المؤلفون: Irissarry, Carla¹ (AUTHOR) carla.irissarry@sciencespo.fr, Burger-Helmchen, Thierry² (AUTHOR) carla.irissarry@sciencespo.fr

المصدر: Businesses. Sep2024, Vol. 4 Issue 3, p453-472. 20p.

مصطلحات موضوعية: *ARTIFICIAL intelligence, DRUG discovery, ORPHAN drugs, DRUG development, THERAPEUTICS

المؤلفون: Nguyen, Thi Thuy Dung¹ (AUTHOR), Lee, Yu-Hsuan^1,2 (AUTHOR), Lin, Yu-Jr³ (AUTHOR), Chang, Shu-Chen³ (AUTHOR), Hsiao, Fei-Yuan^1,4,5,6 (AUTHOR), Chang, Chee-Jen^1,3,7,8 (AUTHOR), Ou, Huang-Tz^1,2 (AUTHOR) huangtz@mail.ncku.edu.tw

المصدر: Value in Health. Feb2025, Vol. 28 Issue 2, p241-249. 9p.

مصطلحات موضوعية: *ORPHAN drugs, *VALUE (Economics), *UNIVERSAL healthcare, *DECISION making, *ANTINEOPLASTIC agents

المؤلفون: Hong, Jihyung¹ (AUTHOR), Bae, Eun-Young² (AUTHOR) eybae@gnu.ac.kr, Cha, Sohee² (AUTHOR), Lee, Joohyun² (AUTHOR)

المصدر: BMC Health Services Research. 1/14/2025, p1-14. 14p.

مصطلحات موضوعية: *ORPHAN drugs, *TECHNOLOGY assessment, *MEDICAL technology, *VALUE (Economics), *ACQUISITION of data

المؤلفون: Simonsen, Mikkel Runason^1,2 (AUTHOR) mikkel.simonsen@rn.dk, Jensen, Jonas Faartoft¹ (AUTHOR), Larsen, Thomas Stauffer³ (AUTHOR), Gade, Inger‐Lise^1,4 (AUTHOR), Nully Brown, Peter⁵ (AUTHOR), El‐Galaly, Tarec Christoffer^6,7,8,9,10 (AUTHOR)

المصدر: European Journal of Haematology. Dec2024, p1. 8p. 3 Illustrations.

مصطلحات موضوعية: *DIFFUSE large B-cell lymphomas, *ORPHAN drugs, *DISEASE prevalence, *SURVIVAL rate, *RARE diseases

المؤلفون: Lakdawalla, Darius N.¹ (AUTHOR), Phelps, Charles E.^1,2 (AUTHOR) chuckphelps2@gmail.com, Nguyen, Hong-Hanh² (AUTHOR)

المصدر: Value in Health. Dec2024, Vol. 27 Issue 12, p1648-1655. 8p.

مصطلحات موضوعية: *ORPHAN drugs, *WILLINGNESS to pay, *MONETARY incentives, *PUBLIC investments, *THERAPEUTICS

المؤلفون: Clifford, Katherine A.¹ (AUTHOR), Levine, A. Alex¹ (AUTHOR), Enright, Daniel E.¹ (AUTHOR), Neumann, Peter J.¹ (AUTHOR), Chambers, James D.¹ (AUTHOR) jchambers@tuftsmedicalcenter.org

المصدر: Value in Health. Dec2024, Vol. 27 Issue 12, p1656-1661. 6p.

مصطلحات موضوعية: *ORPHAN drugs, *VALUE (Economics), *DRUG approval, *DISEASE prevalence, *UNITS of time

المؤلفون: Shin, Eun Young¹ (AUTHOR), Hong, Young Jun¹ (AUTHOR), Lim, Kyung Min¹ (AUTHOR), Kim, Tae Hyang¹ (AUTHOR), Lee, Jong Hyuk¹ (AUTHOR) assajh@cau.ac.kr

المصدر: Journal of Pharmaceutical Policy & Practice. Dec2024, Vol. 17 Issue 1, p1-18. 18p.

