When children are sick, the first thing parents do is to seek medical treatment and take medicine, but many parents find that there are very few drugs produced specifically for children, and most of them are reduced on the basis of adult drugs, which directly leads to the frequent occurrence of adverse drug reactions in children.
To improve this situation, clinical research on pediatric drugs is an effective way to do so. But the question is, they are all their own babies, who wants to let their children participate in the clinical trial research of drugs? Does this treat the child like a guinea pig? Today, we have the honor to invite Professor Wang Wei, Deputy Secretary of the Party Committee of Shanghai Children's Medical Center affiliated to Shanghai Jiao Tong University School of Medicine, to answer questions about the clinical research of children's drugs for us.
【Clinical research, the only way for new drugs to go to market】
Zhang Yixin:
We often hear the word clinical research, but in fact, our concept of this word is very vague, so I would like to ask you to explain to us what clinical research is and what to do.
Wang wei:
Clinical research is the study of disease diagnosis and prevention as the main content. The definition of clinical research can be divided into two types: clinical research in the narrow sense is a systematic trial that only takes the human body as the object of study, including healthy people and patients; generalized clinical research is as long as the sample data applied to patients, including blood and other disease-related studies, can be counted as clinical research. These studies are sometimes not felt by patients, and we are talking about clinical studies that focus on the narrow sense.
For example, a new drug, or a new clinical device, before or after the market, needs to obtain data on the patient's treatment process, as well as its efficacy or side effects. We need to obtain the patient's treatment process and results, determine the efficacy or side effects of the drug, and sometimes there will be some unexpected additional effects, which need to be confirmed or confirmed by clinical research.
In fact, from this point of view, full and complete clinical research is still very necessary and important for the development and use of our new drugs.
Yes, without the support of clinical research, there is a great risk in the rush to market new drugs. There is a very painful lesson in human history, and it is also one of the largest drug injury events in history, the "reaction stop event", which is a typical adverse consequence of insufficient clinical trials.
Around the 1960s, at least 15 countries were circulating the drug, which can alleviate women's pregnancy reactions after eating, and many people have significantly improved their symptoms of nausea and vomiting after taking the drug, so it has become an ideal choice for pregnant women. As a result, the drug was also produced and sold in large quantities, with more than 1 million people in the Federal Republic of Germany alone having taken the reaction stop, but with it, many babies born developed short-limb deformities, which resembled seals, known as "seal deformities".
In 1961, this symptom was finally confirmed to be caused by pregnant women taking a reaction-stop drug, which was banned, but more than 12,000 babies were affected. Such a serious side effect is actually an irreparable consequence of the drug not having undergone adequate clinical trials.
【More rigorous clinical drug research in children】
This shows that clinical research is really important, but there is a question, what is the difference between pediatric clinical research and adult clinical research? What are the difficulties in clinical research in pediatrics?
I think there are a lot of differences between pediatric clinical research and adult clinical research, and the main thing is that there are two aspects. First of all, children are vulnerable groups, not all clinical trials can be carried out in children, such as the phase I clinical trial of drugs, mainly to determine the toxic side effects of drugs, including the pharmacokinetics of new drugs, etc., only used in healthy people, is not allowed to be carried out in children. Only drugs that directly target children with the disease can directly conduct Phase I and Phase II clinical trials for children with that disease, which means that clinical drug research for children will be more cautious.
Another difference is that children are not fully capable people, so the recognition of clinical research is based on the consent of parents or guardians, and children over 8 years old also need their own consent, which means that the informed consent of children's clinical trials is more rigorous. In particular, many parents do not understand too much, think that clinical trials are to treat me as a guinea pig, do experiments if not drugs can have an immediate effect, then I do not agree to do such a clinical trial, so it is generally more difficult to obtain informed consent for children's clinical research. For these two reasons, the number of clinical studies in children is small and more difficult.
As you just said, some parents think that their children's clinical trials are equivalent to acting as guinea pigs, so we also want to know what the real situation is.
