Adhere to "patients first", decentralized clinical trial special seminar held

On August 21, the "Patients as the first" decentralized clinical trial seminar held by the Roude Rare Disease Center was successfully held in Hangzhou. The purpose of this seminar is to study decentralized clinical trials (DE-CENTRALIZED Clinical TRIAL (DCT ") that may face the problems and challenges that may face in the rare disease field in China, as well as the new opportunities brought about by this. A new clinical practice allows the life science industry to collect high -quality clinical and real clinical practice data, and at the same time can better feedback patients' needs.

The conference was hosted by the Rou Dehan Rare Disease Center (CORD), supported by Takeda China, IQVIA). The seminar is divided into two parts: keynote speech and open discussion. The patient representatives reflected the voices on the spot. The clinical medical experts expressed their opinions. Research in rare diseases in China.

The conference was chaired by Huang Rufang, co -founder & Secretary -General of the Ruide Hulk Center, the co -founder & Secretary -General of the Rui Ou Public Welfare Foundation. Director Huang Rufang welcomed the arrival of all guests and participants, introduced the guest lineup of the conference one by one, and the topic of the discussion. The key to the theme of this conference is two points: First, in the context of the global epidemic, talk about DCT's difficulties and challenges, and even the opportunities that DCT faces in China. Research and discussing how DCT is used in the field of rare diseases to benefit patients, and it also brings benefits to research institutions.

(Huang Rufang spoke picture)

The voice of patients with rare diseases

In traditional clinical trials, patients need to go to medical institutions with clinicians for multiple face -to -face interactions. In DCT, patients can remotely interact with clinicians and submit information. There is no need to contact face -to -face, which can provide patients with a more convenient experience and provide more choices for patients who cannot go to the clinical trial point.

(Lu Xiaochun Speaking Picture)

Rare Disease Network Volunteer and the Secretary -General of Rare Diseases Lu Xiaochun (the Secretary -General of Wang Lixin, the Development of the Network Wang Lixin, on behalf of the Rare Disease Organization) believes that decentralized clinical trials can achieve remote/mobile medical interaction. Patients provide more convenient experience, providing more choices for patients who cannot go to clinical trials, and bring hope to patients. At the same time, most patients with rare diseases have caused family or self -difficulties due to diseases, and they should give more support to participating in clinical practice.

The impact of DCT on rare diseases in China

He Jieru, the person in charge of Takeda Digital Strategy, gave a keynote speech based on the "Application and Case Sharing of DCT in Rare Disease Studies". She believes that due to the large types of rare diseases, the number of patients is small and the region is scattered, the condition is severe and the course of the disease is long, and the characteristics of children are more accumulated, which makes patients participate in clinical trials full of challenges. The promotion of DCT in the field of rare diseases can reach more patients, reduce the burden on patients, and allow patients to join in clinical research without regional restrictions. In the test design centered on patients, the DCT elements contained in the patients such as Internet recruitment, long -range knowledge and video diagnosis, support patients multiple scenes (research centers, home, local clinics, etc.) follow -up, to patients' drug transportation, remote patient monitoring And data collection, etc., can provide convenience for patients and reduce the burden of participation research. For applicants, while improving the patient experience, using digital medical technology in DCT, if wearable devices, can more diverse collection data, so as to better evaluate the safety and efficacy of rare disease research drugs.

(He Jieru Speaking)

DCT's practice in China is not enough

Shen Yifeng, director of the Shanghai Mental Health Center Pharmaceutical Clinical Test Institution Office and a member of the Shanghai Pharmaceutical Society of Pharmaceutical Clinical Research Committee, believes that DCT's practice in China is not enough, and there are still many practical problems that need to be resolved. For example, in terms of risk monitoring, patient privacy protection, protection of the right to know, etc.

DCT is centered on patients. At the current stage, the most important thing is to choose the most suitable clinical trial items for DCT. It is suitable for what is suitable for, and everything is based on patient needs. For a long time in the future, the traditional model and DCT mode of clinical trials may coexist.

DCT promotes dilemma and opportunities in China

In the open discussion session, the guests and doctors and patients were carried out research discussions on the two themes of "demand and challenges under multiple perspectives" and "opportunities and challenges under multi -party perspective".

Decentralized clinical trials (DCT) transferred clinical trials from offline to online, and the operation process is relatively hidden. How can the legal regulatory issues, the privacy of patients/subjects, and the protection of the right to know the right to solve it? The patient -centric DCT purpose is to reduce the obstacles of patients to participate in clinical trials, but it is necessary to increase the investigation of the doctor. How to support the doctor to provide patients with more clinical trial services? The discussion session and interactive link of the "demand and challenge of multiple perspectives" were chaired by Ni Shaoqing, director of the Office of the Pharmaceutical Clinical Test Initiative of the Children's Hospital of Zhejiang University, and the guests expressed their views at the seminar.

(Director Ni Shaoqing)

Chen Xiaoyuan, a researcher at the School of Medicine of Tsinghua University, believes that the patient is centered and clinical value -oriented is the core concept of the current drug research and development, and it is also the direction of the regulatory authorities of various countries. It is reflected in the implementation of clinical trials to reduce the burden on patients and improve the experience of patient participation. Either DCT, artificial intelligence, or long -range intelligence, are a patient -centered means and tools. The experience of DCT practice in China is still very limited, and you need to explore and try. In this process, you should always follow this concept. Our purpose is not to practice DCT, but to explore a clinical trial mode that is really conducive to patients. (Chen Xiaoyuan's online speech map)

Li Shuting, deputy dean of the GCP Center of the Jimin Cancer Hospital affiliated to Anhui Medical University, believes that although decentralized clinical trials make clinical trials more convenient, whether the data can be recognized by the Drug Administration is still a problem to solve. In addition, when we are decentralized, when the patient -centric, we have a evaluation standard for the control of the patient's risk during the implementation process. Basic standards.

