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Supplementary Online Content

Tibau A, Ocana A, Anguera G, et al. Oncologic Drugs Advisory Committee recommendations and approval of drugs by the US Food and Drug Administration. JAMA Oncol. Published online March 3, 2016. doi:10.1001/jamaoncol.2015.6479.

eTable 1. Characteristics of included meetings.

eTable 2. List of meetings, drugs discussed and citations to published trials.

eTable 3. Breakdown of conflicts of interest with designated monetary values for advisory committee members and voting consultants.

This supplementary material has been provided by the authors to give readers additional information about their work.

© 2016 American Medical Association. All rights reserved.

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Online Supplement

eTable 1. Characteristics of included meetings.

eTable 2. List of meetings, drugs discussed and citations to published trials.

eTable 3. Breakdown of Conflicts of Interest With Designated Monetary Values for Advisory Committee Members and Voting Consultants.

© 2016 American Medical Association. All rights reserved.

Downloaded From: https://jamanetwork.com/ on 09/26/2021 eTable 1.

Product Meetin ODAC FDA Approv Type of Approv Indication( Num Primary Stud Trial g date Recomme Appro al submiss al s) ber endpoin y size nded val Date iona Type of t Desi Approval trials gn 03/16/2 Yes Yes 04/20/2 S RA First-line Multi TTP RCT 683 hydrochloride 000 000 metastatic ple CRC 03/16/2 No No N/A I RAg First-line Multi PFS RCT 420 000 metastatic ple CRC Gemtuzumab 03/17/2 Yes Yes 05/17/2 I AA Second- Multi RR 3 59; Ozogamicin 000 000 line AML ple SAT 25; 20 Histamine 12/13/2 No No N/A I RAg Metastatic Singl OS RCT 129h hydrochloride 000 melanoma e (305) to the liver Letrozole 12/13/2 Yes Yes 01/10/2 S RA First-line Multi TTP RCT 916 000 001 advanced ple BC 12/14/2 Yes Yes 05/07/2 I AA Third-line Multi RR SAT 93 000 001 therapy for ple B- CLL /Epin 09/10/2 No No N/A I AAg Recurrent Multi RR and RCT 92 ephrine 001 or ple Patient refractory Benefit SCCHN Ibritumomab 09/11/2 Yes Yes 02/19/2 I AA Relapsed Multi RR SAT 54 tiuxetan 001 002 or ple refractory low-grade or follicular in refractory patients No Yes AAf Relapsed Multi RR RCT 143 or ple refractory low-grade or follicular lymphoma in patients who have not failed rituximab Yes Yes AA Relapsed Multi RR RCT 13 or ple refractory low-grade or follicular lymphoma in transforme d patients 3

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Carmustine 12/6/20 Yes Yes 02/25/2 S RA Recurrent Multi OS RCT 207h 01 003 glioblasto ple (240) ma multiforme Zoledronic 01/31/2 Yes Yes 02/22/2 S RA Multiple Multi SRE RCT 1,648 acid 002 002 myeloma ple and BC bone metastases Prostate RCT 643 cancer bone metastases Solid RCT 773 tumors bone metastases 09/ Yes Yes 05/05/2 I AA Third-line Multi RR SAT 142h 24/200 003 NSCLC ple (216) 2 12/17/2 Yes Yes 06/27/2 I AA Refractory Multi RR SAT 61 002 003 or relapsed ple low-grade follicular lymphoma not treated with rituximab Bicalutamide 12/18/2 No No N/A S RAg Adjuvant Multi TTP RCT 3603 002 therapy ple 3292 prostate 1218 cancer Immediate treatment of localized non- metastatic in patients for whom therapy of curative intent is not indicated 05/03/2 No No N/A I RAg First-line Multi OS RCT 771 sodium 004 advanced ple melanoma 09/06/2 No No N/A I AAg Relapsed Singl RR RCT 241 006 or e refractory CLL 05/03/2 No No N/A I RAg Adjunctive Multi OS RCT 115h sodium 004 therapy to ple (538) whole 4

