Patients & families

If your clinician has joined the INPDR, they’ll contact you to offer you the opportunity to take part in the Clinician Reported Database (CRD). 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 securely add your clinical data to the CRD. 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. Although your clinician will need to join to add your clinical data to the Clinician Reported Data part, you can join the Patient Reported Data through the INPDR website – inpdr.org/sign-up 

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.

If you take part in the Patient Reported Database, the INPDR team manages the PRD and can see the data of all participants. INPDR team accesses this data to support participants with enrolment and verification, as well as providing help with any issues. 

By joining the INPDR, you’re helping collect key clinical and lived experience data which can be used to support a wide range of research areas, including natural history development, treatment effectiveness and patient and caregiver reported outcomes. Additionally, your data can be used to support multiple studies as data is reuseable across different research. 

It’s really important for you to know that taking part in the CRD and PRD is voluntary and you are free to withdraw at any time without a reason. If you decide to stop taking part, you’ll be asked  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.

INPDR is responsible for managing and protecting the data held within the registry. The data you contribute remains yours, and participation in the INPDR is entirely voluntary.  Participants provide consent for their information to be included and may withdraw that consent at any time, in accordance with the terms of the consent process and applicable data protection laws.

As a community-driven registry, decisions about how data is used are made through the INPDR’s governance processes, including oversight by the Board of Trustees. Data is only used for approved purposes that are ethical, scientifically valuable, consistent with participant consent, and intended to benefit the Niemann-Pick community. INPDR is committed to ensuring that the interests of patients and families remain at the heart of all decisions relating to registry data.

INPDR maintains processes and controls built into the database to ensure high-quality data is collected.  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 information collected by the INPDR is used to support research that improves understanding, care, treatment, and future outcomes for people affected by Niemann-Pick disease. The registry helps create a global resource that can be used to answer important research questions and support the development of new therapies.​

As a community-driven registry, decisions about how data is used are made through formal governance processes, including oversight by the INPDR Board of Trustees. Data is only shared for approved projects that are consistent with participant consent and are expected to benefit the Niemann-Pick community, with robust safeguards in place to protect privacy.

Traditionally, many disease registries are drug specific and pharmaceutical company owned. Instead, the INPDR is a patient registry that covers all Niemann-Pick diseases, including treated and untreated patients. This model removes the need for multiple drug registries, which can cause data fragmentation, barriers to data sharing and unnecessary duplication.  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. ​

All work INPDR undertake 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. Registry data sharing with pharmaceutical companies is strictly controlled and monitored and is line with data access requests approved by INPDR’s Board of Trustees. ​