The importance of investing science and technology into drug product development has become evident as different product types, higher protein concentrations, and doses and requirements for improved delivery of biological drug products have increased. The need to give patients larger and more concentrated doses has challenged formulation scientists and driven development of new technologies that can deliver those doses. Delivery devices fall under device regulations and have distinctly different design, development, and validation requirements from those of protein drug products alone. The regulatory environment also has evolved, whereby a biological drug product (in even simple delivery systems) is now considered a combination product, making additional development consideration and additional requirements applicable.

A CMC Strategy Forum on drug products for biological medicines, including novel delivery devices, challenging formulations and combination products was held in July 2012 in Bethesda, MD. The purpose of this forum was to promote an understanding of how best to increase the speed and effectiveness of drug product and device development for both large and small companies. Participants focused on areas that improve the likelihood for regulatory success, reduce risk, and decrease the time it takes to get a combination product through development. Topics discussed included drug product formulation challenges, device development, design verification and validation, and clinical testing. The forum included input from regulators on how to prevent delays during review of regulatory applications. Biopharmaceutical companies and regulatory agencies both presented case studies, and open discussions provided opportunities to gain common understanding and consensus on a range of topics.

Four Sessions
The forum comprised four sessions, each followed by an interactive discussion with a panel and a moderator facilitating questions and comments from the audience. Part 1 of this article describes the first two sessions (1). The first session on developing formulations was cochaired by Jay Gerondale (Amgen Inc.), Joel Goldstein (ImClone Systems Corporation), Gerd Kleemann, (Amgen Inc.), and Kathy Lee (CDER, FDA). The second session on early design and characterization of combination products was cochaired by Andrew Donnelly (MedImmune), Jennifer Mercer (Genentech, a Member of the Roche Group), and Lana Shiu (CDRH, FDA).

Part 2 includes sessions three and four (2). The third session covered human-factors studies and clinical testing for combination products. Mark Marley (Eli Lilly and Company) and Jacqueline Ryan (US Food and Drug Administration’s Center for Devices and Radiological Health, CDRH) were session cochairs. Cochaired by Jeanmarie Sales (Medtronic Neuromodulation) and Nikhil Thakur (CDRH), the fourth session covered regulatory pathways for combination products, including marketing applications and postlicensure. Lana Shiu (CDRH) spoke about navigating FDA channels for combination products approval.

1 Mire-Sluis A, et al. Drug Products for Biological Medicines: Novel Delivery Devices, Challenging Formulations, and Combination Products, Part 1. BioProcess Int. 11(4) 2013.

2 Mire-Sluis A, et al. Drug Products for Biological Medicines: Novel Delivery Devices, Challenging Formulations, and Combination Products, Part 2. BioProcess Int. 11(6) 2013.

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