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Patients and families

FAQs

If you or someone in your family is affected by Niemann-Pick disease, we’re always here to help with questions. As a starter for ten, here are some of the questions we’re most often asked about the International Niemann-Pick Disease Registry (INPDR).

If your clinician has joined the INPDR, they’ll contact you to offer you the opportunity to take part in the INPDR. They’ll also provide you with all the information and instructions you need – so you can be comfortable giving your consent to join the INPDR (your data cannot put into the registry without your consent).

Once you’ve given your consent, your clinician will then be able to add your anonymised data to the INPDR.

If your clinician has not joined the INPDR yet, do also feel free to discuss this with them – the more people who join the registry, the more valuable it will be in helping to improve the lives of Niemann Pick disease patients.

Yes absolutely – 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 still join the Patient Reported Data directly through the website.

Yes. For you to participate in the Clinician Reported Database, your doctor will need to work with the INPDR to conduct this registry. By signing the consent form, you’re allowing your Personal Clinical Data to be shared with and used by INPDR. Data collected will be in line with the registry protocol. You can also decide your level of data sharing on the Informed Consent Form.

In terms of other people who will know you’re helping us at INPDR – your doctor may tell your GP and other specialized doctors about patients taking part in the CRD. Your doctor may also need to ask for more information from your GP about your medical history/medications you have taken, and your GP may be told about your progress during the study.

By joining the INPDR, you’re helping the Niemann-Pick diseases 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.

It’s really important for you to know that taking part in this study is voluntary and you are free to withdraw at any time without a reason.

If you decide to stop taking part in the study, we’ll ask you to complete a simple Withdraw Consent Form. You will have choices about how you wish to withdraw and these will be explained to you. This will not affect your standard of care or the benefits to which you are entitled. Any research results already generated from your clinical data cannot be destroyed or recalled– but please be assured they are completely anonymised.

Finally, a very important point: remember you do not have to take part in this study to be treated for your condition.

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

The Clinician Reported Data is provided by your clinician and includes diagnostic information, genetic information, clinical history, treatments, medicines and test results captured as part of your initial appointment and during follow-up visits.

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

The Information contained within the registry is made up of Personal Data like your name & date of birth, plus ‘Special Category Data’ which includes your medical information (e.g. disease, medical treatments and test results).

Your Personal and Special Category Data will be held extremely securely. It’s confidentiality, integrity and availability will be protected by advanced technical and organisational measures. We may use Data Processors (including OpenApp Ltd) to process INPDR data on our behalf. Where this is done, its use will be strictly controlled and governed by legally binding Data Sharing Agreements.

All data is stored on the registry – an electronic database that has been developed and is maintained by OpenApp Ltd. OpenApp Ltd. who have vast experience in health IT solutions and have developed the Registry so it is compliant with strict data protection regulation.

Within the Clinician Reported Data (CRD), participant data is stored anonymously meaning the data cannot be linked to you directly. Instead, your data is allocated a unique patient identification code, which is known only to your doctor and medical institute.

In the case of the Patient Reported Data (PRD), information that can directly identify you is stored within the Registry. Identifying information (such as name, phone number, and address) is held separately from your medical records. As with other online services, if you share your PRD account information or password with others, they may access your account and your information.

The INPDR have strict data protection policies and procedures. Access to all data is restricted to individuals at INPDR who are trained in handling sensitive data.

We will never share identifiable data with other third parties (also known as Data Controllers). Only Anonymised Data will be made available to carefully chosen research partners, with binding agreements in place to ensure your privacy is protected.

Data is owned by you, the participants.

Participants consent to having their data captured by the INPDR, but you can withdraw your consent at any time. You also have control over what happens with your data and who can access the data.

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

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

INPDR has developed a Quality Management Systems to ensure that data within the registry is of the highest standard. Verification and validation checks are performed regularly by Data Managers to ensure the validity and consistency of data. 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.

The overall aim of the INPDR is to develop and sustain a unique patient-led disease registry that supports research and improves care and treatment for NPD patients.

Requests for data are reviewed by our Registry Management Team and Scientific Advisory Committee to determine their scientific quality and the benefit to the NPD community.

Based on the advice from our Scientific Advisory Committee, the INPDR board will approve access to anonymised data in such a way as to protect patient privacy.

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.

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, info@inpdr.org