For Patients

FAQs

If you are an individual or family members affected by Niemann-Pick Disease, you can find a range of answers to FAQs regarding the International Niemann-Pick Disease Registry (INPDR) below.

We review and update these FAQs on a regular basis so if you have any further questions which have not been covered please get in touch.

  • Is my information safe?

    All data is stored on the Registry, an electronic database that has been developed and is maintained by OpenApp Ltd.

    OpenApp Ltd. have vast experience in health IT solutions and have developed the Registry so it is compliant with strict data protection regulation.

    Patient data is stored anonymously, meaning the data cannot be linked to a patient. Instead, all Clinician Reported Data (CRD) is linked to a unique patient identification code, which is known only to the patient’s doctor. In the case of the Patient Reported Data (PRD), the patient and/or their family member should be the only ones who know the patient’s identification code. You should keep these details safe.

    The INPDR also have strict data protection policies and procedures.

  • What if I change my mind after joining the INPDR?

    You can withdraw your consent to take part in the INPDR at any time without reason.

    If you would like to withdraw from the Patient Reported Data, please contact us directly or use our website contact form.

    If you would like to withdraw your consent from the Clinician Reported Data, please speak to your clinician.

  • How do I know if my clinician has joined the INPDR?

    If your clinician has joined the INPDR, they will contact you to offer you the opportunity to take part in the INPDR. They will provide you with information and instructions so you can give your consent to join the INPDR. Once your consent has been given, your clinician will be able to add your anonymised data to the INPDR. Your data cannot put into the Registry without your consent.

    If your clinician has not joined the INPDR yet, you may want to discuss this with them because the more people who join the Registry the more valuable it will be in helping to improve the lives of Niemann Pick Disease patients.

  • Can I take part without my clinician?

    Yes – there are two parts to the Registry: Clinician Reported Data and Patient Reported Data.

    Your clinician will need to join to add your information to the Clinician Reported Data part but you can join the Patient Reported Data directly through the website.

    Even though if both you or your family and your clinician provide information it will have a bigger impact on research and what we can learn about NPDs, data can be added in isolation.

  • How can I help with clinical studies?

    By joining the INPDR, you are helping the Niemann-Pick Disease community to document the natural history of the disease and potentially help researchers understand the impact of new treatments which will improve the long-term safety of new drugs.

    However, the Registry can also aid clinical trial feasibility assessment by enabling pharmaceutical industry groups to understand where potentially eligible patients are in the world and how many patients may be eligible to take part in a proposed clinical trial.

  • 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.

  • 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.

  • 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 audit and review using programs built into the database, as well as manual, random checks. As appropriate, 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 happens to the data in the Registry?

    The overall aim of the INPDR is to develop and sustain a unique patient-led disease registry 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 consider if it will help to achieve the overall aim of the INPDR. The INPDR will then decide to either provide anonymous summary data for analysis and to share results with patient advocates, clinicians, researchers, scientists and/or the pharmaceutical industry, ask for the request to be amended to ensure it aligns with the aims of the INPDR or reject the request completely.
    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-led 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 requests.

    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.