Results 51 to 60 of about 22,199 (155)

Orphan medical devices: addressing the regulatory and access gaps in the EU and US. [PDF]

Front Public Health
Akodad S, Haon B, Rochaix L, Stevens H.
europepmc   +1 more source

Medicine Development and Access for Rare Diseases: Can We Do Better? [PDF]

J Inherit Metab Dis
Hollak CEM, Rosenberg N, Post C, Stolwijk N, Manders E, Schoenmakers D, Penninx B, Reijnhout N, Verdeyen K, Jonker R, Bosch AM, Langeveld M, van der Wel V, de Visser S, van den Berg S.   +14 more
europepmc   +1 more source

Priced Out of Treatment: The Exorbitant Cost of Antiparasitic Drugs in the United States. [PDF]

Open Forum Infect Dis
Alperin C, Nguyen N, Duffey M, Clark EH.
europepmc   +1 more source

Methodological challenges in Dutch HTA of non-oncological orphan drugs: a retrospective analysis and price comparison using different pricing models. [PDF]

Orphanet J Rare Dis
Walraven J, Kaveh M, Uyl-de Groot C.
europepmc   +1 more source

Trends, lag and characteristics of rare disease drug approval in the USA and China, 1983-2022. [PDF]

Orphanet J Rare Dis
Wang S, Liu X, Zou Y, Tang Y, Zuo W, Jiang R, Shang J, Tian X, Liu Q, Xu T, Zhang B, Zhang S.   +11 more
europepmc   +1 more source

Safety population size and duration of exposure prior to approval of new medicines: A database analysis of medicines centralised approved in the European Union between 2011 and 2023. [PDF]

PLoS One
Bouwman L, Leufkens H, Sepodes B, Torre C.   +3 more
europepmc   +1 more source

Advances in orphan drug development for alpha-1 antitrypsin deficiency: a 2025 update from the FDA and EMA. [PDF]

Ther Adv Respir Dis
Höger P, Ries M, Olivares Rivera A, Ersöz H, Buschulte K, Fähndrich S, Kontogianni K, Herth F, Trudzinski FC.   +8 more
europepmc   +1 more source

Advancing hypoparathyroidism treatment: FDA approval of Palopegteriparatide as a promising orphan drug. [PDF]

Ann Med Surg (Lond)
Shahid SR, Shahid K, Ehsan S, Khalid M, Waafira A.   +4 more
europepmc   +1 more source

Informing Dose for Pediatric Rare Diseases-A Survey of Recent Orphan Drugs Approvals. [PDF]

Clin Transl Sci
Fletcher EP, Kim C, Mei H, AbuAsal B, Samuels S, Madabushi R, Ramamoorthy A.   +6 more
europepmc   +1 more source

Pathing the way from regulatory approval to market access for gene therapy products (GTPs): An integrative review in the US, EU5, Japan and China. [PDF]

Regen Ther
Shi J, Hu H, Ung COL.
europepmc   +1 more source