Global consequences of the US FDA's accelerated approval of cancer drugs

Akhade, Amol; Sirohi, Bhawna; Gyawali, Bishal

doi:10.1016/s1470-2045(21)00709-9

Cited by 17 publications

(6 citation statements)

References 7 publications

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“…The US can set a precedent for available therapies in other countries, thus influencing care that is provided outside of the US. This is especially true in low‐to‐middle‐income countries who may lack the resources to do their own drug evaluation 14 . The regulatory approval process should be designed to document patient benefit—if not at approval, at some point in the lifecycle—and if drugs fail to achieve this benchmark, arguably they should be removed from the market.…”

Section: Discussionmentioning

confidence: 99%

An empirical analysis of overall survival in drug approvals by the US FDA (2006–2023)

Elbaz,

Haslam,

Prasad

2024

Cancer Medicine

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BackgroundThe US Food and Drug Administration (FDA) has expanded the use of surrogate markers in drugs approved for oncology/hematology indications. This has likely resulted in a greater number of approvals and possibly drugs coming to market faster, but it is unknown whether these drugs also improve overall survival (OS) for patients taking them.MethodsWe sought to estimate the percentage of oncology drugs that have shown to improve OS in a cross‐sectional analysis of US FDA oncology drug approvals (2006–2023). We searched for OS data in registration trials and the peer‐reviewed literature.ResultsWe found 392 oncology drug approvals. Eighty‐seven (22%) drug approvals were based on OS, 147 drug approvals were later tested for OS benefit (38% of all approvals and 48% of drugs approved on a surrogate), and 130 (33%) have yet to be tested for OS benefit. Of the 147 drug approvals later tested for OS, 109 (28% of all approvals and 74% of drugs later tested for OS) have yet to show OS benefit, whereas 38 (10% of all approvals and 26% of drugs later tested for OS benefit) were later shown to have OS benefit. In total, 125 out of 392 (32%) drugs approved for any indication have been shown to improve OS benefit at some point, and 267 (68%) have yet to show approval.ConclusionAbout 32% of all oncology drug approvals have evidence for an improvement in OS. Higher standards are needed in drug regulation to ensure that approved drugs are delivering better patient outcomes, specifically in regards to survival.

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Section: Discussionmentioning

confidence: 99%

An empirical analysis of overall survival in drug approvals by the US FDA (2006–2023)

Elbaz,

Haslam,

Prasad

2024

Cancer Medicine

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“…The under-representation of LMIC in cost-effectiveness research overestimates the cost-effectiveness of novel therapies and deepens access inequities worldwide. The lack of pharmaceutical regulatory agencies and HTA committees [ 10 ] in many LMICs makes them more susceptible to importing regulatory decisions from the Food and Drug Administration (FDA) or European Medicines Agency (EMA), without careful analysis of implications [ 17 ]. For example, in India, a drug already withdrawn from the FDA accelerated approval (revoked approval) was still being marketed and locally promoted [ 18 ].…”

Section: Discussionmentioning

confidence: 99%

Global representativeness and impact of funding sources in cost-effectiveness research on systemic therapies for advanced breast cancer: A systematic review

Lazar Neto,

de Melo,

Hidalgo Filho

et al. 2024

The Breast

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“…As the USA leads the world in new drug research and development and is primarily the first market to launch new cancer drugs, many LMICs rely on a drug’s FDA approval status to inform its use in their populations. 36 In addition, the clinical trials considered by the FDA are often the only studies available evaluating the efficacy of new cancer drugs. We compared the documented evidence of OS benefit between WHO-TRS and pivotal trials reported in FDA labels and found that benefit evidence differed.…”

Section: Discussionmentioning

confidence: 99%

Overall survival benefits of cancer drugs in the WHO Model List of Essential Medicines, 2015–2021

Zhou,

Naci,

Chen

et al. 2023

BMJ Glob Health

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IntroductionWe examined overall survival (OS) benefits for targeted cancer drugs recommended for List of Essential Medicines (EMLs) since 2015. We assessed consistency of decisions in 2019 and 2021 with more specific criteria: OS benefit >4 months and high scores on European Society for Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS).MethodsWe identified applications for cancer drug in WHO EMLs from 2015 to 2021. We extracted evidence of OS benefit documented in WHO Technical Report Series (TRS) and compared it to evidence from pivotal trial(s) documented in Food and Drug Administration-approved labels. We retrieved published ESMO-MCBS scores. We summarised availability and magnitude of OS benefit and ESMO-MCBS scores and assessed consistency of inclusion decisions against WHO criteria.Results22/54 targeted cancer drug indications were recommended. Among them, 68.2% and 31.8% had OS benefit evidence documented in WHO-TRS and pivotal trials, respectively. Among those not recommended, 59.4% and 56.3% had OS benefit evidence documented in WHO-TRS and pivotal trials, respectively. Of 11 cancer drug indications recommended in 2019 and 2021, 54.5% and 9.1% had evidence of OS benefit >4 months in WHO-TRS and pivotal trials, respectively; 45.5% met ESMO-MCBS criteria. Ten targeted cancer drugs had more than one application for the same indications. Five of those were eventually recommended, including three without new evidence of OS benefit. Additional factors, such as reduced cost, and increased treatment options, seemed to be important factors in the selection.ConclusionWhile WHO has defined approval criteria for cancer drugs EML, we identified areas where adherence of these criteria and communication of the EML approval decision-making processes can be improved.

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Global consequences of the US FDA's accelerated approval of cancer drugs

Cited by 17 publications

References 7 publications

An empirical analysis of overall survival in drug approvals by the US FDA (2006–2023)

An empirical analysis of overall survival in drug approvals by the US FDA (2006–2023)

Global representativeness and impact of funding sources in cost-effectiveness research on systemic therapies for advanced breast cancer: A systematic review

Overall survival benefits of cancer drugs in the WHO Model List of Essential Medicines, 2015–2021

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