Clinical Trials for Medical Device Innovators Part I: Introduction and Designing Clinical Trials

The first, in a blog series about clinical trials for medical device innovators.

In 2017, Cook was approached to co-author the recently published book, “Medical Innovation Concept to Commercialization.”  William D. Voorhees III, Ph.D., Vice President and Chief Science Officer and Theodore Heise, Ph.D., RAC, Vice President Regulatory and Clinical Services, both of MED Institute, collaborated to write the chapter, “Clinical Trials for Medical Device Innovators.” Overall, the purpose of this book is to provide physician-inventors and entrepreneurs with a practical, step by step approach to move a novel concept from the back of a napkin to a tangible, commercially successful product.

Cook News August 2018 MED InstituteWilliam D. Voorhees III, left, and Theodore Heise, holding a copy of “Medical Innovation Concept to Commercialization”.

Dr. Voorhees earned his A.B. in Biology from Hamilton College and a Ph.D. from the Department of Veterinary Physiology & Pharmacology at Purdue University.  As a faculty member of the Hillenbrand Biomedical Engineering Center at Purdue, Bill conducted a wide variety of original research in applied physiology including CPR, transchest cardiac pacing, electroventilation, hyperthermia therapy for solid tumors, hypothermia to protect the myocardium during myocardial infarction, and respiratory function monitoring.  He has authored over 70 refereed scientific articles resulting from this research.  He also served as liaison with the R&D Department of Methodist Hospital of Indiana in Indianapolis.  Bill has been with MED Institute for 28 years, joining the newly formed Clinical Trials/New Product Approvals Group in September 1990.  He was named Vice President/Chief Science Officer in September 2001.  Bill also serves as the Director of Regulatory Affairs and Director of Scientific Communications.  He takes seriously his commitment to patients to ensure the quality and scientific integrity of the research conducted by MED Institute.

Dr. Heise has 25 years’ experience in regulatory affairs, currently serving as Vice President of Regulatory and Clinical Services at MED Institute.  In this capacity, Ted leads the company in designing scientifically robust regulatory and clinical study strategies for its clients: entrepreneurs, consultants and physicians bringing novel medical products through the complex steps of the development process. Graduating with a BS in chemistry from the University of Nebraska at Omaha, Ted went on to earn a Ph.D. in analytical chemistry from Iowa State University.   He has been a member of the Regulatory Affairs Professionals Society since 1993, and the American Chemical Society since 1988. Ted is a U.S. delegate to the technical committee for international consensus standards that governs biocompatibility testing and clinical investigations of medical devices, and serves as convener of its working group on chemical characterization. Ted is also active in developing processes to generate real world evidence for medical devices, representing Cook Medical on the corporate stakeholder board for the SVS/Vascular Quality Initiative and participating in projects within MDEpiNet and Harmonization by Doing.

In their chapter from the book, “Medical Innovation Concept to Commercialization,” presented here as a series of 5 blog posts, Bill and Ted share the wisdom they have gained from their 20 plus-year adventure designing, conducting, and analyzing clinical trials on novel medical devices.

Designing Clinical Trials

Perhaps the most exciting and rewarding time in the development of a new medical device is the first time one sees it work successfully in a patient, but the ensuing clinical trial can be the most exhausting, frustrating, expensive, and time-consuming part of the entire enterprise. So before rushing headlong into the unknown, consider the following: “Is it necessary for me to conduct a clinical trial of my new device?” The answer is “It depends.”

It depends on what you are trying to accomplish. Maybe you just want to show that your device works (i.e., the Proof of Concept). Perhaps you have reached a point at which no more can be learned without testing in humans, for example, pain relief cannot be studied on the bench at all and rarely well in animals. Your goal may be to demonstrate product effectiveness to investors and to raise additional funds. Maybe all you want is enough clinical data to improve your chances of selling the rights to the intellectual property. Perhaps you just want to enhance market awareness and exposure to the device (such as having a few key opinion leaders use it and write a paper), or maybe you want to take it all the way to regulatory approval with proof of safety and effectiveness, which is collected typically in what is called a pivotal study. Even if you want to own the regulatory approval of your device, keep in mind that a clinical study may not be needed if you can show that all relevant risks can be mitigated by bench and/or animal testing or with appropriate labeling. This is why a rigorous risk-analysis is imperative to your plan for development of the device.

Assume you have developed your device to the point that you have made prototypes that you have bench-tested adequately to satisfy yourself that they function as expected and they appear safe to use in humans. The “Grandmother Test” is a good benchmark; would you let your grandmother be treated with this device? Even if the answer is “Yes,” you probably need a clinical feasibility study first. Conducting a clinical feasibility study may also be a good first step when only formal testing in humans can provide the evidence that the device offers the expected benefit (Proof of Concept) or is required for making any final changes in design (e.g., to apply to specific human anatomy). Such data can simply be gathered no other way than to test in humans. Even if you know you will need a pivotal study for regulatory approval, it is likely that you will need initial information about how large a therapeutic effect the device will have before you can design a larger pivotal study to compare its results with state-of-the-art therapy (often a currently marketed device) in a statistically rigorous clinical study. A feasibility clinical study can be used to provide this crucial information.

If your interest lies only in developing the new device to the point that the concept (intellectual property) can be sold to another company to finalize its development and take it to commercialization, then conducting a clinical feasibility study may be adequate to provide the information that the company planning to purchase your device will need to decide to close the deal.

For more information, please visit our website at https://medinstitute.com/.

Continue to follow our blog to view the upcoming Part II: Regulatory Considerations for Clinical Studies.