How to Treat the Rare Diseases That Big Pharma Leaves Behind

More than a half billion people around the world suffer from as many as 8,000 "orphan" diseases, for which treatments haven't yet been developed. Unfortunately, the majority of those people live in poverty in developing countries. For the limited purchasing power of people around the globe who suffer from a rare or orphan disease, the financial investment in drug research is prohibitively expensive and unprofitable. So, there is little or no financial incentive for pharmaceutical companies to invest in seeking those missing treatments.

Solutions may lie in partnerships among pharmaceutical companies and not-for-profit research organizations. We know this can work, because it's worked before.

In the 1960s, researchers at the Population Council, the international nonprofit research group with which I've been associated since 1956, made a breakthrough in developing the modern IUD. FEIWomen's Health was our partner in the development process. Clearly, the physical products were and are enormously important, but we also learned another valuable lesson: Cooperation between the public and private sectors can produce triumphs that neither entity could do alone.

Based on this fruitful partnership, we intensified our collaborative efforts with the biopharmaceutical industry. Soon thereafter, a groundbreaking collaboration among the scientific, social and philanthropic communities resulted in the development of a contraceptive implant. It is extremely unlikely that the initial product -- and all its successors on the market today -- would have been possible without personal, corporate, competitive and financial interests being set aside by Wyeth and others for the sake of helping people in dire need.

Many people, in fact. These long-acting contraceptives and the product families that followed have been used by over 200 million women globally to manage the size of their families. Reducing unwanted pregnancies and allowing safe birth spacing reduced maternal and infant mortality.

There are many examples of what is possible with public/private collaboration. Doxycycline, which now treats drug-resistant malaria and filariasis, was developed by Pfizer as an antibiotic in 1967. The Carter Center, Merck, Lions Clubs and others have collaborated on getting Mectizan® to the poorest citizens of 11 countries to prevent river blindness. And new drugs for the treatment of cystic fibrosis, the result of a partnership between Galapagos NV and the Cystic Fibrosis Foundation, improve life expectancy and quality.

In the U.S., the Orphan Drug Act allows the Food and Drug Administration (FDA) to provide incentives for companies to invest in research and development of treatments for orphan diseases. For more than 20 years, this government support has helped bring a few drugs to market. But it's not enough. Other wealthy nations need to step up as well. Thirty members of the Organization of Economic Cooperation and Development, a Paris-based consortium of the richer countries, have together contributed only $43 million to drug development over the past five years. Compare this to the $15 billion directed to PEPFAR by the U.S. government alone from 2003-2008 to fight HIV/AIDS, tuberculosis and malaria, and to the $48 billion committed for 2009-2013. As the United Nations prepares for the forthcoming General Assembly, the agenda should include intensified research on long-neglected diseases, along with the current investments in treatment for the world's most needy.

A mere one in 10,000 compounds identified in the laboratory eventually receives approval from the FDA, and the road to approval is long, costly and uncertain. Bringing to the global marketplace drugs that have the capacity to greatly influence and improve the human condition requires creativity.