Empower patient organization to help with disease diagnosis and drug development

A few days ago, China's first large-scale inter-disciplinary patient organization experience exchange platform - the first China Experience Exchange for Patient Organizations (CEEPO) was successfully held in Shanghai.

The conference was initiated by Shanghai Roche Pharmaceutical Co., Ltd., and attracted more than 30 patient organizations from various disease fields including cancer, nervous system diseases, blood diseases and rare diseases.

After the meeting, Arterial Network interviewed Professor Shi Anli, President of the Cancer Rehabilitation Association of China Anti-Cancer Association, and Ms. Li Wei, Vice President of Roche Pharmaceuticals China Medical Department, who shared their views on patient organizations from different angles.

赋能患者组织,助力疾病诊疗和药物研发

Domestic patient organizations are increasing, and professional capabilities still need to be improved.

The ancient Chinese saying that "the same disease is pity" means that people with the same experience can feel the same feelings and communicate with each other. Patient organizations come from this, looking for professional knowledge, communicating with the disease, and getting comfort. Various patient organizations have been established to facilitate patients and their patients. Family members communicate.

The Cancer Rehabilitation Association of China Anti-Cancer Association is an established authority of patient organizations. It was established in 1990 and is affiliated to the China Anti-Cancer Association. It is a mass, non-profit, academic organization that combines doctors and patients with cancer patients. There are 99 group members in 27 provinces, cities and counties across the country. The main responsibility of the association is to organize and support cancer prevention, scientific advancement of cancer rehabilitation and patient education activities.

Professor Shi Anli told reporters that the number of existing patient organizations in China has increased year by year. A network of patient organizations based on disease types, regional and online and offline has been initially established, and a number of successful cases have been accumulated in the rehabilitation management of patients. Played an important role. However, compared with international advanced experience, domestic patient organizations still have obvious shortcomings such as late start, organizational dispersion and limitations of professional skills.

赋能患者组织,助力疾病诊疗和药物研发

Professor Shi Anli, President of the Rehabilitation Association of China Anti-Cancer Association

In addition, foreign patient organizations pay more attention to humanistic care, such as disease-related psychological counseling and interest groups, because with the advancement of medical science, many cancers and serious diseases have become "chronic diseases", and they have to live with the disease for a long time. Learning to adapt, psychological construction is very important, requires long-term intervention and counseling by doctors and counselors, and this aspect is currently being followed up in China.

In view of this, the Anti-Cancer Association Rehabilitation Association organized overseas study and exchange meetings, such as the global patient organization experience exchange meeting, cross-strait three-person patient organization exchange meeting, etc., and exchanged experiences with internationally excellent patient organizations.

"We hope to take this opportunity to establish a platform for patients to organize their own communication, fully integrate the resources and advantages of all parties, promote the development of professional skills of patient organizations, build a scientific and efficient collaborative management system, and lead the comprehensive upgrade of patient organizations at all levels. Improve the quality of life of patients, significantly reduce medical expenses and social burden." Professor Shi Anli said.

Patient organizations should play a greater role in assisting diagnosis and treatment

Patients' tissues are often associated with rare diseases. According to Tian Miao and others, "Analysis of the status quo of rare diseases", rare diseases, also known as rare diseases or "orphan diseases", refer to diseases with low incidence and relatively few patients. The World Health Organization (WHO) defines rare diseases as diseases or lesions that account for 0.65% to 0.1% of the total population. Since 2009, the last day of February each year has been internationally designated as the International Rare Disease Day. There are about 20 million rare patients in China.

Difficulties in diagnosis, misclassification and lack of proper coding of rare diseases have made their disease burden unclear, which is one of the main problems encountered in the development of a rare disease health plan.

Rare diseases are also known as "orphan drugs." Of the approximately 7,000 rare diseases currently identified, the vast majority are drug-free, and only a few have the cure, but the price is very expensive and most patients are unbearable. Since the 1980s, the development of orphan drugs has gradually attracted the attention of the society. Many countries and regions have established rare diseases related institutions or organizations, and have formulated special laws and regulations to promote the development of orphan drugs. The content is the monopoly system of orphan drugs, and other measures include providing tax credits, exemption of supervision fees, and priority review.

Compared with developed countries, China's data on the types and incidence of rare diseases, the research on disease pathogenesis and the development of orphan drugs are in a backward state. Therefore, in the future development, I hope that China can first scientifically define rare diseases and orphan drugs, formulate rare disease prevention and control laws as soon as possible, establish an orphan drug monopoly system, emulate foreign law to authorize orphan drug owners to attach market monopoly rights, new drug pricing, taxation. Preferential policies, etc., open up market mechanisms; change orphan drug introduction policies, become passive and take the initiative to ensure that patients can receive timely treatment in the country; at the same time, include rare diseases into the medical security system, so that orphan drugs can enter the medical insurance drug list, and effectively reduce rare patients The burden.

