What is an Experimental Treatment?

Mary McMahon
Mary McMahon

An experimental treatment is a treatment not yet recognized by the medical community, as it is still under investigation to learn more about its efficacy. Also known as an investigational treatment, it can offer a patient a chance to access innovative therapies for a condition, but it also comes with significant risks. Patients who qualify for such treatment will need to go through counseling to make sure they understand the nature of the treatment, so they can make an informed decision about whether to go forward.

Patient undergoing experimental treatments may be asked to fill out questionnaires and keep diaries.
Patient undergoing experimental treatments may be asked to fill out questionnaires and keep diaries.

Most commonly, experimental treatments are in clinical trials. A drug or medical device maker invests in the development of the product and needs to test it to see how and if it works. As the testing proceeds, the trial gets bigger, admitting more patients so the company can generate a larger pool of data. People receiving an experimental treatment have assurances that it has passed basic safety tests, but it could still be dangerous or useless.

Experimental treatments are most commonly used in clinical trials.
Experimental treatments are most commonly used in clinical trials.

A patient may qualify for an experimental treatment if she does not respond to conventional treatment, has a condition that has progressed beyond regular treatment, or has an unusual condition that does not have an established treatment yet. Usually people access the treatment and supportive care for free because they are participating in medical research. Drug companies may also offer experimental treatments on compassionate grounds if a patient doesn't qualify for a clinical trial but could still benefit.

Experimental treatments come with significant risks.
Experimental treatments come with significant risks.

While on an experimental treatment protocol, a patient will need to report for regular medical appointments. The doctor will assess the patient's response to treatment, check for side effects, and take blood and tissue samples, if necessary, for members of the research team. Patients may need to fill out regular questionnaires, keep diaries, and perform other tasks to assist the researchers. This will allow researchers to identify side effects and other issues with the medication or device. Much of this information will go into the packaging and recommendations if government agencies approve the treatment for sale.

Insurance companies typically do not cover this type of treatment. They expect doctors to pursue conventional means first and may decline to cover investigational therapies or drugs. Patients can appeal to see if the company will change its mind in special circumstances, but successful appeals are rare. There are concerns about liability and other legal issues that companies usually try to avoid by simply refusing all requests for coverage in experimental treatment situations.

A patient who does not respond to conventional treatment may qualify for an experimental treatment.
A patient who does not respond to conventional treatment may qualify for an experimental treatment.
Mary McMahon
Mary McMahon

Ever since she began contributing to the site several years ago, Mary has embraced the exciting challenge of being a wiseGEEK researcher and writer. Mary has a liberal arts degree from Goddard College and spends her free time reading, cooking, and exploring the great outdoors.

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Discussion Comments


Although it may seem that clinical trials are inhumane at best, we must remember that most of the trials that actually get to a human level of experimenting have already been tested extensively on other life forms.

Now, they are being tested to make sure that they are safe, but the truth is that the powers that be are fairly certain of their safety to some degree before they begin human trials.

I personally believe that these kinds of experiments will eventually lead to not only the treatment for HIV and cancer, but also the cure.


Although I definitely understand the significance and importance of clinical trials, even on human subjects, I must say that they terrify me. It seems that we are so advanced in so many ways, but it would not take very much to bring down our world as we know it.

For example, in the movie (based on the book) “I Am Legend” a team of doctors has found a supposed cure for cancer. They have utilized a virus already in existence (measles) and mutated it to be good for the human body.

However, before long the human experiments begin to mutate into an entirely different group of beings which are predators. Then it is realized that because this ‘cure’ came from a common virus, it is transmitted in the same ways that many viruses are – both through bodily fluid and through the air.

I know this is fiction, but it just seems to ring as a huge possibility with all of the advancements that we have today. The people in the movie were actually almost eradicated because of their inability to deal with such a mutated virus that began with a clinical trial.


If you are participating in an experimental trial, spare a thought for the nurses who are helping to run it.

They are often having to work with three or four medical trials at once, and they are the ones who have to be on call twenty four hours a day, so that if something goes wrong there is someone there to talk to the patient.

And, you know, I'm sure they get a lot of calls that have nothing to do with the trial, but are just a panicked person who had too much to eat the night before, or whatever.

I have nurse friends who do it, and love it, but it is a super hard job and you should bear it in mind if you ever decide to do one of these experimental treatments.


Experimental treatments might feel like they are on the cutting edge, and they often are in terms of human subjects, but usually the drug has been in development for many years before it reaches the stage where it can be given to human patients, even ones who have agreed to this kind of thing.

Either that, or it is only a slight variation on an already widely used drug. Drug companies who want to keep their patents on particular chemicals will often reuse them in a slightly different way, so they can re-release them and get the patent extended. Even though they know there's no real difference, they still have to go through with the trials.

Anyway, my point is that most of the time there is no real risk in participating in these trials. While there have been people who got sick, or died from participating in experimental treatments, they are few and far between.

I know a few people who pretty much put themselves through college just doing medical trials. You can't make a lot, but you can make some money.

And other people who managed to get treatment they might not have had any other way.


I am a female college student, and I see advertisements for clinical trials all over my campus, and especially in the women's bathrooms. Most of the trials are looking for late teen or young adult women to participate.

I did not really know much about experimental treatment, so I never really follow up on any of these advertisements. I am glad I stumbled across this article, it has taught me a little more about what these trials are all about. Maybe next time I see an advertisement looking for young adult participants, I will look more into it.


I once heard someone say that "Science requires victims." That line has always stuck with me because it is so haunting but also so true. It is often the case that the most scientific knowledge is generated from failed experiments. When science can examine what doesn't work it can lead to huge advances. It is sad to think that people get hurt and sometimes even killed in the process, but there deaths are not totally in vain. Their sacrifice can lead to treatments and practices which can save the lives of countless others


I participated in some experimental medical research when I was in college in order to make some extra money. In one study I was observed while I slept. In another I was asked to use a skin cream that helps to treat rashes and in a third one I tested and experimental cold medication.

I never had any problems in any of the studies. The scientists warned of risks, but I never had any negative reaction. In fact the entire experience was a positive one. I made some much needed extra money and I did my part to help advance science. I think that is a win win


I have a seven year old son with a rare heart condition. Last year, he participated in a clinical trial for a pediatric heart medication. The doctors cannot prescribe him any other medication because they are all too strong for his young body to handle. When his pediatrician told me about this experimental drug made specifically for children, I was immediately intrigued.

Of course, there were risks involved in my son taking this drug that had no proof of being successful or government approval. But, his doctors told me that this drug had been taken by other children with similar heart conditions, and the results were positive. So, my husband and I decided to give this drug a try.

Thankfully, my son's body reacted well to the drug. After taking it, his heart became stronger. He now has the strength to play with his older sister. Me and my husband do not have to worry about him getting too weak and fainting. I am thankful for this drug, and I encourage others to take a chance on experimental treatment.


Experimental treatments, while risky, are important for pushing the boundaries of science. If we are going to make progress and have a series of productive breakthroughs we have to push the limits a little bit. The key is maintaining a balance. Experimental treatments have to ensure the safety of the subject being tested while also exploring territory that science is still unsure of. Good scientists will put in a lot of thought and planning before they ever treat a subject.

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