Provisional prices for surrogate-endpoint approvals, reset when survival data arrive
Drugs approved on early signs of benefit are paid for as if they had proved they extend life. Pay a provisional price and adjust it, up or down, when the survival data come in.
Most accelerated oncology approvals rest on response rate or progression-free survival, and a substantial share later fail to show survival benefit, yet prices are set at launch and rarely fall. Coverage-with-evidence-development schemes exist (the UK Cancer Drugs Fund, Germany's post-launch benefit reassessment) but price adjustment is negotiated rather than automatic. The proposal is a contractual price schedule fixed at launch: a provisional price, a higher price if the confirmatory trial shows survival benefit above a threshold, a sharply lower price or rebate if it does not, with the payer co-funding registry follow-up.
- Prices and value · New cancer drugs routinely cost over $150,000 a year, often for months of benefit. Systems cannot afford them and patients go bankrupt.
- Regulatory divergence between regions · Regulatory divergence means a drug approved in one country can take years to reach another, or never arrive.
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not linked directly; found by shared links- IdeaEvery patient on an accelerated-approval drug enrolled in a registry until confirmation
Shares Accelerated approval, Overall survival (OS), Regulatory divergence between regions.
- IdeaOne evidence plan agreed by regulator and payer before the pivotal trial
Shares Regulatory divergence between regions, Prices and value.
- IdeaSponsors deposit the confirmatory trial budget in escrow at accelerated approval
Shares Accelerated approval, Regulatory divergence between regions.
- IdeaSeamless phase 2/3 with pre-registered go rules as the default for new agents
Shares Overall survival (OS), Progression-free survival (PFS).
- IdeaReport time toxicity, the days a treatment consumes, in every trial and decision aid
Shares Overall survival (OS), Progression-free survival (PFS).
- IdeaConditional approvals that lapse automatically if the confirmatory trial is late
Shares Accelerated approval, Regulatory divergence between regions.
- Key paperPrasad: most surrogate endpoints in cancer trials correlate poorly with survival
Shares Accelerated approval, Overall survival (OS), Regulatory divergence between regions, Progression-free survival (PFS).
- IdeaMake post-progression sampling a condition of accelerated approval
Shares Accelerated approval, Regulatory divergence between regions.