Want to participate in a clinical trial? You should know these things first!

Mr. Zhang, 61 years old, was diagnosed with lung cancer (right lung centrally differentiated squamous carcinoma) in October 2013 and had two lobes of his right lung surgically removed. However, less than a year after surgery, he developed hilar and mediastinal lymph node metastases, and the tumor also invaded large blood vessels. The doctor determined that it was stage IV (advanced) and recommended chemotherapy (paclitaxel + carboplatin), but the treatment did not go well and the tumor continued to grow, with side effects such as severe bone marrow suppression and infection.

Mr. Zhang’s supervising physician approached him and described, “In your current situation, you may be considered for a clinical trial of one of our new drugs.”

“Experimenting on me as a human?”

A clinical trial of a new drug, is that taking a person for a test? Zhang was a little apprehensive.

“All the drugs we use now, before they are widely used in patients, go through ‘preclinical studies’ and ‘clinical studies.’ Preclinical studies will verify the drug’s After confirming the safety and toxicity of the drug, it can be used in clinical trials only after strict approval by the relevant state departments, that is, it can be used in human body to study its metabolic process, effects and safety in our body. If you compare new drugs to ‘recruits’, clinical trials are the ‘real-world test’, and you can go to war only after passing the test.”

“There are four phases of clinical trials. The first phase is to see how the drug is absorbed and metabolized in the body, and what adverse reactions people have after using the drug, i.e. safety, and to explore the Maximal Tolerable Dose (MTD), Dose-Limiting Toxicity (DLT), which provides the next phase II and III clinical trial design and dosing protocols. The Phase II trial is the initial look at the efficacy and safety of the drug for patients, and I recommend that you participate in this trial, which is in Phase II; if the Phase II trial gets good results, we will continue with the Phase III trial to expand the patient scale for validation; if the critical Phase III trial achieves satisfactory results, we will push the national drug regulatory authorities to approve the drug for marketing and use in a wide range of patients.”

“Usually the four phases are sequential and cannot be reversed. We are very strict and cautious in our clinical trials. We have to go through the relevant national authorities to review and validate the trial before we start, and we can recruit patients only after we have received approval, and we do close follow-up, monitoring and documentation throughout the trial to protect the safety and rights of patients.”

“So what’s in it for me to participate in this trial?”

“You are relatively sick now, and our conventional treatments such as surgery, chemotherapy, and radiation therapy are not working well and are producing more serious side effects. There have been many new advances in lung cancer treatment in recent years, and the PD-1 inhibitor class of drugs that I recommend you try is one of them. It enhances the anti-tumor activity of our immune system through a special mechanism. There have been several studies abroad that have shown it to be more effective than chemotherapy for patients who meet the indications.”

“In other words, the ones doing clinical trials are often the newest and most advanced treatments available, and may be more effective and have fewer side effects than existing treatments.

In addition, PD-1 inhibitors are expensive, and when you join a clinical trial, the treatment drugs are free to use and some tests are free, greatly reducing the financial burden. However, there is some uncertainty about drug efficacy, predictors of efficacy, and toxicities.”

“Finally, once you are enrolled in a clinical trial, we will arrange for your ‘exclusive’ doctor to see you, follow you over time, and closely observe your changes in your condition, in strict accordance with the medical protocol and trial protocol, and keep detailed records. You are receiving the best medical care possible.”

“And how exactly does this trial work?”

“Clinical trials are not open to anyone who wants to participate, and there are strict rules about which patients can and cannot be enrolled in each clinical trial, which we call ‘inclusion’ and ‘exclusion’ criteria. If we screen and think you are eligible, you will be enrolled. You will then undergo a ‘randomization’ process. As the saying goes, ‘there is no discrimination without comparison’. To verify that a new drug is good, you need a reference, and the reference is often the best, or standard, treatment of the day. ‘Randomization’ is the process of randomizing you to the ‘new drug group’ or the ‘control group.'”

