Selective Safety Data Collection in Clinical Studies of Oncology Drugs for Marketing Approval in the United States
DOI:
https://doi.org/10.21423/JRS-V05N02P029Keywords:
oncology drug, safety data collection, Food and Drug Administration, guidance for industry, clinical study, adverse eventAbstract
Optimization of data collection is a key issue in clinical studies of oncology drugs because it affects the workload and financial burden of the clinical infrastructure. Focusing on oncology drugs, which produce many low-grade as well as serious adverse reactions, we investigated the use of selective safety data collection in the pivotal clinical studies for marketing approval in the United States.Drug labels were examined to find clinical studies that evaluated adverse events with limited data, and found ten drugs approved between 2004 and 2015 whose pivotal studies used selective rather than comprehensive safety data collection. Only three were in accordance with the 2001 FDA Guidance for Industry, "Cancer Drug and Biological Products -- Clinical Data in Marketing Applications", which suggests such selectivity when targeting a similar population to the initial approval. Two selective studies were applied to a drug's initial approval. Three adopted the Guidance criteria for safety data collection of only noting grade 4-5 hematologic and 3-5 non-hematologic toxicities.
No major problems caused by this approach were found in the description of medical reviews, approval letters and post-marketing revisions to the boxed warnings on labels issued by the FDA. Selective safety data collection can be an efficient approach to streamlining the procedure of clinical studies and should be considered for use in pivotal clinical studies for oncology drugs.
https://doi.org/10.21423/jrs-v05n02p036 (DOI assigned 3/11/2019)
References
Abrams, J., Erwin, R., Fyfe, G., & Schilsky, R. L. (2010). Data Submission Standards and Evidence Requirements. The Oncologist, 15, 448-491.
Alberts, S. R., Sargent, D. J., Nair, S., Mahoney, M. R., Mooney, M., Thibodeau, S. N., Smyrk, T. C., Sinicrope, F. A., Chan, E., Gill, S., Kahlenberg, M. S., Shields, A. F., Quesenberry, J. T., Webb, T. A., Farr Jr., G. H., Pockaj, B. A., Grothey, A., & Goldberg, R. M. (2012). Effect of oxaliplatin, fluorouracil, and leucovorin with or without cetuximab on survival among patients with resected stage III colon cancer: a randomized trial. JAMA, 307, 1383-1393.
Avramis, V. I., Sencer, S., Periclou, A. P., Sather, H., Bostrom, B. C., Cohen, L. J., Ettinger, A. G., Ettinger, L. J., Franklin, J., Gaynon, P.S., Hilden, J. M., Labge, B., Majlessipour, F., Mathew, P., Needle, M., Neglia, J., Reaman, G., Holcenberg, J. S., & Stork, L. (2002). A randomized comparison of native Escherichia coli asparaginase and polyethylene glycol conjugated asparaginase for treatment of children with newly diagnosed standard-risk acute lymphoblastic leukemia: a Children's Cancer Group study. Blood, 99, 1986-1894.
Council for International Organizations of Medical Sciences (CIOMS). (2005). Management of Safety Information from Clinical Trials: Report of CIOMS Working Group VI. Retrieved from http://cioms.ch/shop/wp-content/uploads/2017/01/Mgment_Safety_Info.pdf
Giantonio, B. J., Catalano, P. J., Meropol, N. J., O'Dwyer, P. J., Mitchell, E. P., Alberts, S. R., Schwartz, M. A., & Benson III, A. B. (2007). Bevacizumab in combination with oxaliplatin, fluorouracil, and leucovorin (FOLFOX4) for previously treated metastatic colorectal cancer: results from the Eastern Cooperative Oncology Group Study E3200. Journal of Clinical Oncology, 25, 1539-1544.
Hurwitz, H., Fehrenbacher, L., Novotny, W., Cartwright, T., Hainsworth, J., Heim, W., Berlin, J., Baron, A., Griffing, S., Holmgren, E., Ferrara, N., Fyfe, G., Rogers, B., Ross, R., & Kabbinavar, F. (2004). Bevacizumab plus Irinotecan, Fluorouracil, and Leucovorin for Metastatic Colorectal Cancer. New England Journal of Medicine, 350, 2335-2342.
Kaiser, L. D., Melemed, A. S., Preston, A. J., Chaudri-Ross, H. A., Niedzwiecki, D., Fyfe, G., Gough, J., Bushnell, W. D., Stephens, C. L., Mace, M. K., Abrams, J. S., & Schilsky, R. (2010). Optimizing Collection of Adverse Event Data in Cancer Clinical Trials Supporting Supplemental Indications. Journal of Clinical Oncology, 28, 5046-5053.
Mahoney, M. R., Sargent, D. J., O'Connell, M. J., Goldber, R. M., Schaefer, P., & Buckner, J. C. (2005). Dealing With a Deluge of Data: An Assessment of Adverse Event Data on North Central Cancer Treatment Group Trials. Journal of Clinical Oncology, 23, 9275-9281.
