Tobias Polak

Chapter 8 176 61. Munk NE, Knudsen JS, Pottegård A, Witte DR, Thomsen RW. Differences Between Randomized Clinical Trial Participants and Real-World Empagliflozin Users and the Changes in Their Glycated Hemoglobin Levels. JAMA Netw Open. 2020;3(2):e1920949-e1920949. doi:10.1001/ jamanetworkopen.2019.20949 62. Bierer BE, Li R, Barnes M, Sim I. A Global, Neutral Platform for Sharing Trial Data. N Engl J Med. 2016;374(25):2411-2413. doi:10.1056/ NEJMp1605348 63. Villemur L, Cohignac L, Jeanbat V, Bouee S. PNS266 Collecting EARLY Access Real World Evidence DATA for a Successful Reimbursement Appraisal. Value Health. 2020;23:S685-S685. doi:10.1016/j.jval.2020.08.1710 64. Caplan A, Bateman-House A. Compassion for each individual’s own sake. Am J Bioeth AJOB. 2014;14(11):16-17. doi:10.1080/15265161.201 4.957622 65. Polak TB, Cucchi DGJ, van Rosmalen J, Uylde Groot CA, Darrow JJ. Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations. Front Pharmacol. 2022;13. doi:10.3389/ fphar.2022.913567 66. 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 67. Polak TB, Cucchi DG, van Rosmalen J, Uyl-de Groot CA. Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals. BMJ Open. 2022;12(1):e052186-e052186. doi:10.1136/bmjopen-2021-052186 68. Ouzzani M, Hammady H, Fedorowicz Z, Elmagarmid A. Rayyan—a web and mobile app for systematic reviews. Syst Rev. 2016;5(1):210. doi:10.1186/s13643-016-0384-4 69. van der Helm LH, Alhan C, Wijermans PW, et al. Platelet doubling after the first azacitidine cycle is a promising predictor for response in myelodysplastic syndromes (MDS), chronic myelomonocytic leukaemia (CMML) and acute myeloid leukaemia (AML) patients in the Dutch azacitidine compassionate named p. Br J Haematol. 2011;155(5):599-606. 70. Estey E, Hasserjian RP, Döhner H. Distinguishing AML from MDS: a fixed blast percentage may no longer be optimal. Blood. 2022;139(3):323-332. doi:10.1182/blood.2021011304 71. Duetz C, Cucchi DGJ, Polak TB, et al. The wider perspective: twenty years of clinical trials in myelodysplastic syndromes. Br J Haematol. Published online October 10, 2021. doi:10.1111/ bjh.17892 72. Azemar M, Djahansouzi S, Jäger E, et al. Regression of cutaneous tumor lesions in patients intratumorally injected with a recombinant single-chain antibody-toxin targeted to ErbB2/HER2. Breast Cancer Res Treat. 2003;82(3):155-164. 73. Di Lorenzo G, DʼAniello C, Buonerba C, et al. Pegfilgrastim and cabazitaxel in prostate cancer patients. Anticancer Drugs. 2013;24(1):84-89. 74. 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 75. 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. 76. 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 77. 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 78. 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

RkJQdWJsaXNoZXIy MTk4NDMw