R&D for the Poor
Less than 10 percent of investment in health research goes to diseases that affect 90 percent of the world, according to the
Commission on Health Research for Development. Still, companies are more engaged in global health than ever before—and not
just because of the sales they hope to eventually make in poor nations. "All of the large pharmaceutical companies recognize
that the world is changing, and there is also an ethical obligation to enable access to life-saving drugs and vaccines," says
Merck's Feinberg. "Companies will have a difficult time if they approach the world solely from a commercial perspective."
Most recently, the first pharma-funded institute was established dedicated solely to the development of vaccines for neglected
disease. The Novartis Vaccines Institute for Global Health (NVGH), in Siena, Italy, is a companion to the company's nonprofit
Singapore site, focused on drug treatments for tropical diseases. NVGH is Novartis' answer for developing vaccines that target
developing world needs, but it does so in a context where it can work with and trade intellectual property with the company's
commercial arm. NVGH's first project aims to develop a broad-range enteric vaccine to protect children against several Salmonella infections, including typhoid fever, which kills 200,000 children each year outside the West.
"New medicines can only be made efficiently by commercial organizations," says Paul Herrling, head of corporate research at
Novartis. "We have some responsibility to think about access to medicine, and simply allocate the portion of our research
capacity and science technology to these diseases. We think it's a way companies can distinguish themselves from one another."
Still, most companies don't go it alone. In fact, the majority opt to participate in product development partnerships, or
PDPs, which have exploded in number in recent years as a result of the massive influx of Gates Foundation funding. In 2005,
there were 32 PDPs with pharma companies. By 2008, the number had jumped to 67, according to a study undertaken by the International
Federation of Pharmaceutical Manufacturers.
There are many reasons why it makes sense to work within the PDP framework. "The success rate is much higher if pharma works
in collaboration with a product development partner," says Alan Fairlamb, a professor and researcher at the University of
Dundee's school of life sciences, a hub for research on sleeping sickness and other diseases. "They are constantly checking
to make sure that the additional challenges that a product faces in the developed world are being addressed."
The evidence to the contrary, says WHO's EPI medical officer Rudi Eggers, MD, is products like Prevnar. "The pneumo vaccine
comes in a pre-filled syringe, which is very complicated in the public sector because it adds to your cold chain storage,"
he says. "Disposal of pre-filled syringes is also complicated. In terms of developing future vaccines, the interaction between
the advisors, ourselves, and the manufacturers has to be much closer to develop appropriate formulations that actually would
work better in developing-country public-sector markets."
PDPs have been successful in creating a neglected disease pipeline where there was none. In particular, the malaria vaccine
RTS,S—a recombinant circumsporozoite protein fused to a hepatitis-B surface antigen which uses an adjuvant to enhance the
immune response—illustrates the power of PDPs to mount research where the risk would be too large for any one entity to bear.
GlaxoSmithKline first undertook development of a malaria vaccine more than 25 years ago, but progress picked up when Gates
committed funding to the PATH Malaria Vaccine Initiative, and GSK teamed up with the group. The vaccine is expected to move
into Phase III, where it will be tested in 16,000 patients in Africa—the largest clinical trial ever conducted for a vaccine
on that continent.
"We've reached a high in malaria vaccine development, but this high is a real high," says GSK's Joe Cohen, an inventor of
RTS,S. "It will very hopefully take us all the way to registration and implementation of the very first malaria vaccine. That's
a big step forward, a big breakthrough."
As the vaccine progresses through the pipeline, it has generated more excitement, but the majority of neglected disease projects—39
of the 67—are still in early-stage research. "There may be a lot of science and discovery projects," says IFPMA president
and Schering-Plough CEO Fred Hassan. "But frankly, the funnel becomes pretty narrow on the other end."
The cost of early-stage research is just a fraction of full-scale clinical development. "To move these drugs in the pipeline,
it will cost $1 billion a year," says Herrling. "That's what it cost to build the pipeline over the last five to eight years."
Indeed, the IFPMA study estimates that the funding shortfall from now until 2017 is between $6 billion and $10 billion. With
no indication that current donors could meet the need, many fear such research efforts will simply stall.