ASTMH recommends FDA to include neglected infections of poverty in orphan classification

Today, U.S. scientists committed to finding answers to reducing and eliminating what are known as neglected tropical diseases (NTDs) that plague the world's poorest people in developing countries, urged the FDA to include in its orphan classification the neglected infections of poverty that also affect Americans, and expressed support for stronger relationships with the FDA to ultimately halt these ancient scourges.

In testimony presented at the FDA hearing, Advancing the Development of Medical Products Used in the Prevention, Diagnosis, and Treatment of Neglected Tropical Diseases, Peter J. Hotez, MD, PhD, and President-Elect of the ASTMH, offered recommendations for the FDA's consideration that based on its unique regulatory and public health role could position the agency to more effectively address these diseases and conditions.

"The science and manufacturing associated with producing safe and effective treatments is complex," said Hotez, who, is also President, Sabin Vaccine Institute and Chair, Department of Microbiology, Immunology, and Tropical Medicine, George Washington University.  "However, perhaps more daunting are the operational logistics and economic barriers to bringing critical treatments to affected populations."

Most not-for-profit organizations and academic institutions would welcome additional guidance in working with the FDA and the national regulatory authority (NRA) in countries where these diseases and conditions are almost part of the landscape.  Similarly, because there are no major economic markets for drugs to treat NTDs in the U.S., Europe or Japan, most major pharmaceutical companies are equally less likely to conduct their own R&D into these health issues.  While a small group of non-for-profit organizations, Product Development Partnerships (PDPs), have stepped into this void to develop new commercial entities for the NTDs, they are significantly under-resourced.

"The FDA can help enormously by increasing its guidance and mentorship to PDPs to help them meet the needs of NRAs and the FDA as well as finding ways to operationalize more efficiently," said Hotez.  "These organizations would also welcome increased financial support for NTD product development and testing, and an expansion of FDA's Orphan Grants Program in the Office of Orphan Products Development."  

Of the $3 billion global budget allocated for neglected disease R&D, more than 70% of the budget is allocated to the "big three" – HIV/AIDS, malaria, and tuberculosis, leaving less than 30% of the total budget to address other NTDs.  

NTDs also afflict many of the most impoverished, even in the U.S.  For instance, millions of Americans living in the poorest areas of the Mississippi Delta, the border with Mexico, inner cities, and Appalachia are at risk for parasitic and other infections, and in recognition that there is a need to address the special status of these conditions, the Neglected Infections of Impoverished Americans Act of 2010, H.R. 5986 was just introduced in Congress.