Guide for the Assessment of Clotting Factor Concentrates

SECTION 2LICENSING, REGULATION, AND CONTROL OF CLOTTING FACTORCONCENTRATES IN EUROPE AND NORTH AMERICAIntroductionArrangements for the licensing, regulation, and control of medicinal products have been developedand formalized to ensure that the risk-to-benefit relationship, which is involved in any medical intervention,may be optimized to assure patient safety. The responsibilities of national regulatory authorities(NRAs) under such arrangements include:• Establishing and maintaining a system of licensing and control, including- Dossier review and pre-approval inspection- Facility and product registration- Facility and product inspection and enforcement• Providing standards and guidelines• Requiring that licence holders adopt and maintain appropriate quality systems• Providing arrangements for post-marketing surveillance of productsRegulatory systems in Europe and North America are highly evolved and very complex, and arebeyond the capacity of most healthcare systems in developing countries with limited resources.However, it is beneficial if authorities in developing countries are aware of the approaches used by themain regulatory agencies, which may help them to develop their own framework for assessing andchoosing hemophilia treatment products. The approaches of the Food and Drug Administration andthe European Medicines Evaluation Agency (EMEA) are outlined in this section, along with otherapproaches aimed at harmonization.FDA regulations and guidelinesThe U.S. Food and Drug Administration (FDA) is the largest regulatory body, with wide responsibilities forassuring the quality of foodstuffs, medicines, and medical devices manufactured for sale and supplyin the U.S. Regulations to be observed in the manufacture and supply of pharmaceuticals are definedin Title 21 of the Code of Federal Regulations (21CFR) and in sections 1-999 of the United StatesPharmacopoeia (USP). The parts of 21CFR with specific relevance to plasma products are 5 :• Parts 210 and 211, which describe current good manufacturing practices (cGMPs)• Parts 600 to 680, which set out the requirements for biological productsAdditional guidance (distinct from regulations) is provided to manufacturers (and inspectors) in arange of paper and web-based publications, including:5 Available from http://www.access.gpo.govGuide for the Assessment of Clotting Factor Concentrates 13