Structured Qualitative Benefit-Risk Assessment to Inform Go/No-Go Decisions for Phase III Testing of Pharmaceutical Prod
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LEADING ARTICLE
Structured Qualitative Benefit-Risk Assessment to Inform Go/NoGo Decisions for Phase III Testing of Pharmaceutical Products Anne Wolka1 • Christine Radawski1 • Rebecca Noel1
Published online: 13 January 2016 Springer International Publishing Switzerland 2016
Abstract Late-stage attrition of pharmaceutical products has downsides for both patients participating in clinical trials and sponsor companies. This manuscript presents an outline for a structured qualitative benefit-risk assessment process that sponsors can apply at the time of go/no-go decisions for phase III testing of pharmaceutical products. This approach takes into account the therapeutic and comparator context as well as product-specific information on key benefits, key risks, and risk management. The strengths, limitations, and uncertainties of the evidence are critically evaluated at each step to provide a balanced perspective on the data. Application of a rigorous, structured qualitative benefit-risk assessment to critically evaluate the available information at the time of these decisions lends further support to quality decision making by sponsors. Such an approach ensures that decisions are systematic and transparent and helps development teams communicate the rationale for their recommendations to decision makers. In a succinct organized fashion, this approach provides decision makers with the clinical information they need to ascertain whether pharmaceutical products should be advanced from phase II to phase III testing.
& Anne Wolka [email protected] 1
Eli Lilly and Company, Lilly Corporate Center, 893 S Delaware St., Indianapolis, IN 46285, USA
Key Points Structured qualitative benefit-risk assessment is a rigorous data-driven approach to making go/no-go decisions for phase III testing of pharmaceutical products. The structured qualitative benefit-risk assessment process takes into account information about the pharmaceutical product in addition to the decision context, while considering the strengths, limitations, and uncertainties of evidence at each step to provide a balanced perspective on the data. This approach could play a role in mitigating the risk of late-stage attrition of pharmaceutical products owing to clinical considerations, allowing decision making to be more systematic, consistent, and transparent, with potential benefits for both patients and sponsor companies.
1 Introduction Recent studies have shown that, of pharmaceutical products in phase III testing, approximately 40 % do not reach regulatory submission; furthermore, approximately 10–20 % of pharmaceutical products that are submitted do not receive regulatory approval [1, 2]. Reasons for such attrition can include efficacy, safety, and/or a company decision not to file (often influenced by regulatory uncertainty) [2]. Late-stage attrition of pharmaceutical products has downsides for both patients participating in clinical trials and sponsor companies. Higher failure rates for drugs
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tested in humans is one of the main drivers of rising costs
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