A review of patient-reported outcome assessments in registration trials of FDA-approved new oncology drugs (2014–2018)

Gnanasakthy, Ari; Russo, Jon C.; Gnanasakthy, Kajan; Harris, Nimanee; Castro, Colleen

doi:10.1016/j.cct.2022.106860

Cited by 7 publications

(2 citation statements)

References 21 publications

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“…A more updated analysis on 480 published and 537 registered randomised cancer trials identified PRO measures in over 50 of published trials and up to 66 of registered protocols [ 17 ]. Of 55 registration trials used to support new oncology drugs approved by the FDA during 2014–2018, 41 (75%) included PRO assessments [ 18 ]. Indeed, adoption of PRO-related endpoints in clinical trials can in turn influence regulatory decisions.…”

Section: Introductionmentioning

confidence: 99%

The Assessment of Patient-Reported Outcomes for the Authorisation of Medicines in Europe: A Review of European Public Assessment Reports from 2017 to 2022

Meregaglia,

Malandrini,

Angelini

et al. 2023

Appl Health Econ Health Policy

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Objectives Health regulators have progressively increased their attention and focus on patient-reported outcomes (PROs), driven by the diffusion of a patient-centred approach to the drug development process. This study investigates the consideration of PROs and their measures (PROMs) in the authorisation of medicines in Europe. Methods All medicines for human use authorised or refused by the European Medicines Agency (EMA) in the period 2017–2022 were identified, and corresponding European Public Assessment Reports (EPARs) were downloaded for review. Medicine and PROs/PROM characteristics were systematically recorded. A multivariate logistic regression was performed to identify variables associated with the use of patient-reported evidence in EPARs. Results Overall, 497 EPARs of authorised medicines and 19 EPARs of refused medicines were analysed; of these, 240 (48.3%) and 10 (52.6%), respectively, reported any use of PROs/PROMs ( p = 0.710). For authorised medicines, the likelihood of using PROs/PROMs was negatively affected by generic (OR = 0.01, p < 0.001) and biosimilar status (OR = 0.46, p = 0.013) and positively affected by orphan status (OR = 1.41, p = 0.177). The use of PROMs (50.6% in 2017 vs 47.9% in 2022) did not show a clear pattern over the 6-year period considered ( p = 0.758) and was particularly uncommon in some therapeutic areas (e.g., 15.2% in infectious diseases). A total of 816 dyads of PROs/PROMs were identified. On average each EPAR considered 1.6 (range: 0–14) instruments. Patient-reported outcomes were typically secondary (53.3%) and exploratory endpoints (18.8%); in one-third of cases (32.5%), they assessed generic quality of life. Among the PROMs, 227 (27.8%) targeted general population; EQ-5D (11.0%), SF-36/SF-12 (5.9%) and EORTC QLQ-C30 (5.6%) were the instruments most frequently used. Conclusions This study suggests PROs/PROMs are considered in less than half of total medicine assessments and even more rarely in some disease areas. The adoption of PROs is key in EMA strategy to 2025 and would be facilitated by consensus development on their measures and optimisation of data collection.

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Section: Introductionmentioning

confidence: 99%

The Assessment of Patient-Reported Outcomes for the Authorisation of Medicines in Europe: A Review of European Public Assessment Reports from 2017 to 2022

Meregaglia,

Malandrini,

Angelini

et al. 2023

Appl Health Econ Health Policy

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“…31,32 Guidelines targeting early-phase COA use or adaptation of existing strategies for randomized trials may be beneficial. 9,11,12 The growing role of COAs to establish net clinical benefit of novel therapies 33 and impact regulatory decision making 25,34,35 underscores the need to address the low COA use rates in treatment-focused oncology trials. However, an important caveat when interpreting these rates is that they exclude the use of traditional survival and tumor response endpoints in oncology trials, therefore underestimating the use of ClinROs as they pertain to those endpoints.…”

Section: Discussionmentioning

confidence: 99%

Clinical outcome assessment trends in clinical trials—Contrasting oncology and non‐oncology trials

et al. 2023

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BackgroundClinical outcome assessments (COAs) are key to patient‐centered evaluation of novel interventions and supportive care. COAs are particularly informative in oncology where a focus on how patients feel and function is paramount, but their incorporation in trial outcomes have lagged that of traditional survival and tumor responses. To understand the trends of COA use in oncology and the impact of landmark efforts to promote COA use, we computationally surveyed oncology clinical trials in ClinicalTrials.gov comparing them to the rest of the clinical research landscape.MethodsOncology trials were identified using medical subject heading neoplasm terms. Trials were searched for COA instrument names obtained from PROQOLID. Regression analyses assessed chronological and design‐related trends.ResultsEighteen percent of oncology interventional trials initiated 1985–2020 (N = 35,415) reported using one or more of 655 COA instruments. Eighty‐four percent of the COA‐using trials utilized patient‐reported outcomes, with other COA categories used in 4–27% of these trials. Likelihood of COA use increased with progressing trial phase (OR = 1.30, p < 0.001), randomization (OR = 2.32, p < 0.001), use of data monitoring committees (OR = 1.26, p < 0.001), study of non‐FDA‐regulated interventions (OR = 1.23, p = 0.001), and in supportive care versus treatment‐focused trials (OR = 2.94, p < 0.001). Twenty‐six percent of non‐oncology trials initiated 1985–2020 (N = 244,440) reported COA use; they had similar COA‐use predictive factors as oncology trials. COA use increased linearly over time (R = 0.98, p < 0.001), with significant increases following several individual regulatory events.ConclusionWhile COA use across clinical research has increased over time, there remains a need to further promote COA use particularly in early phase and treatment‐focused oncology trials.

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Use of patient-reported outcome measures for oncology drugs receiving accelerated approval

Moore,

Elmes,

Strassels

et al. 2023

Support Care Cancer

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A review of patient-reported outcome assessments in registration trials of FDA-approved new oncology drugs (2014–2018)

Cited by 7 publications

References 21 publications

The Assessment of Patient-Reported Outcomes for the Authorisation of Medicines in Europe: A Review of European Public Assessment Reports from 2017 to 2022

The Assessment of Patient-Reported Outcomes for the Authorisation of Medicines in Europe: A Review of European Public Assessment Reports from 2017 to 2022

Clinical outcome assessment trends in clinical trials—Contrasting oncology and non‐oncology trials

Use of patient-reported outcome measures for oncology drugs receiving accelerated approval

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