메뉴 건너뛰기




Volumn 353, Issue , 2016, Pages

Letting post-marketing bridge the evidence gap: The case of orphan drugs

Author keywords

[No Author keywords available]

Indexed keywords

ANAGRELIDE; CIXUTUMUMAB; CLADRIBINE; HYDROXYUREA; IBUPROFEN; INDOMETACIN; MITOTANE; ORPHAN DRUG; PARACETAMOL; PHOTOFRIN; PLACEBO; ZINC ACETATE; ANTINEOPLASTIC AGENT; ANTINEOPLASTIC HORMONE AGONISTS AND ANTAGONISTS; ANTITHROMBOCYTIC AGENT; NONSTEROID ANTIINFLAMMATORY AGENT; PHOTOFRIN II; QUINAZOLINE DERIVATIVE;

EID: 84976610769     PISSN: 09598146     EISSN: 17561833     Source Type: Journal    
DOI: 10.1136/bmj.i2978     Document Type: Article
Times cited : (25)

References (42)
  • 1
    • 0003187521 scopus 로고    scopus 로고
    • Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products
    • EC. 2000
    • EC. 2000 Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. Off J Eur Communities 2000;L18:1.
    • (2000) Off J Eur Communities , vol.L18 , pp. 1
  • 2
    • 84876743099 scopus 로고    scopus 로고
    • Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU
    • pmid:23090701
    • Joppi R, Bertele' V, Garattini S. Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. Eur J Clin Pharmacol 2013;69:1009-24. doi:10.1007/s00228-012-1423-2. pmid:23090701.
    • (2013) Eur J Clin Pharmacol , vol.69 , pp. 1009-1024
    • Joppi, R.1    Bertele, V.2    Garattini, S.3
  • 3
    • 33644905861 scopus 로고    scopus 로고
    • Orphan drug development is progressing too slowly
    • 16487232
    • Joppi R, Bertele V, Garattini S. Orphan drug development is progressing too slowly. Br J Clin Pharmacol 2006;61:355-60. doi:10.1111/j.1365-2125.2006.02579.x. pmid:16487232.
    • (2006) Br J Clin Pharmacol , vol.61 , pp. 355-360
    • Joppi, R.1    Bertele, V.2    Garattini, S.3
  • 4
    • 65349108095 scopus 로고    scopus 로고
    • Orphan drug development is not taking off
    • pmid:19552743
    • Joppi R, Bertele V, Garattini S. Orphan drug development is not taking off. Br J Clin Pharmacol 2009;67:494-502. doi:10.1111/j.1365-2125.2009.03369.x. pmid:19552743.
    • (2009) Br J Clin Pharmacol , vol.67 , pp. 494-502
    • Joppi, R.1    Bertele, V.2    Garattini, S.3
  • 6
    • 21444434751 scopus 로고    scopus 로고
    • Hydroxyurea compared with anagrelide in high-risk essential thrombocythemia
    • United Kingdom Medical Research Council Primary Thrombocythemia 1 Study pmid:16000354
    • Harrison CN, Campbell PJ, Buck G, et al. United Kingdom Medical Research Council Primary Thrombocythemia 1 Study. Hydroxyurea compared with anagrelide in high-risk essential thrombocythemia. N Engl J Med 2005;353:33-45. doi:10.1056/NEJMoa043800 pmid:16000354.
    • (2005) N Engl J Med , vol.353 , pp. 33-45
    • Harrison, C.N.1    Campbell, P.J.2    Buck, G.3
  • 7
    • 38749101974 scopus 로고    scopus 로고
    • Effect of anagrelide on platelet coagulant function in patients with essential thrombocythemia
    • Pmid18057866
    • Cacciola RR, Di Francesco E, Pezzella F, Tibullo D, Giustolisi R, Cacciola E. Effect of anagrelide on platelet coagulant function in patients with essential thrombocythemia. Acta Haematol 2007;118:215-8. doi:10.1159/000111776. pmid:18057866.