In a nutshell, from the diagnosis and treatment to the supply of medicines, rare diseases still have a long way to go. Previously, patient organizations mostly worked in rehabilitation and human care. As the patient's organizational function became more and more sound, it was also developing in the “upstream” of disease diagnosis and treatment, participating in relevant policy formulation, helping to establish scientific diagnostic methods, and assisting drugs. Research and development.

Li Wei said that patient organizations can play a particularly important role in promoting drug development, especially in rare diseases. Because patient tissue gathers a large number of patients, it can generate a large amount of data, which can help researchers understand the natural course of the disease and the role of drugs.

赋能患者组织,助力疾病诊疗和药物研发

Ms. Li Wei, Vice President of Roche Pharmaceuticals China Medical Department

“Although the interests of pharmaceutical companies and patient organizations are not the same, the common goal of 'providing patients with standardized treatment and high-quality whole-course management' has brought us together. Helping patients to achieve more professional and professional The development of norms, exerting their own strength to participate in clinical practice, will help to maximize the benefits of patients." Li Wei said.

For Roche's identity as a pharmaceutical company to support this patient organization experience exchange meeting, Li Wei also admitted that the company did not set any business issues, only to demonstrate the corporate social responsibility of the patient organization capacity building: "as anti-tumor, anti-tumor A leader in key areas such as viruses and transplants, and innovative companies that create differentiated value in rare diseases, Roche is committed to strengthening communication with patient organizations to better listen to patients, clinical practice and patient practice. Combine needs and improve the patient value-oriented pharmaceutical value chain."

Digital tools empower patient organization and increase productivity

With the development of the Internet and information technology, patient organizations are also developing online, such as the early QQ group, BBS, the current WeChat group, online community, etc., and the online patient community has grown rapidly. Gu Hongfei, the founder of the online platform of “Lymphoma Home”, said that the online patient community has the characteristics of strong record, strong influence, strong immediacy, and breakthrough of geographical restrictions. It can not only help patients learn knowledge online, but also find rehabilitation patients. It can help pharmaceutical companies with social responsibility to optimize the effectiveness of the education program, and a convenient network will ultimately benefit patients.

The online patient community is available at home and abroad. The well-known foreign patient communities include PatientsLikeMe, HealthUnlocked, MyHealthTeams, etc. In China, there are lymphoma homes, sweet homes, and anti-cancer guards. Among them, PatientsLikeMe is best known for its network community for chronically ill patients.

As of 2017, it already has more than 500,000 registered patients, covering more than 2,700 diseases. Patients share treatment experience with each other in PatientsLikeMe, and the community also has professional medical professionals to help. Many pharmaceutical companies collaborate with PatientsLikeMe for data collection and mining, such as Roche's Genentech, Merck, AstraZeneca and so on.

The data collected by patient organizations from the perspective of patient quality of life is different from professional medical data, reflecting the real-world situation of a large number of patients and drugs, and more directly assisting drug companies in drug development and government decision-making. Patient community and pharmaceutical companies collaborate on data mining. The most important consideration is data structuring. Only the data of patients with large data volume is worthwhile. Useful data is structured and high quality. For example, PatientsLikeMe has a series of data scales, and patient data is continuous, so there is value for continuous monitoring. The domestic patient community is still in its infancy in the construction of big data systems.

There has always been a problem in clinical practice that there is often a difficult difference between the actual needs of patients and the solutions provided by medical systems. In order to get out of this predicament, many countries have begun to use the big data tools and methods to implement the “patient fill” measures in the medical system.

For example, patients' opinions are directly involved in the early development of drugs, and further use of real-world data from patient electronic medical records to assist clinical trials and drug approvals; on the issue of medical payment, it also began to use objective analysis of large-scale clinical efficacy data of patients. Convincing conclusions, exploring a payment system based on efficacy--through the correction of each step above, trying to establish a patient-centered value system, improving the efficiency of medical resource allocation from the roots, and then finding it difficult to see a doctor Solutions for medical system problems such as expensive medical treatment and tired doctors.

赋能患者组织,助力疾病诊疗和药物研发

Patient organization participates in the whole process of disease diagnosis and treatment

Li Wei also said that patient data collection cannot be generalized, and the amount of data collected by each disease is different. From the perspective of data generation, data collection using patient organizations can start from an earlier stage, and design tools and methods from the status quo of diagnosis and treatment, patient appeal, and scientific research needs to ensure that the data volume is sufficient and effective.

“We always believe that patient organizations are our very important partners. Hospitals, governments, patient organizations, and pharmaceutical companies are an integral part of the entire pharmaceutical industry chain. Each type of organization has a different role in it. In the process of participation in the treatment process, the future patient organization can increase participation, or push forward," Li said.

Collaboration is the most important aspect of building patient-centric disease solutions. Huang Rufang, director of the China Center for Rare Diseases Development (CORD), said: "Next, the head of the patient organization will continue to explore patient organizations with Chinese characteristics - government, medical structure, enterprises and other multi-party cooperation models, hoping to put patients on this issue Incorporate into government policy, enterprise research, production, and promote the construction of 'patient-centered' clinical medical practice to help achieve the ambitious goal of 2030 healthy China."

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