chapter  11
39 Pages

The CTD and eCTD for the Registration of Pharmaceuticals for Human Use

WithDuane B. Lakings

INTRODUCTION The year is 1999. You, a pharmaceutical or biotechnology company sponsor or a sponsor’s agent or a CRO under contract by the sponsor, have all the drug discovery reports and/or publications, nonclinical study reports, clinical study reports, and other clinical documents, and the chemistry, manufacturing, and control (CMC) information and documentation on a drug candidate necessary for the preparation of a regulatory agency submission for a marketing application. Your charge is to prepare the necessary summaries to “tell the story” of the discovery and development of a drug candidate and to integrate the information in the appropriate formats for submissions to each of the countries where marketing approval is being sought. To complete this endeavor, you need to know the marketing application submission requirements for each of these countries. These requirements vary substantially from country to country and often require the preparation of different summaries to be presented in a different order for each country. In addition, each country has some differences in formatting (e.g., binding and binder size and color, paper and page size, font and font size, heading and subheading type and style) stipulations. Thus, you end up preparing multiple submissions, probably one for each country. The process takes substantial time and resources, sometimes from six months (if everything goes smoothly and according to plan) to a year (or longer if unexpected “surprises” are encountered). After the submissions are made, you start to receive questions and queries from the regulatory agencies in various countries. Each question has to be carefully considered, in light of the information in the submission to that country, and appropriately answered. These activities, both the time necessary to prepare the submissions and to give response to queries from the various regulatory agencies, shorten the time of marketing exclusivity after approvals are received, causing a reduction in revenue. Possibly a substantial reduction if the delays in approval are long (sometimes because additional research studies are necessary to effectively respond to a query from a regulatory agency), and the drug candidate has a projected fifth year sales of $365,000,000 or one million dollars a day.