    • (2007) Acta Haematol , vol.118 , pp. 215-218
    • Cacciola, R.R.1    Di Francesco, E.2    Pezzella, F.3    Tibullo, D.4    Giustolisi, R.5    Cacciola, E.6
  • 8
    • 84876515240 scopus 로고    scopus 로고
    • Anagrelide compared with hydroxyurea in WHO-classified essential thrombocythemia: The ANAHYDRET Study, a randomized controlled trial
    • ANAHYDRET Study Group
    • Gisslinger H, Gotic M, Holowiecki J, et al. ANAHYDRET Study Group. Anagrelide compared with hydroxyurea in WHO-classified essential thrombocythemia: the ANAHYDRET Study, a randomized controlled trial. Blood 2013;121:1720-8. doi:10.1182/blood-2012-07-443770 pmid:23315161.
    • (2013) Blood , vol.121 , pp. 1720-1728
    • Gisslinger, H.1    Gotic, M.2    Holowiecki, J.3
  • 9
    • 84892669231 scopus 로고    scopus 로고
    • Characterization of different regimens for initiating anagrelide in patients with essential thrombocythemia who are intolerant or refractory to their current cytoreductive therapy: Results from the multicenter FOX study of 177 patients in France
    • FOX study investigators
    • Rey J, Viallard JF, Keddad K, Smith J, Wilde P, Kiladjian JJ. FOX study investigators. Characterization of different regimens for initiating anagrelide in patients with essential thrombocythemia who are intolerant or refractory to their current cytoreductive therapy: results from the multicenter FOX study of 177 patients in France. Eur J Haematol 2014;92:127-36. doi:10.1111/ejh.12210. pmid:24118452.
    • (2014) Eur J Haematol , vol.92 , pp. 127-136
    • Rey, J.1    Viallard, J.F.2    Keddad, K.3    Smith, J.4    Wilde, P.5    Kiladjian, J.J.6
  • 10
    • 77249118383 scopus 로고    scopus 로고
    • Anagrelide for the treatment of thrombocythaemia in daily clinical practice: A post-marketing observational survey on efficacy and safety performed in Germany
    • Schmitz S, Stauch M, Schlag R. Anagrelide for the treatment of thrombocythaemia in daily clinical practice: a post-marketing observational survey on efficacy and safety performed in Germany. Onkologie 2010;33:39-44. doi:10.1159/000264611. pmid:20164660.
    • (2010) Onkologie , vol.33 , pp. 39-44
    • Schmitz, S.1    Stauch, M.2    Schlag, R.3
  • 11
    • 84976620072 scopus 로고    scopus 로고
    • Efficacy and safety of anagrelide in the treatment of essential thrombocythemia: Data from an international registry
    • Steurer MGH, Penka M, Sormann S, et al. Efficacy and safety of anagrelide in the treatment of essential thrombocythemia: data from an international registry. Onkologie 2011;34:206-7.
    • (2011) Onkologie , vol.34 , pp. 206-207
    • Steurer, M.G.H.1    Penka, M.2    Sormann, S.3
  • 12
    • 84872413434 scopus 로고    scopus 로고
    • Cytoreductive treatment patterns for essential thrombocythemia in Europe Analysis of 3643 patients in the EXELS study
    • Besses C, Kiladjian JJ, Griesshammer M, et al. Cytoreductive treatment patterns for essential thrombocythemia in Europe. Analysis of 3643 patients in the EXELS study. Leuk Res 2013;37:162-8. doi:10.1016/j.leukres.2012.11.004 pmid:23199894.
