Development perspectives for curative technologies in primary demyelinating disorders of the central nervous system with neuromyelitis optica spectrum disorder (NMOSD) and myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) at the forefront

Pitter, János (avtor), Nagy, László (avtor), Nagy, Balázs (avtor), Hren, Rok (avtor)

Primary demyelinating disorders of the central nervous system (CNS) include multiple sclerosis and the orphan conditions neuromyelitis optica spectrum disorder (NMOSD) and myelin oligodendrocyte glycoprotein IgG-associated disease (MOGAD). Curative technologies under development aim to selectively block autoimmune reactions against specific autoantigens while preserving the responsiveness of the immune system to other antigens. Our analysis focused on target patient selection for such developments, carefully considering the relevant clinical, regulatory, and market-related aspects. We found that the selection of patients with orphan conditions as target populations offers several advantages. Treatments for orphan conditions are associated with limited production capacity, qualify for regulatory incentives, and may require significantly shorter and lower-scale clinical programs. Furthermore, they may meet a higher acceptable cost-effectiveness threshold in order to compensate for the low numbers of patients to be treated. Finally, curative technologies targeting orphan indications could enter less competitive markets with lower risk of generic price erosion and would benefit from additional market protection measures available only for orphan products. These advantages position orphan conditions and subgroups as the most attractive target indications among primary demyelinating disorders of the CNS. The authors believe that after successful proof-of-principle demonstrations in orphan conditions, broader autoimmune patient populations may also benefit from the success of these pioneering developments.

• Jezik: Angleški jezik
• Ključne besede: medicine, nevrology, diseases of the central nervous system, treatment
• Vrsta gradiva: Članek v reviji
• Tipologija: 1.01 - Izvirni znanstveni članek
• Organizacija: FMF - Fakulteta za matematiko in fiziko
• Različica publikacije: Objavljena publikacija
• Leto izida: 2024
• Št. strani: 13 str.
• Številčenje: Vol. 14, iss. 6, art. no. 599
• PID: 20.500.12556/RUL-158310
• UDK: 616.8
• ISSN pri članku: 2075-4426
• DOI: 10.3390/jpm14060599
• COBISS.SI-ID: 197720579
• Datum objave v RUL: 04.06.2024

Gradivo je del revije

• Naslov: Journal of personalized medicine
• Skrajšan naslov: J. pers. med.
• Založnik: MDPI
• ISSN: 2075-4426
• COBISS.SI-ID: 31207641

Licence

• Licenca: CC BY 4.0, Creative Commons Priznanje avtorstva 4.0 Mednarodna
• Povezava: http://creativecommons.org/licenses/by/4.0/deed.sl
• Opis: To je standardna licenca Creative Commons, ki daje uporabnikom največ možnosti za nadaljnjo uporabo dela, pri čemer morajo navesti avtorja.

Sekundarni jezik

• Jezik: Slovenski jezik
• Ključne besede: medicina, nevrologija, bolezni centralnega živčnega sistema, zdravljenje

Projekti

• Financer: EC - European Commission
• Številka projekta: 101080562
• Naslov: ADVANCED ANTIGEN-SPECIFIC DENDRITIC CELL-BASED THERAPY TO RE-ESTABLISH TOLERANCE IN IMMUNE-MEDIATED DISEASES
• Akronim: IMMUTOL