Data Collection and Sharing

We have two priorities when it comes to sharing data, one is to ensure the privacy of patient data, and secondly, to ensure data is used to advance the research and understanding of Niemann-Pick diseases.

The registry supports clinical and epidemiological research. The registry shares data with experts in the Niemann-Pick Research field including clinicians, academics, and industry. As such, the INPDR has implemented a data request process that both protects sensitive patient data and also ensures that the data is accessible to researchers.

"Personal Data" allows an individual to be identified either directly or indirectly. Common types of Personal Data that identify an individual are: name or address. When information is combined with other Personal Data it may indirectly identify an individual, for example: patient number.

Data related to biometrics, health or genetics is known as "Special Category Data". This Special Category Data is personal data that needs more protection because it is sensitive.

Personal (and Special Category Data) can be "Pseudonymised" to help reduce privacy risks by making it more difficult to identify individuals. All identifiable data is removed and replaced by a unique code or key, which is then held separately from the Personal Data. It is only possible to identify an individual by holding both the information and the key.

Your Personal Data will be fully "Anonymised" before it is shared with our research partners. All identifiable data is removed and potentially aggregated into bands. It is not possible to identify an individual from anonymised data.

Your Identifiable Personal and Special Category Data is stored and processed within the INPDR. This may be pooled with other patient clinical data and analysed to generate new knowledge of NPDs in the form of publications in scientific journals or presentations to healthcare professionals.

Under the guidance of the INPDR Board and Scientific Advisory Committee, The Registry will also produce research and analysis to facilitate understanding of Niemann-Pick Disease Type C and ASMD. Anonymised Data may be shared with third parties (such as researchers) who have submitted data requests that have been reviewed and approved by the INPDR Scientific Advisory Committee (SAC) and the Board of Trustees. Anonymised Data may also be shared with pharmaceutical companies as part of applications for approval of new medicines or to comply with responsibilities set by health bodies such as the European Medicines Agency.

We collect data directly from patients that are signed up to the registry. We have distinct data collection for ASMD and NPC patients. We don’t just collect a snapshot, we collect data on an ongoing basis, extracting data over time.

Data is collected by an individual's clinician. The following information is collected:

• Demographic data (sex, date of birth, ethnicity etc.)
• Diagnosis
• Health status
• Treatment
• Laboratory results
• Imaging tests
• Other relevant clinical information

Please view the data dictionaries below, for more information on data the INPDR collects.

The Scientific Advisory Committee and Board of Trustees will ensure that use of registry data is consistent with the mission, vision and values of the INPDR. No data or analysis will be transferred to any user without their approval.

Clinical investigators in the INPDR will receive access to anonymised datasets and analytical services for approved research studies at no cost.

Advocacy organisations, patient groups and media organisations will be granted access to approved analytical services as part of the core service of the INPDR.

Academic and statutory organisations can request access to datasets and analytics for approved research with a single-use charge applied on a case-by-case basis.

A subscription model has been developed to support the
clinical research needs of industry and corporate stakeholders whilst enabling fair access for patients and academic researchers. It offers tiered levels of service that encourage stakeholder support and are reflective of both their needs and status.