FAQs

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

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

  • Does the INPDR have ethical approval?

    The INPDR has received ethics approval in the United Kingdom, Ireland, Spain, Italy, Germany, Czech Republic and the United States of America.

    If you treat patients in one of these countries, you may still need to obtain national ethics approval, depending on the practise of the country.

    Countries not included on this list will require ethical approval in line with their standard research approval practise. Additionally you may need to obtain local institutional review board approval.

    The INPDR will support you throughout the necessary approval process and will provide a Welcome Pack that includes documents required for approval.

  • Can I get the Welcome Pack in another language?

    At the moment, our Welcome Pack is available in English.

    In order to support the global roll out of the INPDR, we are working on translating all our Information Sheets, Informed Consent Forms and supporting documents. This may take some time because the process involves ‘back-translation’ in order to make sure we remain compliant and that patients are given correct details and can provide their informed consent.

    If are interested in joining the INPDR and would like information in another language or format, please get in touch or complete our website contact form.

    The INPDR is a subsidiary of a large global network of patient organisations and Niemann-Pick Disease specialists that can support the process of joining the INPDR.

  • What information is collected?

    There are two parts to the Registry: Clinician Reported Data (CRD) and Patient Reported Data (PRD).

    The Clinician Reported Data is provided by the patient’s clinician and includes diagnostic information, clinical history, treatments, medicines and test results captured as part of the patient’s initial appointment then again during follow-up visits.

    The Patient Reported Data is provided by the patient and/or their family and includes quality of life based questions, which capture the wider impact of Niemann-Pick Disease.

  • Who owns the data in the INPDR?

    Data is owned by patients.

    Patients consent to having their anonymised data captured by the INPDR and can withdraw their consent at any time. Patients also have control over what happens with their data and who can access the data.

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

  • How is data in the Registry verified?

    Clinical data captured within the Registry is entered directly by the patient's clinical care team. There are also controls built into the database to reduce the risk of mistakes or missing information during data entry.

    Every year, a Data Manager from the INPDR does a comprehensive data review using programs built into the database and manual, random checks. The Data Manager will then work with the clinical team to support any training and make sure all the data is accurate and complete.

  • What if a patient changes their mind?

    The data in the INPDR is owned by patients and they can withdraw their consent at any time without reason.

    If your patient no longer wishes to take part in the Clinician Reported Data, you will need to let us know.

    If your patient no longer wishes to take part in the Patient Reported Data, they can let us know directly.

  • What happens to the data in the Registry?

    The overall aim 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 NPD patients through global collaboration.

    What this means is that the INPDR’s Scientific Advisory Committee will carefully review requests for data and if they feel it will help to achieve the overall aim of the INPDR, anonymous summary data will be provided for analysis and to share results with patient advocates, clinicians, researchers, scientists and the pharmaceutical industry.

    The INPDR regulates data security, quality and access.

  • How does the INPDR work with pharmaceutical industry?

    Traditionally, many disease registries are drug specific and pharmaceutical company owned. Instead, the INPDR is a patient disease registry that covers all Niemann-Pick Diseases.

    The work the INPDR is doing with the pharmaceutical industry can help to improve pharmacovigilance (where drugs are monitored for long-term safety), as well as research into new therapies. This new approach to registries is supported by the European Medicines Agency (EMA), who recognise the power of patient-led registries.

    The work we do with the pharmaceutical industry is governed by signed agreements and funding is secured in order to help with the running costs of the Registry and/or directly for the work involved in providing summary data in response to a Scientific Advisory Committee approved data request. The pharmaceutical industry does not have access to ‘raw’ data and companies must complete data requests in the same way that patient advocates, clinicians, researchers and scientists would.