    • (2013) Leuk Res , vol.37 , pp. 162-168
    • Besses, C.1    Kiladjian, J.J.2    Griesshammer, M.3
  • 13
    • 62149092192 scopus 로고    scopus 로고
    • Pharmacokinetics, bioequivalence, tolerability, and effects on platelet counts of two formulations of anagrelide in healthy volunteers and patients with thrombocythemia associated with chronic myeloproliferation
    • Petrides PE, Gisslinger H, Steurer M, et al. Pharmacokinetics, bioequivalence, tolerability, and effects on platelet counts of two formulations of anagrelide in healthy volunteers and patients with thrombocythemia associated with chronic myeloproliferation. Clin Ther 2009;31:386-98. doi:10.1016/j.clinthera.2009.02.008 pmid:19302911.
    • (2009) Clin Ther , vol.31 , pp. 386-398
    • Petrides, P.E.1    Gisslinger, H.2    Steurer, M.3
  • 15
    • 77950396626 scopus 로고    scopus 로고
    • Randomized trial of daily versus weekly administration of 2-chlorodeoxyadenosine in patients with hairy cell leukemia: A multicenter phase III trial (SAKK 32/98)
    • Zenhaüsern R, Schmitz SF, Solenthaler M, et al. Randomized trial of daily versus weekly administration of 2-chlorodeoxyadenosine in patients with hairy cell leukemia: a multicenter phase III trial (SAKK 32/98). Leuk Lymphoma 2009;50:1501-11. doi:10.1080/10428190903131755 pmid:19672771.
    • (2009) Leuk Lymphoma , vol.50 , pp. 1501-1511
    • Zenhaüsern, R.1    Schmitz, S.F.2    Solenthaler, M.3
  • 16
    • 34247339681 scopus 로고    scopus 로고
    • Cladribine in a weekly versus daily schedule for untreated active hairy cell leukemia: Final report from the Polish Adult Leukemia Group (PALG) of a prospective, randomized, multicenter trial
    • Robak T, Jamroziak K, Gora-Tybor J, et al. Cladribine in a weekly versus daily schedule for untreated active hairy cell leukemia: final report from the Polish Adult Leukemia Group (PALG) of a prospective, randomized, multicenter trial. Blood 2007;109:3672-5. doi:10.1182/blood-2006-08-042929 pmid:17209059.
    • (2007) Blood , vol.109 , pp. 3672-3675
    • Robak, T.1    Jamroziak, K.2    Gora-Tybor, J.3
  • 17
    • 80053649383 scopus 로고    scopus 로고
    • Phase 2 study of cladribine followed by rituximab in patients with hairy cell leukemia
    • pmid:21821712
    • Ravandi F, O'Brien S, Jorgensen J, et al. Phase 2 study of cladribine followed by rituximab in patients with hairy cell leukemia. Blood 2011;118:3818-23. doi:10.1182/blood-2011-04-351502 pmid:21821712.
    • (2011) Blood , vol.118 , pp. 3818-3823
    • Ravandi, F.1    O'Brien, S.2    Jorgensen, J.3
  • 19
    • 84873427525 scopus 로고    scopus 로고
    • Treatment of patent ductus arteriosus (PDA) using ibuprofen: Renal side-effects in VLBW and ELBW newborns
    • Bagnoli F, Rossetti A, Messina G, Mori A, Casucci M, Tomasini B. Treatment of patent ductus arteriosus (PDA) using ibuprofen: renal side-effects in VLBW and ELBW newborns. J Matern Fetal Neonatal Med 2013;26:423-9. doi:10.3109/14767058.2012.733775. pmid: 23057804.
    • (2013) J Matern Fetal Neonatal Med , vol.26 , pp. 423-429
    • Bagnoli, F.1    Rossetti, A.2    Messina, G.3    Mori, A.4    Casucci, M.5    Tomasini, B.6
  • 20
    • 84892413316 scopus 로고    scopus 로고
    • Comparison of oral paracetamol versus ibuprofen in premature infants with patent ductus arteriosus: A randomized controlled trial
    • Dang D, Wang D, Zhang C, Zhou W, Zhou Q, Wu H. Comparison of oral paracetamol versus ibuprofen in premature infants with patent ductus arteriosus: a randomized controlled trial. PLoS One 2013;8:e77888. doi:10.1371/journal.pone.0077888. pmid: 24223740.
