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The utility of real‐world evidence for benefit‐risk assessment, communication, and evaluation of pharmaceuticals: Case studies
Author(s) -
Radawski Christine A.,
Hammad Tarek A.,
Colilla Susan,
Coplan Paul,
Hornbuckle Kenneth,
Freeman Emily,
Smith Meredith Y.,
Sobel Rachel E.,
Bahri Priya,
Arias Ariel E.,
Bennett Dimitri
Publication year - 2020
Publication title -
pharmacoepidemiology and drug safety
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.023
H-Index - 96
eISSN - 1099-1557
pISSN - 1053-8569
DOI - 10.1002/pds.5167
Subject(s) - medicine , stakeholder , stakeholder engagement , risk analysis (engineering) , data collection , product (mathematics) , brace , reimbursement , risk assessment , health care , operations management , engineering , computer science , public relations , computer security , statistics , mathematics , geometry , economics , economic growth , mechanical engineering , political science
Purpose In recent years, novel types of real‐world evidence (RWE) have played a role in various decision‐making processes relating to medicinal products, including regulatory approval, patient access, health technology assessment, safety monitoring, clinical use, and post‐approval lifecycle management. We therefore reviewed the potential utility of RWE in the cycle of medicinal product benefit‐risk (BR) assessment, communication/risk minimization and evaluation (“BRACE”). Methods A convenience sample of illustrative studies was drawn from the published literature and examined. Specifically, we examined the purpose for using RWE, the type of RWE used, its novelty and how it might be integrated with other data and activities of the BRACE cycle, and how it contributed to regulatory decision‐making. Results Eight studies were selected with each illustrating a different activity in the BRACE cycle ranging from BR assessment in the preapproval setting, post‐approval assessment of safety or effectiveness, communicating BR information to patients and healthcare professionals, and evaluating the effectiveness of risk minimization initiatives to support a positive BR balance. Conclusions RWE has an important role in informing regulatory decision‐making regarding the BR management of medicines. With increasing digitalization, facilitating data collection and stakeholder engagement in health, this role is only expected to expand in the future. To reach the full potential of RWE, both regulators and sponsors will need to be familiar with a range of existing and emerging methods for generating and analyzing such evidence appropriately and achieve convergence regarding how different types of RWE can best be used to inform BR management and decision‐making.