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1 Recommendations

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1 Recommendations

1.1 The committee was minded not to recommend avatrombopag as an option for treating primary chronic immune thrombocytopenia refractory to other treatments (for example, corticosteroids, immunoglobulins) in adults.

1.2 The committee recommends that NICE requests further clarification and analyses from the company, which should be made available for the second appraisal committee meeting, and should include:

  • a network meta-analysis with the mean platelet count as a continuous outcome that, together with a distributional assumption, can be used to derive response probabilities

  • scenario analyses for comparison with the company's model assumptions that estimate treatment duration or stopping rates based on the:

    • patient-level data from Study 302

    • empirical data from the extension of Study 302

  • details on the:

    • methods of the company's market research that informed the costs in the model

    • how the bleed-related unit costs were derived

  • a probabilistic sensitivity analysis, including probabilistic incremental cost-effectiveness ratios, cost-effectiveness scatter plots and cost-effectiveness acceptability curves for £20,000 and £30,000 per quality-adjusted life year gained.

Why the committee made these recommendations

Current treatment for newly diagnosed primary chronic immune thrombocytopenia usually includes corticosteroids and immunoglobulins. This is followed by thrombopoietin receptor agonists (TPO‑RAs). Avatrombopag is another TPO‑RA.

Clinical trial evidence suggests that avatrombopag is more effective than placebo at increasing the number of platelets in the blood (cells that help the blood to clot) to a level that meaningfully reduces the risk of bleeding. But avatrombopag's clinical effectiveness compared with other TPO-RAs is unclear because of uncertainties in the company's indirect treatment comparison.

Whether avatrombopag is cost effective is unknown because of uncertainties in the clinical evidence and the economic model. So, no recommendations could be made, and the company is invited to provide more analyses for consideration at the second appraisal committee meeting.