Table 1.
Publications with similar or overlapping research questions
| Study characteristics | Characteristics described or analyzed | ||||||||
|---|---|---|---|---|---|---|---|---|---|
| (Reference, publication year) | Objective | Health authority | Time period | Approval type | Disease characteristics | Regulatory* | Trials | Endpoints | Effect sizes |
| 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 | 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 | 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 | 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 | 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 | 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 | x | |||
| 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 | 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 | 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 | x |
This list is based on a systematic search (Additional file 1) but not intended to be exhaustive, as some relevant articles were brought to our attention by experts and could not be found with our limited search approach. *For example, approval pathways such as accelerated approval or orphan-drug status. Other regulatory characteristics (such as approval times, approval probabilities, or availability of pediatric label information) are not considered here. Abbreviations: ASCO American Society of Clinical Oncology, EMA European Medicines Agency, ESMO European Society for Medical Oncology, FDA US Food and Drug Administration. [a] Limited to breast, lung, colorectal, or pancreatic cancers. [b] Limited to breast, colorectal, endometrial, gastric, liver, pancreatic, prostate and renal cancer as well as melanoma and non-small-cell lung cancer. [c] Limited to drugs approved under accelerated approval. [d] Includes any drug approved on the basis of a single pivotal trial, pivotal trials that used surrogate markers of disease as primary endpoints or both. [e] Includes any drug. [f] Limited to orphan drugs only. [g] Limited to therapeutic biologics only. [h] Limited to drugs approved without evidence from randomized trials