مصطلحات موضوعية: *DRUG accessibility, *ORPHAN drugs, *DRUG approval, *DRUG bioavailability, *THERAPEUTICS

المؤلفون: Grand, Tobias Sydendal^1,2 (AUTHOR) tsgrand1@sheffield.ac.uk, Ren, Shijie¹ (AUTHOR), Hall, James³ (AUTHOR), Åström, Daniel Oudin² (AUTHOR), Regnier, Stephane² (AUTHOR), Thokala, Praveen¹ (AUTHOR)

المصدر: PharmacoEconomics. Jun2024, Vol. 42 Issue 6, p619-631. 13p.

مصطلحات موضوعية: *ECONOMIC models, RARE diseases, ORPHAN drugs, ECONOMIC opportunities, TECHNOLOGY assessment

المؤلفون: Mishra, Sangita¹ (AUTHOR), Bhat, Deepa² (AUTHOR), Venkatesh, M. P.^1,3 (AUTHOR) venkateshmpv@jssuni.edu.in

المصدر: Orphanet Journal of Rare Diseases. 11/29/2024, Vol. 19 Issue 1, p1-18. 18p.

مصطلحات موضوعية: *THERAPEUTICS, *ORPHAN drugs, *RARE diseases, *MEDICAL research, *DISEASE management

المؤلفون: Domike, Reuben^1,2,3 (AUTHOR) reuben.domike@lightpharma.com, Raju, G. K.^1,2 (AUTHOR), Sullivan, Jamie⁴ (AUTHOR), Kennedy, Annie⁴ (AUTHOR)

المصدر: Orphanet Journal of Rare Diseases. 11/8/2024, Vol. 19 Issue 1, p1-12. 12p.

مصطلحات موضوعية: *ORPHAN drugs, *DRUG approval, *BIOMARKERS, *MEDICAL genetics, *MONETARY incentives

المؤلفون: Chen, Handong¹ (AUTHOR), Xiang, Yuliang^1,2 (AUTHOR), Tang, Xian¹ (AUTHOR), Hu, Ming¹ (AUTHOR) huming@scu.edu.cn

المصدر: Orphanet Journal of Rare Diseases. 10/20/2024, Vol. 19 Issue 1, p1-11. 11p.

مصطلحات موضوعية: *MULTIPLE criteria decision making, *ORPHAN drugs, *HEALTH insurance, *DECISION making, *DRUG accessibility

المؤلفون: Han-Wook Yoo¹ hwyoo@chamac.co.kr

المصدر: Clinical & Experimental Pediatrics. Jul2024, Vol. 67 Issue 7, p315-327. 13p.

مصطلحات موضوعية: *ORPHAN drugs, *RARE diseases, *DRUG development, *SMALL interfering RNA, *DRUG repositioning

المؤلفون: Lexchin, Joel^1,2 (AUTHOR) jlexchin@yorku.ca

المصدر: JGIM: Journal of General Internal Medicine. May2024, Vol. 39 Issue 7, p1276-1279. 4p.

مصطلحات موضوعية: *ORPHAN drugs, *DRUG approval, *CROSS-sectional method, *BIOMARKERS

الشركة/الكيان: CANADA. Health Canada

المؤلفون: Hu, Yubo¹ (AUTHOR) huyubo202209@163.com, Pang, Zhiqiang¹ (AUTHOR)

المصدر: PLoS ONE. 5/6/2024, Vol. 19 Issue 5, p1-37. 37p.

مصطلحات موضوعية: *STATISTICAL correlation, *ORPHAN drugs, *GROUP decision making, *CLINICAL medicine, *FUZZY sets, *MACHINE translating

المؤلفون: Cicenas, Jonas^1,2 (AUTHOR) j.cicenas@mapkinases.eu, Simkus, Jokubas^1,3 (AUTHOR)

المصدر: Cancers. Apr2024, Vol. 16 Issue 8, p1555. 6p.

