Clinical Trial vs. Standard Treatment

This page is for people weighing standard cancer treatment against joining a clinical trial. It explains what each one means and how they connect.

When you talk about treatment options, you may hear two phrases:

• Standard treatment is care that medical experts accept as proper for a condition and that is widely used.
• A clinical trial is a research study that tests how well new medical approaches work in people.

One is established care. The other is research that may add to what we know. The two are closely connected.

Standard treatment is the care that experts accept and that healthcare professionals widely use for a given condition. It is sometimes called the standard of care. When your care team describes "the usual approach" for your situation, they are usually describing standard treatment.

A clinical trial is a research study that tests new ways to find, prevent, and treat cancer. Trials also help doctors improve quality of life for people with cancer by testing ways to manage side effects of cancer and its treatment. Ask your care team what is and is not known about any trial you are offered.

Clinical trials are not only about new drugs. There are several types:

• Treatment trials test new treatments or new ways of using existing treatments.
• Prevention trials look at ways to prevent cancer, often in people at high risk.
• Screening trials test ways to find cancer before it causes symptoms.
• Supportive and palliative care trials look at ways to improve quality of life.

Each type answers a different research question.

Standard treatment and clinical trials are not opposites. They are connected. People are living longer today thanks to the results of past cancer clinical trials, and NCI says people today live longer because of discoveries from past research studies. When you take part in a trial, you add to knowledge about cancer and help improve cancer care for people in the future.

The fear that stops many people is the placebo, an inactive look-alike treatment. NCI is clear that "placebos are rarely used in cancer treatment clinical trials."

When they do appear, it is in one of two situations. Either no standard treatment exists for that cancer, or the trial uses an add-on design. In an add-on design, everyone gets the standard treatment. One group also gets the new drug, while the other also gets a placebo. The comparison is standard care versus standard care plus something. Nobody is left untreated.

You must be told in advance if a placebo is part of the design.

Trials run in numbered phases, and each phase asks a different question.

• Phase 1 enrolls around 15 to 30 people. It asks whether the treatment is safe, what side effects it causes, and how high a dose people can tolerate. There is usually no standard-treatment arm. You are not comparing anything; you are the first data.
• Phase 2 enrolls roughly 50 to 100 people and asks whether the treatment appears to work against the cancer.
• Phase 3 enrolls 100 to several thousand and is the true head-to-head against standard therapy.
• Phase 4 happens after FDA approval and tracks long-term safety in large, diverse populations.

So "should I take the trial or the standard treatment?" is a different question in phase 1 than in phase 3. In phase 3 you are choosing between two credible options and letting chance decide. In phase 1 you are testing something largely unknown, usually because standard options have run short.

Costs split into two buckets. Mixing them up causes most of the billing panic.

Patient care costs, also called routine costs, are what NCI describes as "costs that you would pay even if you weren't in a trial": visits, hospital stays, standard drugs, scans, labs. These are "often covered by health insurance."

Research costs are the trial-specific ones: the investigational drug, extra scans done only for the study, research-only lab work. NCI notes that "often these costs are not covered by health insurance. But they may be covered by the trial's sponsor."

Ask your insurer three things in writing:

• Is this an approved clinical trial under your rules?
• Is prior authorization needed?
• Is the treating site in network?

Ask both, and compare the answers:

If the cancer grows on the trial treatment, what do I do next? If it grows on standard treatment, what do I do next?

Sometimes the trial preserves your options and sometimes it uses one up. A trial that requires you to have had no prior treatment for a stage may close off a standard drug you could still take later. A trial that accepts heavily pretreated patients may cost you nothing in future options. This ordering question is often more important than the trial's headline results.

Standard treatment is widely accepted, established care. A clinical trial is a research study testing something new. Whether a trial is right for you is a personal decision, not something this page can decide. Talk with your care team about the standard options for your situation, whether a trial might fit, and the possible benefits and risks of each.