Medicamentos órfãos e medicamentos pediátricos : biológicos versus convencionais
Miniatura indisponível
Data
2020
Autores
Título da revista
ISSN da revista
Título do Volume
Editora
Resumo
Tendo em conta as particularidades da farmacologia pediátrica e a existência de doenças raras com elevada prevalência nesta população, é importante caracterizar os medicamentos órfãos aprovados na população pediátrica, nomeadamente quanto à sua origem biológica ou não-biológica.
Foi desenvolvido um estudo observacional, retrospetivo, de investigação transversal da informação regulamentar, com o objetivo da caracterização dos medicamentos órfãos com indicação terapêutica em pediatria, autorizados na União Europeia e atualmente no mercado.
Foi efetuada uma análise crítica da informação científica e regulamentar relativa aos medicamentos órfãos pediátricos aprovados (1995-2019), para permitir a sua caracterização e evidenciar potenciais diferenças em termos de tipo de autorização de introdução no mercado (AIM), origem do medicamento (biológicos versus convencionais), faixas etárias da população pediátrica e áreas terapêuticas.
Foram identificados 1130 medicamentos de uso humano autorizado por procedimento centralizado de AIM no período em análise, que incluem 129 medicamentos órfãos (14,7%), dos quais 65 são indicados na população pediátrica.
Aproximadamente metade destes 65 medicamentos órfãos pediátricos são medicamentos convencionais ou não biológicos (50,7%), 43,1 % são medicamentos biológicos e apenas 6,2% são medicamentos de terapia avançada (ATMP), nomeadamente de terapia génica ou terapia celular somática.
A AIM convencional é a mais prevalente neste grupo de medicamentos órfãos pediátricos, no entanto cerca de um terço destes medicamentos (36%) foram aprovados em circunstâncias excecionais devido ao número reduzido de doentes estudados no caso de doenças raras ou à dificuldade na realização de estudos, para se obter dados suficientes que comprovem eficácia e segurança.
A maioria dos medicamentos órfãos pediátricos pertence à área terapêutica de disfunções do metabolismo (41,5%), seguida pelas áreas da oncologia (20%) e alterações hematológicas (10,8%). A distribuição dos medicamentos órfãos pediátricos por faixa etária de indicação terapêutica revela que a generalidade se encontra aprovada em todas as subpopulações pediátricas, do nascimento até aos 18 anos.
Os resultados obtidos permitiram caracterizar os medicamentos órfãos pediátricos aprovados na EU, que possibilitam às crianças com doenças raras um acesso iii
mais facilitado às inovações terapêuticas, para que sejam tratadas com medicamentos eficazes, na dose adequada e isentos de riscos.
Given the particularities of paediatric pharmacology and the existence of rare diseases with high prevalence in this population, it is important to characterise orphan medicinal products approved in the paediatric population, in particular as regards their biological or non-biological origin. An observational, retrospective, cross-sectional study of regulatory information was developed with the objective of characterizing orphan medicinal products with therapeutic indication in pediatrics, authorised in the European Union and currently on the market. A critical analysis of scientific and regulatory information on approved paediatric orphan medicinal products (1995-2019) was carried out to enable them to be characterised and to show potential differences in terms of type marketing authorisation (MA), origin of the medicinal product (biological versus conventional), age groups of the paediatric population and therapeutic areas. A total of 1,130 medicinal products authorised by centralised MA procedure were identified during the period under review, which include 129 orphan drugs (14.7%), of which 65 are indicated in the paediatric population. Approximately half of these 65 paediatric orphan drugs are conventional or non-biological medicinal products (50.7%), 43.1% are biological medicines and only 6.2% are advanced therapy (MPA) drugs, including gene therapy or somatic cell therapy. Conventional MA is the most prevalent in this sample of paediatric orphan drugs, however about one third of these medicines (36%) have been approved in exceptional circumstances due to the small number of patients studied in the case of rare diseases or the difficulty in carrying out studies to obtain sufficient data to prove efficacy and safety. Most pediatric orphan drugs belong to the therapeutic area of metabolism dysfunctions (41.5%), followed by oncology (20%) and hematological changes (10.8%). The distribution of paediatric orphan drugs by age group of therapeutic indication reveals that the majority is approved in all pediatric subpopulations, from birth to 18 years. The results obtained made it possible to characterize the pediatric orphan drugs approved in the EU, which allow children with rare diseases easier access to therapeutic innovations, so that they can be treated with effective drugs, at an appropriate dose and free of risks.
Given the particularities of paediatric pharmacology and the existence of rare diseases with high prevalence in this population, it is important to characterise orphan medicinal products approved in the paediatric population, in particular as regards their biological or non-biological origin. An observational, retrospective, cross-sectional study of regulatory information was developed with the objective of characterizing orphan medicinal products with therapeutic indication in pediatrics, authorised in the European Union and currently on the market. A critical analysis of scientific and regulatory information on approved paediatric orphan medicinal products (1995-2019) was carried out to enable them to be characterised and to show potential differences in terms of type marketing authorisation (MA), origin of the medicinal product (biological versus conventional), age groups of the paediatric population and therapeutic areas. A total of 1,130 medicinal products authorised by centralised MA procedure were identified during the period under review, which include 129 orphan drugs (14.7%), of which 65 are indicated in the paediatric population. Approximately half of these 65 paediatric orphan drugs are conventional or non-biological medicinal products (50.7%), 43.1% are biological medicines and only 6.2% are advanced therapy (MPA) drugs, including gene therapy or somatic cell therapy. Conventional MA is the most prevalent in this sample of paediatric orphan drugs, however about one third of these medicines (36%) have been approved in exceptional circumstances due to the small number of patients studied in the case of rare diseases or the difficulty in carrying out studies to obtain sufficient data to prove efficacy and safety. Most pediatric orphan drugs belong to the therapeutic area of metabolism dysfunctions (41.5%), followed by oncology (20%) and hematological changes (10.8%). The distribution of paediatric orphan drugs by age group of therapeutic indication reveals that the majority is approved in all pediatric subpopulations, from birth to 18 years. The results obtained made it possible to characterize the pediatric orphan drugs approved in the EU, which allow children with rare diseases easier access to therapeutic innovations, so that they can be treated with effective drugs, at an appropriate dose and free of risks.
Descrição
Orientação: Ana Sofia Fernandes ; co-orientação: Dinah Duarte
Palavras-chave
MESTRADO INTEGRADO EM CIÊNCIAS FARMACÊUTICAS, CIÊNCIAS FARMACÊUTICAS, PHARMACEUTICAL SCIENCES, MEDICAMENTOS, DRUGS (PHARMACOLOGY), MEDICAMENTOS PARA CRIANÇAS, CHILDREN'S MEDICINES, MEDICAMENTOS BIOLÓGICOS, BIOLOGICAL DRUGS, INDICAÇÕES TERAPÊUTICAS, THERAPEUTIC INDICATIONS, MEDICAMENTOS ÓRFÃOS, ORPHAN DRUGS