    • (2013) PLoS One , vol.8 , pp. e77888
    • Dang, D.1    Wang, D.2    Zhang, C.3    Zhou, W.4    Zhou, Q.5    Wu, H.6
  • 21
    • 84894295157 scopus 로고    scopus 로고
    • Oral paracetamol versus oral ibuprofen in the management of patent ductus arteriosus in preterm infants: A randomized controlled trial
    • pmid:24359938
    • Oncel MY, Yurttutan S, Erdeve O, et al. Oral paracetamol versus oral ibuprofen in the management of patent ductus arteriosus in preterm infants: a randomized controlled trial. J Pediatr 2014;164:510-4.e1. doi:10.1016/j.jpeds.2013.11.008 pmid:24359938.
    • (2014) J Pediatr , vol.164 , pp. 510-514e1
    • Oncel, M.Y.1    Yurttutan, S.2    Erdeve, O.3
  • 22
    • 84898486317 scopus 로고    scopus 로고
    • Continuous infusion of ibuprofen for treatment of patent ductus arteriosus in very low birth weight infants
    • Lago P, Salvadori S, Opocher F, Ricato S, Chiandetti L, Frigo AC. Continuous infusion of ibuprofen for treatment of patent ductus arteriosus in very low birth weight infants. Neonatology 2014;105:46-54. doi:10.1159/000355679. pmid:24281435.
    • (2014) Neonatology , vol.105 , pp. 46-54
    • Lago, P.1    Salvadori, S.2    Opocher, F.3    Ricato, S.4    Chiandetti, L.5    Frigo, A.C.6
  • 23
    • 84902548392 scopus 로고    scopus 로고
    • Comparison of oral ibuprofen with oral indomethacin for PDA closure in Indian preterm neonates: A randomized controlled trial
    • pmid:24435507
    • Yadav S, Agarwal S, Maria A, et al. Comparison of oral ibuprofen with oral indomethacin for PDA closure in Indian preterm neonates: a randomized controlled trial. Pediatr Cardiol 2014;35:824-30. doi:10.1007/s00246-014-0861-2 pmid:24435507.
    • (2014) Pediatr Cardiol , vol.35 , pp. 824-830
    • Yadav, S.1    Agarwal, S.2    Maria, A.3
  • 24
    • 84857958293 scopus 로고    scopus 로고
    • Oral ibuprofen versus intravenous ibuprofen or intravenous indomethacin for the treatment of patent ductus arteriosus in preterm infants: A systematic review and meta-analysis
    • Neumann R, Schulzke SM, Bührer C. Oral ibuprofen versus intravenous ibuprofen or intravenous indomethacin for the treatment of patent ductus arteriosus in preterm infants: a systematic review and meta-analysis. Neonatology 2012;102:9-15. doi:10.1159/000335332. pmid:22414850.
    • (2012) Neonatology , vol.102 , pp. 9-15
    • Neumann, R.1    Schulzke, S.M.2    Bührer, C.3
  • 25
    • 84886842665 scopus 로고    scopus 로고
    • Ibuprofen for the treatment of patent ductus arteriosus in preterm and/or low birth weight infants
    • pmid:23633310
    • Ohlsson A, Walia R, Shah SS. Ibuprofen for the treatment of patent ductus arteriosus in preterm and/or low birth weight infants. Cochrane Database Syst Rev 2013;4:CD003481.pmid:23633310.
    • (2013) Cochrane Database Syst Rev , vol.4 , pp. CD003481
    • Ohlsson, A.1    Walia, R.2    Shah, S.S.3
  • 26
    • 84885950748 scopus 로고    scopus 로고
    • Impact of oral versus intravenous ibuprofen on neurodevelopmental outcome: A randomized controlled parallel study
    • Eras Z, Gokmen T, Erdeve O, Ozyurt BM, Saridas B, Dilmen U. Impact of oral versus intravenous ibuprofen on neurodevelopmental outcome: a randomized controlled parallel study. Am J Perinatol 2013;30:857-62. doi:10.1055/s-0033-1333667. pmid:23359230.
