RESUMO
Current European pharmaceutical legislation is not adequate to meet advances in science and technologies that will lead to rapid development of custom-made medicines. Using existing legislation for custom-made medical devices as a template and anti-sense oligonucleotides as model medicinal products, we propose new European pharmaceutical legislation to permit timely access to custom-made anti-sense oligonucleotide medicinal products. The proposals may be more widely applicable to other medicinal products.
Assuntos
Indústria Farmacêutica/legislação & jurisprudência , Legislação de Medicamentos , Oligonucleotídeos Antissenso/provisão & distribuição , Europa (Continente)RESUMO
The European pharmaceutical regulatory system has not yet been challenged by issues related to highly personalized medicines such as those to be found with active substances that affect RNA biochemistry. We review the current status of RNA-based pharmacology and present three possible case histories. The implications for the European pharmaceutical regulatory system are discussed.
Assuntos
Indústria Farmacêutica/legislação & jurisprudência , Legislação de Medicamentos , Oligonucleotídeos Antissenso/uso terapêutico , Medicina de Precisão , RNA/metabolismo , Europa (Continente) , Regulação da Expressão Gênica/efeitos dos fármacos , Humanos , Conformação de Ácido Nucleico , Oligonucleotídeos Antissenso/química , Oligonucleotídeos Antissenso/metabolismo , Patentes como Assunto , Formulação de Políticas , RNA/químicaRESUMO
To obtain a license to be placed on the marketplace, a medicinal product must be accompanied by data on quality and data from preclinical studies and studies of clinical safety and efficacy. At the time of first production, a custom-made medicinal product will have data on quality but will not be accompanied by data from preclinical studies or from studies of clinical safety and efficacy. To span the gap in data, an "imperfect intellectual bridge" between data for a custom-made medicinal product and data for its master medicinal product is described. The imperfect intellectual bridge will allow the custom-made medicinal product to draw on data associated with its master medicinal product. In time, it may be possible for a custom-made medicinal product to transfer to an independent license after collection of data on clinical safety and efficacy by means of a pharmacovigilance exercise.
