Measuring the Value of New Drugs: Validity and Reliability of 4 Value Assessment Frameworks in the Oncology Setting

Abstract

This work was funded by Eisai Inc. Copher and Knoth are employees of Eisai Inc. Bentley, Lee, Zambrano, and Broder are employees of Partnership for Health Analytic Research, a health services research company paid by Eisai Inc. to conduct this research. For this study, Cohen, Huynh, and Neville report fees from Partnership for Health Analytic Research. Outside of this study, Cohen receives grants and direct consulting fees from various companies that manufacture and market pharmaceuticals. Mei reports a grant from Eisai Inc. during this study. The other authors have no disclosures to report. Study concept and design were contributed by Bentley and Broder, with assistance from Elkin and Cohen. Bentley took the lead in data collection, along with Elkin, Huynh, Mukherjea, Neville, Mei, Popescu, Lee, and Zambrano. Data interpretation was performed by Bentley and Broder, along with Elkin, Cohen, Copher, and Knoth. The manuscript was written primarily by Bentley, along with Elkin and Broder, and revised by Bentley, Broder, Elkin, Cohen, Copher, and Knoth. Select components of this work's methods were presented at ISPOR 19th Annual European Congress held in Vienna, Austria, October 29-November 2, 2016, and Society for Medical Decision Making 38th Annual North American Meeting held in Vancouver, Canada, October 23-26, 2016.

Do current approaches to assessing therapy related adverse events align with the needs of long-term cancer patients and survivors?

“…This review captures recommendations and perspectives from a total of 17 peer-reviewed publications [95,[102][103][104][106][107][108][109][110][111][112][113][114][115][116][117][118]. The reviewed studies were almost evenly split between those including a narrative/qualitative comparison of different frameworks and those incorporating case study/quantitative comparisons across different value frameworks.…”

“…However, the need for more robust and/or detailed safety and toxicity data inclusion in these frameworks was the most common recommendation identified. Additionally, several studies called for more overarching changes to clinical trial design with regard to patient inclusion criteria, duration, outcomes measures, and so forth [102,110,112,115,119]. The details of such modifications were not thoroughly addressed in these publications and are the subject of much discussion elsewhere, but they could have significant impact on the type of AE data generated in the future [120].…”

“…Specifically, enhanced clarity and transparency as to the intended audience for the framework outputs [103,104,110,111,121] and improved guidance to enhance reproducibility were common recommendations [107,108,111,114,119,122].…”

“…Additionally, more comprehensive insights into methodological and data input assumptions across these studies could be gleaned by review of key underlying studies cited by the studies reviewed here. [103,112,119,138] Frameworks should incorporate data from sources other than clinical trials (e.g., observational studies) 3 of 17 (18%) [102] Frameworks should incorporate more robust and/or detailed safety and/or toxicity data 7 of 17 (41%) [103,104,108,112,115,116] Frameworks should use integrated quality of life measures in lieu of safety data 1 of 17 (6%) [117] Frameworks should incorporate more longitudinal data 2 of 17 (12%) [102,103] Frameworks should engage patients in the data evaluation and input process 3 of 17 (18%) [102,103,110] …”

Economic challenges of oncological diseases’ pharmacotherapy

“…Одним из наиболее важных способов снижения стоимости противоопухолевой терапии является оценка ее затратной эффективности как в период регистрации препарата, так и в реальной медицинской практике [11]. В настоящее время и ASCO, и ESMO разработали шкалы, содержащие алгоритмы оценки пользы противоопухолевой терапии, однако они нуждаются в дальнейшей доработке и согласовании между собой [54,55].…”