320 related articles for article (PubMed ID: 32065047)
1. Innovative design and analysis for rare disease drug development.
Chow SC; Huang Z
J Biopharm Stat; 2020 May; 30(3):537-549. PubMed ID: 32065047
[TBL] [Abstract][Full Text] [Related]
2. Statistical considerations for rare diseases drug development.
Chow SC; Chang YW
J Biopharm Stat; 2019; 29(5):874-886. PubMed ID: 31454299
[TBL] [Abstract][Full Text] [Related]
3. Demonstrating effectiveness or demonstrating not ineffectiveness - A potential solution for rare disease drug product development?
Chow SC; Huang Z
J Biopharm Stat; 2019; 29(5):897-907. PubMed ID: 31454301
[TBL] [Abstract][Full Text] [Related]
4. Probability monitoring procedures for sample size determination.
Huang Z; Chow SC
J Biopharm Stat; 2019; 29(5):887-896. PubMed ID: 31454274
[TBL] [Abstract][Full Text] [Related]
5. Flexibility in the FDA approach to orphan drug development.
Hunter NL; Rao GR; Sherman RE
Nat Rev Drug Discov; 2017 Nov; 16(11):737-738. PubMed ID: 28860647
[No Abstract] [Full Text] [Related]
6. Raising orphans: how clinical development programs of drugs for rare and common diseases are different.
Orfali M; Feldman L; Bhattacharjee V; Harkins P; Kadam S; Lo C; Ravi M; Shringarpure DT; Mardekian J; Cassino C; Coté T
Clin Pharmacol Ther; 2012 Aug; 92(2):262-4. PubMed ID: 22739137
[TBL] [Abstract][Full Text] [Related]
7. Regulators adopt more orphan drugs.
Reardon S
Nature; 2014 Apr; 508(7494):16-7. PubMed ID: 24695293
[No Abstract] [Full Text] [Related]
8. Novel treatments for rare rheumatologic disorders: analysis of the impact of 30 years of the US orphan drug act.
Lutz T; Lampert A; Hoffmann GF; Ries M
Orphanet J Rare Dis; 2016 May; 11(1):60. PubMed ID: 27176041
[TBL] [Abstract][Full Text] [Related]
9. Regulatory and scientific issues regarding use of foreign data in support of new drug applications in the United States: an FDA perspective.
Khin NA; Yang P; Hung HM; Maung-U K; Chen YF; Meeker-O'Connell A; Okwesili P; Yasuda SU; Ball LK; Huang SM; O'Neill RT; Temple R
Clin Pharmacol Ther; 2013 Aug; 94(2):230-42. PubMed ID: 23588316
[TBL] [Abstract][Full Text] [Related]
10. Trends of Clinical Trials for Drug Development in Rare Diseases.
Sakate R; Fukagawa A; Takagaki Y; Okura H; Matsuyama A
Curr Clin Pharmacol; 2018; 13(3):199-208. PubMed ID: 29866013
[TBL] [Abstract][Full Text] [Related]
11. The impact of the Orphan Drug Act on Food and Drug Administration-approved therapies for rare skin diseases and skin-related cancers.
Karas L; Lu CY; Agrawal PB; Asgari MM
J Am Acad Dermatol; 2019 Sep; 81(3):867-877. PubMed ID: 31103566
[TBL] [Abstract][Full Text] [Related]
12. FDA orphan products clinical trial grants: assessment of outcomes and impact on rare disease product development.
Miller KL; Mueller C; Liu G; Miller Needleman KI; Maynard J
Orphanet J Rare Dis; 2020 Sep; 15(1):234. PubMed ID: 32883327
[TBL] [Abstract][Full Text] [Related]
13. Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?
Brabers AE; Moors EH; van Weely S; de Vrueh RL
Orphanet J Rare Dis; 2011 Sep; 6():59. PubMed ID: 21892964
[TBL] [Abstract][Full Text] [Related]
14. Precision Medicines' Impact on Orphan Drug Designation.
Mueller CM; Rao GR; Miller Needleman KI
Clin Transl Sci; 2019 Nov; 12(6):633-640. PubMed ID: 31297924
[TBL] [Abstract][Full Text] [Related]
15. Post-approval Studies for Rare Disease Treatments and Orphan Drugs.
Maier WC; Christensen RA; Anderson P
Adv Exp Med Biol; 2017; 1031():197-205. PubMed ID: 29214573
[TBL] [Abstract][Full Text] [Related]
16. Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval.
Harada K; Toriyabe K; Ono S
J Clin Pharmacol; 2020 Jan; 60(1):117-124. PubMed ID: 31364772
[TBL] [Abstract][Full Text] [Related]
17. Characteristics of orphan drug applications that fail to achieve marketing approval in the USA.
Heemstra HE; Leufkens HG; Rodgers RP; Xu K; Voordouw BC; Braun MM
Drug Discov Today; 2011 Jan; 16(1-2):73-80. PubMed ID: 21094692
[TBL] [Abstract][Full Text] [Related]
18. Innovative thinking of clinical investigation for rare disease drug development.
Wang P; Chow SC
Orphanet J Rare Dis; 2023 Sep; 18(1):299. PubMed ID: 37740206
[TBL] [Abstract][Full Text] [Related]
19. Pediatric Orphan Drug Indications: 2010-2018.
Kimmel L; Conti RM; Volerman A; Chua KP
Pediatrics; 2020 Apr; 145(4):. PubMed ID: 32127360
[TBL] [Abstract][Full Text] [Related]
20. Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010.
Pariser AR; Slack DJ; Bauer LJ; Warner CA; Tracy LA
Drug Discov Today; 2012 Aug; 17(15-16):898-904. PubMed ID: 22564486
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]