Developing cures for neglected diseases: Overcoming the barriers

by Geoffrey Horwitz on July 16, 2013


Who will invest in the clinical development of drugs that offer limited commercial opportunities?(scottchan/

The desire to impact areas of great need drives many academic medical researchers. Unfortunately, a variety of challenges can prevent even the most promising innovations and technologies from reaching the patients who would benefit most. When the target population is primarily in the developing world, these challenges are magnified. Only a fraction of research and development funding goes toward treatments that target neglected diseases and the needs of low- and middle-income countries, posing a particularly frustrating situation.

The Universities Allied for Essential Medicines (UAEM)’s recent forum on Global Access Licensing of Biomedically Relevant Technologies delved into this pressing issue. According to the UAEM philosophy, the accessibility of medicine to developing nations “depends critically on how universities manage their intellectual property.” Further, the UAEM suggests that obtaining patents means that “anyone who can’t afford the asking price will be unable to access the product” and that “further innovation is hampered or outright blocked.”

In contrast, many of the panelists at the forum didn’t see intellectual property licensing as the primary obstacle—rather, they viewed it as a requirement to attract industry partners. The experts included leaders of intellectual property offices at the Massachusetts Institute of Technology, Harvard University, Tufts University, Northeastern University, Partners Healthcare, Boston University and Boston Children’s Hospital.

They agreed that the most pressing challenge is getting industry partners to invest in the clinical development of drugs that offer limited commercial opportunities. The cost of getting a drug approved is astounding, and this is a hurdle universities cannot overcome alone.

New government incentives, for-profit initiatives and creative not-for-profit partnerships are helping academic centers translate scientific discoveries into viable products.

While the cost to approve drugs is lower in developing countries, clinical testing can present numerous challenges that limit global access. Developed nations often can’t test treatments for tropical disease in their own countries since the patient base is too limited. And testing in the target countries often isn’t feasible either: The Center for Global Development asserts that “the research and regulatory capacity in many neglected disease-endemic settings is not adequate to support the clinical trials that need to occur there in order to complete the development of these products.”

Global solutions

Initiatives at various levels are helping overcome global access barriers. For example, a Boston Children’s project to prevent infant hypothermia in resource-poor countries received funding from the Lawrence Berkeley National Laboratory’s Institute for Globally Transformative Technology (LIGTT), supported by the Department of Energy. The United States government also offers for-profit companies incentives to develop cures for neglected diseases. In exchange for approving a treatment for tropical diseases, the Food and Drug Administration will provide a priority review voucher to fast-track a subsequent drug of the company’s choice. This has already paid off for Johnson & Johnson with the approval of Sirturo, the first new drug to target tuberculosis (TB) in 40 years.

Many for-profit companies are also working to promote global access to medicine. GlaxoSmithKline (GSK) was recently ranked #1 in the Access to Medicines ranking, in part because of its work in developing treatments for malaria, HIV and TB. GSK and Merck also recently announced huge price reductions for certain vaccines to improve coverage in low-income countries. Nearly 85 percent of patients who die from cervical cancer live in nations that cannot afford the $100-per-shot vaccines. By dropping the cost below $5, these two companies hope to reach many more vulnerable women.

Academic centers also are teaming up proactively to ensure that innovations benefit global populations. A recent paper signed by more than 70 universities, titled “Nine Points to Consider in Licensing University Technology,” acknowledges the key contribution of academic research to public health. The points (PDF) encourage technology transfer strategies that “promote the availability of health-related technologies in developing countries for essential medical care.”

At the forum, panelist Abbie Meyer, a licensing manager at the Technology and Innovation Development Office (TIDO) at Boston Children’s, noted that Boston Children’s has partnered with non-governmental organizations, such as the Gates Foundation and PATH, to support the hospital’s work on vaccines targeting pneumococcal disease and typhus. The hospital also works closely with the World Health Organization, the Red Cross and Red Crescent, Doctors without Borders and other international agencies.

The combination of need and moral obligation drives much of the research targeting neglected diseases. As more government incentives, for-profit initiatives and creative not-for-profit partnerships take root, exciting scientific discoveries will have more opportunity to be translated into viable commercial products.

Geoffrey Horwitz, PhD, is a business development associate in the Technology and Innovation Development Office (TIDO) at Boston Children’s Hospital. Follow him on Twitter @GeoffHorwitz.


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