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:

Papers of special note have been highlighted as: • of interest; •• of considerable interest

References

1. Siegel RL, Miller KD, Jemal A. Cancer statistics, 2018. CA: Cancer J. Clin. 68(1), 7–30 (2018).
MedlineGoogle Scholar
2. Dizon DS, Krilov L, Cohen E et al. Clinical Cancer Advances 2016: annual report on progress against cancer from the American Society of Clinical Oncology. J. Clin. Oncol. 34(9), 987–1011 (2016).
Medline CASGoogle Scholar
3. Abdin SM, Zaher DM, Arafa EA, Omar HA. Tackling cancer resistance by immunotherapy: updated clinical impact and safety of PD-1/PD-L1 inhibitors. Cancers (Basel) 10(2), (2018).
MedlineGoogle Scholar
4. Keytruda^® (pembrolizumab) Prescribing Information (2019). www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf
Google Scholar
5. Opdivo^® (nivolumab) Prescribing Information (2019). https://packageinserts.bms.com/pi/pi_opdivo.pdf
Google Scholar
6. Bavencio^® (avelumab) Injection Prescribing Information (2019). www.emdserono.com/content/dam/web/corporate/non-images/country-specifics/us/pi/bavencio-pi.pdf
Google Scholar
7. What's Next in US Payor Communications: The Impact of FDA's Proposed Guidance on Communication of Healthcare Economic Information (2018). https://www.slideshare.net/npw1980/whats-next-in-us-payor-communications-the-impact-of-fdas-proposed-guidance-on-communication-of-healthcare-economic-information
Google Scholar
8. Bach PB, Pearson SD. Payer and policy maker steps to support value-based pricing for drugs. JAMA 314(23), 2503–2504 (2015).
Medline CASGoogle Scholar
9. Ramsey S, Blough D, Kirchhoff A et al. Washington State cancer patients found to be at greater risk for bankruptcy than people without a cancer diagnosis. Health Aff. (Millwood) 32(6), 1143–1152 (2013).
MedlineGoogle Scholar
10. National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology (NCCN Guidelines^®) with NCCN Evidence Blocks^™ (2018). www.nccn.org/evidenceblocks/default.aspx •• Describes the value assessment framework adopted by the National Comprehensive Cancer Network, using Evidence Blocks™, intended as a visual representation of measures that provide important information about therapeutic recommendations
Google Scholar
11. Schnipper LE, Davidson NE, Wollins DS et al. Updating the American Society of Clinical Oncology value framework: revisions and reflections in response to comments received. J. Clin. Oncol. 34(24), 2925–2934 (2016). •• Describes the American Society of Clinical Oncology value framework and the revisions made to the original framework based on comments received.
MedlineGoogle Scholar
12. Memorial Sloan Kettering Cancer Center. DrugAbacus tool: evidence driven drug pricing project (2018). https://drugpricinglab.org/tools/drug-abacus/methods/ •• Describes the criteria and methods used by the Memorial Sloan Kettering Cancer Center to determine the value of a drug.
Google Scholar
13. Slomiany M, Madhavan P, Kuehn M, Richardson S. Value frameworks in oncology: comparative analysis and implications to the pharmaceutical industry. Am. Health Drug Benefits 10(5), 253–260 (2017).
MedlineGoogle Scholar
14. Mandelblatt JS, Ramsey SD, Lieu TA, Phelps CE. Evaluating frameworks that provide value measures for health care interventions. Value Health 20(2), 185–192 (2017).
MedlineGoogle Scholar
15. Wilson L, Lin T, Wang L et al. Evaluation of the ASCO value framework for anticancer drugs at an academic medical center. J. Manag. Care Spec. Pharm. 23(2), 163–169 (2017).
MedlineGoogle Scholar
16. Bentley TGK, Cohen JT, Elkin EB et al. Validity and reliability of value assessment frameworks for new cancer drugs. Value Health 20(2), 200–205 (2017).
MedlineGoogle Scholar
17. Schnipper LE, Bastian A. New frameworks to assess value of cancer care: strengths and limitations. Oncologist 21(6), 654–658 (2016).
MedlineGoogle Scholar
18. Shah-Manek B, Wong W, Ravelo A, Dibonaventura M. Oncologists' perceptions of drug affordability using NCCN Evidence Blocks: results from a national survey. J. Manag. Care Spec. Pharm. 24(6), 565–571 (2018).
MedlineGoogle Scholar
19. Value frameworks for rare diseases: Should they be different? (2018). www.healthaffairs.org/do/10.1377/hblog20170412.059563/full/ • Discusses the unique challenges associated with valuing treatments for rare disease.
Google Scholar
20. Mincarone P, Leo CG, Sabina S et al. Reimbursed price of orphan drugs: current strategies and potential improvements. Public Health Genom. 20(1), 1–8 (2017).
MedlineGoogle Scholar
21. Orphan Drug Act of 1983. Public Law No. 97–414, 96 Stat. 2049 (2018). www.govinfo.gov/app/details/STATUTE-96/STATUTE-96-Pg2049
Google Scholar
22. Institute for Clinical and Economic Review. Modifications to the ICER value assessment framework for treatments for ultra-rare diseases (2018). https://icer-review.org/wp-content/uploads/2017/11/ICER-Adaptations-of-Value-Framework-for-Rare-Diseases.pdf
Google Scholar
