September 13, 2021, Beijing
To achieve the strategic requirements of “sticking to innovation-driven development” in the 14th Five-Year Plan period, China Pharmaceutical Innovation and Research Development Association (PhIRDA) and the R&D-based Pharmaceutical Association Committee (RDPAC) have been jointly launching report series on “Fostering China Pharmaceutical Innovation System” ever since early 2021. With top priority given to three main aspects saying competitiveness of pharmaceutical innovation industry, accessibility of innovative drugs, and sustainability of the pharmaceutical innovation industry, the report series aim to provide multi-dimensional and constructive suggestions on the sustainable growth of China’s pharmaceutical innovation system and empower high-quality development of China’s pharmaceutical innovation industry.
Through joint efforts of PhIRDA and RDPAC, the “Fostering China Pharmaceutical Innovation System Report 4: Promote Simultaneous R&D, Registration and Review of Innovative Drugs” (hereinafter referred to as the “sub-report” or “report”) is officially released today. The achievements in pharmaceutical innovation driven by China’s drug regulatory reform over the past five years is the bedrock that underpins this report. On this basis, the attention is focused on two themes saying clinical studies and regulatory approval throughout the process of simultaneous development, registration and review. The purpose of this report is to promote the translational research and clinical development of innovative drugs in China to keep in alignment with other countries in the world.
In recent years, the reform of drug review and approval system has greatly shortened the time consumed for new drug approval and makes it possible to synchronize the registration and marketing of innovative drugs at home and abroad. However, there remains a “time lag”. It is pointed out in this report that for China, there are three approaches available to achieve global simultaneous R&D, registration and review of innovative drugs.
To begin with, China should participate in global simultaneous R&D as soon as the early clinical trials has started up and advance simultaneous R&D at all stages to ensure simultaneous registration and marketing;
Or alternatively, China could join in mid- and late-stage global multi-center trials to achieve simultaneous registration and marketing;
Last but not least, the bridging trials and separate clinical trials required for simultaneous registration and marketing in China should be planned earlier.
In view of this, improving regulatory science on registration and the efficiency of clinical studies are the keys to accelerate global simultaneous R&D, registration and review of innovative drugs. That entails China to further optimize regulatory policies, regulatory standards and procedures, and regulatory system on the one hand. On the other hand, it also requires China to strengthen implementation of clinical studies and clinical research capabilities, and put in place an effective security mechanism for clinical studies. Again, efforts should also be made to enhance regulatory capabilities, intensify talents training and establish digital platforms. In terms of these important aspects pertaining to simultaneous R&D, registration and review, ten specific recommendations are made therein.
China’s active participation in global simultaneous R&D is of great significance to the construction of China's pharmaceutical regulatory system, the enhancement of innovation capabilities and improvement of patient benefits.
The pharmaceutical innovation in China has embarked on a road for high-quality development ever since the regulatory reform was carried out in 2015 in a bid to keep in line with international standards. The technical standards for drug development and registration that prevail globally have been comprehensively introduced to China especially since China's regulatory authority joined ICH in 2017. By now, a certain number of companies have successfully accomplished global simultaneous registration of new drugs under the guidance of ICH E17 (General Principles for Planning and Design of Multiregional Clinical Trials Guidance for Industry). Conducting global multi-center clinical trials in multiple regions including China is gradually becoming a strategic choice for domestic and foreign companies that aim to register and market drugs in multiple countries.
Given that the global multi-center clinical trials are conducted simultaneously by multiple sites in multiple countries or regions as per the same clinical trial protocol, and there should be rigorous protocol design, and strict requirements, the scientific and standard practices required in formulation, analysis, regulation, and execution of the clinical trial protocol represent great challenges for all participants involved. If feasible, China's active participation in global multi-center clinical trials will significantly improve its overall clinical study and management capabilities, and accelerate China’s pharmaceutical innovation to be in line with the international innovation.
It is emphasized in the report that the efficiency and quality in clinical R&D period are mainly hindered by the implementation of clinical protocol and the ability to execute the clinical study. In this regard, efforts should be made to strengthen the security mechanism for clinical studies from the following aspects. With respect to implementation of clinical studies, attention should be paid to optimizing and realizing harmonized intra- and inter-site processes, simplifying ethical review and approval, and improving the efficiency and quality of regional or central ethic review; In capacity building, actions should be taken to promote the establishment of clinical study platforms and dedicated clinical study teams, so as to help clarify the positioning of the institutions and accumulate management experience on exploratory clinical trials. In addition, it is necessary to increase incentives for clinical studies, activate incentive mechanism for clinical studies oriented to doctors and hospitals, and increase the fund proportion of clinical medical studies in medical research funds, or set up dedicated scientific research programs and funds.
