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.
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.
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.
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.