Clinical Trials: Everything Cancer Patients Should Know

You may have heard about “clinical trials” or “clinical research” from your doctor or read it in an article. The term, itself, is technical-sounding. Here’s more about what they actually mean – in “human terms” – in the cancer space, with examples from actual patient stories (scroll below).

Clinical trials are studies that are key to advancing scientific development. They offer researchers an opportunity to understand the potential impacts of new drugs and give patient’s access to medicine that may not have been available to them otherwise.

What Are Clinical Trials?

Clinical trials are a stage in medical research that tests the effectiveness of a treatment on human patients. The FDA allows researchers to conduct clinical trials once a treatment has shown promise in animals. Clinical trials can be used to find treatments that are better at helping a medical condition, or to determine if a new type of treatment is safer than the current standard of care.

What Do Clinical Trials Study?

Clinical trials test new interventions -meaning treatments like drugs, or other procedures- that have the potential to be more effective or safer than the current standard of treatment for a disease or condition. Researchers don’t yet know if the intervention used in a clinical trial works, and many treatments never make it past the clinical trial stage.

Without clinical trials, doctors would have no way of knowing if a better treatment exists for a disease. Clinical trials can result in new treatments or even cures for diseases that were previously considered terminal.

It’s important that patients understand that clinical trials aren’t a magic bullet — ultimately, only a small percentage of drugs in clinical trials will receive FDA approval.

Are Clinical Trials Safe?

When you elect to participate in a clinical trial, you will have the risks of the trial explained to you. Potential risks include uncomfortable treatment, treatment that is not better than the current standard of care, and the fact that you may end up in a control group (group that does not receive the experimental treatment, instead receiving a placebo or typical existing treatment).

In the past, clinical trials were sometimes unsafe and unethical. Now, clinical trials must adhere to very strict standards that protect patients. In 1978, the National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research created The Belmont Report, a set of fundamental ethical principles and guidelines that assist researchers in resolving ethical principles when conducting research involving human subjects.

Modern-day clinical trials require explicit consent from the patient. Doctors will never involve you in a clinical trial without your consent, and you are allowed to end your participation in a clinical trial at any time.

Clinical trials must also adhere to strict schedules. While this may mean your appointments are longer or not as flexible, it also means that you will know exactly when and for how long you are receiving treatment.

What Are the Main Phases of a Clinical Trial?

Clinical trials can go through up to four rigorous phases of testing to ensure that they are safe for the general population. Only about 33% of all clinical trial drugs make it through Phase II.

Phase I

Phase I trials test drugs that often have never been administered to humans. The purpose of a Phase I trial is to determine whether the new intervention has the potential to help more than harm. Researchers use Phase I clinical trials to find out what a safe human dosage is.

Study groups in Phase I trials are usually very small. Participants are closely monitored for side effects. Phase I trials are not testing the effectiveness of a treatment — they are primarily focused on how safe the treatment is for humans.

Phase II

Interventions move on to a Phase II trial when they are determined to be safe in Phase I. The goal of a Phase II clinical trial is to see if the drug is effective at treating the target disease in humans.

Like Phase I trials, Phase II trials do not use placebos. Instead, participants either all receive the same dose, or receive a range of doses that researchers determined safe in Phase I in order to determine the optimal effective dose. Because more people are involved in Phase II trials, researchers will likely see side effects that did not occur in Phase I.

Phase III

If a drug shows promise in a Phase II clinical trial, it moves on to Phase III. Phase III trials test whether a drug or treatment is better than the current standard of care. For example, a new type of chemo might effectively kill cancer cells in Phase II — but does that mean it works better than the drug that is already used to treat that type of cancer?

Placebos are used in Phase III trials, but patients are not given an inactive treatment if a treatment that already works for the disease exists. Phase III trials are often very large, taking place concurrently at medical facilities across the country or world.

Phase III clinical trials are often the last step before new drugs are submitted for FDA approval. After a Phase III trial, drugmakers will submit a new drug application (or NDA) to the FDA. The FDA can then accept the application, or return it with a request for further study.

Phase IV

Once a drug is approved by the FDA, it may enter a Phase IV trial. Phase IV clinical trials look at the effects of treatments over a longer period of time, often several years. Drugs in Phase IV trials have already been proven safe and effective — there is little risk to the patient in a Phase IV trial. Instead, the point of Phase IV trials is to help researchers know more about the long-term effectiveness and side effects of the treatment.

---

Note: Several quotes from this article come from The Patient Story’s interview with Dr. Raphael Fonseca, a practicing hematologist and myeloma specialist with over three decades of experience. To watch the interview click below.

---

What are the FDA’s Drug Approval Processes?

According to the FDA, “Over the past three decades, Congress has established five programs aimed at expediting patient access to important drugs that treat serious or life-threatening conditions.”

These five programs are:

• Fast Track designation – designation isgranted if there is an unmet medical need, and a drug’s data thus far shows promise.
• Breakthrough Therapy designation – designation can be given to drugs that show a significant improvement over therapies or drugs that are currently available.
• Regenerative Medicine Advanced Therapy designation (RMAT) – for getting regenerative medicine therapies (i.e., stem cell transplants) with the potential of meeting an unmet need for a serious condition on the market quicker.
• Priority Review – designation is opposed to the FDA’s standard review. Many of the new treatments the FDA approves fall under standard review. This is a process that usually takes about 10 months for review. With Priority Review, the goal is six months. This is for treatments that have shown proven, significant improvements for a serious condition.
• Accelerated Approval – a little different than the other four programs. It allows a drug to be approved based upon what’s called a “surrogate endpoint.” That’s medical jargon for “a blood test” or “a CT scan” that shows improvement that proves the drug is working.   For example, if you had a tumor and were on a drug in the Accelerated Approval program, and the tumor was shrinking every few weeks, that might prove the drug was working without having to wait to see how you feel or how long the treatment extends your life since those things can take years to find out. Source

If a drug or therapy has one of these designations, it could mean that the FDA likely sees that it has the potential to fill a need. It can be helpful to search these designations for new and upcoming treatments that could meet your specific needs.

Clinical Trial Resources

Clinical Trials Patient Stories

Learn about cancer patient’s experiences with enrolling and participating in clinical trials from real-life patients.

Breast Cancer

Cervical Cancer

Kidney Cancer

Leukemia

Lung Cancer

Melanoma

Multiple Myeloma

Non-Hodgkin’s Lymphoma

Ovarian Cancer

Sarcoma

Clinical Trials Experts

Discover medical professionals and experts thoughts and opinions on clinical trials.

---

Additional Resources for Clinical Trials

• https://clinicaltrials.gov – Here you can search your condition and see the status of clinical trials for it. Look for ones that say “recruiting.” Even if you don’t understand all the jargon, you can always present it to your doctor and see if they think it’s a viable option.
• https://www.drugs.com/newdrugs.html – If you’d rather not be part of a clinical trial and want something that the FDA has already granted full approval, you can search for your condition here. This is a constantly updated list of newly approved treatments. Simply search for your condition, and you can read the press release (or show it to your doctor) about the new drug.