(Li Shuting online speech map)

Shen Yifeng, Director of the Office of the Pharmaceutical Clinical Test Institution of Shanghai Mental Health Center and a member of the Shanghai Pharmaceutical Society Drug Clinical Research Committee, believes that Shen Yifeng, director of the Shanghai Mental Health Center Institution Office Effectively understand the relevant content.

In terms of compensation and payment of the subject, Director Shen Yifeng believes that under the support of the hospital's leadership, a third party can be introduced to complete the cost payment, which is much faster than transferred to the subject by the hospital. In addition, the supplier of the contracting business can be required to provide emergency prepaid money to solve patients' urgent burning, so that subjects who encounter accidental damage will not be treated due to cost delay. You can also pay for inspection and inspection costs, so that the subject will not lose the opportunity to participate in clinical trials because of no money to check.

In addition, Director Shen Yifeng also mentioned that the traffic compensation costs of the current fixed amount are actually different from the transportation reporting of ethical requirements. At present, pilot subjects are directly enjoying the innovative model of traffic services without spending money.

(Shen Yifeng's speech map)

Wang Wanqing, the senior director of the clinical operation department of the Takeda Asia Development Center, said that the patient's experience and rights and interests of Takeda attaches great importance to it. Whether the subject can simplify the process of receiving the drug, such as directly from the manufacturer to the patient. At the same time, improve the problems of the previous payment time on the premise of legal compliance.

(Wang Wanqing Speaking Picture)

Representatives of patients with Du's muscle malnutrition in Zhejiang said in the discussion that they hope that DCT will help the poor information recruitment of doctors and patients and strengthen communication of doctor -patient relationships.

The theme discussion of the theme of "Opportunities and Challenges in multiple perspectives" was chaired by Li Gaoyang, the person in charge of the long -range intelligent clinical trial of Yudu Technology, and representatives of the DCT's difficulties and development opportunities brought about by DCT. Different views.

(Li Gaoyang Speaking Picture)

Liu Jun, deputy director of the global project management of the global R & D China Center, believes that promoting DCT in the field of rare diseases may have some specific difficulties. For example, grass -roots hospitals and medical workers have relatively little perception of rare diseases. Whether it can provide the clinical support and security guarantee for patients like a high -level hospital in DCT, the research team needs to consider entering when promoting DCT. Essence At the same time, the implementation of DCT requires some investment in pharmaceutical companies in the early stage, and also hopes that companies will make long -term and multi -dimensional considerations during decision -making.

(Liu Jun spoke picture)

Ma Yan, the person in charge of the long -range intelligent clinical research of IQVIA Ai Kunwei Pharmaceutical Technology (Shanghai) Co., Ltd., believes that in the process of promoting DCT Easy -to -understand user guidance and ensure the user experience. When encountering problems, a fast response measure and startup plan should be formulated. In the direction, the specific implementation also hopes that the regulations and guidance of relevant departments will be gradually improved under the guidance of practice; the third is that the DCT has more advantages in the short term reflected in the recruitment and reservation of patients, and the quality of data quality. The cost has not been significantly reduced in the short term.

(Ma Yan's speech map)

Wu Baolin, the person in charge of the operation of the E-SITE Excellence Center of Tiger Medicine: The state encourages the development of rare diseases and innovative drugs and improved new drugs for rare diseases. The approval time limit is greatly shortened, the verification and approval are prioritized, and the technical information can be supplemented after being confirmed. The dispersion of rare disease sources is a good entry point for promoting the DCT model. The research and development of new technologies accelerating rare diseases. Under the impact of the epidemic, DCT's remote surveillance module has been quickly popularized. Other modules of DCT, such as electronic informed part, can also get a major breakthrough in combination with the particularity of rare diseases.

(Wu Baolin Speaking)

The host Li Gaoyang mainly discussed the "Principles of the Technical Guidance of Patients as the Centers". The guests made valuable suggestions based on rare disease research and development. The guests learned the latest "Consensus of Expert Experts Expert Experts of Remote Intelligent Clinical Test". Among them, the DCT digital platform model was proposed to provide theoretical prototypes for the development of industry technology platforms. The current domestic DCT pilot project experience It has important reference significance. DCT is a service that meets the GCP specifications of pharmaceutical research and development, daily diagnosis and treatment practice, and services that focus on patients' needs. The team and the patient organized two -way to meet the fundamental needs of the patient. (Digitalization platform for remote intelligent clinical trials)

DCT is a service that meets the GCP specifications of pharmaceutical research and development, daily diagnosis and treatment practice, and services that focus on patients' needs. Two -way with patients to meet the fundamental needs of patients.

In general, promoting decentralized clinical trials (DCT) in domestic rare disease research will face the model transformation brings technical adaptation, increased cost, and legal supervision. It still needs a process of continuous practice and continuously solving difficulties. But DCT is also a trend, and it does bring new development and changes to clinical trials. This does not mean to abandon the traditional clinical trial model, but to provide more clinical trial methods on this basis.

The promotion of DCT must adhere to the core concept of "patients first". While providing a convenient and efficient clinical trial method for pharmaceutical companies and medical institutions, it is necessary to fully protect the legal rights and interests of patients/subjects, realize that enterprises and patients shared together The win -win situation of winning.

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