“I’m so sick, you can put me in the new drug group and pick a few people who are less sick and put them on the old drug ……”

“This grouping is not up to me. ‘Randomization’ means that a set of scientific methods is used to ensure that every patient who is enrolled in the group has an equal chance of being in the ‘new drug group’ or ‘control group’. In fact, no one knows which drug you are using, including the doctor in charge, the nurse and yourself. Only in this way can we observe and evaluate the drug effects accurately without interference from subjective factors. It’s like tasting food blindfolded, and which plate you taste will not be revealed until the moment you remove the blindfold. This is why we also call it “blinding” (note: there are many unblinded studies in clinical trials). But don’t worry, even if you’re in the control group, you’re still on the best treatment available in the clinic, and there’s no delay in your condition.”

“I get it, and I’d love to be included!”

“Don’t worry, let me also talk to you about what you need to do with us, and the risks you may take.”

“First of all, this is a phase II trial, and although the phase I trial has initially proven safety and similar drugs that are available overseas have been shown to be safe and effective, any drug can have side effects, and this varies from person to person. Therefore, it cannot be ruled out that you may experience mild or severe adverse reactions during the use of the drug. New drugs are applied for a short period of time and some adverse reactions we lack understanding and expectation, unlike old drugs that have been used in the clinic for many years. I cannot guarantee that the new drug will be safe and effective for you, but, from the data we already have from domestic and international studies, overall we are confident in the safety and efficacy of this type of drug. At the same time, your supervising physician will closely monitor your various reactions to the medication and make thorough preparations to be alert and proactive in the event of an adverse reaction. This also requires your understanding and cooperation.”

“Second, you need to cooperate with us for follow-up reviews. Your home is out of town, but according to our treatment schedule, you need to come to our hospital on the first day of each course of treatment to receive IV fluids and then go home and rest for 20 days; this continues until the end of treatment. In order to ensure the safety and efficacy of the treatment, we need you to follow the doctor’s instructions strictly and make sure you come to the hospital for treatment and follow-up visits. At the same time, according to the trial protocol, you need to do some tests at each follow-up visit for the purpose of better observation of your condition; these tests are free of charge and require you to ‘listen’ and cooperate with your doctor.”

“One final caveat is that there is a risk of not achieving the desired outcome. Lung cancer patients have very different conditions and physical profiles, and a drug that has performed brightly in other studies and patients can’t be predicted to work for sure when used on you. In fact, you will face this problem with any treatment you receive. For example, the paclitaxel + carboplatin chemotherapy that you have used has worked well for some patients, but not for you, and you have had serious adverse effects.”

“I can understand all that, and I’ve also heard that getting into a clinical trial is ‘easy to get on board and hard to get off’, is that really true?”

“Once you are enrolled, we certainly want you to do your best to cooperate with treatment, testing, and follow-up to complete the trial, while keeping your safety and rights in mind. But this does not mean that you must adhere unconditionally. In fact, we respect the safety and wishes of our patients more than we value the results of the research.”

“Every patient enrolled in a clinical trial is required to sign an informed consent form, which states that, in accordance with national law, clinical trials must be approved by national regulatory authorities before they can be conducted, and that the entire process is overseen by a special ethics committee. You have the right to be fully informed when deciding whether or not to join, and any questions you have related to the trial will be answered truthfully. The decision about whether to participate is entirely yours, and even if you do not participate, it does not affect our ability to provide you with other treatments to the best of our ability. Any risks that we anticipate will be communicated to you in advance. You have the right to withdraw at any time during the trial, without reason. We will not ‘think’ of you for this reason and it will not affect your subsequent treatment with us. You have the right to be informed and to decide again whether to continue in the trial if any information about the trial process or the drug is updated. If you develop a physical injury related to the trial drug, we will promptly address it and provide appropriate compensation in accordance with the trial protocol. We will also provide a transportation allowance if you come from your home country for follow-up visits.”

“In short, we will ensure your safety and rights at every step of the way, you have the right to be fully informed and decide, and there is no ‘easy on board, hard off’ situation.”

When it was all said and done, Zhang felt enlightened and entered the clinical trial with great anticipation. He was randomized to the new drug group, and by the end of the first course of treatment, the tumor had begun to shrink and various symptoms had been significantly reduced.

For him, it was a happy ending like a rebirth. But the reality is that there are many patients who have misconceptions and concerns about joining clinical trials. Click below to see what we’ve sorted out and clarified.

Disclaimer:

Tumor disease and treatment options are extremely complex, and treatment should be fully individualized, and this case does not represent a treatment decision for a “similar patient. Please seek professional advice from your supervising physician regarding your specific treatment plan.