New technologies continue to affect the practice of otolaryngology–head and neck surgery. Numerous financial and regulatory barriers must be overcome to develop an idea into a useful device or intervention. US Food and Drug Administration (FDA) approval focuses on safety, often leaving the medical community, in general, to determine the efficacy of the device after FDA approval has been granted. Physicians are involved throughout the technology development process, generating conflicts of interest that must be effectively managed. It is essential that physicians ethically maintain their leadership in developing and evaluating new advances in medical technology.
Technology has always been a part of the practice of medicine. Stethoscopes and sphygmomanometers were as revolutionary a change when they were developed as advanced imaging or minimally invasive surgical techniques have been more recently. Physicians are driven to provide the best possible care for their patients, which often leads to the development, evaluation, and adoption of new medical technologies. This phenomenon is seen in otolaryngology–head and neck surgery, where diagnostic or therapeutic advances can make disease processes more accessible (eg, endoscopy) or allow them to be treated with less morbidity (eg, microscopic surgical advances in laryngology).
Integration of a new technology in medicine would ideally be preceded by a rigorous objective evaluation of safety and efficacy. An underpinning of evidence-based medicine (EBM) is that physicians provide the best care for their patients when it is based on such objective evidence. With regard to new technologic advances, however, practicing strict EBM is often not practical. Medical device developers are subject to numerous financial pressures that require them to bring their products to market and facilitate their use before efficacy is rigorously demonstrated. Moreover, current device regulation takes a rather permissive approach, with most developers using an approval mechanism that compares novel devices to currently approved products.
Rhinology, which has arguably undergone revolutionary changes in the last 20 years, provides multiple examples of generalized adoption of technology preceding evidence of efficacy, often by several years. Endoscopic sinus surgery was first practiced in the United States in the early 1980s, having been adopted from practices in Europe. The first widely published report of the technique appeared in the United States in 1985, followed by a generalized adoption of this technique during the ensuing decade. It is instructive to point out, however, that evidence of efficacy of this new technique did not appear until the early 1990s. Powered instrumentation is another technique that has significantly affected rhinology, whereby its description and adoption preceded evidence of its efficacy by about 5 years. Image guidance has made an increasing impact on rhinology, going from a technology limited to tertiary rhinology practices to a tool available to most rhinologic surgeons. Nevertheless, the evidence supporting its use in endoscopic sinus procedures is limited. These examples illustrate that despite physicians’ dedication to the principles of EBM, valuable new technologies are often adopted before the demonstration of their efficacy.
Developing an idea into a medical device
A new device is invented by a physician and/or an engineer responding to an unmet clinical need. The inventor (or his or her employer, particularly in an academic setting) thus becomes the owner of intellectual property (IP), which is usually protected by patenting the invention. This process typically costs $5000 to $10,000, a cost borne by the owner of the IP. This cost is in addition to any development costs in materials or time that the inventor has already expended. The next step is to develop the invention into a mass-producible and mass-useable device. This is another time- and investment-intensive process, whereby the physician-inventor either partners with or becomes an entrepreneur, a person who strongly believes in the potential of the investment and is accustomed to high risk, hoping for what is often a long-delayed future reward. Indeed, it has been suggested that the definition of an entrepreneur is an individual willing to work very hard for free.
As the device is further refined, a final product eventually results. The final steps in this process must be performed using regulated Good Manufacturing Practices and Good Laboratory Practices, known separately as GMP and GLP. Toxicity measurements must also be performed according to international standards. These methods are necessary to satisfy the regulations that govern devices in the United States and Europe. The processes necessary to bring a device to practice are obviously beyond the capabilities of a physician’s basement workshop. The development of a medical device from idea to final product thus requires between 10 and 20 million dollars. These extensive financial resources are often supplied by a venture capital group, and increased risks are adopted in the hopes of increased potential returns on that investment. Alternatively, an established medical device company may partner with the physician-inventor, expending similar resources and expecting a similar return for that investment.
Obtaining FDA approval
Medical devices in the United States are regulated by the Center for Devices and Radiological Health (CDRH) of the FDA, a branch of the Department of Health and Human Services. The FDA’s drug-regulation and device-regulation mandates have evolved separately, so that these 2 classes are regulated differently. The Food and Drug Act of 1906 established the FDA’s legal basis for regulation. The Federal Food, Drug, and Cosmetic Act of 1938 required the FDA to establish the safety of drugs, and it was not until the Kefauver-Harris Drug Amendments of 1962 that proof of drug effectiveness was required. It was not until 1976 that medical devices were specifically addressed by the Congress. The Medical Device Amendments of 1976 expanded the FDA’s role in device regulation and specifically required the CDRH to assign devices into 1 of 3 classes, based on their complexity and potential risk and thus their need for regulation.
