Experimental Medical Treatment

If you could choose the most cutting-edge medical cure, would you want to?

When the reality of a serious illness hits, most patients and their loved ones want the best possible treatment available. For many, this means getting the safest, most up-to-date, intervention possible. But the definitions of “best” and “most up to date” differ for different individuals.

Doctor and patient using digital tablet in office
JGI / Tom Grill / Getty Images

Medical research is moving at a fast pace. Medical scientists are discovering new ways to treat illnesses that may take years to validate and to deem safe enough for the public. The process of developing a new medical treatment often requires methodical experimental trials. When innovative therapy is ready for real live patients, volunteers are often recruited to help assess whether the treatment is safe and effective through clinical trials.

What Are Clinical Trials?

Clinical trials are experiments designed to determine if a new medication or treatment is safe and effective in humans. The foundation of any clinical trial is the comparison between 2 groups of participants—usually one group that is receiving a type of intervention and another group that is receiving a different intervention or no intervention. The treatment and data are carefully monitored by a research team and then evaluated to assess the difference in outcomes between the 2 groups.

Who Oversees Clinical Trials?

Approval and oversight of clinical trials are quite stringent—requiring detailed applications and approvals at multiple levels. Researchers must be experienced and qualified in order to obtain authorization to implement clinical trials. Generally, a hospital or university or pharmaceutical manufacturer requires preliminary data on safety, sometimes obtained through animal testing, before allowing a human study. Usually, a federal agency, such as the Food and Drug Administration, provides structured oversight and criteria.

Is a Clinical Trial Right for Me?

Pharmaceutical companies, medical device manufacturers, government grants, foundations or non-profit charities often fund the experimental costs. Sometimes, research scientists at universities receive funding from one or more of these sources and may work in collaboration with teams from multiple clinics.


  • Some trials provide reimbursement to patients for medical costs or even cash compensation
  • Volunteers have the opportunity to try new therapeutic options
  • Volunteers do not have to miss out on new treatments just because they haven’t been approved yet
  • Some volunteers have no other medical option besides experimental treatment when faced with a bad prognosis
  • Volunteers can usually end participation at any time if they do not like the intervention
  • The experiment might require more medical visits and monitoring, and thus possibly more personal attention and better health care as a “built-in” benefit
  • Some volunteers appreciate the opportunity to play an active role in scientific progress


  • In some research experiments, the safety is not well established
  • The benefits of many experimental treatments are not well established
  • There are many unknowns
  • Volunteers and their healthcare providers usually don’t know if they are in the treatment group or the no-treatment group
  • The experiment might require more medical visits and monitoring, which can be time-consuming

How Can You Find a Clinical Trial? 

In some cases, your healthcare provider might suggest a clinical trial for you to give you access to the treatment you might not otherwise be able to get.

You can ask your healthcare provider if there is an experimental treatment that you qualify for. You can also search your hospital’s website or the websites of nearby universities by searching for your condition and “clinical trials” or “research.”

You can look for trials through the National Institutes of Health’s clinical trials database or the agency’s health information site. Also, specialized professional groups may list resources for certain diseases. For example, the American Heart Association provides information about stroke research. Non-profit organizations and disease-specific foundations may also help provide some direction when they fund clinical trials.

A Word From Verywell

For some, the best treatment means the safest and most thoroughly tested available. For others, the best intervention means the absolute best around—anywhere—even if information about its safety and effectiveness is incomplete.

No matter where you see yourself on the spectrum, it never hurts to learn about experimental treatments for your illness, and learning about them does not require you to sign up.

By Heidi Moawad, MD
Heidi Moawad is a neurologist and expert in the field of brain health and neurological disorders. Dr. Moawad regularly writes and edits health and career content for medical books and publications.