ADVANCING NEW BIOTECHNOLOGY PRODUCTS INTO CLINICAL TRIALS AND MARKETING APPLICATIONS

Evan B. Siegel

Regulatory Department, Ground Zero Pharmaceuticals, USA

Abstract

Due to actual and perceived patient need, particularly for serious or life-threatening conditions in oncology, cardiovascular, infectious and autoimmune diseases, sponsors of medical products and regulatory agencies alike have developed new approaches to accelerating the movement of biotechnology concepts into the clinic and beyond. Special regulatory programs, particularly in the USA, are facilitating exploratory “bench to patient” preclinical and clinical approaches to shortening lifecycle time, attempting to reduce costs, and relieving the resource-intensive nature of drug and biologic development. These programs include frequent and intensive discussions with the FDA, Orphan Drug, Fast Track and Breakthrough Therapy designations, Accelerated Approval, Priority Review, and Compassionate Use. University spinouts, virtual firms, and well - established SMEs, as well as larger sponsors, can all benefit from such approaches. The melding of good science, targeted product profiles, assessment of patient need, clearly delineated corporate strategy, investor education and underlying sound regulatory principles have led to a significant shortening of clinical development times for a number of US approved/licensed products since 2013. This trend has accelerated in 2016. It is critical to use an integrated CMC, Preclinical, Clinical and Regulatory strategy to move biotechnology programs forward, rapidly build value to sponsor firms, and increase the probability of success in the biotechnology arena. This presentation will touch on key approaches to these goals and open up opportunities for further discussion.

Keywords: Regulatory, CMC, Biotechnology, Medical, Pharmaceutical, Biologic, FDA, Clinical, Preclinical.