Trends at the FDA in Medical Device Approvals

Dr. Carol Stephens delivered a speech at the LifeSciences BC McCarthy Spotlight Speaking Series event in Vancouver, BC on November 17, 2016. The speech was focused on the trends at the Food and Drug Administration regarding medical device approvals.   View and download the full presentation View and download the full presentation About the Author Dr. Carol Stephens has 28 years of experience in drug development and support with focus on regulatory planning, negotiation, execution, and compliance. In her spare time she likes to design new gardens, spoil her dog Moxie, and travel with her...

Trends at the FDA in Drug Approvals

Dr. Thomas Stephens delivered a speech at the LifeSciences BC McCarthy Spotlight Speaking Series event in Vancouver, BC on November 17, 2016. The speech was focused on the trends at the Food and Drug Administration regarding drug approvals.   View and download the full presentation View and download the full presentation   About the Author Thomas W. Stephens, Ph.D. has 28 years of pharmaceutical development preceded by 12 years of in vitro diagnostic experience with emphasis in research, regulatory, project management and administration. In his spare time Tom pursue various interests including astrophotography, underwater photography, swimming, gardening and...

How Would the Biotech Landscape Change After the Election?

This is the 10th US Election I am following personally. I remember US elections going back to 1980 (Carter vs Reagan) at a time when I was attending a kindergarten in Charlotte, North Carolina and Iran hostage crisis was unfolding. The last time I remember a contested and controversial US election was between George W. Bush and Al Gore of Election 2000. This year’s Election has taken everything to a new level (high or low) depending on what you’d be measuring. Besides all the rhetoric and unbelievable factoids we are learning about candidates everyday, we wonder how this election would impact the FDA, reimbursement and generally the life sciences sector. As many of you know, the head the the FDA (the Commissioner) reports to the Secretary of Health and Human Services who reports to the President and is part of the President’s cabinet. Both are appointed by the President and confirmed (or not) by the Senate. So the choice Americans make on the president impacts who he or she chooses for the head of the FDA and the direction FDA would take. Also, we are entering unchartered waters in the US regarding the reimbursement debate. There has not been another time in the US history where drug pricing and the value of new medicines have been so controversial and debated. In my opinion, the few bad apples (Martin Shkreli, Mylan and others) are giving the whole industry a bad reputation. At a BIO CEO/Investor Forum last week in San Francisco, a number of protestors interrupted the plenary lunch session and prevented many of us from attending the session. Drug...

505(b)(2) Regulatory Submissions Require Flexibility, Creativity and Resilience

The FDA Guidance describing the pathway for development and approval of a 505(b)(2) application covers a lot of ground, but as with any Guidance, it serves only as a general outline. A key element of this Guidance is described in Section II.B.2. “For changes to a previously approved drug product, an application may rely on the Agency’s finding of safety and effectiveness of the previously approved product, coupled with the information needed to support the change from the approved product. The additional information could be new studies conducted by the applicant or published data. This use of section 505(b)(2), described in the regulations at 21 CFR 314.54, was intended to encourage innovation without creating duplicate work and reflects the same principle as the 505(j) application: it is wasteful and unnecessary to carry out studies to demonstrate what is already known about a drug.” In my experience the phrase “coupled with the information needed to support the change from the approved product.” is not always straightforward or predictable. The negotiation with FDA involves the product profile desired by the Sponsor and the historical approval and market information within the Agency. Perhaps the best way to examine some potential challenges and diversity of requirements is to present a few real life situations. I discovered these challenges when I joined a virtual company, replacing the Head the Research and Development, Quality and Regulatory groups, where my predecessor, a tenured generic manager, approached the regulatory requirements for a 505(b)(2) NDA from a generic application viewpoint. One NDA had already been submitted for an injectable product but was given the “Refuse to File” designation...

How Do We Create the Most Value With Limited Resources?

Questions to Consider From the Beginning: Drug development is a time and resource expensive proposition and most if not all companies pursuing such a path must find ways to make the greatest progress as fast as possible within their resource limitations. This article intends to help the reader understand some of the necessary considerations to achieve this efficiency by providing answers to these questions: How do we utilize limited time, money and talent to achieve the most value the most efficiently? How do we focus these resources on the critical questions and risks or opportunities? How do we assure that expended resources are delivering the intended product? How do we prepare for our final or intermediate submissions as the work are being conducted minimizing the turnaround time? How do we organize, manage, store and maintain the integrity of our data? Keeping the End in Mind From the Beginning: What are the attributes of a therapeutic product that are needed to achieve a successful launch and commercialization? How do the current marketed products stand up? What are the products in the development pipeline and how will they differ in hurdles and potential? Keeping the end in mind from the beginning is a fundamental requirement to achieving the ultimate goal effectively. Of course at first there are goals that must be met on the way to keep the ball rolling and the funds coming. How do these intermediate goals impact the ultimate success or failure of the intended product? There are many approaches to creating a map for the final product profile. The FDA and industry have worked together to provide...

The Evolving Biotechnology Regulatory Landscape

From blood products and vaccines to gene markers, gene therapy and RNA interference, the technology of biological based therapeutics is changing at an exponential rate. Challenges for the efficacy and safety of these stem from characterization of a therapy, manufacture, quality control, distribution and use. Regulatory authorities in the USA and around the globe are working to keep up with technology which results in a rapidly changing regulatory landscape. This document is intended to summarize some of the key changes that have occurred over the last several decades. Contents: Primary Regulatory Institutions The Transition from Small Drugs to Biologics Humanization and Engineering of Biologics Biosimilars Elucidation of the Human Genome Companion Diagnostics Gene Therapy and RNA Interference Monoclonal Antibodies Lead New Biotechnology Development Medical Devices and Diagnostics Primary Regulatory Institutions In the USA, federal regulation of drugs and devices including diagnostics begins with the Food, Drug and Cosmetic Act approved by Congress in 1938 and is supported by the US Constitution’s requirement for the Federal Government to regulate interstate commerce. The intent is to protect the American public from products that have limited or no efficacy or significant safety concerns when used as recommended by a manufacture either for the treatment of a medical condition or to aid physicians in making decisions about medical treatment. The FDA is currently structured in several divisions: Center Drug Evaluation and Research (CDER) for drugs, Center for Biologics Evaluation and Research (CBER) biologics, and Center for Devices and Radiological Health (CDRH) for devices including diagnostics. In Europe and internationally, centralized regulation of drugs and devices originated with the formation of the European Agency...