23. National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology. Merkel Cell Carcinoma with NCCN Evidence Blocks^™. Version 2.2018. June 15, 2018 (2018). www.nccn.org/evidenceblocks/default.aspx
Google Scholar
24. Alexiou D, Chatzitheofilou I, Pi Blanque A. A review of NICE technology appraisals in oncology using single arm trials evidence. Value Health 21(Suppl. 1), S224 (2018).
Google Scholar
25. Logviss K, Krievins D, Purvina S. Characteristics of clinical trials in rare vs. common diseases: a register-based Latvian study. PLoS ONE 13(4), e0194494 (2018).
MedlineGoogle Scholar
26. Schnipper LE, Davidson NE, Wollins DS et al. American Society of Clinical Oncology Statement: a conceptual framework to assess the value of cancer treatment options. J. Clin. Oncol. 33(23), 2563–2577 (2015).
MedlineGoogle Scholar
27. Nghiem P, Bhatia S, Brohl AS et al. Two-year efficacy and safety update from JAVELIN Merkel 200, part A: a registrational study of avelumab in metastatic Merkel cell carcinoma progressed on chemotherapy. Abstract 9507 presented at 2018 Annual Meeting of the American Society of Clinical Oncologists, Chicago, IL. J. Clin. Oncol. 36(15_suppl), 9507–9507 (2018).
Google Scholar
28. Cowey CL, Mahnke L, Espirito J, Helwig C, Oksen D, Bharmal M. Real-world treatment outcomes in patients with metastatic Merkel cell carcinoma treated with chemotherapy in the USA. Future Oncol. 13(19), 1699–1710 (2017).
CASGoogle Scholar
29. Proskorovsky I, Lanitis T, Ambavane A et al. Projecting overall survival with immuno-oncology treatments: Application of alternative approaches in metastatic Merkel cell carcinoma. Abstract CN4 presented at ISPOR 2018; Baltimore, MD; May 19–23. Value Health 21(Suppl. 1), S6 (2018).
Google Scholar
30. Phatak H, Proskorovsky I, Lanitis T, Ambavane A, Hunger M, D'angelo SP. Projecting long term survival for avelumab in refractory Merkel cell carcinoma. J. Clin. Oncol. 36(15 Suppl.), e21623–e21623 (2018).
Google Scholar
31. Kaufman HL, Hunger M, Hennessy M, Schlichting M, Bharmal M. Nonprogression with avelumab treatment associated with gains in quality of life in metastatic Merkel cell carcinoma. Future Oncol. 14(3), 255–266 (2018).
CASGoogle Scholar
32. National Institute for Health and Care Excellence. Single technology appraisal. Avelumab for treating metastatic merkel cell carcinoma [TA517] (2018). https://www.nice.org.uk/guidance/ta517/documents/committee-papers
Google Scholar
33. IBM Micromedex^® RED BOOK^® (2018). www.ibm.com/us-en/marketplace/micromedex-red-book
Google Scholar
34. Sacco JJ, Botten J, Macbeth F, Bagust A, Clark P. The average body surface area of adult cancer patients in the UK: a multicentre retrospective study. PLoS ONE 5(1), e8933 (2010).
MedlineGoogle Scholar
35. Iyer JG, Blom A, Doumani R et al. Response rates and durability of chemotherapy among 62 patients with metastatic Merkel cell carcinoma. Cancer Med. 5(9), 2294–2301 (2016).
Medline CASGoogle Scholar
36. Becker DJ, Lin D, Lee S et al. Exploration of the ASCO and ESMO value frameworks for antineoplastic drugs. J. Oncol. Pract. 13(7), e653–e665 (2017). • Applies the American Society of Clinical Oncology and European Society for Medical Oncology value frameworks to 55 drugs for solid tumors and examines the relationships between costs and benefits.
MedlineGoogle Scholar
37. Joppi R, Bertele V, Garattini S. Orphan drug development is progressing too slowly. Br. J. Clin. Pharmacol. 61(3), 355–360 (2006).
Medline CASGoogle Scholar
38. Joppi R, Bertele V, Garattini S. Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. Eur. J. Clin. Pharmacol. 69(4), 1009–1024 (2013).
MedlineGoogle Scholar
39. Chandra A, Shafrin J, Dhawan R. Utility of cancer value frameworks for patients, payers, and physicians. JAMA 315(19), 2069–2070 (2016). • Discusses the methodological differences across different value assessment frameworks in oncology and provides recommendations for improving them.
Medline CASGoogle Scholar
40. National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology. Merkel Cell Carcinoma. Version 1. (2018). www.merkelcell.org/wp-content/uploads/2017/11/NCCN-Evidence-Blocks-Clinical-Practice-Guidelines-In-Oncology-Merkel-Cell-Carcinoma-Version-1.2018-October-6-2017.pdf
Google Scholar
41. Davies A, Briggs A, Schneider J et al. The ends justify the mean: outcome measures for estimating the value of new cancer therapies. Health Outcomes Res. Med. 3(1), e25–e36 (2012).
Google Scholar
42. Hoos A. Evolution of end points for cancer immunotherapy trials. Ann. Oncol. 23(Suppl. 8), viii47–52 (2012).
MedlineGoogle Scholar

Vol. 15, No. 35

Metrics

Downloaded 101 times

History

Received 31 July 2019

Accepted 11 October 2019

Published online 5 November 2019

Published in print December 2019

Information

Keywords

Supplementary data

To view the supplementary data that accompany this paper please visit the journal website at: www.futuremedicine.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.

PDF download