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FDA approval based on early evidence (like tumour shrinkage) on condition that a confirmatory trial follows.
United States, federal regulation and statute. Accelerated approval was created by FDA rule in December 1992 (Title 21 of the Code of Federal Regulations, part 314, subpart H for drugs and part 601, subpart E for biologics), written into section 506(c) of the Federal Food, Drug, and Cosmetic Act by the Modernization Act of 1997, and reformed by FDORA in December 2022. Primary text: the FDA accelerated approval programme page links the regulations and the statute.
Accelerated approval is the FDA route that grants approval on early evidence such as tumour shrinkage, measured by surrogate endpoints like ORR or pCR, on condition that a confirmatory trial follows. Approvals are withdrawn when confirmation fails, as with atezolizumab in TNBC after IMpassion131, belantamab in 2022 and sacituzumab in urothelial cancer, and Project Confirm and the 2023 FDORA reforms tightened the requirements. The term is referenced by the Glioma & glioblastoma entry, the drug records for Dordaviprone and Melphalan flufenamide, Project FrontRunner and Real-Time Oncology Review (RTOR). It features in the bottlenecks on trial design, regulatory divergence, prices and value and misaligned incentives, and in an idea on making post-progression sampling a condition of approval.
Patients with newly diagnosed metastatic colorectal cancer whose tumour carries a BRAF V600E mutation, which is about 8-12% of cases, should now be offered encorafenib and cetuximab together with FOLFOX from the start rather than after chemotherapy fails; median survival has roughly doubled to about two and a half years. BRAF testing at diagnosis is therefore essential, alongside RAS and mismatch repair testing. The regimen is more toxic than chemotherapy alone.
Patients with high-risk bladder cancer confined to the lining whose disease has not responded to BCG now have a bladder-sparing option that clears the cancer in most cases, delivered through a simple outpatient procedure. It may allow many to avoid or defer cystectomy, a life-changing operation. Whether responses translate into avoided progression and cystectomy over the long term, and how it compares with cystectomy on survival, remain to be shown.
Patients with advanced synovial sarcoma, a rare cancer of young adults with few effective drugs, now have an approved cell therapy that produces responses lasting about a year in a substantial minority, if their tissue type and tumour antigen match. It proves that engineered T cells can work against a solid tumour when a good target is present, which had been elusive. It is not a cure for most, requires specialised centres, and only a minority of patients are eligible.
Patients with KRAS G12C lung cancer that has progressed after chemo-immunotherapy can take an oral KRAS inhibitor instead of docetaxel and gain a somewhat longer time to progression with fewer severe side effects, but should understand that most tumours become resistant within a year and that survival is not improved. KRAS G12C testing is worthwhile, but first-generation inhibitors are a step rather than a cure; combinations and next-generation inhibitors are the active research fronts.
Patients with small-cell lung cancer that has relapsed after chemotherapy now have a drug that works far better than topotecan or lurbinectedin, and it is the first T-cell engager approved for a solid tumour. Treatment requires inpatient monitoring for the first doses because of cytokine release syndrome, which most centres now manage on a short-stay basis. It does not yet apply to first-line treatment, where trials are ongoing.
Sacituzumab govitecan is a standard second-line or later treatment for metastatic triple-negative breast cancer, roughly doubling survival compared with the chemotherapies it was tested against. Patients should expect neutropenia and diarrhoea, which are manageable with growth factor support and loperamide. Trials are now testing it earlier, in first-line combinations with pembrolizumab and after surgery for residual disease.
The dose on the label is often not the best dose for patients; it is the highest one that was tolerable for a few weeks. Project Optimus means new cancer drugs should arrive with evidence on dose, and it gives clinicians licence to consider dose reduction for toxicity. For older drugs, the evidence gap persists.
A drug that shrinks tumours or delays progression on scans has not necessarily been shown to help patients live longer or better. Patients and clinicians should ask what the endpoint was; regulators should insist on timely confirmatory trials; and trialists should validate surrogates before relying on them.
Shares Real-Time Oncology Review (RTOR) and Assessment Aid, Transferable priority vouchers for first-in-class drugs, with price conditions, Sakigake designation (Japan), Fast Track and RMAT designations.
Shares Real-Time Oncology Review (RTOR) and Assessment Aid, Label, indication and label expansion, Fast Track and RMAT designations, Full (traditional, regular) approval.
Shares Duration of response, Duration of response (DoR) and disease control rate (DCR), Provisional prices for surrogate-endpoint approvals, reset when survival data arrive, Patent term extension scaled to proven survival gain.
Shares Conditional approvals that lapse automatically if the confirmatory trial is late, A public registry of cancer treatments that were later shown not to work, FDA Oncology Center of Excellence, Weak real-world evidence and registries.
Shares Sakigake designation (Japan), Japan conditional early approval and time-limited approval, China Drug Administration Law (2019) and expedited pathways, Conditional marketing authorisation (EU).
Shares Cheap long-term survival follow-up by linking trial participants to registries, Every patient on an accelerated-approval drug enrolled in a registry until confirmation, Provisional prices for surrogate-endpoint approvals, reset when survival data arrive, Patent term extension scaled to proven survival gain.
Shares Fast Track and RMAT designations, Expanded access (compassionate use), Conditional marketing authorisation (EU), Indication withdrawal.
Shares Project FrontRunner, Refractory, Relapsed / refractory (R/R), BREAKWATER: encorafenib plus cetuximab with chemotherapy as first treatment for BRAF V600E-mutated colorectal cancer.