From the Lab to a Patient's Bedside: The Innovation Pathway for New Medicines and Devices

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Dr. James B. Hermiller, The Care Group at The Heart Center, Indianapolis, IN, describes new treatments on the horizon for cardiovascular disease.

Now more than ever, people with cardiovascular disease can be assured there are effective medications, procedures, and other treatments available to treat their conditions. As knowledge of cardiovascular disease has expanded, so has the ability to treat the disease. Yet the medical community is always working toward new innovations to improve patient care, especially considering cardiovascular disease is the leading cause of death in the United States, with nearly 80 million Americans estimated to have a form of the disease.

New medications, devices, and other medical advancements and innovations often begin with a scientist focusing on a specific target, such as a molecule or a mechanism that plays a critical role in a particular disease. The scientist then builds a team with expertise in that molecule or mechanism. For a medicine, this may mean assembling a team of chemists, pharmacologists, and biologists to screen thousands of chemical or genetically engineered compounds to test how they may affect the targeted molecule. For a device, this may mean the inventor working with a team of physicians, engineers, designers, and material scientists to conceptualize and design a device to alter or treat the disease mechanism.

Questions are also raised at the early stage of conception, such as:

• Does the innovation pose any initial safety threats to the patient and, if so, how can those threats be minimized?
• Is the new medication or device likely to be more effective than existing therapies?
• How can it be effectively delivered (injection, inhaler, pill, etc.) or implanted?
• Can it be mass produced with the highest quality?

Every year in the United States countless ideas for new medical devices and medication are explored, but only a few will ever make it to the U.S. Food and Drug Administration (FDA), the agency responsible for protecting consumers through the regulation of products such as drugs and medical devices. New medications and devices introduced for approval by the FDA must meet strict regulations, undergo intense scrutiny, and are only approved when the FDA deems them "safe and effective when used as directed."

Sometimes a medication or device that is not approved by the FDA will be available in Europe or other parts of the world. This does not necessarily mean the device or medication is unsafe. For a device to be used in Europe it must have CE Marking. The letters "CE" are the abbreviation of French phrase "Conformité Européene," which means "European Conformity." A CE Marking on a product is a manufacturer's declaration that the product complies with the essential requirements of the relevant European health, safety, and environmental protection legislation

Approval studies, often called clinical trials, are required for approval of a drug or device in all parts of the world. Different regulatory requirements in individual countries can lead to approval in certain countries prior to other countries.

In the United States, the FDA regulates new medications and medical devices separately, but the process leading to approval, which delivers a new therapy from the laboratory to patient care, follows the same overall approach.

If a new medication or device is similar to one already in use, then the research path will be abbreviated or less stringent than for a novel (new) medication or device. For instance, new medical devices go through a preliminary market approval (PMA) if they are brand new and unlike anything currently approved and in use (on the market). In contrast, if a new device is "substantially equivalent" to a device on the market, then it will go through what the FDA calls a 510(k) process in which clinical trials are typically not required and the FDA review process is often quicker.

Medications and devices that are considered novel - unlike therapies currently on the market - are subject to intense scrutiny in the form of clinical trials to ensure they are both safe and effective. To get a sense of how intense the process for approval is, the Tufts Center for the Study of Drug Development estimates the cost to bring a medicine from the testing stage to final market approval at $802 million and the process can take five years or more.

Learn More About How Clinical Trials and Other Studies Work

• Studies are conducted using varying methods. Read about these types of studies here.
• For more information about the stages of new medicine or medical device development, click here.
• Learn how clinical trials work by clicking here.  
• There are questions you may want to ask before you decide to participate in a clinical trial. SecondsCount suggests several here.
• These days news reports often feature short summaries of clinical trials and other studies that may be relevant to your health care. Unfortunately, these new stories are usually too brief to tell the whole story. But they can still be helpful by prompting you to do some research, such as on this website. As you explore the findings of any study, we suggest the following six questions to help you make sense of health headlines. Check them out here.
• Check out additional resource on clinical trials. Several are listed here.