title: The identification of benefit in medical intervention: an overview and suggestions for process.
abstract: Benefit and risk assessments are not only important to regulatory authorities but also important to the providers, patients, pharmaceutical industry, and payers. In order for patients and providers to continue to have access to new innovative medicines, which have some level of inherent risk, it is critical to have a systematic and balanced focus on understanding the safety risks and benefits to the patient during drug development, at the time of approval and postmarketing. There has been a significant amount of activity around efforts to improve the ability to assess risks in the postmarketing environment. However, there is no widely accepted, systematic approach or process for the ongoing evaluation of benefit. This article introduces 4 critical components in the process of identifying and assessing benefit with a goal of providing a framework that is transparent, comprehensive, applicable to various perspectives, and simple to communicate and implement. We propose the development of a catalog applied to a particular disease to identify the optimal data sources and methods to address the interests of a given perspective. Two key resources will need to be developed to support the catalog development: (1) a summary of benefit measures and preferences by disease and from various perspectives and (2) an investment in a simple visual communication mechanism with minimal statistical language. As the emphasis is on transparency, relevance, applicability, and communication, this approach to assessing benefit should maximize the impact of these data to all stakeholders and decision makers.
authors.affiliation: Research and Development, Johnson & Johnson Pharmaceuticals, Titusville, NJ, USA.
authors.forename: Paul E
authors.initials: PE
authors.lastname: Stang
authors.forename: Sissi V
authors.initials: SV
authors.lastname: Pham
authors.forename: Kraig
authors.initials: K
authors.lastname: Kinchen
authors.forename: Sandra B
authors.initials: SB
authors.lastname: Raff
authors.forename: Filip
authors.initials: F
authors.lastname: Mussen
authors.forename: Kathleen
authors.initials: K
authors.lastname: Gondek
mesh_terms: D002986:Clinical Trials as Topic / Q000706:statistics & numerical data; D003142:Communication; D003625:Data Collection / Q000379:methods; D017277:Drug Approval / Q000458:organization & administration / Q000706:statistics & numerical data*; D004353:Drug Evaluation, Preclinical / Q000706:statistics & numerical data; D004345:Drug Industry / Q000706:statistics & numerical data*; D006801:Humans; D011358:Product Surveillance, Postmarketing / Q000706:statistics & numerical data; D018570:Risk Assessment; D014481:United States; D014486:United States Food and Drug Administration