Analysis of patient-reported outcomes in the approval of novel oncology drugs in the United States, 2017–2022

Ge, Chenghao; Guo, Ke; Li, Yang; Li, Guanqiao; Zhang, Hong; Yang, Jiaxuan; Liu, Yang; Chen, Yin; Liu, Sen; Song-mei, Xie; Chen, Xiaoyuan

doi:10.1016/j.eclinm.2023.101953

Cited by 11 publications

(4 citation statements)

References 38 publications

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“…However, QoL data must be measured and analyzed validly and reliably to provide valuable insights to decision-makers and clinicians. The FDA has hesitated to grant QoL labeling of the product’s benefits, which may be attributed to uncertainties and a lack of quality in the measurement and analytical standards for QoL data [ 35 , 36 ]. There are several calls for standardization of design, reporting, and statistical analyses of QoL data, including initiatives by the, e.g., SPIRIT-PRO [ 37 ], CONSORT-PRO [ 29 ], and SISAQOL [ 30 ] consortiums.…”

Section: Discussionmentioning

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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Section: Discussionmentioning

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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“…Although the regulatory bodies do not typically require PROMs as a tool for the process of approving new drugs, it is anticipated that their significance will grow in the future. 27 …”

Section: Discussionmentioning

confidence: 99%

Guidance on mucositis assessment from the MASCC Mucositis Study Group and ISOO: an international Delphi study

Abdalla-Aslan,

Bonomo,

Keefe

et al. 2024

eClinicalMedicine

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“…freedom from disease and overall survival), adding relevant information for cancer decision-making. 119 , 120 Patients with cancer can provide their own input to guide treatment using PROMs related to pain. However, the literature shows that, because of ineffective transfer of information and frequent clinician inertia in acting on this information, interventions based on PROMs have achieved only modest reductions in cancer pain intensity.…”

Section: Outcomes In Pain Management As Reported By Patientsmentioning

confidence: 99%

Fentanyl in cancer pain management: avoiding hasty judgments and discerning its potential benefits

Cuomo

2023

DIC

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Cancer pain is an important challenge in treatment and requires a rapid onset of action for its control. In particular, breakthrough cancer pain (BTcP) should be adequately controlled with a stable dose of a short-acting oral opioid. Fentanyl is a synthetic, highly selective opioid with many advantageous chemical properties, including high lipophilicity and distinct pharmacokinetic properties. It is recommended for pain management in a variety of settings, including acute pain, chronic pain and BTcP. To date, its variously designed formulations allow non-invasive administration; amongst others, sublingual fentanyl has proven useful in the management of BTcP and in improving the quality of life of patients with cancer. This review provides an update on the management of BTcP with fentanyl, with consideration of safety, as it remains an important tool in the treatment of cancer pain.

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Analysis of patient-reported outcomes in the approval of novel oncology drugs in the United States, 2017–2022

Cited by 11 publications

References 38 publications

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

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

Guidance on mucositis assessment from the MASCC Mucositis Study Group and ISOO: an international Delphi study

Fentanyl in cancer pain management: avoiding hasty judgments and discerning its potential benefits

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