Understanding Clinical Trials: Key Issues

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.

Here are some factors that might influence your thinking about participating in a clinical trial:

1. Your type of cancer and stage of cancer
2. 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.
3. 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 any side effects you may experience.
4. 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.

Don’t hesitate to bring these issues up when you talk to your doctor or the clinical trials coordinator.

As you think about your goals and values, it can also be helpful to consider the ways clinical trials are changing. There are new ways of thinking about clinical trials when it comes to:

• How they are structured
• How patients are recruited 
• How the success or failure of a trial is measured

New models of clinical trials 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.

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

Learning from 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.

Focusing 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

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. These 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.