TABLE 2.
Published Surrogate Correlation Studies for Traditional Approvals Based on a Surrogate
| Tumor type | Approved drug (year) | Basis of traditional approval | Studies assessed level 2 surrogacy | Studies assessed level 1 surrogacy |
|---|---|---|---|---|
| Basal cell carcinoma | Vismodegib (2012) | ORR | - | - |
|
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| Breast cancer | Pertuzumab (2012) | PFS | Ciccarese et al,26 2007 (abstract): rOS vs DFS=0.77 Matsubara et al,27 2011: HROS vs PFS=0.93, P=.31 |
Hackshaw et al,28 2005: rHR OS vs TTP=0.75 Burzykowski et al,29 2008: rHR OS vs PFS=0.48 Miksad et al,30 2008: rHR OS vs PFS=0.7 for anthracyclines Miksad et al,30 2008: rHR OS vs PFS=0.59 for taxanes Ng et al,31 2008: rdiff OS vs DFS=0.62 Sherrill et al,32 2008: rHR OS vs TTP=0.55 Beauchemin et al,33 2014: rdiff OS vs PFS/TTP=0.43 |
|
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| Chronic lymphocytic leukemia | Idelalisib (2014) Ofatumumab (2014) Obinutuzumab (2013) |
PFS | - | - |
|
| ||||
| Chronic myeloid leukemia | Bosutinib (2012) | MCyR Complete hematology response Overall hematologic response |
Rosti et al,21 2003: rOS vs MCyR=0.66 Oriana et al,20 2013: P=.02 at 5 y, CCyR vs no CCyR (no report on coefficient) Jain et al,19 2013: P=.01 at 3 y, CCyR vs no CCyR |
- |
|
| ||||
| Cutaneous T-cell lymphoma | Romidepsin (2009) | ORR | - | - |
|
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| Differentiated thyroid cancer | Sorafenib (2013) | PFS | - | - |
|
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| Follicular lymphoma | Rituximab (2011) | PFS | - | - |
|
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| Gastroenteropancreatic neuroendocrine tumor | Lanreotide (2014) Sunitinib (2011) for pancreatic NET Everolimus (2011) for pancreatic NET |
PFS | - | Singh et al,45 2014: rHR OS vs PFS/TTP=0.17 |
|
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| Giant cell tumor | Denosumab (2013) | ORR and DOR | - | - |
|
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| Mantle cell lymphoma | Lenalidomide (2013) | ORR and DOR | - | - |
|
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| Medullary thyroid carcinoma | Cabozantinib (2012) Vandetenib (2011) |
PFS | - | - |
|
| ||||
| Melanoma | Trametinib (2013) Dabrafenib (2013) Peginterferon-alpha 2b (2013) |
PFS | - | Flaherty et al,46 2014: rHR OS vs PFS=0.89 |
|
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| Non-small cell lung cancer | Nab paclitaxel (2012) | ORR | Splinter,34 1991: rOS vs ORR=0.57 Paesmans et al,35 1997: rOS vs ORR=2.20, P<.0001 Sekine et al,36 1998: rOS vs ORR=0.504 Bruzzi et al,37 2007 (abstract): OS HRresponse vs no response=0.50, P<.001 Tsujino et al,38 2009: rOS vs ORR=0.258 Mandrekar et al,39 2010: OS HRresponse vs no response=0.66, P=.009 Li et al,40 2012: rOS vs ORR=0.91 |
Blumenthal et al,41 2015: rHR OS vs ORR=0.3 Johnson et al,42 2006: rdiff OS vs ORR=0.4 |
| Crizotinib (2014) Afatinib (2014) Erlotinib (2013) |
PFS | Hayashi et al,47 2012: rPFS vs OS=0.43 Sekine et al,48 1999: rPFS vs OS=0.80 |
Blumenthal et al,41 2015: rHR OS vs PFS=0.28 Suzuki et al,49 2014: rHR OS vs PFS=0.41 Hayashi et al,50 2013: rdiff OS vs PFS=0.29 Hotta et al,51 2013: rHR OS vs PFS=0.37 Mauguen et al,52 2013: rHR OS vs DFS=0.96 Hotta et al,53 2009: rOS ratio vs TTP ratio=0.57 |
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| Ovarian cancer | Olaparib (2014) | ORR | Rose et al,44 2010: rOS vs ORR=0.56 | - |
|
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| Ovarian/fallopian/primary peritoneal cancer | Bevacizumab (2014) | PFS | Rose et al,44 2010: rOS vs PFS=0.66 | - |
|
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| Renal cell carcinoma | Axitinib (2012) Pazopanib (2009) Bevacizumab (2009) Everolimus (2009) |
PFS | Halabi et al,54 2014: HROS vs progression at 6 mo=2.8, P<.0001 Heng et al,55 2011: rOS vs PFS=0.66 |
Bria et al,56 2015: r12 mo OS vs 3 mo PFS=0.82, r12 mo OS vs 6 mo PFS=0.85 Johnson et al,57 2015: rdiff OS vs PFS=0.7 Petrelli and Barni,58 2013: rdiff OS vs PFS=0.36 Delea et al,59 2012: rdiff OS vs PFS/TTP=0.53 rHR OS vs PFS/TTP=0.79 |
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| Soft-tissue sarcoma | Pazopanib (2012) | PFS | Penel et al,43 2013: rOS vs PFS=0.4 | - |
CCyR =complete cytogenetic response; CRi =complete response with incomplete blood count recovery; DFS =disease-free survival; DOR =duration of response; HR =hazard ratio; MaHR =major hematologic response; MCyR =major cytogenetic response; MMR =major molecular response; MRD =minimal residual disease; NET =neuroendrocine tumor; ORR =objective response rate, OS =overall survival; pCR =pathologic complete response; PFS =progression-free survival; TTP =time to progression.