Miller, K., Wang, M., Gralow, J., Dickler, M., Cobleigh, M., Perez, E. A., Shenkier, T., Cella, D., & Davidson, N. E. (2007). Paclitaxel plus Bevacizumab versus Paclitaxel Alone for Metastatic Breast Cancer. New England Journal of Medicine, 357, 2666-2676.
O'Leary, E., Seow, H., Julian, J., Levine, M., & Pond, G. R. (2013). Data collection in cancer clinical trials: Too much of a good thing? Clinical Trials, 10, 624-632.
Pujade-Lauraine, E., Hilpert, F., Weber, B., Reuss, A., Poveda, A., Kristensen, G., Sorio, R., Vergote, I., Witteveen, P., Bamias, A., Pereira, D., Wimberger, P., Oaknin, A., Mirza, M. R., Follana, P., Bollag, D., & Ray-Coquard, I. (2014). Bevacizumab Combined With Chemotherapy for Platinum-Resistant Recurrent Ovarian Cancer: The AURELIA Open-Lab Randomized Phase III Trial. Journal of Oncology, 32, 1302-1308.
Pujade-Lauraine, E., Hilpert, F., Weber, B., Reuss, A., Poveda, A., Kristensen, G., Sorio, R., Vergote, I., Witteveen, P., Bamias, A., Pereira, D., Wimberger, P., Oaknin, A., Mirza, M. R., Follana, P., Bollag, D., & Ray-Coquard, I. (2016). Bevacizumab Combined With Chemotherapy for Platinum-Resistant Recurrent Ovarian Cancer: The AURELIA Open-Lab Randomized Phase III Trial [Protocol]. Retrieved July 2, 2017 from http://ascopubs.org/doi/suppl/10.1200/jco.2013.51.4489/suppl_file/Protocol_JCO.2013.51.4489.pdf
Rico-Villademoros, F., Hernando, T., Sanz, J-L, Lopez-Alonso, A., Salamanca, O., Camps, C., & Rosel, R. (2004). The role of the clinical research coordinator--data manager--in oncology clinical trials. BMC Medical Research Methodology, 4:6.
Robak, T., Solal-Celigny, P., Warzocha, K., Loscertales, J., Catalano, J., Afanasiev, B. V., Larratt, L., Geisler, C. H., Montillo, M., Zyuzgin, I., Ganly, P. S., Dartigeas, C., Rosta, A., Maurer, J., Mendila, M., Saville, M. W., Valente, N., Wenger, M. K., & Moiseevm, S. I. (2010). Rituximab plus fludarabine and cyclophosphamide prolongs progression-free survival compared with fludarabine and cyclophosphamide alone in previously treated chronic lymphocytic leukemia. Journal of Clinical Oncology, 28, 1756-1765.
Roche, K., Paul, N., Smuck, B., Whitehead, M., Zee, B., Pater, J., Hiatt, M., & Walker, H. (2002). Factors Affecting Workload of Cancer Clinical Trials: Results of a Multicenter Study of the National Cancer Institute of Canada Clinical Trials. Journal of Clinical Oncology, 20, 545-556.
Romond, E. H., Perez, E. A., Bryant, J., Suman, V. J., Geyer, C. E., Davidson, N. E., Tan-Chiu, E., Martino, S., Paik, S., Kaufman, P. A., Swain, S. M., Pisansky, T. M., Fehrenbacher, L., Kutteh, L. A., Vogel, V. G., Visscher, M. D., Lingle, W. L., Klein, P. M., Ingle, J. N., & Wolmark, N. (2005). Trastuzumab plus Adjuvant Chemotherapy for Operable HER2-Positive Breast Cancer. New England Journal of Medicine, 353, 1673-1684.
Salles, G., Seymore, J. F., Offner, F., Lopez-Guillermo, A., Belada, D., Xerri, L., Feugier, P., Bouabdallah, R., Catalano, J. V., Brice, P., Caballero, D., Haioun, C., Pedersen, L. M., Delmer, A., Simpson, D., Leppa, S., Soubeyran, P., Hagenbeek, A., Casanovas, O., Intragumtornchai, T., Ferme, C., Gomes da Silva, M., Sebban, C., Lister, A., Estell, J. A., Milone, G., Sonet, A., Mendila, M., Coiffier, B., & Tilly, H. (2011). Rituximab maintenance for 2 years in patients with high tumour burden follicular lymphoma responding to rituximab plus chemotherapy (PRIMA): a phase 3, randomised controlled trial. Lancet, 377, 42-51.
Sandler, A., Gray, R., Perry, M. C., Brahmer, J., Schiller, J. H., Dowalti, A., Lilenbaum, R., & Johnson, D. H. (2006). Paclitaxel–Carboplatin Alone or with Bevacizumab for Non–Small-Cell Lung Cancer. New England Journal of Medicine, 355, 2542-2550.
