Does participating in a clinical trial mean being a guinea pig? Uncovering the pros and cons of drug clinical trials

Author: Zheng Xin, Wang Weicong, Bai Hua

As a researcher engaged in clinical pharmacology research in a hospital, my relatives and friends are very envious of me...

However, when they heard that my main job was to recruit subjects for drug clinical trials, they would most likely show a hint of fear...

The angel in white with a halo a second ago turned into a little devil with an evil smile when the term "drug clinical trial" was mentioned - she was afraid that she would be caught as a "guinea pig".

Why do people have so many negative impressions about drug clinical trials? How do people view drug clinical trials?

Today, let us work together to determine the right and wrong of this matter.

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Question 1

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What are drug clinical trials?

A drug clinical trial refers to any systematic study of a drug conducted on humans (patients or healthy volunteers) to confirm or reveal the effects, adverse reactions, and/or absorption, distribution, metabolism, and excretion characteristics of the test drug, with the goal of determining the efficacy and safety of the test drug. It may sound a bit obscure, but in layman's terms, it can be summed up in two sentences:

First sentence: Can this medicine be used by patients?

Drug clinical trials are carefully designed medical studies that aim to confirm whether a new drug is effective, safe, has side effects, is it worth using to treat patients, and other unsolved questions. The answers to these questions are also the necessary basis for drug regulatory authorities to determine whether new drugs can be marketed. The development of many new drugs may take more than 10 years, and every process during this period is crucial to ensuring the safety and effectiveness of the drug.

Another sentence is: How should this medicine be used?

Whenever your doctor prescribes you a medicine, or you feel unwell and want to go to the pharmacy to buy medicine, you will definitely think in your mind: Is this medicine effective? Is it suitable for my condition? Should I take it in the morning or at night? Before or after meals? How many times a day? How many pills should I take each time? How many days should I take it in total? What are the side effects? I am pregnant, can I still take this medicine? I have poor kidney function, can I take this medicine?

…

Almost all of your concerns can be answered in the drug instructions, and drug clinical trials are the best way to get these answers.

Therefore, it can be said that without clinical trials, there would be almost no new drugs because no one knows how to use the drug and what will happen after use.

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Question 2

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How many stages are there in clinical trials of new drugs?

Clinical trials of new drugs generally go through three stages, each with specific goals.

If it successfully passes these 3 phases, the drug will be submitted to the regulatory agency (FDA) for approval. After the drug is approved, additional studies may be conducted in the 4th phase.

The four stages are:

Phase I trials generally select a small number of healthy people to test new drugs to evaluate their safety, determine the safe dosage range, and determine what side effects they have;

Phase II trials use the study drug on dozens or even hundreds of patients to evaluate its effectiveness and further assess its safety;

Phase III trials test the study drug in a population of several hundred to thousands of patients to confirm its effectiveness, monitor side effects, and compare it with commonly used treatments;

Phase IV trials, also called postmarketing studies, look at long-term real-world actual experience and provide additional information about a drug's risks, benefits, and optimal use.

As clinical trials progress through each phase, our understanding of a drug and its safety and effectiveness continues to grow.

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Question 3

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Who takes part in drug clinical trials?

"Drug tester"?

"Guinea pig"?

This is a misunderstanding many people have about participating in drug clinical trials.

Participants in drug clinical trials are not only patients, but also healthy people (Phase I clinical trials), whom we call "subjects". They are important participants in clinical trials. In theory, anyone can become a subject in a clinical trial, but they must meet certain physical indicators of the trial, have a correct understanding and knowledge of clinical trials, and have a certain sense of social responsibility.

Participation in clinical trials must be completely voluntary, and you can withdraw at any time during the trial. Please note that you can withdraw at any time without any reason.

Before participating in a trial, you must fully understand the purpose, drugs, process, especially the benefits and risks of the trial. Only after careful consideration and informed consent can you officially enter the trial.

In addition, each clinical trial has strict inclusion and exclusion criteria. Only patients (healthy volunteers) who meet these criteria may be selected for clinical trials and become subjects. For example, if it is known that a certain drug is only effective for patients with a positive gene mutation, then the inclusion criteria in the clinical trial screening stage are patients with a positive gene mutation.

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Question 4

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Are there risks in participating in a drug clinical trial?

Any medical activity has risks, even daily life has risks: eating (overeating can cause pancreatitis), walking (accidentally spraining your ankle or falling), sleeping (many people have experienced waking up early in the morning with neck pain so severe that they can’t move), and of course clinical trials have risks.

Some risks are related to the mechanism of action of the drug. For example, anticoagulants inevitably carry the risk of bleeding.

Some risks are related to drug dosage. For example, taking only one tablet in a small dose may not be a problem, but taking large doses continuously will cause side effects.

Do you remember the scene in the TV series Nirvana in Fire where the Empress falsely accused Concubine Jing of feeding the Emperor poisonous kudzu grass? After investigation, the Emperor said, "The doctors at the Imperial Hospital have calculated that if she wants to use kudzu grass to poison me, she would have to boil a whole room of grass every day for me to drink."

Did you silently say "The Emperor is wise" after reading this? This tells us that "any discussion of toxicity without considering the dosage is just a conspiracy!"

