Tobias Polak

The ethics of expanded access research 243 12 ♠ 21. De Bono JS, Oudard S, Ozguroglu M, et al. Prednisone plus cabazitaxel or mitoxantrone for metastatic castration-resistant prostate cancer progressing after docetaxel treatment: a randomised open-label trial. The Lancet. 2010;376(9747):1147-1154. doi:10.1016/ S0140-6736(10)61389-X 22. Wissing MD, Van Oort IM, Gerritsen WR, et al. Final quality of life and safety data for patients with metastatic castration-resistant prostate cancer treated with cabazitaxel in the UK Early Access Programme (EAP) (NCT01254279). Asia Pac J Clin Oncol. 2013;13(6):975-983. doi:10.1097/CAD.0b013e32835a56bc 23. Wissing MD, van Oort IM, Gerritsen WR, et al. Cabazitaxel in patients with metastatic castration-resistant prostate cancer: results of a compassionate use program in the Netherlands. Clin Genitourin Cancer. 2013;11(3):238-250.e1. 24. Di Lorenzo G, D’aniello C, Buonerba C, et al. Pegfilgrastim and cabazitaxel in prostate cancer patients. Anticancer Drugs. 2013;24(1):84-89. doi:10.1097/CAD.0b013e32835a56bc 25. Heidenreich A, Scholz HJ, Rogenhofer S, et al. Cabazitaxel plus prednisone for metastatic castration-resistant prostate cancer progressing after docetaxel: results from the German compassionate-use programme. Eur Urol. 2013;63(6):977-982. doi:10.1016/j. eururo.2012.08.058 26. Heidenreich A, Bracarda S, Mason M, et al. Safety of cabazitaxel in senior adults with metastatic castration-resistant prostate cancer: Results of the European compassionate-use programme. Eur J Cancer. 2014;50(6):10901099. doi:10.1016/j.ejca.2014.01.006 27. Bracarda S, Gernone A, Gasparro D, et al. Realworld cabazitaxel safety: the Italian earlyaccess program in metastatic castrationresistant prostate cancer. Future Oncol. 2014;10(6):975-983. 28. Castellano D, Antón Aparicio LM, Esteban E, et al. Cabazitaxel for metastatic castrationresistant prostate cancer: safety data from the Spanish expanded access program. Expert Opin Drug Saf. 2014;13(9):1165-1173. doi:10.1517/1 4740338.2014.939583 29. Lee SH, Bang YJ, Mainwaring P, et al. Sunitinib in metastatic renal cell carcinoma: an ethnic Asian subpopulation analysis for safety and efficacy. Asia Pac J Clin Oncol. 2014;10(3):237-245. 30. Parente P, Ng S, Parnis F, Guminski A, Gurney H. Cabazitaxel in patients with metastatic castration-resistant prostate cancer: safety and quality of life data from the Australian early access program. Asia Pac J Clin Oncol. 2017;13(6):391-399. 31. Yokom DW, Stewart J, Alimohamed NS, et al. Prognostic and predictive clinical factors in patients with metastatic castration-resistant prostate cancer treated with cabazitaxel. Can Urol Assoc J. 2018;12(8):E365-e372. 32. Berger ML, Sox H, Willke RJ, et al. Good practices for real-world data studies of treatment and/or comparative effectiveness: Recommendations from the joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making. Pharmacoepidemiol Drug Saf. 2017;26(9):1033-1039. doi:10.1002/pds.4297 33. Polak TB, van Rosmalen J, Uyl - de Groot CA. Expanded access as a source of real-world data: An overview of FDA and EMA approvals. Br J Clin Pharmacol. Published online March 22, 2020. doi:10.1111/bcp.14284 34. Ison G, Beaver JA, McGuinn WDJ, et al. FDA Approval: Uridine Triacetate for the Treatment of Patients Following Fluorouracil or Capecitabine Overdose or Exhibiting Early-Onset Severe Toxicities Following Administration of These Drugs. Clin Cancer Res Off J Am Assoc Cancer Res. 2016;22(18):4545-4549. doi:10.1158/10780432.CCR-16-0638 35. Balasubramanian G, Morampudi S, Chhabra P, Gowda A, Zomorodi B. An overview of compassionate use programs in the european union member states. Intractable Rare Dis Res. 2016;5(4):244-254. doi:10.5582/ irdr.2016.01054 36. Polak TB, Cucchi DGJ, van Rosmalen J, Uyl-de Groot CA. The DRUG Access Protocol: access inequality and European harmonisation. Lancet Oncol. 2022;23(5):e202. doi:10.1016/S14702045(22)00098-5

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