Applying real-world data from expanded-access (“compassionate use”) patients to drug development

Wasser, June S.; Greenblatt, David J.

doi:10.1017/cts.2023.606

Cited by 3 publications

(2 citation statements)

References 22 publications

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“…We note that this approach, which applies clinical trial methods to individual patient care, is adjacent to data collection as done in the context of the Compassionate Use of medications that are not yet approved for a given condition. Increasingly, RWD are collected for such cases, often rare or dire conditions, and yet there are intrinsic biases in this approach [ 23 , 24 ]. The selection of patients for such compassionate use will potentially depend on the patient’s access to specialized care.…”

Section: Discussionmentioning

confidence: 99%

The use of N-of-1 trials to generate real-world evidence for optimal treatment of individuals and populations

Selker,

Dulko,

Greenblatt

et al. 2023

J. Clin. Trans. Sci.

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Introduction: Ideally, real-world data (RWD) collected to generate real-world evidence (RWE) should lead to impact on the care and health of real-world patients. Deriving from care in which clinicians and patients try various treatments to inform therapeutic decisions, N-of-1 trials bring scientific methods to real-world practice. Methods: These single-patient crossover trials generate RWD and RWE by giving individual patients various treatments in a double-blinded way in sequential periods to determine the most effective treatment for a given patient. Results: This approach is most often used for patients with chronic, relatively stable conditions that provide the opportunity to make comparisons over multiple treatment periods, termed Type 1 N-of-1 trials. These are most helpful when there is heterogeneity of treatment effects among patients and no a priori best option. N-of-1 trials also can be done for patients with rare diseases, potentially testing only one treatment, to generate evidence for personalized treatment decisions, designated as Type 2 N-of-1 trials. With both types, in addition to informing individual’s treatments, when uniform protocols are used for multiple patients with the same condition, the data collected in the individual N-of-1 trials can be aggregated to provide RWD/RWE to inform more general use of the treatments. Thereby, N-of-1 trials can provide RWE for the care of individuals and for populations. Conclusions: To fulfill this potential, we believe N-of-1 trials should be built into our current healthcare ecosystem. To this end, we are building the needed infrastructure and engaging the stakeholders who should receive value from this approach.

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Section: Discussionmentioning

confidence: 99%

The use of N-of-1 trials to generate real-world evidence for optimal treatment of individuals and populations

Selker,

Dulko,

Greenblatt

et al. 2023

J. Clin. Trans. Sci.

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“…Use of RWD to inform trial designs and generate RWE to evaluate safety and efficacy [1][2][3][4][5][6][7][8][9][10][11][12] Currently, most of the patient-derived RWD used in biomedical research resides in Electronic Health Records (EHRs). Even though their development was not intended for research purposes, the large volume of information, data richness, continuum of care data collection, and ability to be linked and integrated with additional data from medical encounters such as claims data, imaging, and laboratory information can provide a more comprehensive view of an individual and their health journey.…”

Section: Background and Overarching Categories For Thematic Issuementioning

confidence: 99%

Advancing regulatory science through real-world data and real-world evidence

Cure,

Fessel,

Hartshorn

et al. 2024

J. Clin. Trans. Sci.

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Data Security and Legal Issues in the Application of Medical Big Data

Du,

Zhao,

Wei

et al. 2024

Advances in Medical Technologies and Clinical Practice

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This chapter systematically explores the challenges of data security and legal issues in the application of medical big data. As the application of medical big data expands from clinical diagnostics to drug development, it holds vast potential for growth. However, numerous issues regarding data security and legal ethics also arise. The chapter begins with an overview of the current state of medical big data applications and introduces their applications across various medical fields. It then delves into the various challenges faced in data security. In terms of law and ethics, the chapter discusses the conflict between data ownership and data sharing, introduces international legal frameworks, and examines issues related to artificial intelligence and ethics. Through detailed case studies, this chapter analyzes medical data breach incidents and summarizes the impact of these events and their common security challenges. The intention of this chapter is to guide the secure and lawful use of medical big data while protecting patient privacy and data integrity.

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Applying real-world data from expanded-access (“compassionate use”) patients to drug development

Abstract: Research Methods and Technology Special CommunicationCite this article: Wasser JS and Greenblatt DJ. Applying real-world data from expandedaccess ("compassionate use") patients to drug development.

Cited by 3 publications

References 22 publications

The use of N-of-1 trials to generate real-world evidence for optimal treatment of individuals and populations

The use of N-of-1 trials to generate real-world evidence for optimal treatment of individuals and populations

Advancing regulatory science through real-world data and real-world evidence

Data Security and Legal Issues in the Application of Medical Big Data

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