The utility of real‐world evidence for benefit‐risk assessment, communication, and evaluation of pharmaceuticals: Case studies

2020 ◽  
Vol 29 (12) ◽  
pp. 1532-1539
Author(s):  
Christine A. Radawski ◽  
Tarek A. Hammad ◽  
Susan Colilla ◽  
Paul Coplan ◽  
Kenneth Hornbuckle ◽  
...  
Keyword(s):  
Risk Assessment ◽  
Case Studies ◽  
Real World ◽  
Real World Evidence ◽  
Benefit Risk Assessment

scholarly journals Connected health for growth hormone treatment research and clinical practice: learnings from different sources of real-world evidence (RWE)—large electronically collected datasets, surveillance studies and individual patients’ cases

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Nea Boman ◽  
Luis Fernandez-Luque ◽  
Ekaterina Koledova ◽  
Marketta Kause ◽  
Risto Lapatto
Keyword(s):  
Growth Hormone ◽  
Observational Study ◽  
Case Studies ◽  
Real World ◽  
Growth Disorders ◽  
Injection Device ◽  
Growth Outcomes ◽  
Real World Evidence ◽  
High Adherence

Abstract Background A range of factors can reduce the effectiveness of treatment prescribed for the long-term management of chronic health conditions, such as growth disorders. In particular, prescription medications may not achieve the positive outcomes expected because approximately half of patients adhere poorly to the prescribed treatment regimen. Methods Adherence to treatment has previously been assessed using relatively unreliable subjective methods, such as patient self-reporting during clinical follow-up, or counting prescriptions filled or vials returned by patients. Here, we report on a new approach, the use of electronically recorded objective evidence of date, time, and dose taken which was obtained through a comprehensive eHealth ecosystem, based around the easypod™ electromechanical auto-injection device and web-based connect software. The benefits of this eHealth approach are also illustrated here by two case studies, selected from the Finnish cohort of the easypod™ Connect Observational Study (ECOS), a 5-year, open-label, observational study that enrolled children from 24 countries who were being treated with growth hormone (GH) via the auto-injection device. Results Analyses of data from 9314 records from the easypod™ connect database showed that, at each time point studied, a significantly greater proportion of female patients had high adherence (≥ 85%) than male patients (2849/3867 [74%] vs 3879/5447 [71%]; P < 0.001). Furthermore, more of the younger patients (< 10 years for girls, < 12 years for boys) were in the high adherence range (P < 0.001). However, recursive partitioning of data from ECOS identified subgroups with lower adherence to GH treatment ‒ children who performed the majority of injections themselves at an early age (~ 8 years) and teenagers starting treatment aged ≥ 14 years. Conclusions The data and case studies presented herein illustrate the importance of adherence to GH therapy and how good growth outcomes can be achieved by following treatment as described. They also show how the device, software, and database ecosystem can complement normal clinical follow-up by providing HCPs with reliable information about patient adherence between visits and also providing researchers with real-world evidence of adherence and growth outcomes across a large population of patients with growth disorders treated with GH via the easypod™ device.

Application of the BRAFO tiered approach for benefit–risk assessment to case studies on dietary interventions

2012 ◽  
Vol 50 ◽  
pp. S710-S723 ◽  
Author(s):  
Hans Verhagen ◽  
Rikke Andersen ◽  
Jean-Michel Antoine ◽  
Paul Finglas ◽  
Jeljer Hoekstra ◽  
...  
Keyword(s):  
Risk Assessment ◽  
Case Studies ◽  
Dietary Interventions ◽  
Tiered Approach ◽  
Benefit Risk Assessment

Application of the BRAFO-tiered approach for benefit-risk assessment to case studies on natural foods

2012 ◽  
Vol 50 ◽  
pp. S699-S709 ◽  
Author(s):  
Bernhard Watzl ◽  
Eva Gelencsér ◽  
Jeljer Hoekstra ◽  
Sabine Kulling ◽  
Eva Lydeking-Olsen ◽  
...  
Keyword(s):  
Risk Assessment ◽  
Case Studies ◽  
Tiered Approach ◽  
Benefit Risk Assessment ◽  
Natural Foods

Application of the BRAFO tiered approach for benefit–risk assessment to case studies on heat processing contaminants

2012 ◽  
Vol 50 ◽  
pp. S724-S735 ◽  
Author(s):  
Katrin Schütte ◽  
Heiner Boeing ◽  
Andy Hart ◽  
Walther Heeschen ◽  
Ernst H. Reimerdes ◽  
...  
Keyword(s):  
Risk Assessment ◽  
Case Studies ◽  
Heat Processing ◽  
Tiered Approach ◽  
Benefit Risk Assessment

Real-world experiences with generating real-world evidence: Case Studies from PCORI's pragmatic clinical Studies program

2020 ◽  
Vol 98 ◽  
pp. 106171
Author(s):  
Mark D. Neuman ◽  
Michael D. Kappelman ◽  
Elliot Israel ◽  
Susan S. Ellenberg ◽  
Cindy Girman ◽  
...  
Keyword(s):  
Case Studies ◽  
Real World ◽  
Clinical Studies ◽  
Real World Evidence

scholarly journals Real-World Evidence in Healthcare Decision Making: Global Trends and Case Studies From Latin America

2019 ◽  
Vol 22 (6) ◽  
pp. 739-749 ◽  
Author(s):  
Nahila Justo ◽  
Manuel A. Espinoza ◽  
Barbara Ratto ◽  
Martha Nicholson ◽  
Diego Rosselli ◽  
...  
Keyword(s):  
Decision Making ◽  
Latin America ◽  
Case Studies ◽  
Real World ◽  
Healthcare Decision ◽  
Global Trends ◽  
Healthcare Decision Making ◽  
Real World Evidence
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