The road to the rise of great doctors

Chapter 43 A brief discussion of clinical trials
Although Liu Dong started his internship and was exposed to clinical work for less than one month, he has been in the ward the longest among the five-person team, and he browses the conditions of general patients every day.

I have also seen a few cases of "advanced cancer, which has progressed after standard treatment, and there is no other way to follow".

There is a personal experience that he still remembers vividly.

It was a woman diagnosed with a kidney tumor.She was just over 30 years old and had just been married for half a year. She came to the hospital for treatment because of low back pain. An ultrasound was performed and it was found that the cause of the low back pain was a left kidney tumor.Although there was no metastasis, the tumor broke through the capsule of the kidney and invaded the fat sac outside the kidney.According to staging standards, this is considered a locally advanced renal tumor.

At that time, a major expert in urology performed an operation. Because the tumor was too big to save the kidney, the entire kidney and the outer fat sac were removed.The pathology result was "renal sarcoma".

"Oh, sarcoma! This is a more malignant tumor than clear cell renal cell carcinoma. Surgery can delay the progression of the disease. You have to receive adjuvant treatment after recovery about one month after surgery. But generally speaking, those who are alive are time is limited."

Liu Dong went to consult this great expert specifically and heard these words at that time.

After that, Liu Dong carefully downloaded and consulted domestic and foreign guidelines, and also searched the literature. Basically, the answers were similar, "Sarcomas are relatively rare, highly malignant, metastasize early, have short survival period and poor prognosis."

That was the first time he felt the helplessness and powerlessness of a doctor.

When Liu Dong checked the information on clinical trials, he saw the question "Those who have no standard treatment plan, or have used many treatment plans but still have no effect, the disease has progressed. What should be done?" The first person that comes to mind is that girl. Her happy life just started, but it ended abruptly. What kind of grief will it bring to her family!
How can medicine improve this situation?
Liu Dong continued to read and found: "For this group of people who have failed standard treatment, if their physical condition and organ function are good, and they can withstand the side effects of anti-tumor drug treatment, then clinical trials may be a good choice." .”

why?

This aroused Liu Dong's thirst for knowledge. He continued to explore and discovered:

First, participating in clinical trials is a last resort.

If there is a definite treatment plan and the efficacy is good, from the perspective of patient safety and ethics, this confirmed treatment method must be recommended first. This is also what doctors often mention as "following the guidelines."

However, the existing treatment options have been fully used and proven to be ineffective, or the patients cannot tolerate the toxic side effects and cannot use this option.At this time, if you still want to seek a chance for survival and continue treatment, you can only cross the river by feeling the stones.

Clinical trials represent new treatments.Those mature regimens - regimens recommended by guidelines, have also gone through phases 1, 2, and 3 of clinical trials.

From the perspective of a pharmaceutical company as a developer, scientists and clinical researchers from pharmaceutical companies evaluate that the drug is worthy of development, which means it has the potential to surpass the current existing treatments, or the efficacy is not weaker than the existing efficacy but the safety is not Improved potential.

Because only after surpassing the currently recognized "best" treatment, the national approval agency will allow the entire drug to be put on the market and sold commercially after seeing the data.If it cannot be surpassed, it will be difficult to be approved for marketing. The more money is spent and the longer the clinical trial phase is, the greater the losses will be for pharmaceutical companies.Therefore, the decision-makers of pharmaceutical companies are among the people who are most concerned about the true efficacy and safety data of this drug!

From a doctor's point of view, he can't bear to tell his patients that there is currently "no cure". Just like the saying "sometimes it's a cure; it's often a help; it's always a comfort." Who doesn't want to provide "a cure or help?" , this is the doctor’s responsibility!

Inform the patient or family of the current treatment options, efficacy, and safety, and also tell the patient or family what exploratory treatment options and data are currently available. The decision-making process is based on the decision of the patient or family, with the participation of doctors. To make decisions, this is the embodiment of "the benevolence of doctors".

Introducing products that are in the research and development stage, as well as products that have not been marketed in China but are marketed abroad, is not illegal, but is the responsibility of doctors.Just when buying, you need to follow the laws of the country and the place where you buy.Second, many cancer patients, after previous treatments such as surgery, radiotherapy, chemotherapy, etc., may have “sold off the pot” at home, and are in a situation where “everything is at the end of their rope – they have no money left to live”.

Even the patient may not want to be treated because he does not want to burden his family, but the family still wants to treat him and is reluctant to give up on him.In this case, clinical trials may be an option if the disease and test results meet the requirements of the study protocol.

One of the great benefits of clinical trials is that the cost of treatment and examination is paid by the pharmaceutical company. At least the costs related to the clinical trial as well as the round-trip transportation and accommodation expenses are borne by them.

This fee may also be a "life-saving straw" for patients.For the patient’s family members, it can also reduce some financial burden without giving up their family members.

Third, clinical trials can be divided into phase 1 trials, phase 2 trials, and phase 3 trials from early to late stages.

Phase 1-2 trials are higher risk, and Phase 3 clinical trials are lower risk. In phase 3 trials, efficacy and safety data have been collected for a part of the population. Therefore, the efficacy can be estimated and is generally not inferior to the best standard treatment plan. Moreover, there is also experience in safety. At this time, the cost-effectiveness is the highest.When receiving treatment, many will use random grouping, which means that the treatment drugs after enrollment may be new drugs under development, or they may be the best current standard treatment drugs, so no matter which group they are assigned to, it does not matter to the patient. bad thing.

As written in many materials Liu Dong saw, this is a process of weighing and making decisions.Generally speaking, only when the benefits to patients outweigh the risks will the ethics committee's approval be obtained and clinical research officially launched.

In addition, in order to protect the rights and interests of patients, clinical trials cannot induce patients to participate.Patients must be fully informed of the true situation and allowed to make their own decisions. This is true both at home and abroad.

However, clinical trials are not perfect, and each phase of trial has its own characteristics.

Liu Dong continued to look, and the next thing he saw were shortcomings.

First, new drug research and development is risky.The earlier the clinical trial, the higher the risk.

Why is the risk high?

Because before the start of the Phase 1 trial, the safety data were based on animal experiments or pathogenesis considerations, not clinical data.The safety of real experiments on humans is unpredictable, and animal experiments cannot replace human applications!This has been a painful lesson in the United States and Europe.

However, the risk is high mainly because it is uncontrollable and unpredictable, not because you will die or be dangerous if you use it.

In order to protect the rights and interests of patients, there is a hedging prevention mechanism in the system.

When looking for suitable clinical trials, you must keep your eyes open and find formal clinical trial projects that have been approved by the hospital ethics committee or registered with the country.Those who need to pay a lot of money may want to be cautious.

There are scammers in every industry, don’t be fooled…

(End of this chapter)