Estudi de caracterització del disseny d'assajos clínics realitzats amb medicaments orfes = Study to characterize the designs of clinical trials conducted with orphan medicinal products /

• Autores: Juan Manuel Fontanet Sacristán
• Directores de la Tesis: Caridad Pontes García (codir. tes.), Josep Torrent-Farnell (codir. tes.)
• Lectura: En la Universitat Autònoma de Barcelona ( España ) en 2017
• Idioma: español
• Tribunal Calificador de la Tesis: C. Avendaño Solá (presid.), Maria Queralt Gorgas Torner (secret.), Gonzalo Calvo (voc.)
• Programa de doctorado: Programa Oficial de Doctorado en Farmacología
• Materias:
  • Ciencias médicas
    • Farmacología
• Enlaces
  • Tesis en acceso abierto en:  DDD  TDX 
• Resumen

  • Background: Research and development of new orphan medicinal products has been limited because of lack of knowledge on the diseases, anticipated lack of return of investment in absence of incentives, and lack of methodologies to deal with small populations. Ten years after the European Orphan Drug Regulation (141/2000) the number of medicines for orphan diseases has increased significantly. The analysis of the type of scientific evidence and clinical trials conducted to support the marketing authorisation of orphan medicines in Europe will allow to analyse if alternative methodologies aimed to increase efficiency of clinical studies in small populations are applied, to have a reference to explore the applicability of such methodologies, and to compare the current methods used to conduct clinical trials in the rare diseases field versus the alternative methods proposed.

    Objective: To analyse the characteristics of the main clinical trials conducted with the orphan drugs authorised in Europe since the European regulation on orphan medicinal products entered in force in year 2000, in order to explore if there is room for application of alternative methodologies in the conduction of clinical trials.

    Methods: A review of administrative, pharmacological, regulatory and clinical data for the first 100 orphan medicinal products approved in Europe since the entry into force of the specific European regulation (ER 141/2000) to December 2014 has been conducted and systematized.

    Results: Since the European regulation on orphan medicinal products entered in force in year 2000 and up to December 2014, 100 orphan medicinal products have been approved for 125 indications in Europe covering 84 different diseases. Oncology is the area with higher number of orphan medicines authorised. Alternative methodological designs are seldom used. Overall, data suggest that the current methodological robustness of the evidence supporting approval of indications for orphan medicinal products is generally low. The rareness degree and the type of conditions influenced the characteristics of clinical trials conducted.

    Conclusions: The characteristics of the main clinical trials conducted with the orphan drugs authorised in Europe since the European regulation on orphan medicinal products entered in force in year 2000 suggest that there is room to explore the application of alternative methodologies in the conduction of clinical trials in rare diseases.