    • (2013) Am J Perinatol , vol.30 , pp. 857-862
    • Eras, Z.1    Gokmen, T.2    Erdeve, O.3    Ozyurt, B.M.4    Saridas, B.5    Dilmen, U.6
  • 28
    • 84861888600 scopus 로고    scopus 로고
    • FIRM-ACT Study Group. Combination chemotherapy in advanced adrenocortical carcinoma
    • Fassnacht M, Terzolo M, Allolio B, et al. FIRM-ACT Study Group. Combination chemotherapy in advanced adrenocortical carcinoma. N Engl J Med 2012;366:2189-97. doi:10.1056/NEJMoa1200966 pmid:22551107.
    • (2012) N Engl J Med , vol.366 , pp. 2189-2197
    • Fassnacht, M.1    Terzolo, M.2    Allolio, B.3
  • 29
    • 84904194311 scopus 로고    scopus 로고
    • The combination of insulin-like growth factor receptor 1 (IGF1R) antibody cixutumumab and mitotane as a first-line therapy for patients with recurrent/metastatic adrenocortical carcinoma: A multi-institutional NCI-sponsored trial
    • Lerario AM, Worden FP, Ramm CA, et al. The combination of insulin-like growth factor receptor 1 (IGF1R) antibody cixutumumab and mitotane as a first-line therapy for patients with recurrent/metastatic adrenocortical carcinoma: a multi-institutional NCI-sponsored trial. Horm Cancer 2014;5:232-9. doi:10.1007/s12672-014-0182-1. pmid:24849545.
    • (2014) Horm Cancer , vol.5 , pp. 232-239
    • Lerario, A.M.1    Worden, F.P.2    Ramm, C.A.3
  • 30
    • 26944468635 scopus 로고    scopus 로고
    • Etoposide, doxorubicin and cisplatin plus mitotane in the treatment of advanced adrenocortical carcinoma: A large prospective phase II trial
    • Berruti A, Terzolo M, Sperone P, et al. Etoposide, doxorubicin and cisplatin plus mitotane in the treatment of advanced adrenocortical carcinoma: a large prospective phase II trial. Endocr Relat Cancer 2005;12:657-66. doi:10.1677/erc.1.01025. pmid:16172198.
    • (2005) Endocr Relat Cancer , vol.12 , pp. 657-666
    • Berruti, A.1    Terzolo, M.2    Sperone, P.3
  • 31
    • 77957579373 scopus 로고    scopus 로고
    • German ACC Registry Group. Improved survival in patients with stage II adrenocortical carcinoma followed up prospectively by specialized centers
    • Fassnacht M, Johanssen S, Fenske W, et al. German ACC Registry Group. Improved survival in patients with stage II adrenocortical carcinoma followed up prospectively by specialized centers. J Clin Endocrinol Metab 2010;95:4925-32. doi:10.1210/jc.2010-0803. pmid:20668036.
    • (2010) J Clin Endocrinol Metab , vol.95 , pp. 4925-4932
    • Fassnacht, M.1    Johanssen, S.2    Fenske, W.3
  • 34
    • 22144466565 scopus 로고    scopus 로고
    • Endoscopic ablation of dysplastic Barrett's oesophagus comparing argon plasma coagulation and photodynamic therapy: A randomized prospective trial assessing efficacy and cost-effectiveness
    • pmid:16118910.
    • Ragunath K, Krasner N, Raman VS, Haqqani MT, Phillips CJ, Cheung I. Endoscopic ablation of dysplastic Barrett's oesophagus comparing argon plasma coagulation and photodynamic therapy: a randomized prospective trial assessing efficacy and cost-effectiveness. Scand J Gastroenterol 2005;40:750-8. doi:10.1080/00365520510015737. pmid:16118910.
