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New Paradigms in Clinical Research: Early Access Programs and Clinical Trials for Unmet Medical Needs by Andrea Buschiazzo, MD
Despite a number of novel therapies having gained worldwide approval in recent years, there are still several diseases for which unmet medical needs persist. For this reason, there has been a dramatic increase in the approval of new medicines by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The authorization for commercializing these new drugs is based on the data reported by pharmaceutical companies through their research, which resulted in the development of novel therapeutic approaches, such as new monoclonal antibodies for the treatment of autoimmune diseases and anticancer drugs that act on genetic biomarkers. For patients with serious or life-threatening diseases who have failed to respond to the available treatment options and who are not eligible for participation in clinical trials, Early Access Programs (EAPs) provide access to investigational therapies (prelaunch and/or prior to country approval). Since they are governed by different regulatory authorities, EAPs vary by country. It is a program that may be implemented at different levels of the
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