The urgent need for effective cancer treatments, particularly for advanced and relapsed
cases in which standard therapies are inadequate, has spurred the development of …

Importance Single-arm trials have allowed for transformative therapies to be made available
to patients expeditiously. However, using single-arm trials to support drug approval presents …

Background Comparative studies often cannot be conducted for cancer types with a small
patient population. We reviewed the efficacy evaluations of new drug approvals in Japan …

Background Single-arm trials (SATs) can provide an invaluable opportunity to speed up
cancer drug development and approval, typically for drugs with dramatic activity in small …

Patients with advanced malignancies have high-unmet medical needs, which bring requests
for early availability of new innovative treatments. Calls for expedited trial formats and rapid …

The traditional drug development paradigm, consisting of sequential phases and
randomized studies, has been challenged in oncology and hemato‐oncology. In the …

Single-arm phase II trials can provide compelling results that facilitate the approval of a new
therapy. Designing and interpreting single-arm studies based on four principles—instinct …

e13572 Background: Improved understanding of the underlying biology of cancer has led to
a paradigm shift in cancer drug development and has paved the way for many products to …

Background In recent years, an increasing number of anticancer drugs have been approved
based on the results of a single-arm trial (SAT). The magnitude of the objective response …

PURPOSE Two-stage single-arm designs have historically been the most common design
used in phase II oncology. They remain a mainstay today, particularly for trials in rare …