In the United States and most of the world, medical professionals base treatment decisions upon evidenced based medicine (EBM).
EBM is the conscientious, explicit, judicious and reasonable use of modern, best evidence in making decisions about the care of individual patients. EBM integrates clinical experience and patient values with the best available research information.
Clinical trials are the cornerstone of EBM and without these trials new drug development, treatment strategies and procedural techniques would remain stagnant.
These trials are essential for patients with malignancies as new drug development and treatment paradigms are created based on trial results.
How Do Clinical Trials Work?
To begin with, before a clinical trial can be done, highly qualified scientific panels determine whether it is ethical to ask patients to volunteer.
Clinical Trials Proceed in Four Phases
PHASE I: Once a drug is approved for human studies, it is tested in a small trial to determine the optimal safe dose. Phase I studies often involve patients with different kinds of cancer, or more recently, a single genetic change.
PHASE II: If a drug can be given safely to people in Phase I, it is tested in a Phase II trial. These are larger studies, usually for one or more specific types and stages of cancer. The goals of Phase II studies are to both determine the optimal dosing and to provide an early assessment of whether the drug works.
PHASE III: These trials take place after the drug has shown good results in earlier studies. They are large studies that may involve hundreds or even thousands of patients in multiple locations across the United States and/or abroad. Patients in Phase III trials have specific types and stages of cancer. Many Phase III trials are randomized meaning patients are randomly assigned to receive either the new treatment or the established standard of care. These trials are designed to provide definitive evidence to support FDA approval of the drug or agent for use in the public.
PHASE IV: These trials take place after a drug is approved and are often called post-marketing trials. The goal is to make sure that no safety or other concerns arise after a drug is approved. It is important to follow patients for a number of years to determine if there are any long-term side effects or other issues that may affect the way the treatment is used.
Outcomes in clinical trials are based on predefined quality measures – defined most often as primary and secondary endpoints.
The studies are designed to answer a specific question or questions and these predefined endpoints are the only questions that can truly be answered when reviewing any study.
The metrics are typically based on:
Survival metrics:
a. progression free survival
b. overall survival
c. time to metastasis
d. time to radiographic progression and others
Safety metrics:
a. adverse events
b. drug interactions
c. dosing
d. and others
Safety of a new drug or treatment is also essential in deciding if, how, and when to use a new treatment. During any clinical trial, researchers track all of the side effects, or adverse events, that happen to the people in the study. Participants are often asked to describe their experience, which is known as patient reported outcomes.
Quality of life metrics:
a. mood
b. activity
c. pain scales
d. and others
These metrics do not represent all the types of data that is collected and analyzed, yet they are among the most common.
After completion of some phase II and most phase III/IV clinical trials, the data is analyzed using various statistical algorithms to determine if the primary or secondary endpoints are met and that the outcomes are not by chance alone.
When Should You Think About a Clinical Trial?
A cancer clinical trial may be a treatment option for anyone who has received a cancer diagnosis. Specific clinical trials may be particularly important if:
- A patient is diagnosed with cancer that has spread or the cancer recurs or comes back after primary treatment
- Someone is suffering from a rare or difficult to treat cancer
- Current treatment involves significant side effects and the trial is designed to reduce the impact of these on the patient’s life
- Current treatment that worked in the past has stopped working
- A patient’s cancer requires that he or she remain on treatment for a long period of time, or for the remainder of his or her lifetime
- There is interest in getting access to the newest cancer treatments
- Someone is interested in helping other cancer patients by advancing cancer treatment
- The best way to learn more about your specific options is to talk to your doctor and other members on your health care team.
For more information about clinical trials available at New Jersey Cancer Care, PA, click here.