Sargent, D. J., & George, S. L. (2010). Clinical Trials Data Collection: When Less Is More. Journal of Clinical Oncology, 28, 5019-5021.
Schilsky, R. L. (n.d.). Data collection and optimization in cancer clinical trials seeking sNDA/sBLA [Presentation].
Schilsky, R. (2011, June). Data Collection and Optimization in Cancer Clinical Trials. Presented at 2011 Annual Meeting of the American Society of Clinical Oncology, Chicago, IL. Retrieved from http://meetinglibrary.asco.org/record/64953/video
Sekine, N., Ohashi, Y., & Aruga, A., (2016). Applicability of Selective Data Collection to Cancer Clinical Studies for Supplemental Marketing Approvals: Frequency of Adverse Reactions Observed During Supplemental Approval Compared With First Approval. Therapeutic Innovation & Regulatory Science, 50, 472-479.
U.S. Food and Drug Administration. (n.d.). Drugs@FDA: FDA Approved Drug Products [Drug Database]. Retrieved November 23, 2016 from https://www.accessdata.fda.gov/scripts/cder/daf/
U.S. Food and Drug Administration. (2001). Guidance for Industry: Cancer Drug and Biological Products -- Clinical Data in Marketing Applications. Retrieved from https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071323.pdf
U.S. Food and Drug Administration. (2004, February). Approval Package for Avastin (bevacizumab). Retrieved July 2, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/nda/2004/STN-125085_Avastin_Approv.pdf
U.S. Food and Drug Administration. (2006, June). Approval Package for Avastin (bevacizumab). Retrieved July 2, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2006/125085s074LTR.pdf
U.S. Food and Drug Administration. (2006, June). Approval Package for Avastin (bevacizumab). Retrieved July 2, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/bla/2006/125085_0074_avastin.pdf
U.S. Food and Drug Administration. (2006, July). Approval Package for Oncaspar (pegaspargase). Retrieved July 2, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2006/103411s5052ltr.pdf
U.S. Food and Drug Administration. (2006, October). Approval Package for Avastin (bevacizumab). Retrieved July 2, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2006/125085s085ltr.pdf
U.S. Food and Drug Administration. (2006, October). Approval Package for Avastin (bevacizumab). Retrieved July 2, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/bla/2006/125085_0085_avastin.pdf
U.S. Food and Drug Administration. (2006, November). Approval Package for Herceptin (trastuzumab). Retrieved July 2, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2006/103792%205150ltr.pdf
U.S. Food and Drug Administration. (2008, February). Approval Package for Avastin (bevacizumab). Retrieved July 2, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/nda/2008/125085_s091_avastin.pdf
U.S. Food and Drug Administration. (2008, February). Approval Package for Avastin (bevacizumab). Retrieved from https://www.accessdata.fda.gov/drugsatfda_docs/nda/2008/125085_s091_avastin.pdf
U.S. Food and Drug Administration. (2010, February). Approval Package for Rituxan (rituximab). Retrieved July 2, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2010/103705s5311ltr.pdf
U.S. Food and Drug Administration. (2011, January). Approval Package for Rituxan (rituximab). Retrieved July 2, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2011/103705s5332ltr.pdf
U.S. Food and Drug Administration. (2012). Draft Guidance for Industry: Determining the Extent of Safety Data Collection Needed in Late Stage Premarket and Postapproval Clinical Investigations. Retrieved November 20, 2014 from https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM291158.pdf
U.S. Food and Drug Administration. (2014, November). Approval Package for Avastin (bevacizumab). Retrieved July 2, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2014/125085Orig1s305ltr.pdf
U.S. Food and Drug Administration. (2014, April). Approval Package for Unituxin (dinutuximab). Retrieved July 2, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2015/125516Orig1s000ltr.pdf
U.S. Food and Drug Administration. (2015, March). Addendum to Clinical Review, Unituxin (dinutuximab). Retrieved July 2, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/nda/2015/125516Orig1s000MedR.pdf
U.S. Food and Drug Administration. (2016). Determining the Extent of Safety Data Collection Needed in Late Stage Premarket and Postapproval Clinical Investigations. Retrieved November 23, 2016 from https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM291158.pdf
Yu, A. L., Gilman, A. L., Ozkaynak, M. F., London, W. B., Kreissman, S. G., Chen, H. X., Smith, M., Anderson, B., Villablanca, J. G., Matthaw, K. K., Shimada, H., Grupp, S. A., Seeger, R., Reynolds, C. P., Buxton, A., Reisfeld, R. A., Gillies, S. D., Cohn, S. L., Maris, J. M., & Sondel, P. M. (2010). Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. New England Journal of Medicine, 363, 1324-1334.
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