An appraisal of FDA approvals for adult solid tumours in 2017-2021: has the eagle landed?

Area of Science:

• Oncology
• Drug Approvals
• Clinical Benefit Assessment

Background:

• The Cancer Moonshot initiative aimed to accelerate cancer research and treatment progress.
• The period 2017-2021 saw significant FDA approvals for solid tumor therapies.
• Evaluating the clinical value of these approvals against the Moonshot's goals is crucial.

Purpose of the Study:

• To assess FDA therapeutic agent approvals for solid tumors (2017-2021) in light of the Cancer Moonshot.
• To evaluate the clinical benefit of these approved therapies using a standardized scale.
• To determine if the quality of approved treatments aligns with the Moonshot's ambition for substantial patient benefit.

Main Methods:

• Analysis of FDA approvals for solid tumor oncology agents between 2017 and 2021.
• Application of the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) v1.1.
• Categorization of approved therapies based on the level of clinical benefit provided.

Main Results:

• 161 new therapeutic agent approvals were issued by the FDA for solid tumors in adult patients.
• Only 27% of newly approved medicines demonstrated overall survival benefit; others relied on surrogate endpoints.
• Approximately 20% of new indications showed low clinical benefit, and 44% offered intermediate benefit.

Conclusions:

• Despite a high volume of FDA approvals, a significant portion of new cancer therapies offer limited clinical benefit.
• There is a need to improve the quality and evidence base for approved cancer treatments to ensure substantial patient benefit.
• Aligning drug approval standards with the Cancer Moonshot's goals requires a focus on demonstrable, meaningful clinical outcomes.