Here's an example of a P2 cancer drug with positive results getting approval.
https://www.yahoo.com/news/astrazeneca-daiich...30062.html
https://www.astrazeneca.com/media-centre/pres...02019.html
They enrolled over 250 patients (estimated to be 99 in US) and took time to determine PFS and OS rates (due to no secondary endpoints like CTC).
Quote:Patients on trastuzumab deruxtecan, also known as DS-8201, who had already undergone roughly six prior treatment courses, were saw no further progression for a median of 16.4 months.
https://www.yahoo.com/news/astrazeneca-daiich...30062.html
Quote:Trastuzumab deruxtecan was previously granted US FDA Breakthrough Therapy Designation and Fast Track designation. The BLA is based on the combination of data from the Phase I trial published in The Lancet Oncology and the pivotal Phase II DESTINY-Breast01 trial.1 The response rate observed in DESTINY-Breast01, as assessed by an independent review committee, validated the clinical activity observed in the Phase I trial.
Quote:The Prescription Drug User Fee Act (PDUFA) date for trastuzumab deruxtecan, a HER2-targeting antibody drug conjugate (ADC) and potential new medicine for the treatment of HER2-positive metastatic breast cancer, is set for the second quarter of 2020.
Quote:DESTINY-Breast01 is a pivotal Phase II, open-label, global, multicentre, two-part trial evaluating the safety and efficacy of trastuzumab deruxtecan in patients with HER2-positive unresectable and/or metastatic breast cancer previously treated with trastuzumab emtansine. The primary endpoint of the trial is objective response rate, as determined by independent central review. Secondary objectives include duration of response, disease control rate, clinical benefit rate, progression-free survival and overall survival. Enrolment into DESTINY-Breast01 was completed in September 2018 with 253 patients at more than 100 sites across North America, Europe, Japan and other countries in Asia.
The safety and tolerability profile of trastuzumab deruxtecan in DESTINY-Breast01 was consistent with the Phase I trial data published in The Lancet Oncology, in which the most common adverse events (≥30 percent, any grade) included nausea, decreased appetite, vomiting, alopecia, fatigue, anaemia, diarrhoea and constipation. Cases of drug-related interstitial lung disease and pneumonitis, including grade 5 events, have also been reported in the clinical development programme.
https://www.astrazeneca.com/media-centre/pres...02019.html
They enrolled over 250 patients (estimated to be 99 in US) and took time to determine PFS and OS rates (due to no secondary endpoints like CTC).
Quote:Secondary Outcome Measures :
Duration of Response (DoR) [ Time Frame: within 24 months ]
Disease Control Rate (DCR) [ Time Frame: within 24 months ]
Progression Free Survival (PFS) [ Time Frame: within 24 months ]
PFS is defined as the time from randomization until objective tumor progression or death, whichever occurs first.
Time to Response based on central review [ Time Frame: within 24 months ]
Overall Survival (OS) [ Time Frame: within 24 months ]
Overall survival is defined as the time from randomization until death from any cause and is measured in the intent-to-treat population.
ORR [ Time Frame: within 24 months ]
ORR, as assessed by the investigator based on RECIST Version 1.1.
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