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Characteristics of Preapproval and Postapproval Studies for Drugs Granted Accelerated Approval by the US Food and Drug Administration

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Characteristics of Preapproval and Postapproval Studies for Drugs Granted Accelerated Approval by the US Food and Drug Administration Supplementary Online Content Naci H, Smalley KR, Kesselheim AS. Characteristics of preapproval and postapproval studies for drugs granted accelerated approval by the US Food and Drug Administration. JAMA. doi: 10.1001/jama.2017.9415 eTable 1. Key Data Limitations at the Time of Accelerated Approval eTable 2. Confirmatory Study Details, Status and Associated Regulatory Outcomes eReferences This supplementary material has been provided by the authors to give readers additional information about their work. © 2017 American Medical Association. All rights reserved. Downloaded From: https://jamanetwork.com/ on 09/29/2021 eTable 1. Key Data Limitations at the Time of Accelerated Approval Agent Excerpt from product label Bevacizumab No data available demonstrating improvement in disease-related symptoms or survival with [bevacizumab]. Pralatrexate Clinical benefit such as improvement in progression free survival or overall survival has not been demonstrated. Ofatumumab No data demonstrate an improvement in disease related symptoms or increased survival with [ofatumumab]. Lapatinib [Lapatinib] in combination with an aromatase inhibitor has not been compared to a trastuzumab-containing chemotherapy regimen for the treatment of metastatic breast cancer. Nilotinib The study is ongoing and further data will be required to determine long-term outcome. Dasatinib The trial is ongoing and further data will be required to determine long-term outcome. Everolimus Clinical benefit such as improvement in disease-related symptoms or increase in overall survival has not been demonstrated. Hydroxyprogesterone There are no controlled trials demonstrating a direct clinical benefit, such as caproate improvement in neonatal mortality and morbidity. Romidepsin Clinical benefit such as improvement in overall survival has not been demonstrated. Brentuximab vedotin There are no data available demonstrating improvement in patient reported outcomes or survival with [brentuximab vedotin]. Brentuximab vedotin There are no data available demonstrating improvement in patient reported outcomes or survival with [brentuximab vedotin]. Crizotinib There are no data available demonstrating improvement in patient reported outcomes or survival with [crizotinib]. Deferiprone There are no controlled trials demonstrating a direct treatment benefit, such as improvement in disease-related symptoms, functioning, or increased survival. Everolimus Further follow-up of patients is required to determine long-term outcomes. Carfilzomib Clinical benefit, such as improvement in survival or symptoms, has not been verified. Vincristine sulfate Clinical benefit such as improvement in overall survival has not been verified. liposome Omacetaxine There are no trials verifying an improvement in disease-related symptoms or increased survival with [omacetaxine mepesuccinate]. Ponatinib There are no trials verifying an improvement in disease-related symptoms or increased survival with [ponatinib]. Bedaquiline This indication is based on analysis of time to sputum culture conversion from two controlled Phase 2 trials in patients with pulmonary multidrug resistant tuberculosis. Deferasirox An improvement in survival or disease-related symptoms has not been established. Pomalidomide Clinical benefit, such as improvement in survival or symptoms, has not been verified. Idursulfase In patients 16 months to 5 years of age, no data are available to demonstrate improvement in disease-related symptoms or long term clinical outcome. Pertuzumab No data are available demonstrating improvement in event-free survival or overall survival. Ibrutinib An improvement in survival or disease-related symptoms has not been established. © 2017 American Medical Association. All rights reserved. Downloaded From: https://jamanetwork.com/ on 09/29/2021 eTable 2. Confirmatory Study Details, Status and Associated Regulatory Outcomes Agent Confirmatory study description (according to regulatory letters sent Status Regulatory outcome to sponsors) Bevacizumab To submit an efficacy supplement containing the final study report, Complete. Not available. including summary analyses and datasets and revised labeling based on the results of study AVF4396g/BO20990 entitled “A Randomized, FDA database: Double Blind, Placebo Controlled, Multicenter, Phase III Trial of “Submitted” (no further detail Bevacizumab, Temozolomide and Radiotherapy, Followed by provided) Bevacizumab and Temozolomide Versus Placebo, Temozolomide Followed by Placebo and Temozolomide in Patients with Newly ClinicalTrials.gov identifier: Diagnosed Glioblastoma,” which was accepted under a