| ==== Front | |
| J Manag Care Spec Pharm | |
| J Manag Care Spec Pharm | |
| jmcsp | |
| Journal of Managed Care & Specialty Pharmacy | |
| 2376-0540 | |
| 2376-1032 | |
| Academy of Managed Care Pharmacy | |
| 10.18553/jmcp.2018.24.5.487 | |
| Letters | |
| Letter--Incorporating Real-World Evidence and Patient Value Criteria into Value-Based Frameworks for Relapsed/Refractory Multiple Myeloma | |
| Orlowski Robert Z. MD, PhD 1 * | |
| 1 Department of Lymphoma/Myeloma, Division of Cancer Medicine The University of Texas MD Anderson Cancer Center Houston, TX. | |
| * ROrlowski@mdanderson.org | |
| Orlowski has received research funding from Amgen, BioTheryX, Bristol-Myers Squibb, Celgene Corporation, and Takeda Pharmaceuticals; honoraria from Amgen, Bristol-Myers Squibb, Celgene Corporation, Janssen, Millennium Pharmaceuticals, and Onyx Pharmaceuticals; and is a member of advisory boards for Amgen, Bristol-Myers Squibb, Celgene Corporation, Incyte, Kite, Legend Biotech, Sanofi-Aventis, and Takeda Pharmaceuticals. | |
| 5 2018 | |
| 24 5 10.18553/jmcp.2018.24.5.487Copyright © 2018, Academy of Managed Care Pharmacy. All rights reserved. | |
| 2018 | |
| https://creativecommons.org/licenses/by/4.0/ This article is licensed under a Creative Commons Attribution 4.0 International License, which permits unrestricted use and redistribution provided that the original author and source are credited. | |
| ==== Body | |
| pmcIn the article “Evaluating Oncology Value-Based Frameworks in the U.S. Marketplace and Challenges in Real-World Application: A Multiple Myeloma Test Case,” published in the JMCP January 2018 issue, Djatche et al. report on their evaluation of 3 U.S. value-based frameworks for comparing carfilzomib, elotuzumab, and ixazomib plus lenalidomide-dexamethasone as triplet regimens for relapsed/refractory multiple myeloma. This timely analysis highlights a number of important shortcomings of currently available value-based frameworks, and the authors provide valuable constructive recommendations for future modifications to these tools. Notably, they highlight the limitations of the value-based frameworks in terms of consistency of treatment evaluations and with regards to adaptability to address multiple targeted stakeholder perspectives. | |
| This latter point is of particular relevance in relapsed/refractory multiple myeloma. As the authors highlight, the value-based frameworks mainly rely on evidence from randomized clinical trials and do not necessarily capture other aspects and value criteria of importance to the patient perspective in the real-world setting, such as convenience and preference. In this context, an additional important recommendation may be the inclusion of real-world evidence within value-based framework evaluations, particularly in clinical settings in which there is divergence between the efficacy reported from phase 3 trials and the effectiveness reported in real-world patient populations. Such a scenario has been reported recently in multiple myeloma, highlighting notable discrepancies between real-world effectiveness and clinical trial efficacy for a number of approved regimens, particularly those incorporating agents administered via intravenous or subcutaneous injection.1,2 | |
| This gap between effectiveness and efficacy may be related to the convenience and patient preference associated with oral therapy, as reported recently in multiple myeloma.3,4 The importance of these aspects is magnified in myeloma because they contribute to the feasibility of long-term treatment, and evidence is emerging of a correlation between real-world treatment duration and overall survival.5 In order to achieve this real-world surrogate endpoint for survival, namely long-term treatment duration, regimens are likely to need to fulfill the value criteria of importance to patients, supporting the inclusion of real-world evidence and patient perspectives within revised value-based framework methodologies. | |
| Acknowledging the limitations of the present value-based frameworks, Djatche et al. draw conclusions from their framework evaluations that rank carfilzomib-based therapy as most valued, followed by elotuzumab-based and ixazomib-based therapy. However, recent real-world data in relapsed/refractory myeloma patients indicate that those receiving ixazomib-based therapy have a longer duration of therapy and prolonged time to next therapy compared with those receiving carfilzomib- or bortezomib-based therapy. In addition, the discrepancy between these real-world findings and respective clinical trials appears most striking for carfilzomibbased therapy.2 Considering these findings and the recent reports on patient preference for oral versus injectable therapies, 3,4 it may be hypothesized that the ranking of the regimens would be altered if evaluated using a value-based framework incorporating real-world evidence and value criteria related to patient perspective. Because of its oral route of administration and its similar effectiveness and efficacy,1,2 ixazomib-based therapy could possibly be valued more highly in such a comprehensive value-based framework. | |
| Acknowledgments | |
| Writing assistance was provided by Steve Hill of FireKite (an Ashfield Company, part of UDG Healthcare) and was funded by Millennium Pharmaceuticals, a wholly owned subsidiary of Takeda Pharmaceutical Company, in compliance with Good Publication Practice-3 (GPP3) guidelines (Battisti WP, et al. Ann Intern Med. 2015;163:461-64). | |
| ==== Refs | |
| REFERENCES | |
| 1. Richardson PG, San Miguel JF, Moreau P, et al . Real-world and clinical trial data in relapsed/refractory multiple myeloma (RRMM): evaluating treatment duration and comparing effectiveness and efficacy. Blood. 2017;130 (Suppl 1 ):3149 [Abstract]. | |
| 2. Chari A, Romanus D, Luptakova K, et al . Duration of therapy (DOT) and time to next therapy (TTNT) of bortezomib, carfilzomib and ixazomib combinations with lenalidomide/dexamethasone (VRd, KRd, IRd) in patients (pts) with relapsed/refractory multiple myeloma (RRMM): clinical practice in the United States vs clinical trial experience. Blood. 2017;130 (Suppl 1 ):1818 [Abstract]. | |
| 3. Romanus D, DasMahapatra P, Hoole M, et al . Treatment satisfaction and burden of illness with oral vs injectable multiple myeloma therapy in patients with newly diagnosed disease (NDMM). Value Health. 2017;20 (9 ):A454 [Abstract]. | |
| 4. Bauer S, Mueller S, Ratsch B, et al . Patient preferences regarding treatment options for relapsed refractory multiple myeloma (RRMM). Value Health. 2017;20 (9 ):A451 [Abstract]. | |
| 5. Hari PN, Romanus D, Palumbo A, et al . Prolonged duration of therapy is associated with improved survival in patients treated for relapsed/refractory multiple myeloma in routine clinical care in the United States. Clin Lymphoma Myeloma Leuk. 2018;18 (2 ):152-60.29395837 | |