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SP0069 Registries in musculoskeletal diseases and their regulatory use
  1. R Vesely
  1. Rheumatology, Respiratory, Gastroenterology and Immunology Office, European Medicines Agency, London, United Kingdom

Abstract

Patient registries collect information about individuals sharing health-related characteristics, for example, a particular disorder, a treatment or a procedure. While randomised controlled trials typically provide the primary evidence supporting marketing authorisations for new medicines, the patients studied may not be fully representative of everyone ultimately receiving the medicine and the trials may provide limited information about the natural history of the disorder. Information collected in patient registries is potentially of value for filling these evidence gaps in certain situations and for providing post-marketing safety and effectiveness information. Multiple stakeholders stand to benefit from using registry information in this way including patients, healthcare providers, policy makers, manufacturers and healthcare regulators.

In 2014, the EMA commenced a Registry Initiative aiming to optimise the use of registries in supporting medicines authorisations. Establishing a strategy of early engagement between marketing authorisation applicants and registry holders and a task force to support activities, a pilot phase was undertaken aiming to understand the barriers and enablers in using registries to support marketing authorisation applications and to inform the development of recommendations to optimise their use.

On 28 October 2016 the Agency organised Patient Registries Workshop to collect and discuss the information about experience from different patient registries in various therapeutic areas. The topics included:

  • Benefits of registries for regulators

  • Benefits of registries for HTA and payers

  • Benefits for industry

  • Benefits for clinicians and researchers

  • Benefits for patients

  • Challenges in collaboration between registries

  • Technical challenges

  • Governance

  • Sustainability

Conclusions of the pilot and the workshop have been utilised in the following activities including workshops to support registries in individual diseases.

There are multiple advanced registries in RA and JIA. Utilisation of outcomes of these and other existing or newly planned registries in other musculoskeletal diseases for regulatory purposes current environment offers new opportunities that require further analyses and collaboration.

Disclosure of Interest None declared

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