Request for NCT00943826 Special Protocol Assessment on December 29, 2008. Protocol submitted: October 31, 2008, Complete accrual: June 30, 2013, Complete study: June Trial results are published.1 30, 2015, Supplement submission: December 31, 2015 Pralatrexate 1547-1 A randomized trial of maintenance treatment with pralatrexate in Incomplete. Not available. previously untreated patients with PTCL who have demonstrated a Study has been terminated. response to CHOP or a CHOP-like regimen. Description of trial: This will be a Phase 3 multi-center, randomized clinical trial of sequential FDA database: FOLOTYN versus observation in patients with newly diagnosed “Ongoing” (no further detail aggressive peripheral T-cell lymphoma who have responded following provided) initial treatment with CHOP-based chemotherapy. The primary endpoint will be progression-free survival (PFS). The trial will also be sized to ClinicalTrials.gov: detect a realistic difference in survival. Patients will be enrolled prior to “Study has been terminated.” initiation of the CHOP-based regimen. Patients responding (CR or PR) ClinicalTrials.gov identifier: after CHOP-based treatment will then be randomized 2:1 to FOLOTYN NCT01420679 versus observation. The timetable you submitted on September 20, 2009, states that you will conduct this trial according to the following timetable: Final Protocol Submission Date: December 23, 2009 Trial Completion Date: December 31, 2016 Final Report Submission Date: June 30, 2017 Pralatrexate 1547-2 A randomized trial comparing pralatrexate in combination with Incomplete. Not available. systemic bexarotene versus systemic bexarotene alone in patients with Delayed by more than 12 cutaneous T-cell lymphoma (CTCL) who are refractory to at least one months. prior systemic therapy. Description of trial: This will be a Phase 3 multi- center, randomized clinical trial in patients with CTCL. The primary FDA database: endpoint will be progression-free survival (PFS). Response rate will be a “Thirty evaluable patients are © 2017 American Medical Association. All rights reserved. Downloaded From: https://jamanetwork.com/ on 09/29/2021 eTable 2. Confirmatory Study Details, Status and Associated Regulatory Outcomes Agent Confirmatory study description (according to regulatory letters sent Status Regulatory outcome to sponsors) secondary endpoint. Prior to initiation of the Phase 3 trial, a Phase 1 trial enrolled in the study and the will be conducted to determine the maximum tolerated dose (MTD) of study is ongoing. The Phase 1 the combination. The timetable you submitted on September 20, 2009, trial completion date (August 31, states that you will conduct this trial according to the following timetable: 2011) and the Phase 3 Protocol Protocol Submission Date for Phase 1 Trial: November 15, 2009 Phase 1 submission date (September 30, Trial Completion Date: August 31, 2011 Final Phase 3 Protocol 2011) have been missed.” Submission Date: September 30, 2011 Phase 3 Trial Completion Date: March 31, 2015 Phase 3 Trial Final Report Submission Date: September ClinicalTrials.gov identifier: 30, 2015 Could not be identified. (Non- randomized dose-finding component was completed. ClinicalTrials.gov identifier: NCT01134341) Ofatumumab To submit a final report for ongoing clinical trial OMB110911, entitled, Complete. FDA letter dated 17 April “A Phase III Open-label, Randomized, Multicenter Trial of Ofatumumab 2014 confirms fulfilment Added to Chlorambucil versus Chlorambucil Monotherapy in Previously FDA database: of commitments made Untreated Patients with Chronic Lymphocytic Leukemia” which is Could not be identified. under 21 CFR 601.41. intended to verify the clinical benefit of ofatumumab through demonstration of a clinically meaningful effect on progression-free ClinicalTrials.gov identifier: survival. The protocol for clinical trial OMB110911 was submitted to NCT00748189 FDA on October 24, 2008 and began patient accrual on December 22, 2008. We also acknowledge receipt of the amended protocol submitted Trial results are published.2 August 21, 2009. The timetable you submitted on October 6, 2009 states that you will conduct this trial according to the following milestones: Patient Accrual 50% Completed (222 patients) by August 30, 2010, Patient Accrual 75% Completed (333 patients) by March 30, 2011, Patient Accrual Completed by November 30, 2011, Trial Completion Date: by October 14, 2013, Final Report Submission: by June 30, 2014 © 2017 American Medical Association. All rights reserved. Downloaded From: https://jamanetwork.com/ on 09/29/2021 eTable 2. Confirmatory Study Details, Status and Associated Regulatory Outcomes Agent Confirmatory study description (according to regulatory letters sent Status Regulatory outcome to sponsors) Lapatinib 1586-1. A randomized trial comparing lapatinib in combination with Incomplete. Not available. trastuzumab and an aromatase inhibitor versus trastuzumab in
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