On November 30, 2024, the Innovative Drug R&D for Metabolic Diseases Forum of the 2024 China BioMed Innovation and Investment Conference was successfully held in Guangzhou. Against the backdrop of the increasingly severe global obesity crisis, the treatment and prevention of metabolic diseases will become a major issue in the future healthcare field. The forum invited experts from multiple disciplines to discuss and explore new ideas for research and development.

Breaking the Involution: Exploring New R&D Approaches from Multiple Dimensions

In the face of increasingly fierce competition, clinical and industry experts are joining forces to explore new directions and break free from involution. Experts at the forum offered unique insights on innovative R&D approaches.

Ji Linong, Director of Endocrinology and Metabolism Department,Peking University People’s Hospital, pointed out two distinct paths in drug development. On one hand, companies can choose to follow the safe and proven market paths, such as developing biosimilars of semaglutide, inheriting its established efficacy and safety data. On the other hand, companies can take risks and venture off the mainstream paths to explore unmet clinical needs, despite the larger risks involved. He specifically mentioned that while the current surge in weight-loss drug development has fueled research on GLP-1 receptor agonists (GLP-1RAs) and related products, it has cast a shadow over the development of non-GLP-1 drugs for treating cardiovascular-kidney-metabolic syndrome (CKM). He believes that future drug development should go beyond current popular targets to find more drugs that can effectively improve clinical outcomes.

Ji Linong delivered a keynote speech on “Reflections on the R&D of New Drugs and Clinical Management of Cardiovascular-Kidney-Metabolic Diseases.”

Julia Zhu, Therapeutic Area Head of Cardio-Renal-Metabolic Diseases in Medicine, Boehringer Ingelheim, emphasized the close connection between metabolic diseases and cardiovascular and kidney diseases. Although many domestic companies are still focusing on obesity as a hot research area, there remain huge gaps in the treatment of diseases related to metabolic disorders, such as fatty liver disease, chronic kidney disease, and chronic heart failure. She suggested that companies should expand their treatment goals from short-term weight loss to long-term cardiovascular and kidney protection, and conduct in-depth clinical research to validate the clinical value of these drugs, providing more comprehensive treatment options for patients. 

Yuan Qinghui, Head of Pharmaceutical and Biotechnology Industry Equity Research at China Securities, shared her views on the future development focus of GLP-1 drugs. She emphasized that while improving efficacy and weight loss effects are important, greater attention should be given to the overall benefit of the drugs and the development of new indications. Furthermore, improving drug tolerability and safety, as well as developing new administration methods like monthly injections, should also be key areas of focus. She stressed that while pursuing weight loss, drug tolerability and overall benefits should not be overlooked.

Rao Huiying, Director of Peking University Hepatology Institute, focused on patients with severe liver diseases such as cirrhosis. She pointed out that for these patients, weight improvement is only part of the treatment, and the more important goal is to identify drugs that target the liver to improve liver fibrosis and fatty liver disease. She hopes that in addition to the current focus on metabolic drug development, there will be more attention on liver disease treatment, bringing greater benefits to patients with fatty liver.

Zhu Wenjing participated in the discussion on “How to Advance the Sustainable Development of Innovative Drugs for Metabolic Diseases Through Collaboration.”

Yuan Qinghui delivered a keynote speech on “Market Trends and Industry Analysis of Metabolic Diseases/Weight-Loss Drugs.”

Rao Huiying delivered a keynote speech on “Focusing on the Progress and Clinical Application Prospects of New Drugs for Obesity and MASH.”

With ongoing research and technological innovations, innovative drugs are being continuously brought to market. However, in the context of an aging population and slower medical insurance funding, how to efficiently select valuable drugs and solve the problem of new drug access into hospitals has become a key issue for ensuring a sustainable innovation ecosystem in pharmaceutical innovation and investment.

Innovative Drug Value Differentiation

Jin Chunlin, Director of Shanghai Health Development Research Center, believes that the trend of “replacing old with new” will not change, and medical insurance funds will continue to support highly innovative drugs. The innovativeness of drugs has various dimensions: developers focus on scientific value, such as new mechanisms, new targets, and irreplaceable therapeutic positions; insurers and doctors focus on therapeutic value, such as improving clinical efficacy, reducing adverse reactions, improving quality of life, and increasing compliance; while researchers in health policy and insurance policy emphasize social value, such as reducing disease burden, fostering industrial development, and achieving long-term social benefits like technology localization. Life is priceless, but drugs are priced. The challenge lies in finding a scientific balance between the “priceless” and the “priced,” which requires joint efforts.

Jin Chunlin delivered a keynote speech on “Considerations on Key Points for the Medical Insurance Access and Payment Standards of Innovative Drugs.”

