Potential alternative dosage strategies for patent-protected oral cancer drugs

To identify potential alternative dosage strategies for patent-protected oral oncology drugs that could reduce costs without decreasing efficacy for future post-marketing optimization trials or immediate prescriber practice changes.

All patent-protected oral oncology drugs approved by the Food & Drug Administration (FDA) between 2004 and 2024 were identified. Publications, abstracts, and FDA reviews available at Drugs@FDA were assessed to determine if there was a potential alternative dosage strategy that could reduce cost while maintaining efficacy. Strategies were categorized by level of supporting evidence, whether based on randomized controlled trials, nonrandomized trials, or pharmacologic data. Potential cost savings were calculated using average wholesale price and pricing schemes by drug. The potential reduction in global sales revenue was calculated based on public sales data.

A total of 90 oral oncology drugs were identified, of which we proposed 50 (56 %) potential cost-saving alternative dosage strategies. These strategies yielded a median maximal potential cost savings of 50 % (range 20 %-86 %). Global spending could potentially be reduced by as much as EUR 20.1 billion per year if these strategies are implemented for the 37 drugs with global revenue data available. Randomized trial evidence supported immediate dosage modification for sotorasib and ribociclib; post-marketing optimization trials were warranted for 48 other drugs.

Over half of patent-protected oral oncology drugs have potential alternative dosage strategies that may reduce costs for payors and side effects for patients. Public payors and closed healthcare systems should consider post-marketing dosage optimization trials to evaluate these strategies.