Assessment of the clinical and cost-effectiveness evidence in the reimbursement decisions of new cancer drugs

Strand, G. Chauca; Bonander, Carl; Jakobsson, Niklas; Johansson, N.; Svensson, Mikael

doi:10.1016/j.esmoop.2022.100569

Cited by 6 publications

(6 citation statements)

References 47 publications

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“…In a previous study of the clinical and cost-effectiveness evidence underlying reimbursement assessments in Sweden [ 22 ], we observed a lack of conclusive evidence of improvements in OS and QOL at the time of reimbursement in around 50% of reimbursed cancer drug indications, and suggestions of increased reliance on single-arm studies and surrogate outcomes compared with previous studies. Similar limitations have been reported for different regulatory and reimbursement decisions in Canada, China, and Europe [ 23 – 26 ], and in an overview of accelerated approvals in the USA over the last decades, only 20% of authorized drugs had shown improvements in OS in confirmatory trials [ 22 ].…”

Section: Introductionmentioning

confidence: 62%

“…In a previous review of the clinical and cost-effectiveness evidence used in reimbursement applications, we identified all cancer drug indications seeking reimbursement and claiming an added clinical value between 2010 and 2020 in Sweden [ 22 ]. Briefly, 60 drug indications were included in the initial review of which 46 were granted full or restricted reimbursement.…”

Section: Methodsmentioning

confidence: 99%

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Cancer Drugs Reimbursed with Limited Evidence on Overall Survival and Quality of Life: Do Follow-Up Studies Confirm Patient Benefits?

Chauca Strand,

Johansson,

Jakobsson

et al. 2023

Clin Drug Investig

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Background and Objective Cancer drug costs have increased considerably within healthcare systems, but many drugs lack quality-of-life (QoL) and overall survival (OS) data at the time of reimbursement approval. This study aimed to review the extent of subsequent literature documenting improvements in OS and QoL for cancer drug indications where no such evidence existed at the time of reimbursement approval. Methods Drug indications with claims of added therapeutical value but a lack of evidence on OS and QoL that were reimbursed between 2010 and 2020 in Sweden were included for review. Searches were conducted in PubMed and ClinicalTrial.gov for randomized controlled trials examining OS and QoL. Results Of the 22 included drug indications, seven were found to have at least one trial with conclusive evidence of improvements in OS or QoL after a mean follow-up of 6.6 years. The remaining 15 drug indications either lacked subsequent randomized controlled trial data on OS or QoL ( n = 6) or showed no statistically significant improvements ( n = 9). Only one drug demonstrated evidence of improvement in both OS and QoL for its indication. Conclusions A considerable share of reimbursed cancer drug indications continue to lack evidence of improvement in both OS and QoL. With limited healthcare resources and an increasing cancer burden, third-party payers have strong incentives to require additional post-reimbursement data to confirm any improvements in OS and QoL. Supplementary Information The online version contains supplementary material available at 10.1007/s40261-023-01285-4.

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Section: Introductionmentioning

confidence: 62%

Section: Methodsmentioning

confidence: 99%

Cancer Drugs Reimbursed with Limited Evidence on Overall Survival and Quality of Life: Do Follow-Up Studies Confirm Patient Benefits?

Chauca Strand,

Johansson,

Jakobsson

et al. 2023

Clin Drug Investig

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“…This transition is especially pertinent when integrating emerging technologies like AI, where financial considerations are paramount. The study by Harrison et al underscored the importance of economical scalability in cell therapy manufacturing, highlighting the potential of small-scale microfactories as a cost-effective approach before progressing to larger-scale macrofactories [15,16]. This model calls for careful economic assessment, particularly in automating processes to enhance efficiency and reduce costs, while maintaining quality control and managing the variability in donor cell characteristics.…”

Section: Investment In Infrastructure and Personnelmentioning

confidence: 99%

Integrating Artificial Intelligence for Academic Advanced Therapy Medicinal Products: Challenges and Opportunities

Aguilar-Gallardo,

Bonora-Centelles

2024

Applied Sciences

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Cell and gene therapies represent promising new treatment options for many diseases, but also face challenges for clinical translation and delivery. Hospital-based GMP facilities enable rapid bench-to-bedside development and patient access but require significant adaptation to implement pharmaceutical manufacturing in healthcare infrastructures constrained by space, regulations, and resources. This article reviews key considerations, constraints, and solutions for establishing hospital facilities for advanced therapy medicinal products (ATMPs). Technologies like process analytical technology (PAT), continuous manufacturing, and artificial intelligence (AI) can aid these facilities through enhanced process monitoring, control, and automation. However, quality systems tailored for product quality rather than just compliance, and substantial investment in infrastructure, equipment, personnel, and multi-departmental coordination, remain crucial for successful hospital ATMP facilities and to drive new therapies from research to clinical impact.

