Increasing orphan drug loss in Japan: Trends and R&D strategy for rare diseases.

Rare disease (RD) patients face significant unmet therapeutic needs worldwide. However, orphan drugs approved in the United States, but not approved or developed in Japan, have increased rapidly with recently increasing US approvals, indicating greater RD drug loss in Japan. US/EU-based startups have become key players in RD drug R&D, significantly contributing to this drug loss trend. They successfully develop drugs in the United States by combining in-licensing with in-house drug discovery. Out-licensing to Japanese companies or large pharma is critical for expansion into Japan, with successes attributed to drug innovation, target indications, and transactional capabilities. These findings highlight the need to foster partnerships with startups and cultivate an ecosystem in Japan that nurtures local startups, to address drug loss and ensure access to promising drugs.

Analysis and suggestions on the development trend of drug innovation in China / 中国药科大学学报

@#In order to comprehensivehy evaluate the stage of China in the global drug innovation, to further optimize the environment for drug innovation in China, and to unleash the vitality of drug innovation, this article mainly analyzes through comparison China''s situation of drug innovation in global competition from such perspectives as the current situation of the global drug R&D innovation market, R&D investment, product pipeline, policy support, and development trends, combined with the characteristics of China''s drug innovation development.It can be seen that China''s pharmaceutical innovation is faced with such practical problems as lagging behind some developed countries in terms of innovation development, companies bunching into research and development innovation, sudden research and development rush, and excessive dependence on capital markets for pharmaceutical innovation.Accordingly, this paper puts forward suggestions on continuously improving China''s new drug innovation environment, rationally selecting differentiated competition and new tracks, reasonably formulating drug innovation development strategies, guiding capital to return to innovative research and development, and constructing a "double cycle" strategy for drug innovation.

[Considerations on Clinical Development and Regulatory of the Oversea License-in 
Anti-tumor Drugs].

With the boom of China's innovative pharmaceutical industry, licensing-in model has gradually become an important research and development model for innovative pharmaceutical companies. The in-licensed drugs at different stages need different research and development (R&D) strategy in China. The pharmaceutical companies take the responsibility to comprehensively collate the oversea clinical data and conduct a detailed analysis of clinical pharmacology, safety, efficacy and ethnic sensitivity. Clinical R&D strategy should be made based on the results of the above data and analysis. We encourage high-quality drugs which fill unmet clinical needs licensed in, and as early as possible, so as to conduct multi-regional clinical trials (MRCTs). The clinical R&D strategy in China is particularly important for the drug's approval. Guidelines published by the National Medical Products Administration (NMPA) and clinical associations should be followed. Communications about clinical R&D strategy with Center of Drug Evaluation (CDE) are encouraged.
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Considerations on Clinical Development and Regulatory of the Oversea License-in Anti-tumor Drugs / 中国肺癌杂志

With the boom of China's innovative pharmaceutical industry, licensing-in model has gradually become an important research and development model for innovative pharmaceutical companies. The in-licensed drugs at different stages need different research and development (R&D) strategy in China. The pharmaceutical companies take the responsibility to comprehensively collate the oversea clinical data and conduct a detailed analysis of clinical pharmacology, safety, efficacy and ethnic sensitivity. Clinical R&D strategy should be made based on the results of the above data and analysis. We encourage high-quality drugs which fill unmet clinical needs licensed in, and as early as possible, so as to conduct multi-regional clinical trials (MRCTs). The clinical R&D strategy in China is particularly important for the drug's approval. Guidelines published by the National Medical Products Administration (NMPA) and clinical associations should be followed. Communications about clinical R&D strategy with Center of Drug Evaluation (CDE) are encouraged.
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Research progress on sedative and hypnotic effect of Sini Powder and strategic analysis of its new drug development / 中草药

Sini Powder is the traditional Chinese medicine compound, which comes from Treatise on Febrile Diseases. It is used to treat the syndrome of yang depression and reversal cold of limbs. Sini Powder has a certain effect on treating insomnia caused by liver depression and spleen deficiency. Lots of researches have shown that Sini Powder has a sedative and hypnotic effect similar to diazepam and plays an important role in improving sleep disorders caused by post-traumatic stress disorder (PTSD). Sini Powder is not included in Chinese Pharmacopoeia (2015 edition). It is probably to develop Sini Powder into a new drug for the clinic. We collated and summarized the literature about the sedative and hypnotic effect and the improvement of sleep disorders caused by PTSD of Sini Powder. Based on the modern research on the sedative and hypnotic effect of Sini Powder, we think the effect of "relieve uneasiness of mind and body tranquilization" adapts the theory of traditional Chinese medicine, so that it can improve the applied range of Sini Powder. Meanwhile, we analyzed the possibility of Sini Powder being selected into the ancient classical prescription and key problem in research and development on drug preparation of Sini Powder according to regulations of traditional Chinese medicine. Then we put forward some suggestions for further research from two aspects which are the development of Sini Powder and new drugs of traditional Chinese medicine, to provide a reference for the development of new Chinese medicine preparation of Sini Powder focus on "expelling pathogen and relieving depression, soothing liver and regulating spleen, tranquilizing mind".

Research and Development of New Traditional Chinese Medicine Based on Experienced Prescriptions of Prestigious Chinese Physicians / 中国实验方剂学杂志

The experienced prescriptions of famous prestigious Chinese physicians are effective prescriptions developed by prestigious Chinese doctors during their long-time clinical practice, which reflect the traditional Chinese medicine (TCM) understanding of the disease development regularity and core pathogenesis. These experienced prescriptions provide valuable experience for medication and prescription regularities, and represent the highest level of TCM treatment and the major sources of new drug research, development and technology innovation. It is of great significance to inherit academic thoughts and clinical experiences of prestigious Chinese physicians, explore and summarize experienced prescriptions, and develop new Chinese drugs. The researches of new drugs based on experienced prescriptions are the major direction encouraged by the government, with the maximum amount of new TCM drug applications but a low approval rate in recent years. The main reason for the low number of approved new TCM drug applications is that researchers know less about evaluation concepts and relevant techniques, leading to problems in research and development strategy. To facilitate a smooth advance of the new drug research and development and take full advantage of the experienced prescriptions, in this paper, we focus on the problems about new drug development of experienced prescriptions, lay emphasis on the new TCM drug research and development concepts of clinical value, history of human application and whole-process quality control, and deeply and systematically analyze concerns in such links as pharmacy, pharmacodynamics, toxicology and clinic application. The purpose of this article is to provide the reference in solving actual problems, the reliable basis of further researches on experienced prescriptions, and the important guarantee for developing more safe, effective and high-quality controllable drugs to meets clinical requirements, so as to achieve the strategy of a healthy China.

Catenion Strategies, GmbH.

Catenion Strategies is a strategy consultancy company formed around a long-standing team of Anglo-German partners of various scientific and industrial backgrounds with a common passion for the dynamics of the life-science industry. Catenion provide strategy consulting services exclusively for the top management of pharmaceutical, biotechnology and diagnostics companies. Their focus lies in the areas of corporate development, research and development (R&D), therapeutic area and product strategy, as well as in the emerging field of personalized medicine. Catenion believe their grounding in R&D is unique, and they aspire to be the premier partner of choice for senior management in all core aspects of shaping pharmaceutical strategy.