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brain radiation for BC brain metastasis 07/27/2 Yes Yes 08/19/2 S AA Second- Singl RR RCT 571 004 004 line NSCLC e No No N/A RAg OS 12/01/2 Yes Yes 12/28/2 I AA Refractory Multi RR SAT 49 004 004 or relapsed ple paediatric ALL No No N/A AAg Refractory Multi RR SAT 35 or relapsed ple paediatric AML 09/01/2 No No N/A S AAg AML Multi RR SAT 112h 009 ple (116) 12/01/2 No No N/A I AAg Third-line Multi RR SAT 119 sulfat 004 aggressive ple liposome NHL 03/21/2 Yes Yes 08/09/2 I AA Third-line Multi RR SAT 65 012 012 adult Ph- ple ALL 05/05/2 No No N/A I RAg Newly Singl RR SAT 136 005 diagnosed e AAg poor-risk Singl myeloid e leukemia 09/13/2 No No N/A I RAg HRPC with Multi TTP RCT 690h 005 metastasis ple (809) to bone 09/13/2 Yes Yes 11/02/2 S RA Locally Singl OS RCT 569 005 005 advanced e metastatic pancreatic cancer 12/16/2 No Yes 04/16/2 Sb RA Maintenan Singl PFS RCT 889 009 010 ce therapy e NSCLC 09/14/2 Yes Yes 12/27/2 I RA Myelodyspl Multi RR SAT 148 005 005 astic ple syndromes 09/14/2 Yes Yes 10/28/2 I AA T-cell ALL Multi RR SAT 149 005 005 or T-cell ple lymphoblas tic lymphoma in paediatric population T-cell ALL Multi 39 or T-cell ple lymphoblas tic

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lymphoma adult population 03/13/2 No Yes 07/14/2 Sb RA Second- Multi PFS RCT 356 hydrochloride 006 006 line ple ovarian cancer 06/02/2 Yes Yes 06/28/2 I AA Second- Multi RR 3 86; 006 006 line CML ple SAT 107; 74 RA Second- Multi SAT 39h line Ph- ALL ple (78) Dalteparin 09/06/2 Yes Yes 01/05/2 S RA Extended Singl Time to RCT 676 sodium 006 007 treatment e first of VTE sympto matic recurren t VTE Beclamethaso 05/09/2 No No N/A I RAg Treatment Multi Time to RCT 129 ne 007 of graft vs. ple treatme dipropionate host nt disease failure (GvHD) involving the gastrointes tinal tract Mifamurtide 05/09/2 No No N/A I RAg Newly Singl DFS RCT 678 007 diagnosed e resectable high grade osteosarco ma following surgical resection 07/24/2 No No N/A I AAg HRPC that Multi PFS and RCT 950 capsules 007 has failed ple OS prior chemother apy 12/05/2 No Yes 02/22/2 Sb AA Locally Multi PFS RCT 722 007 008 recurrent ple or metastatic HER2- BC 03/31/2 Yes Yes 05/05/2 S AA Previously Multi RR SAT 167 009 009 treated ple glioblasto ma multiforme 07/20/2 No No N/A S Convers Added to Multi PFS RCT 736 010 ions ple from AA in locally to RAg recurrent or 6

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metastatic HER2- BC Added to RCT 1237 , anthracycli nes, or capecitabin e in locally recurrent or metastatic HER2- BC Added to RCT 722 in locally recurrent or metastatic HER2- BC Raloxifine 07/24/2 Yes Yes 09/13/2 I RA Reduction Multi Incidenc RCT 1010 hydrochloride 007 007 in the risk ple e of 1 of invasive cancer BC in postmenop ausal women with osteoporos is Yes Yes Reduction Multi 1974 in the risk ple 7 of invasive BC in postmenop ausal women at high risk for BC 05/29/2 Yes Yes 10/26/2 I AA Refractory Multi RR SAT 59h(1 009 009 CLL ple 54) 07/15/2 No No N/A I RAg Relapsed Multi PFS RCT 672 009 ovarian ple cancer 07/15/2 No No N/A S RAg Locally Singl TTP RCT 751 liposome 009 advanced e or metastatic BC Laromustine 09/01/2 No No N/A I AAg Remission Multi RR SAT 85 009 induction ple AML

Pralatrexate 09/02/2 Yes Yes 09/24/2 I AA Relapsed Singl RR SAT 109 009 009 or e refractory

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PTCL

Romidepsin 09/02/2 Yes Yes 11/05/2 I RA Second- Multi RR SAT 96 009 009 line CTCL ple

Pazopanib 10/05/2 Yes Yes 10/19/2 I RA Advanced Multi PFS RCT 435 009 009 RCC ple

