Clinical Trials and What Statistics Really Mean

Clinical trials that concentrate on assessing a specific drug or treatment and measuring the outcome of its safety and effectiveness on patients are known as 'interventional' clinical trials.

"We need everyone to be partners with us if we're ever going to truly cure cancer or prevent people from having to die from cancer," says Dr. Beth Karlan, director of the Women's Cancer Center at Cedars-Sinai Medical Center.

How do interventional clinical trials for lung cancer treatments work?

Whether we're talking about small cell lung cancer or non-small cell lung cancer treatment or any kind of treatment trial for that matter, the process is pretty much the same. It starts with the principal investigator probably a doctor who is the working force behind this operation. The principal investigator prepares a 'protocol' that contains all the details of the trial, including the following:

• Aim and reason for the clinical trial
• Who should be included or excluded from the trial (also known as the eligibility criteria)
• Background information about the drug or new treatment that will be tested
• How the drugs will be assigned to the participants
• The number of participants allowed in the trial
• Additional medication or treatments that will be given, its dose, and intake method
• Tests that will be done to assess the outcome result for treatments given
• Duration of the trial
• When the trial should be stopped for safety or efficacy
• Many other details to ensure patients (or participants) are safe

Interventional clinical trials can take many forms when it comes to patient grouping and the type of treatments that will be administered. For instance, some trials divide participants into two groups giving one group the experimental treatment while giving the second group a 'placebo' of a standard of care treatment. This is mainly done to compare a new therapy with conventional treatments of cancer.

Generally, the group given the experimental drug will be called 'the experimental group or treatment group' while the other group given the placebo will be called the control group. This placebo will include a traditional treatment that is currently approved and would be prescribed for the patient under normal circumstances.

Similarly, according to the study design, some trials can employ randomization for their participants, but this is not always the case.

All groups are then followed up by the entire healthcare or research team through all the phases of the trial to collect information needed further on to get approval from the FDA or other relevant institutional bodies.

Patient safety in lung cancer clinical trials

The safety of patients in clinical trials is kept under federal laws and ethics boards to ensure the safety and well-being of all participants. Before participating in a clinical trial, you will be given an "informed consent" to sign, which contains all the information about the phases of the trial, what's expected of you, and the anticipated side effects that may happen.

You should use this time to review this document carefully and ask any questions so that you fully understand everything expected of you in this trial.

Also, the trial will be continually monitored by several competent authorities including, the Institutional Review Board (IRB), Data and Safety Monitoring Boards, trials sponsors, and the research team.

Lung cancer clinical trial phases

There are four different phases in an interventional cancer clinical trial, each having a specific goal to establish. These phases can be summarized through the following points:

• Phase I. The first phase of the trial it's initiated after a drug is approved and aims to find a safe dose for and determine the optimal method for drug administration. This phase involves a small number of volunteers (15-30 participants).
• Phase II. The second phase of the trial is initiated after a safe dose of the drug has been determined from the first phase, and it aims to test the effectiveness of the treatment. This phase involves around 100 volunteers or less.
• Phase III. In stages I and II, a drug has shown promising results. Phase III is initiated on a much larger scale and aims to compare a new treatment with traditional treatments. This phase involves from one hundred to several thousand volunteers.
• Phase IV. This phase starts after the approval of the drug and lasts for several years to make sure that there are no long-term effects or adverse events for the drug that hasn't been shown in the previous phase. This phase is also known as the post-marketing phase.

However, these phases are not written in stone. Some researchers can design protocols that merge phase I with II or phase II with III; this depends on the study design and research question originally put in place by the principal investigator.

Why are clinical trials important for lung cancer patients?

Taking part in a clinical trial may allow lung cancer patients to access the most recent and cutting-edge treatments years before they're available, and these treatments can include immunotherapy, targeted therapy, gene therapy, or even a new combination of currently used treatments like surgery, chemotherapy, or radiation therapy.

Although there's no guarantee that this newly offered treatment will produce great results in curing lung cancer, at the very least, it can ensure proper care and follow-up from the healthcare team running the trial.

There's also the knowledge that you'll be helping doctors make ground-breaking discoveries and produce much-needed treatments empowering other lung cancer patients like you. Additionally, many of the fees or added costs that are directly related to the trial will be covered by insurance or the trial, so you will no longer have to pay to get treated.

Measuring the outcome

There are many factors to consider when measuring the outcome of a clinical trial, and they mainly depend on the parameters set by the researchers at the beginning of the trial. The outcome of the trial that needs measuring is also known as the 'endpoint', and it's classified into 'primary endpoint' and 'secondary endpoint' or ' secondary endpoints'.

The primary endpoint is the outcome that researchers are mostly keen on finding out, and it can be several things like:

• The effectiveness of the treatment
• How long will the patient live after taking the treatment?
• Will their cancer come back? And if so, after how long?
• How does the experimental drug compare to currently approved drugs?
• Can this new treatment cause any fatalities?