"Three initiatives" to break through the current bottlenecks and "five focuses" to ensure a sound system
China is home to rich clinical resources and great potential to contribute to global new drug development. With the current support in policies and funding, the demand for clinical trials in pharmaceutical innovation is rising sharply, and this brings about unprecedented opportunity for China to participate inglobal multi-center clinical trials. However, there is also an urgent need to improve the efficiency of clinical studies and the scientificity of registration regulation. To be more specific, more efforts should be made to optimize resource allocation that has been influenced by homogeneous studies, enhance management ability and experience of all parties concerned, and properly define the proportion of exploratory studies in the overall clinical program. Additionally, the common concern across the industry about the impact that the regulatory process and specific policies would have on the implementation of clinical studies should be relieved. All in all, the impact of various key factors on global simultaneous innovation is becoming increasingly evident.
It is pointed out in the report that to address the current bottlenecks, we need to implement “three initiatives”. In the first place, a scientific and reasonable risk control management system for human genetic resources should be established by developing rational requirements for genetic resources application and optimizing the efficiency of the process, along with the introduction of the Measures for the Regulations on Management of Human Genetic Resources. The second initiative is to define more scientific requirements for the enrollment of Chinese subjects and enhance mutual recognition of global data. On this premise, the goal is to increase the degree of openness to clinical trial data of Northeast Asia. The third is to promote unified, standard, and collaborative processes for clinical sites and ensure efficient implementation, to straighten out the inter-site processes of project establishment, ethical approval and contract execution, and to short start-up timeline for clinical trials through overall planning and simplification. Competent health authorities should standardize the inter-site processes and carry out quality control, and incorporate into institutional assessments key indicators for implementation of clinical studies.
It is recommended in this report that to forge a sound system, we should focus on five aspects. Optimization of review-related processes and clinical value-oriented reviews should be encouraged; The requirements for dossier submitted for review and approval should be improved in line with global standards; The drug marketing authorization holder mechanism should be fully implemented; Clinical study platforms and dedicated clinical study teams should be established to help clarify the positioning of the institutions and accumulate management experience on exploratory clinical trials; and improvement should be made to the incentive mechanism for and resource investment in clinical studies.
China's pharmaceutical innovation is entering a stage of higher level of development. The number of local Chinese innovative companies is on the rise and international cooperation and exchange is further driving the growth of China’s pharmaceutical innovation. Vaccines developed by Chinese companies have been recognized by many countries and two of them have been listed by the WHO for emergency use. This has not only demonstrated China's pharmaceutical innovation capabilities, but more importantly, as a practical example of development and implementation of commonly used international technical standards, it has helped China accumulate valuable experience for a more scientific, international and modernized drug regulation. At the same time, we should not let go the opportunities. Instead, we should continuously improve our regulatory system in line with international standards, and R&D capability and capacity, and follow the trend of globalization in new drug R&D, and build on our strengths to advance the construction of a “powerful country in pharmaceutical innovation. Promoting simultaneous development, registration and review in China will allow innovative drugs to be accessible to Chinese patients at a faster pace and help China be better synchronized with the world in pharmaceutical innovation.
About Report Series on Fostering China Pharmaceutical Innovation System
The report series Fostering China Pharmaceutical Innovation System is co-led by the China Pharmaceutical Innovation and Research Development Association (PhIRDA), and R&D-Based Pharmaceutical Association Committee (RDPAC) in 2021. Starting from the overarching design, this report series aims to analyze in depth the impact of key elements such as basic research, policies, markets (e.g. access, payment and procurement) and funds on improving China's pharmaceutical innovation ability. This report series also aims to identify the weak links and facing challenges in the whole industrial chain at the present stage, put forward constructive suggestions of substantial significance from multiple dimensions, and promote the high-quality development of China 's pharmaceutical innovation, so as to build an international-oriented ecological environment that is suitable for the sustainable development of China's biopharmaceutical innovation, which all make positive contributions to the successful transformation of China into a global leader in pharmaceutical innovation. Please stay tuned for the release of the Global Simultaneous R&D sub-report and the full version of the report to follow.
Click on the following link for the full report:
http://cnadmin.rdpac.org/upload/upload_file/1631519479.pdf
Click on the following links for the published series reports:
