Regulatory Considerations for Medical Aerosol Products

Abstract

This article provides a high-level overview of regulatory considerations for medicinal aerosol products around the world. It builds on our previous review and reflects developments related to more recent trends and innovations in science, business, technology, and regulation. All medicines, including medicinal aerosol products, must be approved by an appropriate government agency prior to marketing authorization. Knowing and complying with the requirements of an appropriate regulatory agency (or agencies) is a prerequisite to successful commercialization of any aerosol therapy. The range of governmental regulatory oversight is broad, including inspections of manufacturing sites, evaluations of preliminary data and study protocols before the start of trials in human subjects, evaluations of the sponsor’s clinical and in vitro data for the proposed product, approval of final labeling and of postapproval changes to any aspect of the product or manufacturing process, monitoring of adverse event reports, and other areas. The specific regulatory requirements vary by the product type and by country, and they also change over time as the science and technology involved in the development, manufacture, and characterization of pharmaceutical and biological products evolve. In general, in order to obtain marketing authorization, the sponsor must demonstrate to regulators, using data from appropriately designed studies and other relevant documentation, (1) that the product is safe and efficacious for the proposed therapeutic indication in the target patient population, (2) that the product’s manufacturing facility follows current good manufacturing practice^2–4 and/or quality management systems,^5,6 and (3) that appropriate quality control and quality assurance programs are in place, including programs to verify that the product released to the market, as well as product kept in stability-testing environmental chambers, complies with preset quality specifications. For aerosol delivery devices and drug-device or biological-device combination products, the sponsor must also assess the influence of human factors, device robustness, and materials’ (bio)compatibility. The sponsor’s responsibility to regulators continues after a product’s approval in the form of, for example, required compliance with ongoing quality testing, pharmacovigilance monitoring, any postapproval commitments, and qualification of any postapproval changes. This article provides an overview of the regulatory considerations for inhaled and nasal products.