Impacts

INVENTS research and innovation activities will significantly improve clinical drug development for small populations and increase the likelihood of developing effective treatments for patients with RD. 

INVENTS project results will impact:

Pharmacokinetic experts

Drug developers

Clinical researchers

Pharmaceutical industry
and small and medium size enterprises (SMEs) specialized in rare and pediatric diseases

Health and Regulatory European agencies

Payers

Health Technology Assessments bodies

Patients and general public

Main outcomes

  • Faster drug development in rare/paediatric diseases
  • To optimise the efficient use of real world data (RWD)
  • Facilitating method development while also ensuring the evaluation and assessment of evidence derived from the use of new computational models for decision-making processes involving small sample sizes and rare diseases
  • Introducing innovative methods for extrapolation, modelling, and simulation based on virtual populations, aimed at minimizing the need for unnecessary clinical trials or modelling processes
  • Create tools like frameworks, checklists, and guidelines that consider health economics, patient preferences, established assessment criteria, and decision support tools for regulatory decision making in rare diseases
  • Improved patient management and prognostics through cost-effective clinical trial designs
  • More consistency between European Medicin Agency (EMA) and national (HTAs) decisions for patients reimbursement.
  • Better consideration of patient preference in decision-making
  • Reduction of off label use in rare and pediatric diseases

Main impacts

Scientific

Optimized evidence use, faster risk/benefit assessments, robust dose rationale for new medicines, medical autoregulation, and increased digital trust.

Societal

Comprehensive value chain integrated in drug development, better quality of life for patients, increased acceptance of digital tools in health.

Economic

Expanded market (incl. SMEs), increased reimbursement rates, cheaper new drugs for rare/pediatrics, stop development of drugs with insufficient baseline evidence to redirect investments.

Public Health

Broader reach, new drugs for rare/pediatric patients with no options, improved and safer alternatives for patients with suboptimal responses or side-effects.