FAQs

If you are a clinician or health professional working in the field of Niemann-Pick diseases, you can find a range of answers to FAQs regarding the International Niemann-Pick Disease Registry below.

  • The INPDR Clinician Reported Dataset is a prospective, multi-centre, multinational, observational registry enrolling approximately 1000 NPD patients, with patients being followed for a minimum of 5 years after enrolment.

    Participating in the CRD involves local site set-up using INPDR provided materials, recruiting eligible patients, undertaking data entry into the electronic data capture (EDC) system, as well as other standard clinical research activities expected for observational registry studies.

  • Data is collected via the CRD, a web-based EDC system that is accessible to pre-approved users at authorised study sites. Access to the CRD is granted once study sites have obtained all required study approval documents.

  • The EDC system is designed to offer flexibility regarding how often data is collected. Data is expected to be collected in line with the patient’s standard of care visit schedule. The CRD protocol does not require any additional visits or investigations outside of the patient’s standard of care. The EDC system also allows for historical data to be entered.

  • The INDPR is the data controller. Patients consent to having their data captured by the INPDR and can withdraw their consent at any time.

    The INPDR ensures that all patient data is maintained and protected with data security in mind.

  • The CRD collects data for both Acid Sphingomyelinase Deficiency and Niemann-Pick Disease Type C, with differing data being collected for each disease. Data to be collected includes demographic information, genetic information, diagnosis, medical history and co-morbidities, treatment history, physical assessments and disease-specific assessments. Only data generated from routine standard of care will be collected in the CRD.

  • Applicable research governance approval, including Institutional Review Board/Research Ethics Committee, is required before any study activities can take place at site. All necessary documents for applying for research governance approvals are provided by INPDR, and any additional documents that may be required can be provided.

    Sponsorship of the CRD is arranged by either the country lead site or with the individual study site, depending on the research governance practice of the country. Additionally data sharing agreements are arranged between the study site and the INPDR to allow for data transfer.

  • Patients can withdraw their consent at any time without reason. If a patient no longer wishes to take part in the Clinician Reported Data, they can decide what will happen to any data already entered into the EDC system, such as whether it is maintained or deleted.

  • The INPDR aims to provide support based on study site requirements. The INPDR provides all set-up documents and works closely with sites to produce any additional documents required for study set-up. Set-up materials are currently provided in 10 languages for non-English speaking sites. Comprehensive training is provided to all site staff and one-to-one support is available throughout the duration of the study.

  • Interested parties of different backgrounds, including clinicians, are invited to submit data access requests to access CRD data to support investigator initiated research. All data access requests will be reviewed by the INPDR Scientific Advisory Committee to assess the feasibility and integrity of received requests.

  • Traditionally, disease registries were created as product registries owned by pharmaceutical companies. This lead to siloing of valuable data and duplication of work, where multiple registries exist for one disease.

    Instead, the INPDR is a patient registry that covers all Niemann-Pick diseases – Acid Sphingomyelinase Deficiency (also known as Niemann-Pick Disease Type A and Type B) and Niemann-Pick Disease Type C. The aim of the INPDR is to be a single disease registry which can support the data requirements of different stakeholders, negating the need for multiple registries for Niemann-Pick diseases.

    The INPDR can serve as a data source to support pharmaceutical industry requirements, including post-authorisation studies which may be required by regulatory agencies such as the European Medicines Agency and the US Food and Drug Administration, as well as opportunities to support the development of new therapies.

    Any work undertaken with pharmaceutical organisations is governed by agreements and managed with oversight from the Scientific Advisory Committee and INPDR Board of Trustees. No access to direct patient-identifiable raw data is provided and pharmaceutical organisation are required to complete data access requests in the same way that patient advocates, clinicians, researchers and scientists would.

We will keep these up to date so if you have any further questions which have not been covered please don’t hesitate to get in touch with us directly at: info@inpdr.org