03/20/2 Yes Yes 04/26/2 S RA Second- Singl PFS RCT 369 012 012 line STS e

Peginterferon 10/05/2 Yes Yes 03/29/2 S RA Stage III Multi DFS RCT 1256 Alfa-2b 009 011 melanoma ple

Omacetaxine 03/22 No Yes 10/26/2 Id AA Third-line Singl RR SAT 66 mepesuccinate 2010 012 CML e 03/22/2 No No N/A I AAg Recurring Singl RR RCT 140 dimaleate 010 or e refractory aggressive NHL Dutasteride 12/01/2 No No N/A S RAg Reduction Singl Incidenc RCT 8231 010 in the risk e e of of prostate cancer cancer Finasteride 12/01/2 No No N/A S RAf Reduction Singl Incidenc RCT 1888 010 in the risk e e of 2 of prostate cancer cancer 12/02/2 Yes Yes 04/06/2 Ic RA Locally Multi PFS RCT 331 010 011 advanced ple or metastatic medullary thyroid cancer 04/12/2 Yes Yes 05/05/2 S RA Advanced Singl PFS RCT 410 011 011 pNET e 04/12/2 Yes Yes 05/20/2 S RA Advanced Singl PFS RCT 171 011 011 pNET e Brentuximab 07/14/2 Yes Yes 08/19/2 I AA Third-line Singl RR SAT 102 vedotin 011 011 HL e Second- Singl 58 line ALCL e 12/07/2 Yes Yes 01/27/2 I RA Advanced Singl PFS RCT 723 011 012 RCC e Denosumab 02/08/2 No No N/A S RAg HRPC at Multi BMFS RCT 1432 012 high risk ple for metastases 02/09/2 No No N/A S RAg AML in Multi OS RCT 485 012 adults 65 ple years of age or older Ridaforolimus 03/20/2 No No N/A I RAg Maintenan Singl PFS RCT 711 8

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012 ce therapy e metastatic STS or bone sarcoma Semuloparin 06/20/2 No No N/A I RAg Prophylaxis Singl Time-to- RCT 1674h sodium 012 of VTE e first (3212 occurren ) ce of VTE 06/20/2 Yes Yes 07/20/2 I AA Relapsed Multi RR SAT 266 012 012 and ple refractory 05/02/2 No No N/A I RAg Ocular Multi PFS RCT 93h 013 melanoma ple (186) metastatic to the liver 05/02/2 No No N/A I RAg Advanced Singl PFS RCT 517 013 RCC e Pertuzumab 09/12/2 Yes Yes 09/30/2 S AA Neoadjuva Multi RR RCT 417 013 013 nt HER2- ple positive locally advanced or early stage BC 06/25/2 No Yes 12/19/2 Ib AA Maintenan Singl PFS RCT 136h 014 014 ce therapy e (265) - sensitive relapsed ovarian cancer 11/06/2 No Yes 02/23/2 Ib RA Relapsed Multi PFS RCT 768 014 015 and ple refractory multiple myeloma

Abbreviations: ALL, acute lymphoblastic leukemia; AML, acute myelogenous leukemia; BMFS, bone metastases free survival; BC, ; CLL, chronic lymphocytic leukemia; CML, chronic myelocytic leukemia; CRC, colorectal cancer; CTCL, cutaneous T-cell lymphoma; DFS, Disease Free Survival; GvHD, graft vs. host disease; HL, Hodgkin lymphoma; HRPC, hormone refractory prostate cancer; LBL, lymphoblastic lymphoma; N/A, Not Applicable, NHL, non–Hodgkin lymphoma; NSCLC, non–small cell ; OS, overall survival; Ph, Philadelphia chromosome; PFS, progression-free survival, pNET, pancreatic neuroendocrine tumors; PTCL, peripheral T-cell lymphoma; RCC, renal (kidney) cell carcinoma; RCT, randomized; RR, response rate; SAT, single-arm trial; SCCHN, squamous cell carcinoma of the head and neck; SRE, skeletal-related event; STS, soft tissue sarcoma; TTP, time to progression; VTE, venous thromboembolism. aI, initial approval; S, supplemental approval. b ODAC did not recommend approval. c ODAC convened to discuss postmarketing safety studies. d ODAC did not recommend approval, and the drug was subsequently approved under a different new drug application. e AA, accelerated approval; RA, regular approval f ODAC did not recommend RA, and the drug was subsequently approved under AA g Proposed approval type, although the drug was not approved 9

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h Proposed approval based on Subgroup Analysis

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eTable 2.

