How to Treat the Rare Diseases That Big Pharma Leaves Behind

Personal Health

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.

What was once a pioneering collaborative approach to drug development and delivery has now been tested and can serve as a model for organizations committed to human health and quality of life. Only through cooperative efforts and the firm allocation of sufficient funds can progress be made in understanding and treating some of the most serious but neglected diseases such as drug-resistant tuberculosis, dengue fever and many other newly emerging hemorrhagic viral diseases such as Ebola.

Today there are FDA-approved drugs, successfully marketed in developed countries that, if properly and efficiently tested against orphan diseases, may prove to be useful in combating them. Other compounds are sitting on shelves of pharmaceuticals companies waiting for nonprofit researchers to explore their potential.

That research is only feasible through cooperation. To better serve those 500 million people by treating and curing orphan diseases, we must pursue a future of biomedical research based on collaboration among major pharmaceutical companies, small start-up companies, universities and independent research organizations where most of the country's biomedical scientists work.

Individuals and organizations can encourage government officials and pharmaceutical companies to adopt policies that support such cooperation. Shareholders can pressure companies to address problems whose solutions may not drive up stock prices, but will improve the lives of millions. Let's all provide financial support for nongovernmental organizations and universities that can help move these formulations to the lab bench and, those with promise, to the field for clinical testing and into the homes of the people whose lives could be saved.

Science must earn the pedestal of respect on which it is placed by society. Scientific work, no matter how basic or applied, has meaning and impact through its contribution toward the betterment of humankind.

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