Review
Year 2023,
Volume: 43 Issue: 3, 261 - 268, 01.09.2023
Abstract
Son yıllarda yeni ilaç geliştirme çalışmaları kapsamında yapılan araştırmalarda, öksüz ilaçların çok önemli bir yerinin olduğu görülmektedir. Öksüz ilaçların geliştirilmesiyle, nadir hastalıkların tedavisinde kullanılacak yararlı yeni ilaçların elde edilmesi mümkün olabileceği gibi, etkinliği kanıtlanmış bu ilaçların, henüz tedavisi mümkün olmayan pek çok hastalığın tedavisi için de öncü olması beklenmektedir. Bu çalışmada, öksüz ilaçların, mevcut durumuna ilişkin araştırma ve değerlendirme yapılması amaçlanmıştır. Çalışma kapsamında yapılan literatür taramaları sonucunda, öksüz ilaçlar hakkında yaşanan son gelişmeler ve bu ilaçlarla ilgili araştırma-geliştirme çalışmalarının artmasını sağlayan yasa ve programlar detaylı olarak sunulmuştur. Öksüz ilaçlarla ilgili sunulan sayısal veriler, bu ilaçların yeni ilaç geliştirilmesindeki yerinin önemini göstermektedir.
Keywords
Supporting Institution
Bu çalışma herhangi bir proje desteği olmadan Çukurova Üniversitesi Eczacılık Fakültesi bünyesinde gerçekleştirilmiştir.
References
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- Pak Güre MD, İnce Ö: Examining of Health Policy in Rare Diseases in Terms of Historical Perspective in Turkey. Journal of International Health Sciences and Management 2021, 7(14): 62-71.
- Aronson JK: Rare diseases and orphan drugs. British Journal of Clinical Pharmacology 2006, 61(3):243-245.
- Dellve L, Samuelsson L, Tallborn A, Fasth A, Hallberg LRM: Stress and well-being among parents of children with rare diseases: a prospective intervention study. Journal of Advanced Nursing 2006, 53(4):392-402.
- Taruscio D, Capozzoli F, Frank C: Rare diseases and orphan drugs. Ann Ist Super Sanita 2011, 47(1):83-93.
- Orofino J, Soto J, Casado MA, Oyagüez I: Global Spending on Orphan Drugs in France, Germany, the UK, Italy and Spain during 2007. Applied Health Economics and Health Policy 2010, 8(5):301-315.
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- Franco P: Orphan drugs: the regulatory environment. Drug Discovery Today 2013, 18(3-4):163-172.
- Attwood MM, Rask-Andersen M, Schiöth HB: Orphan Drugs and Their Impact on Pharmaceutical Development. Trends in Pharmacological Sciences 2018, 39(6):525-535.
- Giannuzzi V, Conte R, Landi A, Ottomano SA, Bonifazi D, Baiardi P, Bonifazi F, Ceci A: Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen. Orphanet Journal of Rare Diseases 2017, 12(1).
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- Ürek D , Karaman S: Önemli Bir Halk Sağliği Sorunu Olarak Nadir Hastaliklar Ve Yetim Ilaçlar. Hacettepe Sağlık İdaresi Dergisi 2019, 22(4):863-878.
- Kılıç P, Koçkaya G, Yemşen Ö, Tan C, Öztunca FH, Aksungur P, Kerman S: Orphan drug regulations in Turkey. Journal of Pharmaceutical Health Services Research 2013, 4(3):151-153.
- Orphanet Türkiye websayfası giriş noktası.; 2022 March 12. Available from: http://www.orpha.net/national/TR-TR/index/orphanet-t%C3%BCrkiye/
- Advancing Health Through Innovation: New Drug Therapy Approvals 2021.; 2022 May 13. Available from: https://www.fda.gov/media/155227/download
- Miller KL, Fermaglich LJ, Maynard J: Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases. Orphanet Journal of Rare Diseases 2021, 16(1).
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- Hadjivasiliou A: Orphan Drug Report 2017. In., 4th edn: EvaluatePharma; 2017.
- Pomeranz K: Orphan Drug Report 2019. In. Edited by Urquhart L, 6th edn: EvaluatePharma; 2019.
