COLUMNS

2018 ◽  
Vol 22 (08) ◽  
pp. 14-29
Keyword(s):  
Mental Illness ◽  
Real World ◽  
New Drugs ◽  
Real World Evidence ◽  
The Future

The future of pharma: Digital, personalized, and dynamic. Using real world evidence in the development of new medicine. Hunting for new drugs to treat mental illness. Clean cow starts now.

Real-world evidence for U.S. Food and Drug Administration-approved oncology products, 2015 to 2020.

2020 ◽  
Vol 38 (29_suppl) ◽  
pp. 280-280
Author(s):  
Bhakti Arondekar ◽  
Mei Sheng Duh ◽  
Rachel Bhak ◽  
Maral DerSarkissian ◽  
Lynn Huynh ◽  
...  
Keyword(s):  
Drug Administration ◽  
Real World ◽  
Food And Drug Administration ◽  
A Priori ◽  
New Drugs ◽  
Pivotal Trial ◽  
Real World Evidence ◽  
Data Source ◽  
Source Study ◽  
Clinical Trial Results

280 Background: Since the 21st Century Cures Act, there has been growing interest in using real-world evidence (RWE) to support regulatory filings for new drugs and indications. The goal of this study was to provide a comprehensive source of RWE use cases of U.S. Food and Drug Administration (FDA) approvals for oncology products. Methods: A systematic review of FDA new drug applications (NDAs) and biologics license applications (BLAs) from 1/2015-2/2020 for approved oncology products was performed. Data on the RWE study were extracted (data source, study design, statistical methods, results), and corresponding FDA comments were synthesized to identify patterns of the FDA’s review. Results: We identified 102 NDAs and BLAs, 8 (8%) of which included RWE, all post-Cures Act (see Table). RWE supporting avelumab, axicabtagene ciloleucel, and avapritinib were received positively and used by the FDA in their approval decision. RWE results were used to provide contextualization to the pivotal trial, rather than statistical comparison. Common data sources included Flatiron (38%) and chart reviews (38%). FDA critiques included lack of a priori study protocol, incomparability with the pivotal trial population and endpoints, and uncontrolled confounding. Conclusions: There have been few examples of RWE in oncology submissions, and most served to complement clinical trial results. To meet FDA standards, RWE studies should be clearly designed and discussed with the FDA and include robust methods to minimize bias. [Table: see text]

scholarly journals Developing a framework to incorporate real-world evidence in cancer drug funding decisions: the Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration

BMJ Open ◽  
2020 ◽  
Vol 10 (1) ◽  
pp. e032884 ◽  
Author(s):  
Kelvin Chan ◽  
Seungree Nam ◽  
Bill Evans ◽  
Claire de Oliveira ◽  
Alexandra Chambers ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Healthcare System ◽  
Real World ◽  
Decision Makers ◽  
New Drugs ◽  
Cancer Drug ◽  
Cancer Drugs ◽  
Value For Money ◽  
Real World Evidence ◽  
Clinical Benefits

BackgroundOncology therapy is becoming increasingly more expensive and challenging the affordability and sustainability of drug programmes around the world. When new drugs are evaluated, health technology assessment organisations rely on clinical trials to inform funding decisions. However, clinical trials are not able to assess overall survival and generalises evidence in a real-world setting. As a result, policy makers have little information on whether drug funding decisions based on clinical trials ultimately yield the outcomes and value for money that might be expected.ObjectiveThe Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration, consisting of researchers, recommendation-makers, decision makers, payers, patients and caregivers, are developing and testing a framework for Canadian provinces to generate and use real-world evidence (RWE) for cancer drug funding in a consistent and integrated manner.StrategyThe CanREValue collaboration has established five formal working groups (WGs) to focus on specific processes in the generation and use of RWE for cancer drug funding decisions in Canada. The different RWE WGs are: (1) Planning and Drug Selection; (2) Methods; (3) Data; (4) Reassessment and Uptake; (5) Engagement. These WGs are acting collaboratively to develop a framework for RWE evaluation, validate the framework through the multiprovince RWE projects and help to integrate the final RWE framework into the Canadian healthcare system.OutcomesThe framework will enable the reassessment of cancer drugs, refinement of funding recommendations and use of novel funding mechanisms by decision-makers/payers across Canada to ensure the healthcare system is providing clinical benefits and value for money.

The Future of Registries in the Era of Real-world Evidence for Medical Devices

2019 ◽  
Vol 4 (3) ◽  
pp. 197 ◽  
Author(s):  
Rachael L. Fleurence ◽  
Kathleen Blake ◽  
Jeffrey Shuren
Keyword(s):  
Medical Devices ◽  
Real World ◽  
Real World Evidence ◽  
The Future

1243-P: Novel Use of Natural Language Processing to Identify Reasons for Insulin Discontinuation in Patients with T2DM: A Real-World Evidence Study

Diabetes ◽  
2019 ◽  
Vol 68 (Supplement 1) ◽  
pp. 1243-P
Author(s):  
JIANMIN WU ◽  
FRITHA J. MORRISON ◽  
ZHENXIANG ZHAO ◽  
XUANYAO HE ◽  
MARIA SHUBINA ◽  
...  
Keyword(s):  
Natural Language Processing ◽  
Natural Language ◽  
Language Processing ◽  
Real World ◽  
Real World Evidence

381-P: Real-World Evidence that Impaired Awareness of Hypoglycemia Increases Severe Hypoglycemia Rates in T2DM (InHypo-DM Study)

Diabetes ◽  
2019 ◽  
Vol 68 (Supplement 1) ◽  
pp. 381-P
Author(s):  
ALEXANDRIA RATZKI-LEEWING ◽  
STEWART B. HARRIS ◽  
NATALIE H. AU ◽  
SUSAN WEBSTER-BOGAERT ◽  
JUDITH B. BROWN ◽  
...  
Keyword(s):  
Real World ◽  
Severe Hypoglycemia ◽  
Real World Evidence ◽  
Impaired Awareness
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