مصطلحات موضوعية: *ORPHAN drugs, *HEMATOLOGIC malignancies, *BREAST tumors, *DRUG approval, *METASTASIS, *MOLECULAR structure, *CYCLIN-dependent kinases, *GENOMES, *CHEMICAL inhibitors

الشركة/الكيان: UNITED States. Food & Drug Administration

المؤلفون: Kostadinov, Kostadin¹ (AUTHOR) ivelina.popova@mu-plovdiv.bg, Popova-Sotirova, Ivelina¹ (AUTHOR) yuliyana.marinova@mu-plovdiv.bg, Marinova, Yuliyana¹ (AUTHOR) nina.musurlieva@mu-plovdiv.bg, Musurlieva, Nina¹ (AUTHOR) georgi.iskrov@mu-plovdiv.bg, Iskrov, Georgi^1,2 (AUTHOR) rumen.stefanov@mu-plovdiv.bg, Stefanov, Rumen^1,2 (AUTHOR)

المصدر: Cancers. Apr2024, Vol. 16 Issue 8, p1489. 19p.

مصطلحات موضوعية: *HEALTH services accessibility, *ORPHAN drugs, *HEALTH insurance reimbursement, *RARE diseases, *HEALTH policy, *GOVERNMENT agencies, *RETROSPECTIVE studies, *DESCRIPTIVE statistics, *MANN Whitney U Test, *MEDICAL records, *ACQUISITION of data, *TUMORS, *HEALTH equity, *DATA analysis software, *MEDICAL care costs

مصطلحات جغرافية: BULGARIA

المؤلفون: Michaeli, Thomas^1,2,3,4 (AUTHOR), Michaeli, Daniel Tobias^1,4,5 (AUTHOR) danielmichaeli@yahoo.com

المصدر: Value in Health. Apr2024, Vol. 27 Issue 4, p449-457. 9p.

مصطلحات موضوعية: *ORPHAN drugs, *DRUG approval, *ANTINEOPLASTIC agents, *PRICES, *GLOBAL burden of disease

الشركة/الكيان: UNITED States. Food & Drug Administration

المؤلفون: Ruggiero, Rosanna^1,2 (AUTHOR) nunziabalzano95@gmail.com, Balzano, Nunzia^1,2 (AUTHOR) gabriella.dimauro@unicampania.it, Nicoletti, Maria Maddalena³ (AUTHOR) maddalenanicoletti9@gmail.com, di Mauro, Gabriella^1,2,4 (AUTHOR) annalisa.capuano@unicampania.it, Fraenza, Federica^1,2 (AUTHOR), Campitiello, Maria Rosaria⁵ (AUTHOR), Rossi, Francesco^1,2,6 (AUTHOR), Capuano, Annalisa^1,2 (AUTHOR)

المصدر: Pharmaceuticals (14248247). Mar2024, Vol. 17 Issue 3, p394. 13p.

مصطلحات موضوعية: *SPINAL muscular atrophy, *ORPHAN drugs, *RARE diseases, *MEDICAL personnel, *LIVER failure

المؤلفون: Brantly, Mark, Kuhn, Brooks, Farah, Humam, Mahadeva, Ravi, Cole, Alexandra, Chang, Catherina, Brown, Cynthia, Campos, Michael, Lascano, Jorge, Babcock, Erin, Bhagwat, Sharvari, Boyea, Teresa, Veldstra, Carson, Andrianov, Vasily, Kalabus, James, Eckelman, Brendan, Veale, Andrew

المصدر: Chronic obstructive pulmonary diseases (Miami, Fla.). 11(3)

مصطلحات موضوعية: alpha-1 antitrypsin deficiency, augmentation therapy, clinical trials, orphan drugs, outcomes biologic agents

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/6qr0j1fh
https://escholarship.org/content/qt6qr0j1fh/qt6qr0j1fh.pdf

المؤلفون: García-Parra, Beliu, Guiu, Josep M., Povedano, MÓnica, Modamio, Pilar

المصدر: Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration; Feb2025, Vol. 26 Issue 1/2, p48-57, 10p

مصطلحات موضوعية: SPINAL muscular atrophy, AMYOTROPHIC lateral sclerosis, ECONOMIC uncertainty, ORPHAN drugs, NEURODEGENERATION