The scope of medical devices is staggering. The CDRH regulates nearly 2000 types of devices, ranging from bandages and gloves to pacemakers and tissue allografts. Of these nearly 2000 types of devices, about 500,000 medical device models from 23,000 different manufacturers are monitored by the CDRH. Medical devices affect the care of nearly every patient in the United States, with 4% of patients having an implanted device. Medical devices comprise a 130-billion-dollar industry worldwide, with nearly half of the consumption taking place in the United States. The FDA’s device-approval process is divided into 2 pathways, depending on the degree of novelty and risk of the proposed device.
Approval for a novel or high-risk device may start with an application for Premarket Approval (PMA), and the process is similar to the review of a new drug. The PMA review process requires clinical evidence of safety and efficacy before approval. Because of the increased time and expense of this more rigorous process, less than 100 of the approximately 4000 applications that the FDA receives each year are PMAs.
The second, more streamlined process available for devices is known as the 510(k) pathway. This process is used when the proposed device can be demonstrated to be “substantially equivalent” to a device that already has FDA approval. In this context, the previously approved device is known as a predicate device. This process is much quicker, with approval occurring within 90 days of application submission, typically without a requirement for clinical evidence of efficacy. In fact, 90% of 510(k) applications have no such clinical data submitted. With more than 95% of medical devices receiving FDA approval as a result of this expedited 510(k) process, most devices reaching the physician’s hands have not objectively demonstrated clinical efficacy. The current regulatory environment thus facilitates the availability of the latest potential clinical advances for patients. This “buyer beware” arrangement implicitly relies on individual practitioners to determine, often based on scant evidence, whether a device is most likely to be beneficial to an individual patient or not. It leaves proof of efficacy to practitioners rather than tying up potential advances in prolonged regulatory processes. In fact, it is during this postmarketing phase that evidence of efficacy is developed (and additional safety data is accumulated). For this reason, medical technologies enter clinical use well before their efficacy is established.
Obtaining FDA approval
Medical devices in the United States are regulated by the Center for Devices and Radiological Health (CDRH) of the FDA, a branch of the Department of Health and Human Services. The FDA’s drug-regulation and device-regulation mandates have evolved separately, so that these 2 classes are regulated differently. The Food and Drug Act of 1906 established the FDA’s legal basis for regulation. The Federal Food, Drug, and Cosmetic Act of 1938 required the FDA to establish the safety of drugs, and it was not until the Kefauver-Harris Drug Amendments of 1962 that proof of drug effectiveness was required. It was not until 1976 that medical devices were specifically addressed by the Congress. The Medical Device Amendments of 1976 expanded the FDA’s role in device regulation and specifically required the CDRH to assign devices into 1 of 3 classes, based on their complexity and potential risk and thus their need for regulation.
The scope of medical devices is staggering. The CDRH regulates nearly 2000 types of devices, ranging from bandages and gloves to pacemakers and tissue allografts. Of these nearly 2000 types of devices, about 500,000 medical device models from 23,000 different manufacturers are monitored by the CDRH. Medical devices affect the care of nearly every patient in the United States, with 4% of patients having an implanted device. Medical devices comprise a 130-billion-dollar industry worldwide, with nearly half of the consumption taking place in the United States. The FDA’s device-approval process is divided into 2 pathways, depending on the degree of novelty and risk of the proposed device.
Approval for a novel or high-risk device may start with an application for Premarket Approval (PMA), and the process is similar to the review of a new drug. The PMA review process requires clinical evidence of safety and efficacy before approval. Because of the increased time and expense of this more rigorous process, less than 100 of the approximately 4000 applications that the FDA receives each year are PMAs.
The second, more streamlined process available for devices is known as the 510(k) pathway. This process is used when the proposed device can be demonstrated to be “substantially equivalent” to a device that already has FDA approval. In this context, the previously approved device is known as a predicate device. This process is much quicker, with approval occurring within 90 days of application submission, typically without a requirement for clinical evidence of efficacy. In fact, 90% of 510(k) applications have no such clinical data submitted. With more than 95% of medical devices receiving FDA approval as a result of this expedited 510(k) process, most devices reaching the physician’s hands have not objectively demonstrated clinical efficacy. The current regulatory environment thus facilitates the availability of the latest potential clinical advances for patients. This “buyer beware” arrangement implicitly relies on individual practitioners to determine, often based on scant evidence, whether a device is most likely to be beneficial to an individual patient or not. It leaves proof of efficacy to practitioners rather than tying up potential advances in prolonged regulatory processes. In fact, it is during this postmarketing phase that evidence of efficacy is developed (and additional safety data is accumulated). For this reason, medical technologies enter clinical use well before their efficacy is established.
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