[Ip-health] Deadly Gaps Persist in New Drug Development for Neglected Diseases - Study published in Lancet Global Health

Thiru Balasubramaniam thiru at keionline.org
Thu Oct 24 06:14:50 PDT 2013

This message is posted on behalf of Gabrielle Landry of DNDi.
Deadly Gaps Persist in New Drug Development for Neglected Diseases - Study
published in Lancet Global Health


*Deadly Gaps Persist in New Drug Development for Neglected Diseases*****

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*New study shows that, despite some progress, only 4% of new drugs and
vaccines approved 2000-2011 were for neglected diseases, and a ‘fatal
imbalance’ remains in R&D for many neglected patients*****

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*[Geneva, Switzerland – 24 October 2013]* – *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.****

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Researchers from DNDi, Médecins Sans Frontières/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, Grenoble, 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).****

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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.****

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*‘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 Pécoul, Executive
Director of DNDi.****

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*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.****

   - *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.****

   - *Nearly 80% of neglected diseases have R&D gaps: *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

   - *Forecasting sees numerous vaccines but few brand-new drugs:* Applying
   attrition rates, the authors forecast 28 new registered products for
   neglected diseases over the next six years, but only five being NCEs.
   Fifteen new vaccines or biological products are forecasted to be registered
   in the next 10 years.****

   - *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.

*‘Although strides have been made in the last decade, we still see deadly
gaps in new medicines for some of the world's least visible patients’,* said
Dr Nathalie Strub-Wourgaft, Medical Director of DNDi. *‘We need to get more
treatment candidates, NCEs or existing ones for repurposing, into and
through the R&D pipeline to fundamentally change the way we manage these

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*‘Our patients are still waiting for true medical breakthroughs’,* said Dr
Jean-Hervé Bradol of MSF, a co-author of the study. *‘People are still
suffering and dying from these diseases, and healthcare providers must be
able to offer all patients - irrespective of their ability to pay - the
best treatment possible. Only then will we say that we have made progress.’

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*Research article link:*****

Pedrique B et al. The drug and vaccine landscape for neglected diseases
(2000-11): a systematic assessment.*Lancet Global Health*, Early Online
Publication, 24 Oct 2013.****


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*Press release link:*****


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*Study background:*****

This study comes a decade after MSF hosted a major conference in New York
to examine the crisis in R&D for neglected diseases and lay the groundwork
for the creation of DNDi in 2003. In a 2001 study carried out by MSF and
the Drugs for Neglected Diseases Working Group, the precursor to DNDi, only
1.1% of new drugs approved between 1975 and 1999 were found to be for
neglected diseases, including NTDs, malaria, and TB, though they accounted
for 12% of the global disease burden.****

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See the original article here:****


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See the original report here:****


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*Media contacts: *****

(North America) Oliver Yun: e-mail: oyun at dndi.org / Tel: +1-646-616-8681 /
Mobile: +1-646-266-5216****

(Europe) Samantha Bolton: e-mail: samanthabolton at gmail.com / Mobile: +44
797 242 8633****

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Gabrielle Landry Chappuis****

Head of Communication and Advocacy****

Drugs for Neglected Diseases initiative – DNDi****

15 Chemin Louis-Dunant, 1202 Geneva, Switzerland****

glandry at dndi.org / tel. +41 22 906 9249 / mobile: +41 79 309 3910****

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