Moving into its initial area of focus this spring, BIO Ventures for Global Health is well along in its mission to serve as a medium in helping the biotechnology industry address Third World drug development needs.
"Our approach is to radically change the equation for companies to be able to invest their own resources in product development and move them forward," said Wendy Taylor, the nonprofit organization's executive director. "It's founded on a belief that market mechanisms are really the critical driver in what the industry needs to move products forward."
To that end, BIO Ventures for Global Health (BVGH) aims to elucidate underlying market opportunities to drive decision making for companies interested in global health issues. According to the Biotechnology Industry Organization in Washington, from which BIO Ventures for Global Health was spun, less than 10 percent of health research funding is targeted to diseases that account for 90 percent of the global disease burden. Defining such diseases as business prospects would go a long way in pushing companies with relevant research and technologies, which undeniably have responsibilities to their investors and "have real opportunity costs," Taylor told BioWorld Today. "We need to find ways to help make these opportunities viable for companies."
That approach deals with the so-called "pull side" of drug development, by defining markets that traditionally have not drawn interest. It contrasts with research-level work conducted through government grants from organizations such as the National Institutes of Health or from charitable donors. Many of those public/private partnerships have been focused on a single disease area, such as AIDS or malaria, angled at the "push side" by providing money to fund research and development.
At the same time, existing distribution channels have been established by global organizations such as the United Nations or World Health Organization. Drugs can get to patients who need them, if suppliers can get beyond problems related to cold storage and difficulties associated with multidose administrations.
But there is a void in the middle of the equation; product development is languishing. For those on the industry side of the continuum, who point to spiraling costs as becoming more and more prohibitive, a pull toward a defined market could prove to be a final piece of the puzzle.
"I have really been pushing to try to get to late-stage development," said Una Ryan, CEO of Avant Immunotherapeutics Inc., "the pothole in the middle of all of this."
Avant, based in Needham, Mass., is developing single-dose oral vaccines for cholera, typhoid fever and E. coli, among other diseases, without a need for refrigerants. Primarily aimed at a travelers' market to earn profits to support Third World development, many have advanced into clinical trials, but Ryan told BioWorld Today that they tend to "get stuck at Phase II." Large Phase III evaluations of such products require massive funding commitments for manufacturing, distribution and recruitment.
Ryan, who sits on the business advisory board of BVGH, noted that her company's most advanced product moved forward by way of a partnership with GlaxoSmithKline plc, the London-based pharmaceutical giant. Rotarix, a vaccine for rotavirus, was tested in 70,000 children "at a cost of $500 million." GSK is working to get it licensed around the world.
Not all late-stage studies carry such enormous costs, but Ryan, nevertheless, warned that "all of these wonderful products that would be great in the developing world are stuck in the pipeline." Avant recently completed a manufacturing facility capable of producing 100 million vaccine doses per year, by way of an inexpensive fermentation process, and Ryan stressed that she wants "to make vaccines under $1." But she cautioned that end-user costs also are made of research and development costs.
Still, her passion for global well-being made her an immediately enthusiastic supporter of BVGH. And Taylor found many in the biotech community are cut from the same cloth.
She spoke of an "excitement that there's an organization trying to help them be able to do that, and that understands their constraints and their responsibilities to their shareholders, but is looking for new ways to make this work."
BVGH was incorporated a year ago and scaled up its activities after receiving a $1 million grant from the Bill and Melinda Gates Foundation, as well as another $100,000 from the Rockefeller Foundation. Its operations well under way, the organization is developing business cases for specific diseases, with an initial focus on tuberculosis. Through such a framework, BVGH aims to show that there are viable markets for a sufficient return on investment, as well as established systems for product registration and distribution channels.
In developing business cases, Taylor said the organization envisions readily defined viable markets, which would make industry investment attractive. Should a market not prove viable to industry, the business case could be brought to charitable donors or governments that could make up shortcomings through market-based incentives such as advanced purchase commitments, which guarantee sales for a specific product, as well as lures for follow-on development.
"There are a number of different things we could work on to try to improve market conditions for diseases," Taylor said, "and we would to try to make sure that happened."
BVGH plans to do two to three business cases per year. With its current small staff, it is contracting with consultants to complete the initial work. And the organization is working in concert with biotech companies, which also have concerns related to intellectual property, tiered pricing and product testing in multiple regulatory systems.
"Part of what we're doing will be bringing together companies that have an interest in this, and involving them in the development of a business case, so we can make sure that the business case is useful, relevant and credible," Taylor said. "Industry has to believe that the information is credible, and that it's information that they can use."
Ryan, who added that she hopes BVGH can influence the FDA in speeding regulations, believes the framework being put forth by the organization can help companies such as hers, "the small companies who are spending our investors' dollars," to reassure them that "their dollars aren't going to low-margin opportunities. We can do good and do well at the same time."