What New Clinical Trial Participants Can Expect

Clinical trials develop necessary information and feedback about new medical products. Participants who have not been satisfied with readily available products and devices may want to consider the advantages and disadvantages of participating in clinical trials. Knowing what to expect will minimize confusion and disappointment.

How can I be sure to get the new medical device?

Most clinical trials are “randomized”, and this means that a certain percent of clinical trial participants will not actually receive the product being tested. Generally, every clinical trial will have a group of subjects who either receive a placebo or receive the currently approved standard of treatment, and another group of participants who receive the new product. The reason for this is that individuals receiving the current standard of care or device serve as a control group, and their outcomes are compared to those outcomes of the group receiving the new product. If everyone received the new product, the sponsor could not make a valid comparison. Having said that, a participant may ask to be in the group receiving the new product, and it will be up to the investigators conducting the clinical trial to determine if that request can or should be honored under the clinical trial guidelines. It is typical for a randomized clinical trial to have 60 to 70% of participants receive the new treatment, so the odds tend to be a little better to land in that group.

How long do clinical trials last?

The duration of clinical trials vary from months to years (including follow-up assessments), depending on the type of clinical trial being conducted and whether a drug or a medical device is being tested. Prevention clinical trials will frequently last 10 to 20 years in order to evaluate the long-term implications of lifestyle changes such as exercise or diet. A more common span is three to seven years for new drugs or devices.

However long a clinical trial lasts, a clinical trial may have several stages. Clinical trials for new drugs proceed through four distinct phases, starting with a small group (fewer than 100) of patients and progressing to larger trials and usually further trials after the drug is approved. (Not all drugs and devices complete the whole protocol. Many times a promising drug or device exhibits complications as the clinical trial progresses and the trial is halted, either permanently or until the protocol can be refined.)

In contrast, a clinical trial for a new medical product that is a device (e.g., an artificial disc or other type of spinal implant) may be considerably shorter. In general, devices receive an Investigational Device Exemption (IDE approval) and then the sponsor is allowed to conduct a multi-center clinical trial to test the device’s safety and efficacy. Therefore, new medical devices are not tested as thoroughly as new drugs which sometimes are tested on thousands of subjects before the drug is approved for general release. Many clinical trials for new medical devices have fewer than 200 participants, so the applicability of the findings to an individual’s case may be marginal.

Both new drug and new medical device clinical trials will often have Phase IV post-marketing studies (also called Phase IV commitments), which are conducted after the drug or device has been approved and released for use in the general patient population. In Phase IV manufacturers agree to conduct follow-up studies to monitor side effects and recommend refinements to dosage, usage, etc. Follow-up studies may also be done to identify new indications or uses for the product.

Can an American participate in a foreign clinical trial for products?

It is possible, but not a frequent occurrence, for a U.S. citizen to participate in a clinical trial overseas. Although it is true that many products are approved faster by European authorities (such as artificial discs), it may not follow that anyone physically in Europe can obtain these products. Aside from the potential logistical hurdles, such as traveling to Europe to participate and finding a clinical trial location in which you are comfortable, the manufacturer conducting the clinical trial may exclude Americans because the maker would not want to put eventual U.S. approval at risk by allowing Americans to use a device that has not won FDA approval for use in the U.S. Sponsors of clinical trials conducted in the U.S. use a case-by-case analysis to determine if non-U.S. citizens can participate.

If I am not selected can I still obtain the new treatment or device?

In some cases, yes. The FDA sometimes enables manufacturers to provide new treatments under expanded access or continued access programs designed to help individuals who have a demonstrable need for the device but did not participate in the formal clinical trials. Generally expanded access to experimental treatments is granted in cases of a life-threatening or serious disease for which there is no alternative treatment, and for which the experimental treatment actually shows promise in clinical trials. Recently the expanded access protocols have been used to deliver drugs to individuals with HIV, the virus that causes AIDS.

Continued access” programs also make it possible to obtain a new medical device quickly. Continued access initiatives allow people to receive the product after the clinical trial has been fully enrolled but before the FDA has approved the product for use in the general patient population. Data is collected on patients who receive devices implanted under continued access programs, although these procedures are not part of a randomized clinical trial. The makers of the Charite artificial disc, for example, provided significant continued access to the device through investigators who conducted the clinical trial while the FDA continued to evaluate the device.1 These programs can serve as a valuable alternative to gain access to innovative products because there can be a two to three year lag between the end of the enrollment in the initial clinical trial and actual approval of the device.

If a particular clinical trial involves a new medical device that is designed to treat a patient’s condition, prospective participants should be sure to ask direct, specific questions about cost, post-trial follow-up, and other issues during the screening process before they agree to participate in a clinical trial for a new medical product or new medical device.

1. The Charite artificial disc was granted approval by the FDA in October 2004.

Please note that the contents of this article synthesize publicly available data and have not been peer-reviewed by Spine-health.com's Medical Advisory Board.

By: Stephanie Burke, CEO, Spine-health.com