    • (2005) Scand J Gastroenterol , vol.40 , pp. 750-758
    • Ragunath, K.1    Krasner, N.2    Raman, V.S.3    Haqqani, M.T.4    Phillips, C.J.5    Cheung, I.6
  • 35
    • 84879489944 scopus 로고    scopus 로고
    • A randomised controlled trial of ALA vs Photofrin photodynamic therapy for high-grade dysplasia arising in Barrett's oesophagus
    • Dunn JM, Mackenzie GD, Banks MR, et al. A randomised controlled trial of ALA vs. Photofrin photodynamic therapy for high-grade dysplasia arising in Barrett's oesophagus. Lasers Med Sci 2013;28:707-15. doi:10.1007/s10103-012-1132-1 pmid:22699800.
    • (2013) Lasers Med Sci , vol.28 , pp. 707-715
    • Dunn, J.M.1    MacKenzie, G.D.2    Banks, M.R.3
  • 36
    • 80052074340 scopus 로고    scopus 로고
    • Photodynamic therapy (PDT) using HPPH for the treatment of precancerous lesions associated with Barrett's esophagus
    • Nava HR, Allamaneni SS, Dougherty TJ, et al. Photodynamic therapy (PDT) using HPPH for the treatment of precancerous lesions associated with Barrett's esophagus. Lasers Surg Med 2011;43:705-12. doi:10.1002/lsm.21112. pmid:22057498.
    • (2011) Lasers Surg Med , vol.43 , pp. 705-712
    • Nava, H.R.1    Allamaneni, S.S.2    Dougherty, T.J.3
  • 37
    • 84994190078 scopus 로고    scopus 로고
    • Sa1828 Comparison of endotherapies for the treatment of Barrett's dysplasia and neoplasia: What are the ideal modalities
    • Waseem J, David BJQ, Massimo R, et al. Sa1828 Comparison of endotherapies for the treatment of Barrett's dysplasia and neoplasia: what are the ideal modalities? Gastroenterology 2014;146(Suppl 1):S305-6.
    • (2014) Gastroenterology , vol.146 , pp. S305-S306
    • Waseem, J.1    David, B.J.Q.2    Massimo, R.3
  • 38
    • 84978615085 scopus 로고    scopus 로고
    • EMA
    • EMA. European Medicines Agency assessment report for zinc. 2015. http://www.ema. europa.eu/ema/index.jsp?curl=pages/medicines/human/orphans/2009/11/human-orphan-000142.jsp&mid=WC0b01ac058001d12b
    • (2015) European Medicines Agency Assessment Report for Zinc
  • 39
    • 16244386202 scopus 로고    scopus 로고
    • Neurologically presenting Wilson's disease: Epidemiology, pathophysiology and treatment
    • Brewer GJ. Neurologically presenting Wilson's disease: epidemiology, pathophysiology and treatment. CNS Drugs 2005;19:185-92. doi:10.2165/00023210-200519030-00001. pmid:15740174.
    • (2005) CNS Drugs , vol.19 , pp. 185-192
    • Brewer, G.J.1
  • 40
    • 84943661661 scopus 로고    scopus 로고
    • Approvals of drugs with uncertain benefit-risk profiles in Europe
    • Banzi R, Gerardi C, Bertele' V, Garattini S. Approvals of drugs with uncertain benefit-risk profiles in Europe. Eur J Intern Med 2015;26:572-84. doi:10.1016/j.ejim.2015.08.008. pmid: 26342723.
    • (2015) Eur J Intern Med , vol.26 , pp. 572-584
    • Banzi, R.1    Gerardi, C.2    Bertele, V.3    Garattini, S.4
  • 41
    • 23444460612 scopus 로고    scopus 로고
    • Regulation (EC) 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency
    • EC
    • EC. Regulation (EC) 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency. Off J Eur Union 2004;L136:1.
    • (2004) Off J Eur Union , vol.L136 , pp. 1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.