Value Assessment of Immuno-Oncology in the Treatment of Rare Tumors in the Era of Accelerated Conditional Approvals

Abstract

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.

Keywords::

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

Supplementary data

To view the supplementary data that accompany this paper please visit the journal website at: www.tandfonline.com/doi/suppl/10.2217/fon-2019-0456

Financial & competing interests disclosure

T Lanitis and A Ambavane are salaried employees of Evidera, London, UK; as such they are not allowed to accept remuneration from Evidera clients. Evidera received funding from EMD Serono in connection with this study. This work was supported by EMD Serono Inc, Rockland, MA, USA, a business of Merck KGaA, Darmstadt, Germany. H Phatak and Y Zheng are employees of EMD Serono Inc (Rockland, MA, USA), a business of Merck KGaA, and M Schlichting is an employee of Merck KGaA (Darmstadt, Germany); these three authors have employee stock incentives. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Medical writing support was provided by S Heiman, PharmD, and M Jardim, PhD from Evidera and funded by EMD Serono Inc, Rockland, MA, USA.

Additional information

Funding

T Lanitis and A Ambavane are salaried employees of Evidera, London, UK; as such they are not allowed to accept remuneration from Evidera clients. Evidera received funding from EMD Serono in connection with this study. This work was supported by EMD Serono Inc, Rockland, MA, USA, a business of Merck KGaA, Darmstadt, Germany. H Phatak and Y Zheng are employees of EMD Serono Inc (Rockland, MA, USA), a business of Merck KGaA, and M Schlichting is an employee of Merck KGaA (Darmstadt, Germany); these three authors have employee stock incentives. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Medical writing support was provided by S Heiman, PharmD, and M Jardim, PhD from Evidera and funded by EMD Serono Inc, Rockland, MA, USA.