[Ip-health] DNDi/MSF: Millions of Patients Still Waiting for Medical "Breakthroughs" Against Neglected Diseases

Rachel Cohen rachel.cohen72 at gmail.com
Thu Dec 13 05:21:13 PST 2012

Doctors Without Borders/Médecins Sans Frontières (MSF)

Drugs for Neglected Diseases initiative (DNDi)

Mount Sinai School of Medicine 


Millions of Patients Still Waiting for Medical “Breakthroughs” Against
Neglected Diseases


Analysis of Health R&D Pipeline Shows Important Progress, but Significant
Gaps in Innovation Remain


New York, NY, December 13, 2012 — Despite important progress in research and
development (R&D) for global health over the past decade, only a small
fraction of new medicines developed between 2000 and 2011 were for the
treatment of neglected diseases, highlighting the ‘fatal imbalance’ between
global disease burden and drug development for some of the world most
devastating illnesses, said Doctors Without Borders/Médecins Sans Frontières
(MSF) and the Drugs for Neglected Diseases initiative (DNDi), in an analysis
to be presented today at an international conference aimed at spurring
medical innovations for these diseases.  


DNDi and MSF found that between 2000 and 2011, 3.8 percent of newly approved
drugs (excluding vaccines) were for tropical diseases, TB, and other
neglected infections, which together account for 10.5 percent of the global
disease burden. Much of the progress in the treatment of neglected diseases
and important patient benefit during this time came about through drug
reformulations and repurposing of existing drugs against these illnesses.
However, only four of the 336 brand-new medicines (new chemical entities)
developed between 2000 and 2011 were for the treatment of neglected


“We have to ask ourselves, how much progress have we really made over the
past decade?” said Dr. Unni Karunakara, MSF International President. “People
are still dying of archaic diseases. Doctors and nurses are still handcuffed
by the shortcomings of available medicines, forced to treat their patients
with decades-old, often brutal drugs. As we speak, there are drug-resistant
TB patients enduring two years on an absolutely horrific course of
treatment—debilitating nausea and pain, depression, social isolation,
hearing loss, and even psychosis, are just some of the few side effects they
can have while on these medicines. Our patients are still waiting for real
scientific breakthroughs.” 


The two-day conference, Lives in the Balance: Delivering Medical Innovations
for Neglected Patients and Populations, also hosted by the Mount Sinai
School of Medicine’s Global Health Program, will look at the progress and
shortcomings of the last decade of medical R&D to fight neglected diseases—a
period during which there has been an increase in new neglected disease R&D
initiatives and funders— and will focus in particular on the need to
accelerate development and delivery of new health technologies to fight
Chagas disease and drug-resistant tuberculosis (DR-TB). It will also examine
the need for field-adapted vaccines to reach the 22.4 million children who
are still not receiving even the most basic package of immunizations every


According to the DNDi and MSF analysis, three of the four brand-new
medicines approved for neglected diseases in the past decade were for
malaria, with none for the 17 neglected tropical diseases (NTDs) defined by
the World Health Organization (WHO), nor TB. Furthermore, as of December
2011, only 1.4 percent of a total of nearly 150,000 registered clinical
trials were focused on neglected diseases.


The conference is taking place 10 years 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 percent of new drugs approved between 1975 and 1999 were for
neglected diseases, including NTDs, malaria, and TB, though they accounted
for 12 percent of the global disease burden. 


Some individual successes have emerged from the proliferation of global R&D
actors over the last decade. For example, product development partnerships
(PDPs) were responsible for over 40 percent of neglected disease products
registered between 2000 and 2011, including new TB diagnostics and malaria
combination treatments.  


“There have been advances, but for many diseases we have yet to see the kind
of ‘game-changers’ that are truly needed,” said Dr. Bernard Pécoul,
Executive Director of DNDi. “Product development partnerships and ad hoc R&D
initiatives cannot be ‘the’ solution to the systemic lack of innovation. We
must make patient needs the driving force for R&D. This is the only way to
build on the successes we have seen in the past ten years and overcome the
fatal imbalance that still exists between drug R&D and global health needs.
Governments must put in place a R&D framework to sustainably coordinate,
finance, and stimulate medical innovation for new drugs, diagnostics and
vaccines for the people who need them most.”


The conference comes on the heels of a recent decision by governments to
further delay a WHO-led, 10-year effort to develop a global framework to
strengthen priority-setting, coordination, and financing of R&D for diseases
that affect millions worldwide. Today’s system for medical R&D is flawed in
that it is predominantly driven by commercial rewards rather than global
health priorities. This means that research is steered towards areas that
are the most profitable, leaving fundamental medical needs—particularly
those that disproportionately affect developing countries like NTDs or


The conference, which will bring together a broad range of researchers,
medical professionals, global health experts, policymakers, pharmaceutical
and biotechnology experts, donors, activists, patient advocates, and
journalists, will feature a keynote address from Dr. Anthony S. Fauci,
director of the National Institute of Allergy and Infectious Diseases at the
US National Institutes of Health, and a video address by World Bank
President Dr. Jim Yong Kim.


About Doctors Without Borders/Médecins Sans Frontières (MSF)

MSF is an international independent medical humanitarian organization that
delivers emergency aid to people affected by armed conflict, epidemics,
malnutrition, natural disasters, and exclusion from health care in more than
60 countries. On any one day, more than 27,000 individuals representing
dozens of nationalities can be found providing assistance to people caught
in crises around the world. They are doctors, nurses, logistics experts,
administrators, epidemiologists, laboratory technicians, mental health
professionals, and others who work together in accordance with MSF’s guiding
principles of humanitarian action and medical ethics. MSF received the Nobel
Peace Prize in 1999.  <http://www.doctorswithoutborders.org>


About Drugs for Neglected Diseases initiative (DNDi)

DNDi is a not-for-profit research and development (R&D) organization working
to deliver new treatments for the most neglected diseases, in particular
sleeping sickness (human African trypanosomiasis), Chagas disease,
leishmaniasis, specific helminth (filarial) infections, and pediatric HIV.
Since its inception in 2003, DNDi has delivered six treatments: two
fixed-dose antimalarials (ASAQ and ASMQ), nifurtimox-eflornithine
combination therapy (NECT) for late-stage sleeping sickness, sodium
stibogluconate and paromomycin (SSG&PM) combination therapy for visceral
leishmaniasis in Africa, a set of combination therapies for visceral
leishmaniasis in Asia, and a pediatric dosage form of benznidazole for
Chagas disease. DNDi was established in 2003 by MSF, the Indian Council of
Medical Research, Brazil’s Oswaldo Cruz Foundation, the Kenya Medical
Research Institute, the Ministry of Health of Malaysia, and the Institut
Pasteur in France, with the UNICEF/UNDP/World Bank/World Health
Organization’s Special Programme for Research and Training in Tropical
Diseases as a permanent observer.  <http://www.dndi.org> www.dndi.org 


About Mount Sinai School of Medicine’s Global Health Program

Mount Sinai Global Health is a new institution-wide interdisciplinary
program at The Mount Sinai School of Medicine. We are working to improve the
health of people around the world by building global partnerships in
research, education, and patient care – in turn creating a forum for
collaboration among the school’s students, physicians, scientists, and
trainees interested and involved in global health.
<http://www.mssm.edu/globalhealth> www.mssm.edu/globalhealth 




Sandra Murillo, MSF: + 1-646-207-0405 / sandra.murillo at newyork.msf.org 

Oliver Yun, DNDi: +1-646-266-5216 / oyun at dndi.org 




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