- Orphan Drug Report 2022.; 2022 May 12. Available from: Orphan Drug 2022 Report | Evaluate
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Year 2023,
Volume: 43 Issue: 3, 261 - 268, 01.09.2023
Abstract
It has recently been observed that orphan drugs have a very important place in the researches carried out within the scope of new drug development studies. With the development of orphan drugs, it will be possible to obtain useful new drugs to be used in the treatment of rare diseases, and these drugs with proven effectiveness are expected to lead to the treatment of many diseases that are not yet curable. In this review study, it is aimed to research and evaluate the current status of orphan drugs. As a result of the literature review carried out within the scope of the study, the laws, and programs that encouraged research and development studies and the latest developments about orphan drugs are presented in detail. The numerical data presented on orphan drugs show the importance of these drugs in the development of new drugs.
Keywords
References
- Azie N, Vincent J: Rare Diseases: The Bane of Modern Society and the Quest for Cures. Clinical Pharmacology & Therapeutics 2012, 92(2):135-139.
- Wirtz VJ: Priority medicines for Europe and the World: setting a public-health-based medicines development agenda. Journal of Pharmaceutical Policy and Practice 2015, 8(S1).
- Anderson M, Elliott EJ, Zurynski YA: Australian families living with rare disease: experiences of diagnosis, health services use and needs for psychosocial support. Orphanet Journal of Rare Diseases 2013, 8(1).
- Pak Güre MD, İnce Ö: Examining of Health Policy in Rare Diseases in Terms of Historical Perspective in Turkey. Journal of International Health Sciences and Management 2021, 7(14): 62-71.
- Aronson JK: Rare diseases and orphan drugs. British Journal of Clinical Pharmacology 2006, 61(3):243-245.
- Dellve L, Samuelsson L, Tallborn A, Fasth A, Hallberg LRM: Stress and well-being among parents of children with rare diseases: a prospective intervention study. Journal of Advanced Nursing 2006, 53(4):392-402.
- Taruscio D, Capozzoli F, Frank C: Rare diseases and orphan drugs. Ann Ist Super Sanita 2011, 47(1):83-93.
- Orofino J, Soto J, Casado MA, Oyagüez I: Global Spending on Orphan Drugs in France, Germany, the UK, Italy and Spain during 2007. Applied Health Economics and Health Policy 2010, 8(5):301-315.
- Designating an Orphan Product: Drugs and Biological Products.; 2022 May 12. Available from: https://www.fda.gov/industry/developing-products-rare-diseases-conditions/designating-orphan-product-drugs-and-biological-products
- Drummond MF, Wilson DA, Kanavos P, Ubel P, Rovira J: Assessing the economic challenges posed by orphan drugs. International Journal of Technology Assessment in Health Care 2007, 23(1):36-42.
- Franco P: Orphan drugs: the regulatory environment. Drug Discovery Today 2013, 18(3-4):163-172.
- Attwood MM, Rask-Andersen M, Schiöth HB: Orphan Drugs and Their Impact on Pharmaceutical Development. Trends in Pharmacological Sciences 2018, 39(6):525-535.
- Giannuzzi V, Conte R, Landi A, Ottomano SA, Bonifazi D, Baiardi P, Bonifazi F, Ceci A: Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen. Orphanet Journal of Rare Diseases 2017, 12(1).
- Scott DL, Alder S, Usui E, Lui K: Orphan Drug Programs/Policies in Australia, Japan, and Canada. Drug Information Journal 2001, 35(1):1-16.
- O'Connor DJ: Orphan drug designation – Europe, the USA and Japan. Expert Opinion on Orphan Drugs 2013, 1(4):255-259.
- Çoban Aİ, Tunca AB, Ertan A, Güneş C, Akdere CG, Taştan C, Başbuğu C, Güngör D, Tunç DH, Özcan E, Öksüz E, Metin F, Yılmaz F, Gülden G, Ceylaner G, Dönmez G, Palacı H, Köse ME, Tarım M, Tekindal M, Çolak MY, Pak MD, Yarar O, Yücel O, İnce Ö, Tekin RN, Özden SA, Bülbül S, İper ST, Ceylaner S, Vatansever S, Malhan S, Soyer T, Kartal Y, Gülhan YB : Tüm Yönleriyle Nadir Hastalıklar: Nobel Akademik Yayıncılık; Ankara , Türkiye, 2019.
