New study shows that, despite some progress, only 4 percent of new drugs and vaccines approved 2000-2011 were for neglected diseases, and a 'fatal imbalance' remains in R&D for many neglected patients
In a study published today in the open-access journal The Lancet Global Health, the Drugs for Neglected Diseases initiative (DNDi) and other researchers report a persistent deficiency in truly new therapeutics for neglected diseases, despite nominal progress and an acceleration in research and development (R&D) efforts. This continued 'fatal imbalance' in medical R&D points to the urgent need to develop and deliver groundbreaking new treatments for the world's poorest and most neglected patients.
Researchers from DNDi, M-decins Sans Fronti-res/Doctors Without Borders (MSF), the Special Programme for Research and Training in Tropical Diseases (WHO-TDR), and three universities (University Hospital of Grenoble, France; Joseph Fourier University, France; University of Oxford, UK) found that of the 850 new drugs and vaccines approved for all diseases, 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.
'While drug and vaccine development shows signs of acceleration for neglected diseases, we must keep pushing to keep these diseases on the international policy agenda and move quickly to deliver truly transformative, life-saving treatments', said Dr Bernard P-coul, Executive Director of DNDi.
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.
Clinical trials are lacking for neglected diseases: Of the nearly 150,000 registered clinical trials for new therapeutic products in development as of December 2011, only 1% were for neglected 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.