When a company decides to sponsor an FDA clinical trial, there are a few phases prior to approval for a particular indication. After the FDA gives approval to conduct the human aspect of studying the medication or a procedure, there is a particular method performing the study to make it valid.
The studies are actually really expensive and have a high risk but high reward for a sponsoring company. They may not be high risk for the patients involved, what I am referring to is the expense weighted against whether or not there's going to show significant improvement in the disease process with what is being studied.
Let's use an example of a stem cell injections study for low back pain. This is actually a real example, as there is an Australian company performing a stem cell injection study for degenerative disc disease at multiple sites in the United States.
In order to show that the treatment works and gets approved by the FDA, the company needs to set up the study with comparison against another treatment. This may be the current gold standard for treatment of the condition, or it may be what is known as a placebo. In the case of the stem cell injection clinical trial, the regenerative medicine injection being studied is being compared versus a placebo. The placebo may simply be saline or a liquid with no medication whatsoever. This should not have a true affect on patients, but it is well-known that it does approximately 30% of the time.
When a patient meets the inclusion criteria for the FDA clinical trial, they will end up being randomized into one of the two groups. Sometimes this comes out of an envelope, or a computer generated sequence ends up telling which treatment the patient ends up receiving.
The usual method is that if 100 patients are being selected for a clinical trial, then 50 will be in the treatment group and the other 50 will end up being in the placebo group with randomization taking place to make sure there's no bias in selection.
For the stem cell treatment clinical trial, patients are then followed at regular intervals checking both objective information along with subjective information. This would mean looking to see how much pain medication the patients are requiring after the injection, and also checking to see what their pain scale scores are as the study moves along. Any adverse events from the injections are noted, and if there is a truly serious situation then the FDA would be notified immediately.
There are fairly strict criteria for who can qualify for an FDA clinical trial. For instance in the stem cell clinical trial, patients need to have degenerative disc disease at only one level. Along with this, patients need to have over 70% of their pain situated in the low back and not more than 30% down into their legs.
Once the enrollment is complete, usually the study is closed. There are times when patients can still be enrolled as a humanitarian exemption. The timeframe for follow up on an FDA clinical trial will vary somewhat, with medical devices the typical for timeframe is two years. With biological substances such as stem cell injections, the timeframe would be substantially less, maybe one year.
Once the study follow up is completed, it is up to the funding company to make sure that the proper reporting is in with the FDA. The results can be published in peer-reviewed journals, and often make for excellent publications because they are what it's called level one evidence studies. This means they are prospective and randomized. With a study such as the stem cell injections, there will also be double blinded. This means neither the pain management doctor nor the patient in study will not know exactly what is being injected.
The FDA clinical trial process is expensive and uses up a lot of resources, but it can pay off huge dividends for a company if the treatment works and the FDA approves the product.
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