Meeti ng Product name Primary Publication Date 16- Saltz et al. N Engl J Med Mar- Irinotecan hydrochloride injection 2000 00 16- de Gramont et al. J Clin Mar- Oxaliplatin Oncol 2010 00 17- Sievers et al. j Clin Oncol Mar- 2001 00 13- Agarwala et al. J Clin Oncol Dec- Histamine hydrochloride injection (1 mg/ml) 2002 00 13- Dec- Letrozole Mouridsen et al. J Clin 00 Oncol 2001 14- Dec- Alemtuzumab 00 Keating et al. Blood 2002 Wenig et al. Arch 10- Otolaryngol Head Neck Sep- Cisplatin/epinephrine Injectable Gel Surg 2002 and Werner et 01 al. Brit J Cancer 2002 11- Witzig et al. J Clin Oncol Sep- 2002 01 11- Witzig et al. J Clin Oncol Sep- Ibritumomab tiuxetan 2002 01 11- Witzig et al. J Clin Oncol Sep- Ibritumomab tiuxetan 2002 01 06- Westphal et al. Neurooncol Dec- 2003 01 06- No published data Dec- Irinotecan hydrochloride injection available 01 31- Zoledronic acid for injection Rosen et al. Cancer J 2001 Jan-02 31- Saad et al. J Natl Cancer Zoledronic acid for injection Jan-02 Inst 2002 11

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31- Rosen et al. J Clin Oncol Zoledronic acid for injection Jan-02 2003 24- Cellla et al. J Clin Oncol Sep- Gefitinib 2005 02 17- Kaminski et al. J Clin Oncol Dec- Tositumomab and 131Iodine 2001 02 18- Dec- Bicalutamide 02 See et al. J Urol 2002 18- Dec- Bicalutamide 02 See et al. J Urol 2002 03- May- RSR13 Suh et al. J Clin Oncol 2006 04 03- Bedikian et al. J Clin Oncol May- Oblimersen sodium 2006 04 27-Jul- Hanna et al. J Clin Oncol Pemetrexed 04 2004 27-Jul- Hanna et al. J Clin Oncol Pemetrexed 04 2004 01- Kantarjian et al. J Clin Dec- Clofarabine Oncol 2010 04 01- Kantarjian et al. J Clin Dec- Clofarabine Oncol 2010 04 01- Rodriguez et al. Cancer Dec- Vincristine sulfat liposome injection 2009 04 05- May- Tipifarnib Lancet et al. Blood 2007 05 05- May- Tipifarnib Lancet et al. Blood 2007 05 13- Moore et al. J Clon Oncol Sep- Erlotinib 2007 05 13- Sep- Atrasentan 05 Carducci et al. Cancer 2007 14- Lenalidomide List et al. N Engl J Med 12

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Sep- 2006 05 14- Berg et al. J Clin Oncol Sep- Nelarabine 2005 05 14- Sep- Nelarabine DeAngelo et al. Blood 2007 05 13- Pfisterer et al. J Clin Oncol Mar- Gemcitabine hydrochloride 2006 06 02- Apperley et al. J Clin Oncol Dasatinib Jun-06 2009 02- Dasatinib Ottmann et al. Blood 2007 Jun-06 06- O'Brien et al. J Clin Oncol Sep- Oblimersen Sodium 2007 06 06- Lee et al. N Engl J Med Sep- Dalteparin sodium 2003 06 09- Meyers et al. J Clin Oncol May- Mifamurtide 2005 07 09- May- Beclamethasone dipropionate Hockenbery et al. Blood 07 2007 10- Erythropoeisis-stimulating agents: Darbepoetin alfa, Epoetin alfa, May- N/A Epoetin alfa 07 24-Jul- Barrett-Connor et al. N Raloxifene hydrochloride 07 Engl J Med 2006 24-Jul- Raloxifene hydrochloride Vogel et al. JAMA 2006 07 24-Jul- Sternberg et al. J Clin Oncol Satraplatin capsules 07 2009 05- Miller et al. N Engl J Med Dec- Bevacizumab 2007 07 13- Darbepoetin alfa, Epoetin alfa, Epoetin alfa, and methoxy polyethylene Mar- N/A glycol-epoetin beta 08 31- Friedman et al. J Clin Oncol Mar- Bevacizumab 2009 09

