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...

What One Tool Is the Most Useful in Guiding Clinical Drug Development?

The title question may seem unrealistic: the scientific depth, medical intricacies, high cost, significant resources, and cross-functional logistics needed to bring a molecule from pre-IND through approval involve head-spinning complexity. Yet seasoned drug developers have found that one tool is central to ensuring a focused path through the labyrinth. That tool is the label. At this point, you might be asking how on earth the label could serve this role. Isn’t the label a long list of details, printed on tissue-like paper, written at the very end of phase III to include conclusions from pivotal trials? The answer is yes and no. Here is how the label, officially called Physicians’ Prescribing Information (PI), serves as an indispensible compass. The First PI—It’s Foundational Many companies use essential label components to drive decisions from pre-IND through approval. Market Planning PI (MPPI) is the first draft of options for claims in the eventual PI, typically including the desired indication statement, efficacy outcomes, key safety features and others. This document is based on an assessment of preclinical results, competitive landscape and clinical outcomes needed for a commercially viable, approvable new drug. Typically, there is a MPPI for each major market for the drug (e.g. US, EU, Japan), and each includes key label attributes in several sections: 1. minimum for registration and break-even commercialization, 2. target for success and 3. high commercial success. The reason for dividing into markets is that they vary in regulatory requirements and competitor drugs, and in some cases variations in clinical trials will be required. For example, the minimum case for US efficacy might be statistically significant superiority vs....

5 Things You Need to Know About Expedited Drug Development

When you are looking for ways to make drug development as effective and efficient as possible, take advantage of all the acceleration options from regulatory authorities. They may be more helpful than you think. The trend is unmistakable Increasingly, FDA and similar regulatory agencies are offering a range of expedited development and review programs, and companies are applying for and receiving the benefits. In the last few years, an average of approximately 60% of recent drug development has been in therapies approved by FDA for at least one of the expedited programs. Of recently approved drugs, about a third qualified for two or more of the programs.1 The options are diverse, and the benefits useful FDA’s four expedited programs for drugs treating serious conditions represent the largest opportunities for most companies. Fast Track Designation may be granted for a qualified infectious disease drug or one with nonclinical or clinical data demonstrating the potential to address an unmet medical need. Breakthrough Therapy designation requires preliminary clinical evidence indicating the drug may demonstrate substantial improvement in a clinically significant endpoint(s) over available therapies. These designations confer more frequent FDA interactions, potential for rolling reviews, and eligibility for accelerated approval and priority review. For Breakthrough Designation, companies receive intensive FDA guidance on their drug’s development program. For the Accelerated Approval Pathway, companies can use and gain approval based on a compelling surrogate endpoint or an intermediate clinical endpoint reasonably likely to predict a drug’s clinical benefit. The Priority Review Designation allows shorter submission review clock of 6 months vs. the usual 10-month standard review. The FDA has developed additional incentives, some quite...