of poverty, excluding HIV/AIDS. Globally these neglected diseases represent an 11% health burden, based on a recent assessment of 2010 disability-adjusted life-years (DALYs).

Most newly developed therapeutic products were repurposed versions of existing drugs. Of the 336 brand-new drugs (new chemical entities, or NCEs) approved for all diseases in 2000-2011, only four, or 1%, were for neglected diseases; three were for malaria, and one for diarrheal disease. None were for any of the 17 WHO-listed NTDs

Of 148,445 phase I-III clinical trials registered as of Dec 31, 2011, only 1% (2,016) were for neglected diseases.

Other study highlights:

  • New drugs for neglected diseases have a measurable medical benefit: Using inclusion on the WHO Essential Medicines List (EML) as a proxy measure for medical benefit, 48% of all new therapeutic products (excluding vaccines/biological products) approved 2000-2011 for neglected diseases were on the EML, compared with 4% for all other diseases.
  • Most new candidates in development are vaccines: 123 new products are currently in development for neglected diseases, with over half (55%; 68) being vaccines or biological products, including 21 for malaria. A little over a quarter (28%; 34) are for the 17 NTDs, with only 3 NCEs (for onchocerciasis, Chagas disease, sleeping sickness).
  • Drug repurposing and NCEs are further along in development than vaccines: 56% (38/68) of vaccine/biological product candidates are in Phase I clinical trials, whereas 85% (29/34) of repurposed-drug products and 63% (10/16) of NCEs are in Phases II-III.
  • Nearly 80% of neglected diseases have R&D gaps: Based on selected evaluation criteria, of the 49 neglected diseases included in the study, 11 (22%) had no R&D gaps; 25 (51%) had R&D gaps and some ongoing R&D; and 13 (27%) had R&D gaps and no ongoing R&D.
  • Public sponsorship leads R&D: Clinical trial sponsors were 54% public (governments, academia, public research institutes), 23% private industry (pharma/biotech), and 15% private non-for-profit (product development partnerships, charities, foundations). Remaining 8% were mixed. These results suggest that public and not-for-profit organizations are largely involved in these clinical trials for neglected diseases.
  • Neglected-disease R&D has accelerated somewhat over the past 35 years: Previous studies reported 0.6-1.3 new products/year for neglected diseases for 1975-1999. This study reports a slight increase of 2.4 new products/year for 2000-2011 and predicts 4.7 new products/year through 2018 with a higher proportion of vaccines over brand new drugs. : Were any of the findings unexpected?

Dr. Pedrique:

  • Few new chemical entities (NCEs) have been developed in the last decade for most neglected diseases, including for all NTDs, and few NCEs are in the current pipeline.
  • We found a consistently low proportion of 1% allocated to R&D for neglected diseases in different areas of analysis: only 1% of approved NCEs (2000-2011), 1% of clinical trials (December 2011), and, in a recent report by other authors (Lancet 2013; 382: 1286-1307), 1% of global health investment. : What should clinicians and patients take away from your report?

 Dr. Pedrique:

  • Major advances were achieved in the last decade in the treatment and prevention of some neglected diseases (i.e. malaria, leishmaniasis, sleeping sickness, diarrhoeal diseases)
  • Nevertheless, new therapeutic products are still needed for many neglected diseases in order to treat patients at the field level. : What recommendations do you have for future research as a result of this study?

  • Researchers should conduct further analyses of patient and R&D needs for prevention, treatment, and diagnosis of neglected diseases and set R&D priorities to address these unmet needs.
  • Researchers should provide regular updates of ongoing clinical trials and timelines, successes, and hurdles for new therapeutics in development for neglected diseases.


The drug and vaccine landscape for neglected diseases (2000–11): a systematic assessment
Dr Belen Pedrique MD,Nathalie Strub-Wourgaft MD,Claudette Some PharmD,Piero Olliaro MD,Patrice Trouiller PharmD,Nathan Ford PhD,Bernard Pécoul MD,Jean-Hervé Bradol MD
The Lancet Global Health – 24 October 2013
DOI: 10.1016/S2214-109X(13)70078-0



Last Updated on November 1, 2013 by Marie Benz MD FAAD