First of all, we must have a very clear point, the establishment and development of clinical research is very rigorous, not casually implemented, clinical research projects, need peer experts and ethics committee approval, the first principle of clinical research is to ensure the rights and interests of subjects, and to minimize the additional possible such a harm.
If a clinical study of a new drug is conducted, the company producing the new drug will be required to purchase the appropriate insurance to protect against possible unexpected situations. A large proportion of clinical research in children is aimed at current incurable diseases, or those with poor treatment effects. If they do not participate in clinical studies, children may not have the right medications to treat them.
So what are the advantages of the child participating in the clinical trial in treatment? Many parents have questions, clinical trials have a trial group, a placebo group, then in case we are selected for the placebo group, is not it a sparring partner?
In fact, there are considerable advantages to participating in clinical studies, whether it is a placebo group, a blank group, or a trial group using mental therapies or drugs. Pediatric clinical such a number of studies, many for the current incurable disease, to participate in this study may use the latest treatment programs or treatment methods, improve the treatment effect of the disease, and even may cure the original disease that can not be treated, for some children without drugs available, may find a new vitality.
Even cases in the placebo or blank group are not meant to be treated, but to be treated with the optimal current therapy. Moreover, patients who participate in clinical research will receive more attention from medical staff, and he needs to record some data content in the treatment process, and medical costs can also be partially reduced.
【Working together to promote clinical trials of children's drugs】
After all, clinical trials are not marketed drugs or treatments, and it still has great uncertainty. So how do we control such a risk?
I think we can control risk in three ways.
First, general drugs can not be directly studied in children, some special drugs, must first pass the adult clinical trial for children, there are adults to help us lay a bottom, but also to ensure the basic safety.
Second, clinical research programs are well-designed and prepared with the participation of many experts, must follow the principle of least harm and lowest risk, and must pass the strictest ethical and scientific review of official institutions, which is the guarantee of the system.
Third, before the clinical study of the drug, the pro researcher must introduce the clinical study of the drug with the legal guardian and the child who has reached a certain age. At this time, the guardian should not easily say no or do, must thoroughly understand the content of the study and the rights and interests of the subjects, and ask the doctor any questions. This includes whether the aforementioned insurance is purchased for the subject and whether it is already used in adults. Under the premise of full understanding, then judge whether to agree to it to ensure their own rights and interests. During the course of the study, subjects can also request to withdraw from clinical trials at any time, so they will not be treated unfairly.
So what do you think is the state of clinical trials in children in our country? What else needs to be improved?
It should be said that the clinical trials for children in our country are still relatively backward. According to current statistics, children account for only 2.8% of clinical trials in China in the same period, far lower than the global proportion of more than 11%.
This may be because the current awareness and mission of this cause is not too full, and this work still has a long way to go. Including the patient's family and the medical staff themselves, they need to have a further deepening understanding of clinical research, and ethical work also needs to be continuously optimized.
There are clear regulations that any ethics committee that meets the norms will definitely require the participation of lawyers and non-medical personnel, including community representatives. They can judge the rationality of the experiment from the perspective of a bystander to better protect the rights and interests of patients.
At present, many hospitals have opened clinical research wards, specifically for clinical research institutes. Shanghai Children's Medical Center has also set up more than 30 clinical research wards for this purpose, and we are constantly summarizing experience in many jobs, and found that some need to change, including we also need to make some improvements in the system.
For example, how to better build a professional clinical research team, as well as the popularization of clinical research knowledge and science, the popularization of relevant laws and regulations, and the change of the social public opinion environment, the government level also needs to pay certain attention. Here, I also appeal to our patients, parents, and medical staff, hoping that everyone will come together to do such a job that should be said to benefit children.
Through Professor Wang Wei's interpretation, we understand that strengthening the clinical research of children's drugs is the only way to solve the problem of children's drugs, but at present, there are few clinical trial projects for children's drugs, which are difficult and involve more ethical issues, making domestic children's drug research and development and clinical trials at a low level. Then the comprehensive development of pediatric clinical research requires multi-angle, multi-dimensional and multi-departmental cooperation, which requires both policy encouragement and regulatory improvement, as well as improving public awareness.