行业对罕见亚型试验的支持.pdf

1、Industry Support of Rare Subtype TrialsSumanta K.Pal,MD,FASCOProfessor&Vice Chair of Academic AffairsCity of Hope Comprehensive Cancer CenterImagine the landscape in metastatic clear cell renal cell carcinoma before doublet therapies were introduced.The waterfall plots on the left represent 3 phase

2、I clinical trials.Should any of these regimens have supplanted sunitinib ahead of phase III evidence?No.Phase III clinical trials have helped us contextualize appropriate use of each of these regimens.ArgumentFallacyWhat are the arguments against phase III trials for non-clear cell renal cell carcin

3、oma?We already have approvals in clear cell renal cell carcinoma!Clearly,non-clear cell subtypes have a distinct biology.Enrollment is too challenging in non-clear cell RCC studies.Phase III trials in non-clear cell RCC have completed accrual.Single arm phase II data is very compelling.How many time

4、s have we been burned by carefully selected single-arm studies?ArgumentFallacyWhat are the arguments against phase III trials for non-clear cell renal cell carcinoma?We already have approvals in clear cell renal cell carcinoma!Clearly,non-clear cell subtypes have a distinct biology.Reference:Marchet

5、ti et al Int J Mol Sci 2021How many times have we been burned by carefully selected single-arm studies?Single arm phase II data is very compelling.ArgumentFallacyWhat are the arguments against phase III trials for non-clear cell renal cell carcinoma?Reference:Kuo et al Nat Rev Clin Oncol 2024How man

6、y times have we been burned by carefully selected single-arm studies?Single arm phase II data is very compelling.ArgumentFallacyWhat are the arguments against phase III trials for non-clear cell renal cell carcinoma?Reference:Kuo et al Nat Rev Clin Oncol 2024How many times have we been burned by car