Table 1 Publications with similar or overlapping research questions

Zeitoun et al. (2018) [28]

To characterize post-marketing trials of cancer drugs

EMA, FDA

2005–2010

Original and supplemental

Solid tumors and hematologic malignancies

x

x

x

Barnes and Amir (2017) [29]

To describe the efficacy, safety, tolerability, and price of new cancer drugs.

FDA

2005–2016

Not described/Unclear

Solid tumors only

x

x

Booth and Del Paggio (2017) [30]

To evaluate the value of novel drugs using the ESMO Magnitude of Clinical Benefit Scale and ASCO Value Framework.

FDA

2015–2016

Not described/Unclear

Selected solid tumors ^[a]

x

x

Brooks et al. (2017) [31]

To understand the consequences of delaying approval of novel drugs until data on overall survival is available

FDA

1952–2016

Original and supplemental

Ten most common solid tumors ^[b]

x

Davis et al. (2017) [11]

To determine the availability of data on overall survival and quality of life benefits of cancer drugs.

EMA

2009–2013

Original and supplemental

Solid tumors and hematologic malignancies

x

x

x

Grossmann et al. (2017) [32]

To investigate the extent of EMA-approved cancer drugs that meet the threshold for “meaningful clinical benefit”, defined by the framework.

EMA

2011–2016

Original and supplemental

Solid tumors and hematologic malignancies

x

x

Naci et al. (2017) [33]

To characterize preapproval and confirmatory clinical trials of drugs granted accelerated approval.

FDA

2009–2013

Original and supplemental

Solid tumors and hematologic malignancies ^[c]

x

x

Naci et al. (2017) [9]

To systematically evaluate the timing and characteristics of clinical trials of drugs receiving accelerated approval.

FDA

2000–2013

Original only

Any disease or medical condition

x

x

x

Pease et al. (2017) [34]

To characterize controlled studies for drugs approved based on limited evidence.

FDA

2005–2012

Original only

Any disease or medical condition ^[d]

x

x

x

Salas-Vega et al. (2017) [35]

To evaluate the comparative therapeutic value of all new cancer medicines.

EMA, FDA

2003–2013

Original only

Solid tumors and hematologic malignancies

x

Smith et al. (2017) [36]

To characterize the primary endpoints used to support FDA approvals for new drug or novel hematologic malignancies indications.

FDA

2002–2015

Original and supplemental

hematologic malignancies only

x

x

x

Tibau et al. (2017) [37]

To derive the clinically meaningful benefit for FDA-approved drugs using the ESMO Magnitude of Clinical Benefit Scale.

FDA

2006–2016

Original and supplemental

Solid tumors only

x

Grossmann and Wild (2016) [38]

To describe the knowledge about the clinical benefit of new cancer therapies at the time of approval.

EMA

2009–2016

Original and supplemental

Solid tumors and hematologic malignancies

x

Hoekman et al. (2015) [39]

To describe the marketing authorization of oncology medicines granted based on the conditional marketing authorization pathway

EMA

2006–2013

Original only

Solid tumors only

x

x

x

Kim and Prasad (2015) [7]

To describe how often cancer drugs are approved based on a surrogate endpoint, whether subsequent studies for these drugs are reported, and whether the drugs improve overall survival.

FDA

2008–2012

Not described/Unclear

Solid tumors and hematologic malignancies

x

x

Wang and Kesselheim (2015) [40]

To characterize the types of comparators and endpoints used in efficacy trials for approvals of supplemental indications, compared with the data supporting these drugs’ originally approved indications.

FDA

2005–2014

Supplemental only

Any disease or medical condition ^[e]

x

x

x

Winstone et al. (2015) [41]

To characterize the clinical trial evidence of orphan drugs.

EMA

2006–2014

Not described/Unclear

Solid tumors and hematologic malignancies^[f]

x

x

x

Downing et al. (2014) [6]

To characterize pivotal efficacy trials for newly approved novel therapeutic agents.

FDA

2005–2012

Original only

Any disease or medical condition ^[e]

x

x

x

Fojo et al. (2014) [42]

To determine the availability of data on overall survival and quality of life benefits of cancer drugs.

FDA

2002–2014

Not described/Unclear

Solid tumors only

Hartmann et al. (2013) [12]

To review the outcomes of marketing authorization applications for cancer drugs

EMA

2006–2011

Original only

Solid tumors and hematologic malignancies

x

x

x

Martell et al. (2013) [43]

To describe approval trends and characteristics.

FDA

1949–2011

Original and supplemental

Solid tumors and hematologic malignancies

x

x

x

Thomas et al. (2013) [44]

To describe pre- and post-approval availability of published comparative efficacy studies.

FDA

2000–2010

Original only

Any disease or medical condition ^[g]

x

x

Goldberg et al. (2011) [45]

To quantify the availability of comparative efficacy data for new molecular entities.

FDA

2000–2010

Original only

Any disease or medical condition

x

x

Johnson et al. (2011) [46]

To provide an overview of the regulatory history of accelerated approved oncology products.

FDA

1992–2010

Not described/Unclear

Solid tumors and hematologic malignancies^[c]

x

x

Kesselheim et al. (2011) [47]

To define characteristics of orphan cancer drugs and their pivotal clinical trials and to compare these with non-orphan drugs.

FDA

2004–2010

Not described/Unclear

Solid tumors and hematologic malignancies^[f]

x

x

x

Ocana and Tannock (2011) [48]

To determine if a difference in outcome between the experimental and control groups was detected that was equal to or greater than the value predefined in the protocol

FDA

2000–2010

Not described/Unclear

Solid tumors only

x

Sridhara et al. (2010) [13]

To conduct an overview of products that were reviewed by the FDA’s Office of Hematology and Oncology Products for marketing approval and the regulatory actions taken during July 2005 to December 2007.

FDA

2005–2007

Original and supplemental

Solid tumors and hematologic malignancies

x

x

x

Tsimberidou et al. (2009) [10]

To review the long-term safety and efficacy or cancer drugs approved without evidence from randomized trials.

FDA

1973–2006

Original only

Solid tumors and hematologic malignancies^[h]

x

x

x