Loading
Progression-free survival (PFS) is how long patients live without their cancer growing.
Progression-free survival (PFS) measures the time from random assignment until the cancer progresses or the patient dies, whichever comes first. It is a common primary endpoint because a trial can read out with fewer patients and in less time than one powered for overall survival, but a PFS gain does not always translate into an OS benefit, as TROPION-Breast01 illustrated; PFS2 tracks progression on the next therapy. PFS is referenced by the Small-cell lung cancer, Sarcomas and Hodgkin lymphoma entries, the POLARIX trial and Michael LeBlanc, and it features in the bottlenecks on trial design, real-world evidence and quality of life. Ideas engaging with it include clonal clearance as an endpoint, a six-week ctDNA switch and seamless phase 2/3 designs with pre-registered go rules.
The 48 most recent of 57 papers; see them all →
The latest in a line of negative targeted-therapy trials in triple-negative disease (EGFR, VEGF, iniparib, now AKT): a modest delay in progression that did not translate into survival, in the same year that an antibody-drug conjugate did. It is why the roadmap treats pathway-targeted small molecules as the road not taken.
The first-line overall survival result that made an antibody-drug conjugate the standard for PD-L1-negative or immunotherapy-ineligible metastatic triple-negative disease, and the reason the sequencing question (which TROP2 drug first, what after it) is now urgent.
Chinese patients in the trial saw the same pattern as the global population: a clear progression-free survival gain, no proven survival gain, and a different rather than heavier side-effect burden, with mouth and eye toxicity in nearly half. It supports use of datopotamab deruxtecan in this setting in China but does not resolve the global trial's missing survival benefit.
AMPLIFY delivered the first all-oral, fixed-duration doublet for front-line CLL and supported its approval, giving fit patients a way to avoid both chemotherapy and years of continuous BTK inhibitor. It does not settle whether a doublet or triplet is best, or how AV compares with venetoclax-obinutuzumab. Patients with TP53 aberration were excluded and still need different strategies.
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.
CEPHEUS extends the quadruplet standard to patients who are not going to transplant, closing the gap between transplant-eligible and ineligible populations. It is also one of the first phase 3 trials to be designed around MRD-negativity as the primary endpoint, which could shorten future myeloma trials by years. Frailer patients still need dose-adapted approaches.
For the first time a new drug has beaten pembrolizumab, the global first-line standard, in a randomised lung cancer trial, and it did so by combining checkpoint blockade with anti-angiogenesis in one molecule. For patients outside China nothing changes yet: the drug is not approved in the West, the trial was single-country, and survival benefit has not been shown. If confirmed in the global HARMONi-3 and HARMONi-7 trials, PD-1 x VEGF bispecifics could replace PD-1 antibodies as the immunotherapy backbone.
The evidence behind the 2026 approval of Optune Pax for locally advanced disease, the first new approval in that setting in decades, and an unusual case of a survival gain without a progression-free survival gain.
Shares Require a randomised phase 2 before any phase 3, Blinded independent central review (BICR), Duration of response, Duration of response (DoR) and disease control rate (DCR).
Shares Progressive disease and radiographic progression, Progression, NAPOLI-3: NALIRIFOX versus gemcitabine plus nab-paclitaxel as first treatment for metastatic pancreatic cancer, RELATIVITY-047: relatlimab plus nivolumab, the first LAG-3 checkpoint combination, in untreated advanced melanoma.
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 Provisional prices for surrogate-endpoint approvals, reset when survival data arrive, Launch prices indexed to the ESMO benefit scale, revisited when survival matures, Power trials to detect a benefit patients would value, not the smallest detectable one, ISPOR (The Professional Society for Health Economics and Outcomes Research).
Shares Publish the disagreement between trial doctors and independent reviewers for every trial, Validate real-world progression endpoints so pragmatic trials can use them, An independent programme that validates surrogate endpoints, setting by setting, POLARIX: swapping vincristine for the antibody-drug conjugate polatuzumab vedotin in first-line treatment of diffuse large B-cell lymphoma.
Shares Use a blood test at six weeks to decide whether to keep going, RELATIVITY-047: relatlimab plus nivolumab, the first LAG-3 checkpoint combination, in untreated advanced melanoma, RUBY: dostarlimab with chemotherapy for advanced or recurrent endometrial cancer, HARMONi-2: ivonescimab, a PD-1 x VEGF bispecific, beats pembrolizumab head-to-head in PD-L1-positive lung cancer.
Shares Seamless phase 2/3 with pre-registered go rules as the default for new agents, Require a randomised phase 2 before any phase 3, Validate real-world progression endpoints so pragmatic trials can use them, AI-assisted central imaging reads to cut endpoint cost and variability.
Shares Time to progression (TTP) and time to next treatment (TTNT), Clinical benefit response (the gemcitabine trial endpoint), Primary, secondary and co-primary endpoints, Endpoint.