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You can find answers to the most common questions about the INPDR here. This page will be updated on a regular basis depending on the feedback we get.
If you have any questions that aren't answered here, please get in touch.
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 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 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-owned 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.
Is my information safe?
All data is stored on the Registry, an electronic database that has been developed by 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 protect regulation.
Patient data is stored anonymously, meaning the data cannot be linked to a patient. Instead, all Clinician reported patient data 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 to offer more data security.
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 instructions so you can give your consent to join the INPDR. Once your consent has been given, your clinician will be able to data 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 NPD 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.
Although we can learn more if both you or our family and your clinician provide information, 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.
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.
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.
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.