scholarly journals Report on the current status of the use of real‐world data (RWD) and real‐world evidence (RWE) in drug development and regulation

2019 ◽  
Vol 85 (9) ◽  
pp. 1874-1877 ◽  
Author(s):  
Alasdair M. Breckenridge ◽  
Ross A. Breckenridge ◽  
Carl C. Peck
Keyword(s):  
Drug Development ◽  
Real World ◽  
Current Status ◽  
Real World Data ◽  
World Data ◽  
Real World Evidence

Current Status and Examples of US FDA or EMA’s Regulatory Decision using Real World Data/Real World Evidence

Yakhak Hoeji ◽  
2020 ◽  
Vol 64 (2) ◽  
pp. 136-155
Author(s):  
Hi Gin Sung ◽  
Han-Heui Park ◽  
Gyu-Won Jung ◽  
Ju-Young Shin
Keyword(s):  
Real World ◽  
Current Status ◽  
Real World Data ◽  
World Data ◽  
Regulatory Decision ◽  
Real World Evidence ◽  
Us Fda

scholarly journals Development of an Innovative Real-World Evidence Registry for the Herpes Simplex Virus: Case Study

10.2196/16933 ◽  
2020 ◽  
Vol 3 (1) ◽  
pp. e16933 ◽  
Author(s):  
Michelle Helena van Velthoven ◽  
Ching Lam ◽  
Caroline de Cock ◽  
Terese Stenfors ◽  
Hassan Chaudhury ◽  
...  
Keyword(s):  
Herpes Simplex Virus ◽  
Herpes Simplex ◽  
Real World ◽  
Physical Symptoms ◽  
Use Cases ◽  
Real World Data ◽  
World Data ◽  
Real World Evidence ◽  
Simplex Virus

Background Infection with the herpes simplex virus (HSV) is common but not well understood. Furthermore, there remains a social stigma surrounding HSV that can have psychosocial implications for those infected. Despite many patients infected with HSV experiencing mild-to-severe physical symptoms, only one subeffective treatment is available. A registry collecting real-world data reported by individuals potentially infected with HSV could help patients to better understand and manage their condition. Objective This study aimed to report on the development of a registry to collect real-world data reported by people who might be infected with HSV. Methods A case study design was selected as it provides a systematic and in-depth approach to investigating the planning phase of the registry. The case study followed seven stages: plan, design, prepare, collect, analyze, create, and share. We carried out semistructured interviews with experts, which were thematically analyzed and used to build use cases for the proposed registry. These use cases will be used to generate detailed models of how a real-world evidence registry might be perceived and used by different users. Results The following key themes were identified in the interviews: (1) stigma and anonymity, (2) selection bias, (3) understanding treatment and outcome gaps, (4) lifestyle factors, (5) individualized versus population-level data, and (6) severe complications of HSV. We developed use cases for different types of users of the registry, including individuals with HSV, members of the public, researchers, and clinicians. Conclusions This case study revealed key considerations and insights for the development of an appropriate registry to collect real-world data reported by people who might be infected with HSV. Further development and testing of the registry with different users is required. The registry must also be evaluated for the feasibility and effectiveness of collecting data to support symptom management. This registry has the potential to contribute to the development of vaccines and treatments and provide insights into the impact of HSV on other conditions.

Real-World Evidence and Real-World Data for Evaluating Drug Safety and Effectiveness

JAMA ◽  
2018 ◽  
Vol 320 (9) ◽  
pp. 867 ◽  
Author(s):  
Jacqueline Corrigan-Curay ◽  
Leonard Sacks ◽  
Janet Woodcock
Keyword(s):  
Drug Safety ◽  
Real World ◽  
Real World Data ◽  
World Data ◽  
Real World Evidence

scholarly journals Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development

2022 ◽  
Vol 11 ◽  
Author(s):  
Timothy A. Yap ◽  
Ira Jacobs ◽  
Elodie Baumfeld Andre ◽  
Lauren J. Lee ◽  
Darrin Beaupre ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Real World ◽  
External Control ◽  
Control Group ◽  
Precision Oncology ◽  
Efficacy And Safety ◽  
Control Groups ◽  
Real World Data ◽  
World Data

Randomized controlled trials (RCTs) that assess overall survival are considered the “gold standard” when evaluating the efficacy and safety of a new oncology intervention. However, single-arm trials that use surrogate endpoints (e.g., objective response rate or duration of response) to evaluate clinical benefit have become the basis for accelerated or breakthrough regulatory approval of precision oncology drugs for cases where the target and research populations are relatively small. Interpretation of efficacy in single-arm trials can be challenging because such studies lack a standard-of-care comparator arm. Although an external control group can be based on data from other clinical trials, using an external control group based on data collected outside of a trial may not only offer an alternative to both RCTs and uncontrolled single-arm trials, but it may also help improve decision-making by study sponsors or regulatory authorities. Hence, leveraging real-world data (RWD) to construct external control arms in clinical trials that investigate the efficacy and safety of drug interventions in oncology has become a topic of interest. Herein, we review the benefits and challenges associated with the use of RWD to construct external control groups, and the relevance of RWD to early oncology drug development.

scholarly journals 3. Utilizing Real World Data in Drug Development　―Expectations from Pharmaceutical Companies―

2019 ◽  
Vol 24 (1) ◽  
pp. 19-30 ◽  
Author(s):  
Kanae TOGO ◽  
Shinya KAWAMATSU ◽  
Ryo KIGUCHI ◽  
Yasuhiko IMAI
Keyword(s):  
Drug Development ◽  
Real World ◽  
Pharmaceutical Companies ◽  
Real World Data ◽  
World Data

scholarly journals Mobile apps for real-world evidence in health care

2020 ◽  
Vol 27 (6) ◽  
pp. 976-980
Author(s):  
Madison Milne-Ives ◽  
Michelle Helena van Velthoven ◽  
Edward Meinert
Keyword(s):  
Health Care ◽  
Mobile Health ◽  
Real World ◽  
Patient Empowerment ◽  
Real World Data ◽  
Privacy And Security ◽  
World Data ◽  
Health Apps ◽  
Real World Evidence ◽  
Mobile Health Apps

Abstract The use of real-world evidence for health care research and evaluation is growing. Mobile health apps have often-overlooked potential to contribute valuable real-world data that are not captured by other sources and could provide data that are more cost-effective and generalizable than can randomized controlled trials. However, there are several challenges that must be overcome to realize the potential value of patient-used mobile health app real-world data, including data quality, motivation for long-term use, privacy and security, methods of analysis, and standardization and integration. Addressing these challenges will increase the value of data from mobile health apps to inform real-world evidence and improve patient empowerment, clinical management, disease research, and treatment development.

Sign in / Sign up

Export Citation Format

Share Document