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29- William et al. J Clin Oncol May- Ofatumumab 2010 09 15-Jul- Sparano et al. J Clin Oncol Doxorubicin Hcl liposome injection 09 2009 15-Jul- Monk et al. J Clin Oncol Trabectedin 09 2010 01- Schiller et al. J Clin Oncol Sep- Laromustine 2010 09 01- Kantarjian et al. J Clin Sep- Clofarabin Oncol 2010 09 02- Sep- Coiffier et al. J Clon Oncol 09 2012 02- O'Connor et al. J Clin Oncol Sep- 2011 09 05- Oct- Peginterferon Alfa-2b Eggermont et al. Lancet 09 2008 05- Sternberg et al. J Clin Oncol Oct- hydrochloride 2010 09 16- Cappuzzo et al. Lancet Dec- Erlotinib 2010 09 22- Mar- Omapro Cortes et al. Blood 2012 10 22- Mar- Pixantrone dimaleate Pettengell et al. Lancet 10 Oncol 2012 20-Jul- Miles et al. J Clin Oncol Bevacizumab 10 2010 20-Jul- Robert et al. J Clin Oncol Bevacizumab 10 2011 20-Jul- Miller et al. N Engl J Med Bevacizumab 10 2007 01- Andriole et al. N Engl J Med Dec- Dutasteride 2010 10 01- Thompson et al. N Engl J Dec- Finasteride Med 2003 10 14

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02- Wells et al. J Clin Oncol Dec- Vandetanib 2012 10 12- Yao et al. N Engl J Med Apr- Everolimus 2011 11 12- Raymond et al. N Engl J Apr- Sunitinib malate Med 2011 11 14-Jul- Younes et al. J Clin Oncol 11 2012 14-Jul- Brentuximab vedotin Pro et al. J Clin Oncol 2012 11 07- Dec- Axitinib Rini et al. Lancet 2011 11 08- Feb- Denosumab Smith et al. Lancet 2012 12 09- Kantarjian et al. J Clin Feb- Decitabine Oncol 2012 12 20- Demetri et al. J Clin Oncol Mar- Ridaforolimus 2013 12 20- Van der Graaf et al. Lancet Mar- Pazopanib hydrochloride 2012 12 21- O'Brien et al. J Clin Oncol Mar- Vincristine sulfate liposomes injection 2013 12 20- Carfilzomib Siegel et al. Blood 2012 Jun-12 20- Agnelli et al. N Engl J Med Semuloparin sodium injection Jun-12 2012 02- Motzer et al. J Clin Oncol May- Tivozanib 2012 13 02- May- Melphalan N/A 13 12- Sep- Pertuzumab Gianni et al. Lancet, 2012 13 25- Ledermann et al. Lancet Olaparib Jun-14 2012 15

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06- San-Miguel et al. Lancet Nov- Panobinostat capsules 2014 14

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eTable 3.

Conflict Type No. (%) b Total 194 (100) Investment ($ per firm) c 0- $5,000 22 (24) $5,001 - $25,000 41 (45) $25,001 - $50,000 21 (23) $50,001 - $100,000 5 (5) > $100,000 2 (2) Employer grant or contract ($ per year) d 0- $100,000 9 (64) $100,001 - $300,000 2 (14) > $300,000 3 (22) Paid Speaker ($ per year) 0 - $ 5000 1 (14) $ 5001 - $ 10 000 4 (57) $10 001 - $ 50 000 2 (29) Research ($ per year) 0 - $ 50000 6 (67) $ 50001 - $ 100 000 2 (22) $100 001 - $ 300 000 1 (1) Consulting ($ per year) e 0 - $ 10 000 64 (90) $10 001 - $ 50 000 6 (8) No amount details 1 (2) Lecturing / Honoraria ($ per year) $ 5001-10 000 2 (100)

a Individuals could have more than 1 type of conflict of interest. We did not include one member declaring one

conflict of interest not remunerated and members recused.

b Percentage of conflicts of this type.

c Including those shared with spouses.

d Including salary support to immediate family member from research grant.

e Including member of scientific or other advisory board or steering committee, blinded endpoint reviewer or

member of data safety monitoring board and Consultancy Competitor Personal/Immediate Family.

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