Towards the implementation of gene therapy in the clinical practice

Juan Bueren(2) Cristian Smerdou(1)

1:Cima Universidad de Navarra & Idisna, Pamplona, Spain; 2:CIEMAT, CIBERER & IIS-FJD, Spain

After many years of efforts developed by the scientific community, including academic institutions and private companies, several gene therapy products have obtained marketing authorization in Europe. Furthermore, a large number of gene therapy trials are currently in advanced development stages. All this strongly suggests that in the coming years many gene therapy products will be available in different countries, including Spain. Consequently, in addition to the need to progress in developing more efficient therapies for debilitating diseases, further aspects will have to be considered to facilitate access of patients to these novel therapies. Indeed, the efficient implementation of gene therapy in Europe will rely on regulatory agencies and national health systems, but it will also depend on other factors, like strategic considerations from companies, hospitals, clinicians, scientists, and patient associations. To address challenges derived from the imminent approval of many gene therapy products, and to facilitate the access of patients to these therapies under conditions of quality, equity and with an optimal cost-effectiveness ratios, a multidisciplinary panel of experts from different areas, including gene therapy scientists, healthcare providers involved in rare diseases, healthcare managers, lawyers, economists, and patients, among others, have met to discuss these questions. Based on their deliberations, a document gathering multidisciplinary and comprehensive suggestions was elaborated. In addition to the Program of Advanced Therapies developed by the Spanish National Health System, this document aims to facilitate a sustainable application of gene therapy in Spain. A summary of the main conclusions raised in this document will be presented.