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Future Oncol. 2019 Dec;15(35):4057-4067. doi: 10.2217/fon-2019-0456. Epub 2019 Nov 5.

Value assessment of immuno-oncology in the treatment of rare tumors in the era of accelerated conditional approvals.

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Evidera, The Ark, 201 Talgarth Road, Hammersmith, London W6 8BJ, United Kingdom.
EMD Serono Inc, One Technology Place, Rockland, MA 02370, USA.
Merck KGaA, Frankfurter Straße 250, Darmstadt 64293, Germany.


Aim: To conduct a value assessment of an immuno-oncology (IO) therapy for a rare cancer and evaluate whether existing frameworks consider challenges associated with valuing IOs for rare cancers. Materials & methods: Value frameworks developed by American Society of Clinical Oncologists, Memorial Sloan Kettering Cancer Center and National Comprehensive Cancer Network were used to estimate the value of an IO therapy in a rare cancer based on single-arm trial data and retrospective studies. Results: Paucity of direct evidence comparing rare cancer treatments and lack of acceptance of indirect comparisons hinder appropriate value assessment. Measurement of value based on short-term outcomes may not capture the value of IOs, where survival is often characterized by a plateau. Conclusion: Further work is required to factor in nuances associated with rare cancers and guide end users of the frameworks. To capture true value, multiple or more holistic value assessments are required.


ASCO value framework; Abacus price; immuno-oncology therapy; rare cancer; value assessment; value framework


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