Understanding Clinical Trials: Key Issues

A sense of clarity

Your Values and Clinical Trials

"I would tell every cancer patient who is making decisions about their treatment, to slow down and take the time to understand the options—and to think about how those options fit into their lives."

Meg Gaines, JD, patient advocate

Enrolling in a clinical trial is a choice you make because you hope and believe it will benefit you. It’s an important decision and it makes sense to think about it in terms of what is important in your life and what is happening with your cancer.

Below are some factors that might influence your thinking about participating in a clinical trial. Don’t hesitate to bring these issues up when you talk to your doctor or the clinical trials coordinator.

  • Your type of cancer and stage.
  • Your life: This is a very good time to think about what you want and hope to accomplish. It is also important to have an honest discussion with your doctor about the realistic expected outcomes for your treatment
  • Possible side effects: When you discuss the trial with your doctor, ask about the likely side effects. Think about how these side effects may impact your life and what you want to do. Ask about what plans are in place to help manage side effects you experience.
  • What you want to get out of the trial: Each person has their own reason for why they participate in a trial, and there is no right or wrong reason. For some it’s the hope of living longer or better. For others it can be contributing to a better future for cancer treatment

New Models for Clinical Trials

There are new ways of thinking of clinical trials—in how trials are structured, how patients are recruited and how their success or failure is measured. These include:

Matching Patients to Targeted Therapies

Instead of looking for people with a specific type or stage of cancer, these trials match people with specific genetic mutations to targeted therapies.

Flexible Approaches

Instead of waiting until the trial ends to assess whether it is working, these “adaptive approaches” add and subtract treatment arms as the trial progresses, based on what is working and what is not.

Exceptional Responders

In the past, a trial in which a very small percentage of people benefitted would have been a failure. Today, there is great interest in understanding why these people did so well and using that information to develop new targeted therapies.

Focus on Preventing Recurrence

The best chance for curing most cancers is to treat it effectively the first time. Neoadjuvant and adjuvant trials aim to reduce the risk that a cancer will return or spread by improving the primary treatment.

Childhood Cancer and Clinical Trials--Another Perspective
"We evaluate every patient who comes here for a clinical trial and more than 95 percent of our patients are entered into one at some point in their treatment. We are also actively involved in research on cancer survivorship and distress screening for our patients and families. Both are critical to improving the outcomes for our patients."

Aziza Shad, Medstar Georgetown Cancer Center

Less than 5% of adults with cancer participate in a clinical trial—but over 60% of children and young adults enroll in a trial at some during their treatment. These include trials to improve survival but also many studies aimed at reducing both short and long term side effects of treatment. While cure rates vary for specific cancers, overall almost 80% of children diagnosed with cancer are now considered cured.

Much of this progress is because of clinical trials. There are various reasons for why more children than adults participate in clinical trials, but factors include:

  • Most children with cancer are treated in academic medical centers where clinical research is the norm
  • Parents of children with cancer are willing to enroll their children in studies to improve their chances of survival and their quality of life
  • Insurance companies have traditionally covered all the cost of clinical trials for children

There are differences between adults and children when it comes to cancer treatment, but there is also much to be learned from the clear positive results of having such a high percentage of young cancer patients enrolled in these studies.