The Burrill Report

Just 4 percent of new drugs and vaccines approved 2000-2011 were for neglected diseases— diseases that are leading causes of death, disability, and poverty afflicting people in mostly low-income countries, a new study finds.

The study from the Drugs for Neglected Diseases and other researchers offers a grim view of the efforts to develop new drugs to combat neglected disease saying that a “fatal imbalance remains in R&D for many neglected patients.”

Neglected diseases represent 11 percent of the health burden globally, but just 4 percent of the new drugs and vaccines approved between 2000 and 2011.

“Our patients are still waiting for true medical breakthroughs,” says Jean-Hervé Bradol of Médecins Sans Frontières/Doctors Without Borders and 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.”

Of course, there’s a reason these are called neglected diseases. The study, published in The Lancet Global Health, found that of the 850 new drugs and vaccines approved for all diseases, only 37 were for neglected diseases. These diseases include malaria, tuberculosis, 17 neglected tropical diseases, 11 diarrheal diseases, and 19 other diseases of poverty, excluding HIV/AIDS.

Of the new drugs for neglected diseases approved during the study period, most were repurposed drugs developed for other indications. Of the 336 new chemical entities approved for all diseases in 2000-2011, only four, or 1 percent, were for neglected diseases. Of those, three were for malaria and one for diarrheal disease.

The study also found a lack of clinical trials for neglected diseases. Of the nearly 150,000 registered clinical trials for new therapeutic products in development as of December 2011, only 1 percent were for neglected diseases.

In fact, most of the new candidates in development for neglected diseases are vaccines. Sixty eight of the 123 new products in development, or 55 percent, are vaccines or biological products, including 21 for malaria. A little more than a quarter of these are for the 17 neglected tropical diseases.

A 2001 study carried out by Médecins Sans Frontières and the Drugs for Neglected Diseases Working Group, argued that simple economics resulted in the lack of development of new drugs for neglected diseases. “Potential return on investment, not global health needs, determines how companies decide to allocate R&D funds,” the report said.

“According to the drug industry, the low purchasing power of developing countries—coupled with the high cost of R&D and drug registration—rationalizes their focus on wealthy country markets.”

It said drug development, which is largely conducted by private industry, will follow its own priorities. It said since multinational pharmaceutical companies cannot be relied on to develop drugs needed to treat diseases afflicting the world’s poor, government must take action. “The current crisis in R&D for neglected diseases is a result not only of the failure of the market, but also of the failure of public policy.”

In fact, the new report found public sponsorship leads R&D with 54 percent of clinical trial sponsors being public (governments, academia, public research institutes), 23 percent private industry (pharma/biotech), and 15 percent private non-for-profit (product development partnerships, charities, foundations). The remaining 8 percent were mixed.

“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,” says Nathalie Strub-Wourgaft, medical director of Drugs for Neglected Diseases. “We need to get more treatment candidates, [new drugs] or existing ones for repurposing, into and through the R&D pipeline to fundamentally change the way we manage these diseases.”


October 30, 2013
http://www.burrillreport.com/article-study_finds_neglected_diseases_remain_so.html