About the Registry

The INPDR has the power to increase knowledge and understanding of Niemann-Pick Disease through reaching out to patients across the Globe and harnessing quality data.

The INPDR is truly a global collaboration between patient associations, clinicians and scientists working together for the benefit of patients and their families everywhere.

The INPDR is a not-for-profit, registered company based in the UK.

It is owned and managed by the International Niemann-Pick Disease Alliance (INPDA), an umbrella organisation of 16 NPD patient support groups and charities in 15 different countries, with support from professional partners involved in the care of patients affected by all types of Niemann-Pick Disease (ASMD & NP-C).

The purpose of the INPDR is to develop and sustain a unique patient-led database that supports research and therapy development, and improves care and treatment for Niemann-Pick Disease patients through global collaboration. This is done by:

  • Continually using the power of data to improve the broader understanding of Niemann-Pick disease
  • Supporting and facilitating consistent research and therapy development
  • Encouraging earlier diagnosis and equitable access to diagnostic testing
  • Establishing the INPDR as a unifying initiative much-needed by patients and professional alike.

We hope that this pioneering initiative encourages further global collaboration between patient groups, clinicians, scientists, researchers and pharmaceutical companies, all of whom wish to collectively improve care and treatment options for Niemann-Pick disease patients everywhere.

 

Working with Industry

Guidelines from the European Medicines Agency (EMA) aimed at raising standards for studies looking at the long-term safety of drugs (pharmacovigilence) encourage the use of independent patient disease specific registries. The INPDR currently works with a number of companies considering the use of the Registry to conduct long-term safety studies or in the development of new therapies.

The pharmaceutical companies that we collaborate with do not have access to patient-level data and any data requests we receive are carefully considered by the Scientific Advisory Committee (SAC). Working with industry helps to improve their research and studies, as well as secure resources to support the ongoing work of the INPDR.