ABSTRACT
Drug development for rare diseases, classified as diseases with a prevalence of < 200 000 patients, is limited by the high cost of research and low target population. Owing to a lack of representative disease models, research has been challenging for orphan drugs. Human-on-a-chip (HoaC) technology, which models human tissues in interconnected in vitro microfluidic devices, has the potential to lower the cost of preclinical studies and increase the rate of drug approval by introducing human phenotypic models early in the drug discovery process. Advances in HoaC technology can drive a new approach to rare disease research and orphan drug development.
Subject(s)
Drug Discovery/methods , Lab-On-A-Chip Devices , Models, Biological , Orphan Drug Production/methods , Rare Diseases/drug therapy , Drug Approval , Drug Discovery/instrumentation , Humans , Orphan Drug Production/instrumentationABSTRACT
El tratamiento de las enfermedades raras muchasveces resulta complicado debido a las dificultades queplantean la investigación y desarrollo de medicamentoshuérfanos por parte de la industria farmacéutica.Una de las mayores preocupaciones de este grupo depacientes tan escaso y diverso al mismo tiempo, es elacceso a los fármacos disponibles.La Agencia Europea del Medicamento establece ladenominación de medicamentos huérfanos y los incentivosy bases necesarias para su comercialización.La mayoría de los medicamentos huérfanos porsus especiales características, exigen una adecuadavigilancia y control. Los servicios de farmacia hospitalariosestán activamente implicados en su gestión y dispensación,así como en un correcto seguimiento farmacoterapéuticode los pacientes
Treatment of rare diseases is often complicated due to the difficulties posed by the research anddevelopment of medicines by the pharmacological industry. One of the greatest concerns of this group ofpatients, which is both scarce and diverse, is access toavailable medicines.The European Medicine Agency establishes theterm orphan medicines and the incentives and basesfor their commercialisation.Due to their special characteristics, the majority oforphan medicines require suitable vigilance andcontrol. The hospital pharmacy services are activelyinvolved in their management and supply, as well as ina correct pharmacotherapeutic monitoring of thepatients
