Descripción del proyecto
Diseños y herramientas innovadores para la evaluación de medicamentos para enfermedades raras
La evaluación de la eficacia y seguridad de nuevos medicamentos para enfermedades raras, en concretos las infantiles, plantea varios retos. Entre ellos se incluyen el pequeño tamaño muestral de pacientes, la heterogeneidad de los pacientes y de las enfermedades y el conocimiento limitado sobre las enfermedades. Todo ello dificulta el acceso a un tratamiento eficaz y limita las opciones disponibles. En este contexto, el proyecto INVENTS, financiado con fondos europeos, pretende perfeccionar las trayectorias de evolución de enfermedades basadas en modelos longitudinales, mejorar los modelos de extrapolación e implementar ensayos «in silico», teniendo en cuenta las necesidades de los pacientes, para mejorar la toma de decisiones normativas. Se prevé que los resultados del proyecto permitan a los investigadores, los organismos reguladores y la industria farmacéutica europea aprovechar diseños innovadores de ensayos clínicos, ensayos «in silico» y métodos de análisis de datos de la vida real. Los pacientes con enfermedades raras se beneficiarán de un acceso mayor y más rápido a tratamientos eficaces y seguros.
Objetivo
The evaluation of new medicines for rare diseases (RD) including rare paediatric RDs is challenging for several reasons, among which are the small patient sample sizes, heterogeneity of patients and diseases and heterogeneity in disease knowledge. Due to these difficulties, access to effective treatments and the number of treatment options are often limited in RDs.
INVENTS aims to provide clinical trial trialists, researchers and regulators with a global framework encompassing methods, workflows and evidence assessment tools to be implemented in orphan and paediatric drug development. Our ambition is to significantly improve the evaluation of evidence and regulatory decision-making through the development and validation of: refined longitudinal model-based diseases trajectories and treatment effect, improved extrapolation models, in silico trials (e.g. virtual patient cohorts), optimised model-based clinical trial designs and evidence synthesis methods. These will be evaluated through simulation studies and tested on extensive data from a range of use cases provided by our industrial partners Roche and Novartis and Real World data (RWD) from RD registry. The INVENTS framework will improve consistency and efficiency of the drug evaluation process for RD by augmenting clinical evidence without compromising its scientific integrity and providing regulators assessment credibility criteria.
At the end of this 5 years project, the European industry will be able to exploit novel and improved clinical trial designs, in silico trials and RWD analysis approaches supporting drug development in RD. The European Medicine Agency and European national regulators (including Health Technology Assessment bodies) will be supplied with a general framework allowing better informed decision-making. Most importantly, RD patients will benefit from an increased and faster access to efficacious and safe treatments.
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HORIZON-RIA - HORIZON Research and Innovation ActionsCoordinador
75654 Paris
Francia