FDA Publish Guidance on Neglected Tropical Diseases

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The purpose of this guidance is to assist sponsors in the clinical development of drugs for the treatment or prevention of neglected diseases of the developing world. Specifically, this guidance addresses the Food and Drug Administration’s (FDA’s) current thinking regarding the overall drug development program for the treatment or prevention of neglected tropical diseases (NTDs), as defined in section 524(a)(3) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), including clinical trial designs and internal review standards to support approval of drugs. This draft guidance is intended to serve as a focus for continued discussions among the review divisions in the Office of Antimicrobial Products, pharmaceutical sponsors, the academic community, and the public.

This guidance addresses section 740 of the Agriculture, Rural Development, Food and Drug Administration and Related Agencies Appropriations Act, 2010 (Public Law 111-80), dated October 21, 2009, that directed the FDA to provide guidance in the form of general recommendations and regulatory considerations for drugs being developed for the treatment or prevention of NTDs.3 Section 740 references the NTDs included in section 524(a)(3) of the FD&C Act, as follows:

• Tuberculosis
• Malaria
• Blinding trachoma
• Buruli Ulcer
• Cholera
• Dengue/dengue hemorrhagic fever
• Dracunculiasis (guinea-worm disease)
• Fascioliasis
• Human African trypanosomiasis
• Leishmaniasis
• Leprosy
• Lymphatic filariasis
• Onchocerciasis
• Schistosomiasis
• Soil-transmitted helminths
• Yaws

This guidance is intended to clarify the regulatory requirements for drug approval in the United States as well as the internal review standards for drugs for these NTDs. Specifically, this guidance is directed to sponsors who lack general knowledge about drug development issues. Pharmaceutical sponsors with experience in drug development will find this guidance to be basic, but we acknowledge that sponsors interested in evaluating investigational drugs for NTDs may have little experience in working with the FDA on drug development issues, and this guidance is intended to help them better understand the FDA processes.

Potential sponsors should understand that: (1) we will review and comment on clinical development programs for NTDs under an investigational new drug application (IND) submission, regardless of where the clinical development will take place; (2) we can approve a drug for treatment of an NTD not endemic in the United States; (3) the regulatory pathways and internal review standards for approval of drugs for NTDs are the same as for approval of drugs for diseases endemic in the United States; and (4) we are committed to exercising our regulatory authorities to facilitate access to therapies that can help reduce morbidity and mortality associated with NTDs. Specifically, FDA regulations give the FDA considerable latitude “to exercise its scientific judgment to determine the kind and quality of data and information an applicant is required to provide . . . to meet the statutory standards [for approval]” (21 CFR 72 314.105(c)). FDA regulations also specifically require that we consider the severity of disease and the absence of alternative satisfactory therapy in weighing whether the benefits of therapy outweigh known and potential risks (21 CFR 312.84(a)). In addition, there may be circumstances when one trial provides adequate evidence of efficacy.

For Assistance with Developing Products in the Developing World Click Here

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

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