Solving the “Last Mile” Problem

Pan Huijuan, Director of the Medical Insurance Department of Guangzhou, emphasized that the medical insurance department is committed to providing better protection for insured individuals.In promoting the access of innovative drugs into hospitals, medical institutions hold timely pharmacy meetings to ensure drugs are “as available as possible.” For drugs that have not entered hospitals, they expand access through a dual-channel system,extending the service coverage from nationally negotiated drugs to outpatient and special-use medicines, making it more convenient for patients to access medication.In terms of medical insurance coverage, national negotiated drugs are paid separately, not subject to outpatient and special use limits, ensuring that insured individuals can use these drugs with confidence. Deng Xijin, Director of the Guangzhou Municipal Health Commission, introduced three initiatives currently being implemented in Guangzhou: first, requiring hospitals to establish innovation drug working groups under their pharmacy committees to proactively discuss innovation drug issues; second, setting up the Regulatory Innovation and Development of Pharmaceutical and Medical Device in the Guangdong-Hong Kong-Macao Greater Bay Area professional committee to introduce overseas innovative drugs based on clinical needs; and third, promoting deep integration between hospital resources and high-quality enterprise resources to accelerate innovative drug R&D transformation, helping hospitals and doctors better understand innovative drugs in the early stages and expedite the provision of safer and more effective treatment options to patients.

On the Widespread Societal Concern About Obesity

Ji Linong pointed out that international society’s understanding of obesity is undergoing a profound change. Obesity is no longer viewed simply as weight gain, but is now redefined as a clinically significant disease. Its diagnosis now requires combining weight gain with dysfunction and psychological disorders. This shift not only affects the diagnostic and treatment standards for obesity but also prompts international discussions on the reimbursement of weight-loss drugs.

Jin Chunlin emphasized that the current Chinese medical insurance system mainly focuses on medical insurance rather than health insurance, which has, to some extent, limited insurance funding for the prevention of obesity and other chronic diseases. He believes that while obesity will eventually evolve into a disease, efforts to prevent obesity involve both medical treatment and prevention, and the intersection between these two needs further clarification.

Looking to the Future: Synergistic Development of Disease Management and Clinical Development

In the vast field of metabolic drug development, multidisciplinary collaboration and innovative drug development will be key drivers of future growth.Experts shared their views on the future trends of metabolic drug development from different perspectives.

Jiang Hongwei, Vice President of the First Affiliated Hospital of Henan University of Science & Technology, noted that obesity, as the root cause of many diseases, requires multidisciplinary collaboration, including endocrinology, metabolism, surgery, and cardiology. As drugs targeting single, double, and triple GLP-1 pathways are developed, more specialties are being incorporated into the treatment system for obesity and its related diseases. Through multidisciplinary collaboration, more benefits can be brought to patients.

Li Yanbing, Director of the Department of Endocrinology, First Affiliated Hospital of Sun Yat-sen University, emphasized that low awareness and insufficient attention are major obstacles in the clinical management of diabetes and MASH. In routine diagnosis and treatment, there is still a lack of interaction between endocrinology and other departments, such as hepatology. He looks forward to deepening communication through more interdisciplinary cooperation, further revealing the intrinsic mechanisms of diseases and their interactions.

Zou Xiantong , Assistant Professor of Peking University, discussed the disease mechanisms and stated that fatty liver is not merely a complication of obesity but involves multiple target pathways during its development. Developing drugs targeting these key pathways will provide new directions for fatty liver treatment. She emphasized that weight loss plays a central role in the treatment of fatty liver, but treating liver fat alone has a minimal impact on overall metabolism. Therefore, combining weight loss with drugs targeting other pathological pathways like liver fibrosis or oxidative stress will be a crucial strategy for future fatty liver treatment.

From a clinical trial management perspective, Angela Xiao, Cardiovascular-Renal-Metabolic CD & O TAH, Boehringer Ingelheim (China) Investment Co., Ltd., emphasized that high-quality research is essential for ensuring the successful launch of innovative drugs. As a sponsor, it is important not only to speed up the development process but also to focus on the quality of clinical trials. She shared her experience in establishing early-stage patient and researcher advisory committees, which offer valuable insights for the research protocol, ensuring operability and high patient adherence, providing more accurate and effective guidance for metabolic drug development.

From a drug development strategy perspective, Ji Linong stated that with the updated definition of obesity, the development path for obesity drugs will become more diversified. On one hand, weight-loss drugs will become more widely used by the general public to help people reduce weight, and these drugs may no longer require hospital administration in the future. On the other hand, the development of weight-loss drugs will face challenges due to reimbursement restrictions, highlighting the need to focus on developing drugs that not only aid in weight loss but also prevent the progression of metabolic diseases, such as cardiovascular and liver diseases, into more serious conditions. He emphasized that to gain clinical recognition and secure reimbursement, drug developers will need to conduct in-depth research and clinical validation for each specific indication.

The forum provided a valuable platform for industry professionals to exchange ideas on research and development strategies, injecting new energy into the innovative R&D of metabolic disease treatments. We look forward to seeing clinical experts and drug developers bring more comprehensive and effective treatment options to patients in the future.