Strategies to Address Current Challenges in Real-World Evidence Generation in Japan

Laurent, Thomas; Lambrelli, Dimitra; Wakabayashi, Ryozo; Hirano, Takahiro; Kuwatsuru, Ryohei

doi:10.1007/s40801-023-00371-5

Cited by 4 publications

(1 citation statement)

References 69 publications

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“…Nonetheless, there are not yet many examples of the real application of PPs in the real-world setting, according to the literature. This limited incorporation of PP might be due to PP’s methodological challenges related to capturing and using real-world data for decision-making (limited access to databases and registries, data quality, complex analytical methodologies and confounding, for example) ( 74 ).…”

Section: Patient Experience Data Use In Medicines’ Life Cyclementioning

confidence: 99%

Leveraging patient experience data to guide medicines development, regulation, access decisions and clinical care in the EU

Almeida,

Umuhire,

Gonzalez-Quevedo

et al. 2024

Front. Med.

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Patient experience data (PED), provided by patients/their carers without interpretation by clinicians, directly capture what matters more to patients on their medical condition, treatment and impact of healthcare. PED can be collected through different methodologies and these need to be robust and validated for its intended use. Medicine regulators are increasingly encouraging stakeholders to generate, collect and submit PED to support both scientific advice in development programs and regulatory decisions on the approval and use of these medicines. This article reviews the existing definitions and types of PED and demonstrate the potential for use in different settings of medicines’ life cycle, focusing on Patient-Reported Outcomes (PRO) and Patient Preferences (PP). Furthermore, it addresses some challenges and opportunities, alluding to important regulatory guidance that has been published, methodological aspects and digitalization, highlighting the lack of guidance as a key hurdle to achieve more systematic inclusion of PED in regulatory submissions. In addition, the article discusses opportunities at European and global level that could be implemented to leverage PED use. New digital tools that allow patients to collect PED in real time could also contribute to these advances, but it is equally important not to overlook the challenges they entail. The numerous and relevant initiatives being developed by various stakeholders in this field, including regulators, show their confidence in PED’s value and create an ideal moment to address challenges and consolidate PED use across medicines’ life cycle.

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Section: Patient Experience Data Use In Medicines’ Life Cyclementioning

confidence: 99%

Leveraging patient experience data to guide medicines development, regulation, access decisions and clinical care in the EU

Almeida,

Umuhire,

Gonzalez-Quevedo

et al. 2024

Front. Med.

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The emerging role of real-world data in oncology care in Japan

Bando,

Tajima,

Aoyagi

et al. 2023

ESMO Real World Data and Digital Oncology

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Transparency in the secondary use of health data: Assessing the status quo of guidance and best practices

R. van den Akker,

Thibault,

Ioannidis

et al. 2024

Preprint

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We evaluated what guidance exists in the literature to improve the transparency of studies that make secondary use of health data. To find relevant literature, we searched PubMed and Google Scholar and drafted a list of health organizations based on our personal expertise. We quantitatively and qualitatively coded different types of research transparency: registration, methods reporting, results reporting, data sharing, and code sharing. We found 54 documents that provide recommendations to improve the transparency of studies making secondary use of health data, mainly in relation to study registration (n = 27) and methods reporting (n = 39). Only three documents made recommendations on data sharing or code sharing. Recommendations for study registration and methods reporting mainly came in the form of structured documents like registration templates and reporting guidelines. Aside from the recommendations aimed directly at researchers, we found 31 recommendations aimed at the wider research community, typically on how to improve research infrastructure. Limitations or challenges of improving transparency were rarely mentioned, highlighting the need for more nuance in providing transparency guidance for studies that make secondary use of health data.

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Strategies to Address Current Challenges in Real-World Evidence Generation in Japan

Cited by 4 publications

References 69 publications

Leveraging patient experience data to guide medicines development, regulation, access decisions and clinical care in the EU

Leveraging patient experience data to guide medicines development, regulation, access decisions and clinical care in the EU

The emerging role of real-world data in oncology care in Japan

Transparency in the secondary use of health data: Assessing the status quo of guidance and best practices

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