01 Nov Neglected Diseases: Research and Development Landscape

MedicalResearch.com Interview with :
Dr Belén Pedrique
Epidemiologist
Drugs for Neglected Diseases initiative
DNDi, 15 Chemin Louis Dunant
1202 Geneva, Switzerland

MedicalResearch.com : 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.

MedicalResearch.com : 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.

MedicalResearch.com : 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.

MedicalResearch.com : 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.

Citation:

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

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