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“…At the time of regulatory market authorization of cancer drugs, it is frequent that data on OS or QoL confirming clinical benefits is lacking. Market authorization decisions are instead often based on intermediate (surrogate) endpoints such as Progression-Free Survival (PFS) and Response Rates [ 6 – 9 ], which frequently lack validation as predictors of long-term OS and QoL benefits. Following market authorization, many healthcare systems rely on subsequent Health Technology Assessments (HTA) to decide on reimbursement and to inform pricing negotiations [ 10 – 12 ].…”

Section: Introductionmentioning

confidence: 99%

“…Specifically, we analyze cancer drugs introduced in Swedish healthcare with limited evidence, defined as lacking randomized trial data or analysis showing any statistically significant OS or QoL benefits at the time of reimbursement. In Sweden, the Pharmaceutical and Benefits Agency (TLV) decides on the reimbursement of prescription drugs, and a large majority of all cancer drugs with European Medicines Agency (EMA) market authorization receive reimbursement [ 9 ]. In a previous study, we identified all reimbursed cancer drugs between 2010 and 2020 that had limited evidence of OS and QoL benefits at the time of reimbursement.…”

Section: Introductionmentioning

confidence: 99%

Analyses of quality of life in cancer drug trials - a review of measurements and analytical choices in post-reimbursement studies

Svensson,

Strand,

Bonander

et al. 2024

BMC Cancer

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Objectives For drugs reimbursed with limited evidence of patient benefits, confirmatory evidence of overall survival (OS) and quality of life (QoL) benefits is important. For QoL data to serve as valuable input to patients and decision-makers, it must be measured and analyzed using appropriate methods. We aimed to assess the measurement and analyses of post-reimbursement QoL data for cancer drugs introduced in Swedish healthcare with limited evidence at the time of reimbursement. Methods We reviewed any published post-reimbursement trial data on QoL for cancer drugs reimbursed in Sweden between 2010 and 2020 with limited evidence of improvement in QoL and OS benefits at the time of reimbursement. We extracted information on the instruments used, frequency of measurement, extent of missing data, statistical approaches, and the use of pre-registration and study protocols. Results Out of 22 drugs satisfying our inclusion criteria, we identified published QoL data for 12 drugs in 22 studies covering multiple cancer types. The most frequently used QoL instruments were EORTC QLQ-C30 and EQ-5D-3/5L. We identified three areas needing improvement in QoL measurement and analysis: (i) motivation for the frequency of measurements, (ii) handling of the substantial missing data problem, and (iii) inclusion and adherence to QoL analyses in clinical trial pre-registration and study protocols. Conclusions Our review shows that the measurements and analysis of QoL data in our sample of cancer trials covering drugs initially reimbursed without any confirmed QoL or OS evidence have significant room for improvement. The increasing use of QoL assessments must be accompanied by a stricter adherence to best-practice guidelines to provide valuable input to patients and decision-makers.

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Assessment of the clinical and cost-effectiveness evidence in the reimbursement decisions of new cancer drugs

Cited by 6 publications

References 47 publications

Cancer Drugs Reimbursed with Limited Evidence on Overall Survival and Quality of Life: Do Follow-Up Studies Confirm Patient Benefits?

Cancer Drugs Reimbursed with Limited Evidence on Overall Survival and Quality of Life: Do Follow-Up Studies Confirm Patient Benefits?

Integrating Artificial Intelligence for Academic Advanced Therapy Medicinal Products: Challenges and Opportunities

Analyses of quality of life in cancer drug trials - a review of measurements and analytical choices in post-reimbursement studies

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