- Haffner ME, Whitley J, Moses M: Two decades of orphan product development. Nature Reviews Drug Discovery 2002, 1(10):821-825.
- Cheung RY, Cohen JC, Illingworth P: Orphan drug policies: implications for the United States, Canada, and developing countries. Health Law J 2004, 12:183-200.
- Wellman-Labadie O, Zhou Y: The US Orphan Drug Act: Rare disease research stimulator or commercial opportunity? Health Policy 2010, 95(2-3):216-228.
- Results for Approved Products.; 2022 May 12. Available from: https://www.accessdata.fda.gov/scripts/opdlisting/oopd/listResult.cfm
- Pryde DC, Groft SC. Chapter 1. Definitions, History and Regulatory Framework for Rare Diseases and Orphan Drugs. In: Pryde DC (eds), Orphan Drugs and Rare Diseases, Royal Society of Chemistry, Cambridge, UK. 2014: pp 3-31.
- Sakushima K, Takeda H, Aoi Y: Orphan drug designation and development in Japan: 25 years of experience and assessment. Nature Reviews Drug Discovery 2021, 20(12):893-894.
- Grogan P: Therapeutic Goods Administration Orphan Drugs Program: Discussion paper, 2015.
- European regulation on orphan medicinal products: 10 years of experience and future perspectives. Nature Reviews Drug Discovery 2011, 10(5):341-349.
- Joppi R, Bertele’ V, Garattini S: Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. European Journal of Clinical Pharmacology 2012, 69(4):1009-1024.
- Singh J: The portal for rare diseases and orphan drugs. J Pharmacol Pharmacother 2013, 4(2):168-169.
- Ürek D , Karaman S: Önemli Bir Halk Sağliği Sorunu Olarak Nadir Hastaliklar Ve Yetim Ilaçlar. Hacettepe Sağlık İdaresi Dergisi 2019, 22(4):863-878.
- Kılıç P, Koçkaya G, Yemşen Ö, Tan C, Öztunca FH, Aksungur P, Kerman S: Orphan drug regulations in Turkey. Journal of Pharmaceutical Health Services Research 2013, 4(3):151-153.
- Orphanet Türkiye websayfası giriş noktası.; 2022 March 12. Available from: http://www.orpha.net/national/TR-TR/index/orphanet-t%C3%BCrkiye/
- Advancing Health Through Innovation: New Drug Therapy Approvals 2021.; 2022 May 13. Available from: https://www.fda.gov/media/155227/download
- Miller KL, Fermaglich LJ, Maynard J: Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases. Orphanet Journal of Rare Diseases 2021, 16(1).
- Iervolino A: World Preview2017,Outlook to 2022 In. Edited by Urquhart L, 10th edn: EvaluatePharma; 2017.
- Williams C. Chapter 4. Treating Rare Diseases: Business Model for Orphan Drug Development. In: Thurston D (eds) Orphan Drugs and Rare Diseases, Royal Society of Chemistry, Cambridge, UK. 2014: pp 83-115.
- Hadjivasiliou A: Orphan Drug Report 2017. In., 4th edn: EvaluatePharma; 2017.
- Pomeranz K: Orphan Drug Report 2019. In. Edited by Urquhart L, 6th edn: EvaluatePharma; 2019.
- Orphan Drug Report 2022.; 2022 May 12. Available from: Orphan Drug 2022 Report | Evaluate
- Orphan Drugs Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2022-2027.; 2022 May 13. Available from: https://www.researchandmarkets.com/reports/5547179/orphan-drugs-market-global-industry-trends
There are 37 citations in total.
Details
| Primary Language | Turkish |
|---|---|
| Subjects | Pharmacology and Pharmaceutical Sciences |
| Journal Section | Review Articles |
| Authors | |
| Publication Date | September 1, 2023 |
| Acceptance Date | December 25, 2022 |
| Published in Issue | Year 2023 Volume: 43 Issue: 3 |