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S.R. Hill



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    MS25 - Translating Research into Practice (Applied Statistics) (ID 42)

    • Event: WCLC 2013
    • Type: Mini Symposia
    • Track: Statistics
    • Presentations: 1
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      MS25.4 - Assessing New Treatments for Lung Cancer - Regulatory and Cost-Effectiveness Implications (ID 582)

      15:05 - 15:25  |  Author(s): S.R. Hill

      • Abstract
      • Presentation
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      Abstract
      Over the last several years, there have been a number of new classes of drugs approved for the management of lung cancer. Regulatory pathways are evolving in many ways – for example, to recognize that targeted therapies, such as the EGFR inhibitors, will require evaluation of the drug alongside a diagnostic test; or that the increasing specificity of targets may result in smaller and shorter trials with different endpoints; or that regulatory authorities might identify potential significant advances in therapy by the use of special evaluation pathways, such as the ‘breakthrough’ therapy designation from the US FDA. Sometimes the trials now also measure quality of life outcomes or patient preferences. But these trials are still fundamentally designed to ask the ‘regulatory’ question – can the new product work – as well as assessing the risk-benefit ratio. But where does this changing regulatory environment leave payers? The information set that is available to guide assessments of value of money generally seems to becomes more limited as the regulators increase the pace of their decision-making. Trials are truncated or treatment groups are crossed over to the new treatment at the earliest possible opportunity, often before there is a confident estimate of the effect size in terms of final clinical outcomes. The ethical imperative to offer access to possibly effective treatments outweighs ensuring adequate trial design to be confident in the estimate of effect. Statistical techniques to ‘adjust’ for limitations in design become more and more complex, and more prone to uncertainty. And at the same time, most countries are still struggling with faltering economies and diminishing health budgets. Patients still want access to the latest treatments, but are less and less willing to pay increasing prices. Payers then have to compare the limited information set available for new drugs with what is known about current treatments. Arguments over the value of differences such as a 1.4 months gain in overall survival compared to existing treatment become conflated with the cost of this gain. That is assuming, of course, that there is a gain in overall survival, which has not been often shown to date in the trials of the new drugs for lung cancer. When high prices and high prevalence are combined, the value-for-money question is rightly raised – why pay so much more for so much uncertainty? Arguments about incremental advances in therapy, innovation and technological developments then dominate the discussion, rather than the appropriate focus on health gain for communities and individuals. So what is the solution? Options suggested include ‘managed entry’ of new products with additional data collection as a condition of price negotiation, ‘paying for performance’ with outcome data collection, or ‘value-based pricing’ that allows a premium for ‘innovation’. However, given the high clinical need for effective treatments, the emphasis should be on getting the best health outcomes for an affordable price, to the community and for individuals.

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