案例库 · 战略与领导 · 战略决策 · 1992
这条还没译成中文，下面是英文原文。
FDA's accelerated approval got drugs out early on a surrogate, then demanded proof.
Since 1992 FDA has approved serious-unmet-need drugs on surrogate markers, then required confirmatory trials.
U.S. Food and Drug Administration
那一手
For life-threatening diseases, running the long trial needed to prove real clinical benefit could delay access to a promising drug for years.
In 1992 the FDA created accelerated approval: a drug for a serious, unmet condition can be approved on a surrogate marker — a lab value, scan or sign reasonably likely to predict real benefit, such as tumour shrinkage for cancer.
The sponsor must then complete a confirmatory trial. If it shows real clinical benefit, the approval is converted; if not, the drug can be withdrawn. AACR's 2024 review found only 43% of 2013–2017 cancer accelerated approvals showed clinical benefit in confirmatory trials within five years.
为什么管用
- A surrogate lets a useful drug reach patients years sooner than waiting for survival data
- The mandatory confirmatory trial keeps the early access conditional rather than permanent
- The threat of withdrawal disciplines sponsors to finish the proof
可以搬走什么
When the direct measure is years away, condition approval on a good proxy plus a mandatory check, and tie the next decision to that evidence.
后来呢
The pathway sped drugs to people with HIV, cancer and rare diseases and was codified in the 2012 FDASIA. But critics note that many accelerated approvals do not confirm benefit, so the FDA has since tightened expectations around confirmatory trials and created Project Confirm to review them.
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