The US Food and Drug Administration Provides a Pathway for Licensing Vaccines for Global Diseases

Abstract

Responding to an urgency for new vaccines for global diseases, in September 2008 the Center for Biologics Evaluation and Research (CBER) at the US Food and Drug Administration (FDA) published an important FDA Guidance Document (see Box 1), ‘‘General Principles for the Development of Vaccines to Protect Against Global Infectious Diseases,’’ [1] that should expand the FDA’s role in facilitating the development of new vaccines for global bacterial, viral, and parasitic diseases affecting millions of people in developing countries worldwide. In part, this action is in response to a changing paradigm in advocacy for global diseases and recognition on the part of developed nations that the facilitation of vaccines and other products for neglected diseases disproportionately affecting those living in poverty is an effort that benefits industrialized as well as low-income countries. Moreover, through the effort of researchers, the pharmaceutical and biotechnology industries, and guided by nongovernmental product development partnerships, many funded by the Bill & Melinda Gates Foundation, preventative vaccines for global diseases are becoming a reality. Recent progress in this area is exemplified by both a meningococcal vaccine [2] and a new malaria vaccine moving forward into phase III efficacy trials in several African nations [3] and by several candidate vaccines for the prevention of tuberculosis being tested in phase I and II trials [4]. Vaccines and therapeutics for other viral [5], bacterial [6], and parasitic [7] neglected diseases are also in various stages of progress. As these new products enter into clinical trials, it has become clear to product development partners that it is critically important to identify regulatory pathways leading to the timely evaluation and acceptance of safe and effective life-saving interventions. However, this effort is impeded by the fact that regulatory agencies in developing countries where tropical diseases are endemic often lack the capacity to review applications for new vaccines, resulting in lengthy delays in obtaining permission to conduct clinical trials. Better-resourced regulatory agencies such as the US FDA, Health Canada, and the European Union’s European Medicines Agency (EMEA) have been willing to help strengthen regulatory authorities in developing countries, mostly through capacity-building and training programs coordinated by the World Health Organization (WHO), yet their ability to license new vaccines for global diseases has been restricted by the paucity of these diseases within their countries [8]. Some new approaches are being undertaken by the EMEA and by WHO and are briefly described here. In 2004 the European Union introduced a resolution (Article 58) stating that the EMEA could provide a scientific opinion equivalent to a marketing authorization in cooperation with WHO for the evaluation of medicinal products intended