As for the secondary endpoint, these include measurable outcomes of the trial that the researchers are interested in, but it's not the main focus or aim of the trial, for instance:

• How the drug interacts in the body
• Side effects
• The emotional state of patients

Measuring these outcomes is extremely important because they showcase the importance of the trial and how it can benefit or harm patients. So, here are some terminologies used by researchers when they assess the endpoints of the trial:

• Overall survival: This refers to how long people live after getting the treatment
• Progression-free survival: This refers to the time between controlling cancer by inhibiting its growth and signs it started growing again
• Disease-free survival: This refers to the time between observable progress in curing cancer and signs it started growing again

Getting familiar with these terminologies can help you make a choice to either join a clinical trial or not. However, it's crucial to understand the differences in these outcomes and how they can affect you.

As best explained by Dr. Scott Strome of the University of Maryland School of Medicine, "There are drugs available now that can improve the progression-free survival and overall survival of late-stage lung cancer patients who have not responded well to primary therapies. It's important to understand the distinction between these two types of survival statistics. Improvement in overall survival means the study shows that you actually live longer because of the treatment. Progression-free survival means that you don't live longer, but you have more time when your cancer does not get any worse."

This means that even though there are a lot of advantages to taking part in a clinical trial, it can also have unpredictable or unwanted side effects on some of its participants.

Possible risks of participating in a lung cancer clinical trial

Since it's always better to get the big picture before making any decisions, here's a list of some of the possible risks of participating in a clinical trial:

• The experimental treatment may be worse or work just the same as the conventional treatment
• The unpredictability of the side effects that can happen
• It's impossible to know whether a patient will benefit from the effects of the new treatment or not at all
• This happens rarely, but with this level of unpredictable side effects, it can cause fatalities
• Some groups may get the placebo (standard treatment) and later find out that the experimental drug had great effects, but they weren't fortunate enough to get it
• More monitoring, visits, or travel might be warranted
• The insurance may not cover all the costs

It's easy to focus on the benefits of treatment, especially for a complex disease like lung cancer, but it's imminent to consider the risks as well as the benefits and to keep in mind that each patient's case is different and what might work for someone may not have the same effect on someone else. As Dr. Strome outs it, "It's very exciting for doctors to be able to offer these new therapies, but it's important to remember that they don't work for every patient. There are plenty of patients who participate in clinical trials, are exposed to the toxicities of a drug and do not benefit at all."

Similarly, it's important to think about the desired outcome of the treatment, and whether it's worth all the hassle or not. This is a question that needs to be asked of each patient personally. Dr. Strome also shares his thoughts on this one saying, "Some experts question the importance of a drug that improves progression-free survival by a few months. But others say that if a drug can give the patient more months when the cancer does not get worse, then that is an important improvement."

Thus, with these varying opinions, it's pivotal to look at each patient's case individually and consider their needs and how best to accommodate them.

There have been an increasing number of lung cancer clinical trials to improve the following:

• Small cell lung cancer and non-small cell lung care treatment
• Quality of life of lung cancer patients
• Increase survival time for lung cancer patients
• Lung cancer side effects

Lung Cancer Side effects

• Persistent coughing
• Coughing up blood
• Chest infections
• Fatigue
• Breathlessness
• Painful breathing
• Wheezing
• Swallowing difficulty
• Unexplained weight loss
• Face and neck swelling

So far, there have been a lot of advancements in immunotherapy, and targeted therapy for lung cancer patients, and some of these drugs have been successfully approved by the FDA after showing promising and ground-breaking results.

Deciding to take part in a clinical trial

After reviewing all the data, the decision to join a lung cancer clinical trial remains up to you. If you decide this is something you want, we suggest you start by consulting your doctor first and searching the official database for US clinical trials for the type of your lung cancer, whether it's small cell lung cancer, non-small cell lung cancer, or carcinoids and you will find all the data you need. You can also find more information through the  National Cancer Institute and the Center for Information & Study on Clinical Research Participation.  

SurvivorNet can also help you connect to clinical trials for treatment through a new artificial intelligence tool, providing an interactive and user-friendly option compared to scrolling through lists alone in the hope of finding something.

The Bottom Line

Today, joining a clinical trial for lung cancer treatment can mean many things. It can mean a cure, a longer survival time, manageable symptoms, and improved quality of life, but at the same time, it can also mean side effects, toxicity, uncertainty, or even death. But now you know the pros and cons of taking part in a clinical trial, so you're more eligible to take that decision depending on your health condition and what you want.

Nevertheless, as time goes by, scientific breakthroughs are discovered, and there is plenty of evidence in the scientific literature that proves that those who invest in the future of cancer treatment play a great role in empowering themselves while saving other lives in the process.

Learn more about SurvivorNet's rigorous medical review process.

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