Use of real world data to improve drug coverage decisions in China

Wen, Wang; Tan, Jing; Wu, Jiang; Huang, Shiyao; YunXiang, Huang; Xie, Feng; Sun, Xin

doi:10.1136/bmj-2021-068911

Cited by 5 publications

(3 citation statements)

References 30 publications

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“…Additionally, it is not possible to fully explore all new categories of antineoplastic drugs for off-label use because the clinical requirements are intricate and varied, such as the requirements of patients with advanced cancer, multiple diseases, adherence, affordability, and tolerability, physician decision-making, and the same drug from different manufacturers or different specifications and dosage forms from the same manufacturer, which can also lead to the unapproved use of antineoplastic drug. Nevertheless, to promote the standardized management and rational use of unapproved anticancer drugs, the scope of categories on unapproved antineoplastic drug use can be further extended from electronic medical records, patient-reported outcome studies ( Khan and Butler, 2022 ), and the real-world database from China ( Wang et al, 2023 ).…”

Section: Discussionmentioning

confidence: 99%

Advancing perspectives on the off-label use of anticancer drugs: an updated classification and exploration of categories

Chen,

Ou,

Luo

et al. 2024

Front. Pharmacol.

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To date, the definition that the off-label usage of drugs refers to the unapproved use of approved drugs, which covers unapproved indications, patient populations, doses, and/or routes of administration, has been in existence for many years. Currently, there is a limited frequency and prevalence of research on the off-label use of antineoplastic drugs, mainly due to incomplete definition and classification issues. It is time to embrace new categories for the off-label usage of anticancer drugs. This review provided an insight into an updated overview of the concept and categories of the off-label use of anticancer drugs, along with illustrating specific examples to establish the next studies about the extent of the off-label usage of anticancer drugs in the oncology setting. The scope of the off-label use of current anticancer drugs beyond the previous definitions not only includes off-label uses in terms of indications, patient populations, doses, and/or routes of administration but also off-label use in terms of medication course, combination, sequence of medication, clinical purpose, contraindications scenarios, etc. In addition, the definition of the off-label usage of anticancer drugs should be added to the condition at a given time, and it varies from approval authorities. We presented a new and relatively comprehensive classification, providing extensive analysis and illustrative examples of the off-label usage of antineoplastic drugs for the first time. Such a classification has the potential to promote practical adoption and enhance management strategies for the off-label use of antitumor drugs.

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Section: Discussionmentioning

confidence: 99%

Advancing perspectives on the off-label use of anticancer drugs: an updated classification and exploration of categories

Chen,

Ou,

Luo

et al. 2024

Front. Pharmacol.

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“…RWE can inform clinical trial strategy and design in the research and development stage by generating hypotheses and predicting free analysis. In China, specific utilization involves describing disease epidemiology and treatment patterns, assessing comparative effectiveness, safety, disease progression, and healthcare resource utilization, and estimating treatment costs [ 26 ]. In the launch and post-launch stages, RWE can be combined with evidence from clinical trials through conducting hybrid studies in safety monitoring and pharmacovigilance to produce generalizable results for supporting regulatory applications.…”

Section: Rwe Enablement In China (Second Eac Meeting)mentioning

confidence: 99%

Maximizing the Value of Real-World Data and Real-World Evidence to Accelerate Healthcare Transformation in China: Summary of External Advisory Committee Meetings

Sun,

Bedenkov,

Liu

et al. 2024

Pharm Med

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Use of real-world data (RWD) is gaining wide attention. To bridge the gap between diverse healthcare stakeholders and to leverage the impact of Chinese real-world evidence (RWE) globally, a multi-stakeholder External Advisory Committee (EAC) and EAC meetings were initiated, aiming to elucidate the current and evolving RWD landscape in China, articulate the values of RWE in ensuring Chinese patients’ equitable access to affordable medicines and solutions, and identify strategic opportunities and partnerships for expansion of RWE generation in China. Chinese and international experts who are clinicians and academic researchers were selected as EAC members based on their professional background and familiarity with RWD/RWE. Three EAC meetings were held quarterly in 2023. Various topics were presented and discussed for insights and suggestions. Nine experts from China, one from South Korea, and two from Europe were selected as EAC members and attended these meetings. Experts’ presentations were summarized by theme, including the RWD landscape and RWE enablement in China, as well as global development of a patient-centric ecosystem. Experts’ insights and suggestions on maximizing the RWD/RWE value to accelerate healthcare transformation in China were collected. We concluded that though data access, sharing, and quality are still challenging, RWD is developing to support evidence generation in the medicinal product lifecycle, inform clinical practice, and empower patient management in China. RWD/RWE creates value, accelerates healthcare transformation, and improves patient outcomes. Fostering a patient-centric ecosystem across healthcare stakeholders and maintaining global partnerships and collaboration are essential for unlocking the power of RWD/RWE. Supplementary Information The online version contains supplementary material available at 10.1007/s40290-024-00520-3.

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“…China is still in the initial stage of using RWE to support the development and evaluation of pharmaceutical products, and the relevant guidelines issued thus far are all trial documents. While the framework design and technical guidelines from the FDA and EMA have been valuable references for China, 16–20 it is important to acknowledge that the foreign experience may not be fully applicable to the Chinese healthcare data environment due to variations in data quality, limited data sharing, etc. There is an urgent need for regulatory agencies to improve relevant policies and regulations, and for experts to develop relevant technical guidelines to support them.…”

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confidence: 99%

The Use of Real‐World Evidence for Regulatory Decisions in China

Xu,

Wu,

Zhang

et al. 2024

Clin Pharma and Therapeutics

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There is a growing demand for the use of high‐quality real‐world evidence (RWE) to support regulatory decision‐making worldwide and in China, which highlights the need for conducting literature reviews to evaluate the available data and evidence. This study aims to review the use of RWE in Chinese regulatory decisions and to summarize relevant regulatory and methodological considerations to inform the future use of RWE in China. We identified policy documents, technical guidance documents, and cases on official Chinese government websites and extracted their contents separately. We consulted experts from the National Medical Products Administration (NMPA) and academic institutes and searched case‐related articles for enrichment. We also searched and included articles related to the use of RWE/Real‐world data in Chinese regulatory decisions. Six trial versions of technical guidance documents, 7 case studies, and 40 articles related to the Chinese regulatory decisions were included in this study. Based on the technical guidance, data quality, and appropriate study design and statistical analysis are the main concerns for RWE generation. The cases and articles related to regulatory decisions revealed 9 main concerns, including data sources and applicability, data quality, strength of existing evidence, appropriate study design and statistical analysis, regulated and transparent process for analysis and evidence generation, product safety and efficacy, product characteristics and clinical needs, ethical considerations and data security, and communicate adequately with regulatory authorities. Among these concerns, data issues are central. Preliminary attempts have been made by the NMPA to promote the use of RWE, but substantial challenges still remain.

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Use of real world data to improve drug coverage decisions in China

Abstract: Wen Wang and colleagues discuss the rationale and propose a framework for using real world evidence to support coverage decisions in Chinese setting

Cited by 5 publications

References 30 publications

Advancing perspectives on the off-label use of anticancer drugs: an updated classification and exploration of categories

Advancing perspectives on the off-label use of anticancer drugs: an updated classification and exploration of categories

Maximizing the Value of Real-World Data and Real-World Evidence to Accelerate Healthcare Transformation in China: Summary of External Advisory Committee Meetings

The Use of Real‐World Evidence for Regulatory Decisions in China

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