Clinical Trials

Clinical trials are designed to evaluate both safety and effectiveness. What the trial is testing depends on its phase:

Phase 1: The treatment is being tested in people for the first time, with a focus on safety.

Phase 2 and Phase 3: These look at both safety and efficacy—how well the treatment works—often compared to standard care. Phase 3 trials typically include more participants and longer follow-up periods to provide stronger evidence for FDA review.

Some trials also explore whether a certain drug is more likely to help patients with specific biomarkers or genetic features. If a treatment proves unsafe or ineffective, the trial can be stopped early—your safety comes first.

Clinical trials can be funded in a few different ways:

• Government support also plays a role. For example, Dana-Farber conducts trials funded by the National Cancer Institute (NCI) as part of the National Clinical Trials Network.
• Pharmaceutical companies may sponsor trials to test therapies they’ve developed. These companies provide funding, but independent clinical teams run the actual trial. Strict regulations ensure results are ethical and trustworthy.
• Investigator-initiated trials are created by doctors or researchers based on what they see in practice. They may seek funding from drug companies, grants, or philanthropic donors.

In cancer clinical trials, most participants receive either the current standard of care or an investigational treatment. Sometimes, a placebo is added to standard treatment in addition to regular care, not instead of it.

If a placebo might be involved in any way, you’ll be told up front during the informed consent process, where every aspect of the trial is explained and you get to ask questions before you choose to enroll.

Not at all. Some trials are designed as a first-line treatment, meaning they could be your initial therapy, based on emerging evidence that it may be more effective than current options. Your care team can help you understand where a trial might fit into your overall plan.

It depends on the trial. If standard treatment is working for you, your doctor likely won’t recommend stopping it or recommend a clinical trial at that time.

Some studies focusing on quality of life or supportive care will allow you to stay on standard treatment.

Not necessarily. Each clinical trial has unique eligibility rules, often based on safety or scientific factors. Being ineligible for one doesn’t disqualify you from others. For instance, you might not qualify for a treatment study but could still be eligible for trials focused on quality of life or supportive care. Always ask your doctor about alternatives.

In many cases, yes. If you’re benefiting from the treatment and tolerating it well, trials often allow you to continue, even after the official study period ends. These decisions are usually guided by the informed consent agreement and your trial’s institutional review board (IRB), which advocates for your safety and well-being.

Usually not. Most trial-related medications and special tests are covered by the trial sponsor. Standard medical care—like labs or imaging—may still be billed to your insurance, just like with regular treatment. Some states (like Massachusetts) require insurance companies to cover costs related to cancer research trials.

Be sure to ask during the informed consent process if there are any potential out-of-pocket costs. You may still be responsible for deductibles or co-pays tied to standard care.

Every medical treatment has risks—and clinical trials are no different—but you’ll never face them alone. Cancer research today is more advanced than ever. Scientists now understand cancer at a molecular level, which helps them design therapies that are more targeted and come with fewer side effects.

In phase 1 trials, doses start low and increase gradually to find what’s safe. Later-phase trials use data from earlier studies to start at doses already shown to be tolerable.

Because you’ll be closely monitored, you might even feel like you’re getting more attention than in standard care. Trials often include dedicated research nurses and providers trained to recognize and manage side effects quickly. If a treatment isn’t working for you, your trial team will help you make a plan for what’s next.

Yes, absolutely!  Diverse participation is essential to improving cancer care for everyone. Right now, certain populations are underrepresented in research. By participating, you’re not only helping yourself, you’re helping expand access and equity in cancer care.