Some risks are related to the patient's physical condition (also called individual differences). Different people will have different reactions to the same medicine. As the saying goes, "one man's meat may be another man's poison." For example, most people will be fine with penicillin, but some individuals will suffer from anaphylactic shock; some people will develop coughing after taking a certain antihypertensive drug, while others will experience edema.

The size of the risk cannot be generalized. Some adverse reactions have a high incidence rate but are mild and can be tolerated by most people. Some are very rare but can be fatal. Even if the severity is the same, they should be treated differently depending on the purpose of treatment. For example, severe leukopenia after medication is common and acceptable for chemotherapy drugs used to treat cancer, but if it is used for a blood pressure drug, I'm afraid no one will use this drug.

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Question 5

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Are there benefits to participating in drug clinical trials?

Healthy subjects who participate in Phase I drug clinical trials will not benefit from them. Their participation is entirely voluntary to contribute to medical development and social needs, so they should be paid the highest respect (applause should be given here).

Most patients who participate in drug clinical trials are likely to benefit from the use of new drugs. For diseases that currently have no treatment, this may even be the only hope for patients. Of course, the efficacy and safety of drugs in the research stage are still uncertain, so the benefits are also uncertain.

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Question 6

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Who will protect my interests when participating in drug clinical trials?

Each drug clinical trial has different risks and benefits. Since the participants are different, the risks and benefits they experience are also different. However, the design of each link of the drug clinical trial will maximize the safety of the subjects.

First of all, drug clinical trials are generally conducted in research hospitals with excellent medical conditions, and the participating doctors and nurses are all experienced professionals. When designing the research plan, they will control the risks to the minimum range based on authoritative professional knowledge, and do their best to ensure the safety of the subjects throughout the trial. Once a serious adverse event occurs, it will be judged and handled immediately, and reported to the regulatory authorities and ethics committees in accordance with very strict reporting procedures.

The "Ethics Committee" is an institution dedicated to reviewing and supervising clinical trials. They are composed of many medical/pharmaceutical experts, lawyers, ethicists, and representatives from the community and patients who have no vested interest in the research. They are responsible for approving whether clinical trials can be carried out and supervising whether the rights and interests of the subjects are protected during the clinical trials. They can be said to be the "mother-in-law" of the subjects.

No matter how scientifically sound a clinical trial is, if the safety and rights of the subjects are not guaranteed, the ethics committee will not approve it, and subsequent research and development cannot be carried out. In addition, if anything that damages the rights of the subjects occurs during the clinical trial, the ethics committee will stop it at any time.

Secondly, every pharmaceutical company that initiates clinical trials must be fully responsible for any health damage to the subjects that occurs during the trial. The research doctors will actively and appropriately treat these damages, and the treatment costs will be borne by the pharmaceutical company or the insurance purchased. When financial compensation is required in accordance with regulations, the pharmaceutical company must also bear it.

Finally, pharmaceutical companies will not conduct human trials easily. They will only start clinical trials after sufficient verification in the laboratory and on animals, and when they are fully confident of the efficacy and safety. The reason is simple. In addition to the strict access requirements for human trials, clinical trials are too expensive. The investment in each clinical trial is astronomical. On average, the research and development of a new drug may cost more than $8 billion and take decades.

Therefore, drug development manufacturers are extremely cautious in including subjects, as they absolutely hope that the new drug is safe and effective for every subject.

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Question 7

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Why does clinical trials require everyone’s participation?

To advance medicine, clinical trials require participants of various disease backgrounds, ages, races and ethnicities to help verify which drugs are effective. It takes a lot of work and resources to get a new drug from laboratory discovery to development to the first human trial and then to approval for marketing.

We can use the greatest scientists, the most advanced equipment, and billions of dollars to conduct research, but if no one participates in the clinical trials, it would be like fighting a war with only planes and tanks but no soldiers. The trials will not be able to proceed and medicine will stagnate.

In conclusion

Finally, we would like to say that during the entire process of drug clinical trials, research doctors, pharmaceutical companies, and regulatory authorities at all levels have extremely strict management and supervision of clinical trials in order to protect the interests of the subjects from infringement and to ensure that the valuable data obtained from clinical trials are authentic and reliable. If clinical trial data is falsified, severe criminal penalties may be imposed depending on the severity of the circumstances. Therefore, drug clinical trials are a sunny cause that is beneficial to the present and future generations under the protection of science, ethics, and regulations!

Without clinical trials, there would be no new drugs or new therapies that would benefit patients. The subjects who participate in clinical trials have contributed to the health of all mankind and are worthy of respect and protection. Clinical trials are the only way to develop new drugs. In order to promote the progress of medicine, the participation and help of the whole society are needed.

In this light, do you still think that the subjects participating in drug clinical trials are treated as "guinea pigs"?

Let’s stop here for now on clinical trials of new drugs. If you need a deeper understanding, you can leave a message in the interactive area below and we will do our best to answer your questions.

Acknowledgements

Health Science Action Column in this issue of Peking Union Medical College Journal

Main creators: Zheng Xin, Wang Weicong, Bai Hua

About the Author

Source: Peking Union Medical College Journal

丨Editor: Liu Yang Zhao Na

丨Reviewed by: Li Na, Li Yule, Dong Zhe

丨Producer: Wu Wenming

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