Reassessing the net health benefit (NHB) of FDA approved cancer drugs with evolution of evidence using the American Society of Clinical Oncology Value Framework (ASCO-VF).

Authors

null

Seanthel Delos Santos

Sunnybrook Research Institute, Toronto, ON, Canada

Seanthel Delos Santos , Noah Witzke , Vanessa Sarah Arciero , Amanda Putri Rahmadian , Louis Everest , Kelvin K. Chan

Organizations

Sunnybrook Research Institute, Toronto, ON, Canada, Sunnybrook Health Science Centre, Toronto, ON, Canada, Odette Cancer Centre, Sunnybrook Health Sciences Centre, University of Toronto, Toronto, ON, Canada, Sunnybrook Odette Cancer Centre, University of Toronto, Toronto, ON, Canada

Research Funding

No funding received
None

Background: Regulatory approval of oncology drugs are often based on data presented in the primary publication of clinical trials (CT). However, clinically relevant data, such as long-term overall survival (OS) and quality of life (QOL), are often reported in subsequent publications. Therefore, this study aimed to evaluate the ASCO-VF NHB at the time of drug approval and over time as further evidence is published. Methods: All FDA approved oncology drug indications from 01/06-12/16 were reviewed to identify CTs that were scorable using the ASCO-VF version 2. Subsequent publications of included CTs relevant for scoring were identified from Web of Science with a follow-up time of 3 years from approval. Using ASCO-defined threshold scores of ≤40 for low benefit and ≥45 for substantial benefit, changes in classification of benefit were assessed at 3-years post-FDA approval. Results: We identified 57 FDA approved indications (40.4% OS, 59.6% progression-free survival (PFS) as primary endpoints) with scorable ASCO-VF CTs. Among those 57 indications, 36.8% at the time of FDA approval demonstrated substantial benefit, 10.5% demonstrated intermediate benefit, and 52.6% demonstrated low benefit. We then identified 96 subsequent publications relevant to scoring within 3-years of FDA approval, consisting of primary endpoint updates (29.2%; 14.6% OS, 12.5% PFS), secondary endpoint updates (44.8%; 16.7% OS, 7.3% PFS), new reporting of secondary endpoint (4.2% OS), safety updates (28.1%), and QOL reporting (43.8%). Upon reassessment of the NHB in subsequent publications, there was an overall change from initial classification of benefit in 36.8% of trials (17.5% became substantial, 8.8% became low, and 10.5% became intermediate). Changes in scores were mainly the result of an updated hazard ratio (35.1%), change in scoring endpoints from PFS to OS as per ASCO-VF endpoint hierarchy (8.8%), toxicity updates (57.9%), new tail of the curve bonus (12.3%), palliation bonus (14.0%), or QOL bonus (22.8%). Overall, at reassessment at 3 years post-FDA approval, 42.1% were substantial, 10.5% were intermediate, and 47.3% were low benefit. Conclusions: Only a modest proportion of FDA approved drugs have demonstrated substantial NHB at time of approval. As further evidence was published, a substantial proportion of indications have a change in classification of NHB, resulting in a small increase in the overall proportion of indications being deemed to have substantial benefit at 3 years post-approval.

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Abstract Details

Meeting

2020 ASCO Virtual Scientific Program

Session Type

Poster Discussion Session

Session Title

Health Services Research and Quality Improvement

Track

Quality Care/Health Services Research

Sub Track

Value/Cost of Care

Citation

J Clin Oncol 38: 2020 (suppl; abstr 7011)

DOI

10.1200/JCO.2020.38.15_suppl.7011

Abstract #

7011

Poster Bd #

283

Abstract Disclosures