Bringing in health technology assessment and cost‐effectiveness considerations at an early stage of drug development. Issue 5 (23rd October 2014)

• Record Type: Journal Article
• Authors: Jönsson, Bengt
• Other Names: Mendelsohn John specialEditor.; Ringborg Ulrik specialEditor.; Schilsky Richard specialEditor.
• Is Part Of: Molecular oncology. Volume 9:Issue 5(2015:May)
• Page Start: 1025
• Page End: 1033
• Publication Date: 2014-10-23
• Subjects: Health technology assessment -- Cost-effectiveness -- Clinical trials -- Outcome research -- Early development -- Relative effectiveness; Cancer -- Molecular aspects -- Periodicals; 616.994005
• DOI: 10.1016/j.molonc.2014.10.009
• Languages: English
• ISSNs: 1574-7891

Abstract

Abstract : This paper reviews the issues involved in undertaking HTA studies early in the development of new cancer therapies, and discusses the data and methods for estimating the cost‐effectiveness of new diagnostics and treatments. The value for patients of new cancer therapies is based on access to the treatment and optimal use. Realising potential value depends on successful completion of a series of steps, from the initial economic evaluations based on clinical trial data, to the reimbursement decisions based on the evaluations and the implementation of these decisions in clinical practice. Considerable resources have been devoted to the study of the cost‐effectiveness of new cancer drugs as a basis for decisions about payment and use. Such resources could be used much more effectively if industry and HTA agencies were to collaborate at an early stage in the development process. The traditional clinical trial approach of using progression‐free survival and cross‐overs has serious shortcomings, producing data that cannot be used to determine outcomes and, so, cost‐effectiveness. A new standard is needed; both regulatory and HTA authorities should be involved in its development. Highlights: Aspects of drug development create particular challenges for early HTA of cancer drugs. Technology assessments require estimates of gains in mean survival, whereas trials are designed to study differences in progression‐free or overall median survival. The use of biomarkers to identify the relevant patient population can help create a close link between the population in the clinical trial and the use of the drug once it reaches the market. HTA studies need to be adapted to fit the requirements of different decision makers. Early modelling of cost‐effectiveness improve the chance of selecting high value development strategies.