Clinicians

​The INPDR Clinician Reported Dataset is a prospective, multi-centre, multinational, observational registry aiming to enroll at least 1,000 NPD patients, including Acid Sphingomyelinase Deficiency (ASMD) and Niemann-Pick disease Type C (NPC). Participants are expected to be followed for a minimum of 5 years after enrolment.​

The INPDR team will support you in opening the  CRD study, and we have a range of site support approaches to address barriers to your participation. ​

Once the CRD study is approved at your centre, eligible patients can be enrolled and data entry using INPDR’s electronic data capture (EDC) system can begin. As an observational study, no additionally clinic visits, assessments or examinations are required, and treatment status is not an eligibility criteria.

INPDR’s data quality team will support your site with ensuring data entered means data quality standards, including through on-site monitoring visits and remote meetings where possible.

Data is collected through a web-based EDC system that’s accessible to pre-approved users at authorised study sites. The EDC system is hosted and maintained by OpenApp (an IQVIA company), based in Dublin, Ireland. ​

The CRD study protocol is designed to offer flexibility regarding how often data is collected. Data is expected to be collected in line with a patient’s standard of care visit schedule. The study protocol does not require any additional visits or investigations outside of the patient’s standard of care, and retrospective data can be entered.

The INPDR is the data controller – but we don’t own any data. Patients consent to having their data captured by the INPDR and can withdraw their consent at any time.​

​What we do make sure is that all patient data is maintained and protected to the highest regulations and standards.​

INPDR is the data controlled for all registry data and is responsible for managing and protecting the data held within the registry.  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​.

The CRD collects data for both ASMD and NPC, with differing data being collected for each disease. Data to be collected includes demographic information, genetic information, diagnosis, medical history and co-morbidities, treatment history, physical assessments and disease-specific assessments. Only data generated from routine standard of care will be collected in the CRD.​

Applicable research governance approval, including Institutional Review Board/Research Ethics Committee, is required before any study activities can take place at site. We can provide all necessary documents for applying for research governance approvals, as well as any additional documents that may be required.​

Sponsorship of the CRD is arranged by either the country lead site or with the individual study site, depending on the research governance practice of the country. In addition, data sharing agreements are arranged between the study site and the INPDR to allow for data transfer.

Patients can withdraw their consent at any time without reason. If one of your patients no longer wishes to take part in the Clinician Reported Data, they can decide what will happen to any data already entered into the EDC system, such as whether it is maintained or deleted. Any research results already generated from your clinical data cannot be destroyed or recalled– but please be assured they are completely anonymised.

Our aim at INPDR is to provide as much support for study sites as we can. We have a range of site support approaches designed to reduce barriers to opening the CRD registry study. Additionally, registry study set-up documents, including translations, are provided to all sites, and we can produce any additional documents required.​

Furthermore, INPDR encourages participating clinicians to undertake registry investigator-initiated research. This includes using registry data to support local, national or global research projects, co-authorship and collaboration opportunities with other registry investigators, and ability to utilise the CRD to inform a national registry partnership with clinical colleagues  within your country. INPDR will support registry investigators as much as possible to maximise the value of this global registry. 

Yes – as a clinician you can submit a data access request to access CRD data to support any registry investigator-initiated research. 

The INPDR isn’t just an international registry and can function as your national registry. You have the autonomy to collaborate with your clinical colleagues and conduct your own national independent analyses without needing permission from the INPDR. All we request is that you inform us about your research and acknowledge the INPDR’s contribution. ​

​While you have the option to seek peer-review from our Scientific Advisory Committee and enlist the support of the INPDR team for your analysis, this remains at your discretion since the INPDR is fundamentally your registry. ​​

Of course, the global reach of the INPDR is one of its greatest strengths, The international database is available to support your research. For this, we will require your study to be peer-reviewed by the Scientific Advisory Committee and approved by our Board. We are keen to witness the registry being utilised to its fullest potential, so please don’t hesitate to reach out.​

Traditionally, disease registries were created as product registries owned by pharmaceutical companies. This led to siloing of valuable data and duplication of work, where multiple registries exist for one disease. ​

Instead, the INPDR is a patient registry that covers all Niemann-Pick diseases regardless of a patient’s treatment status. The aim of the INPDR is to be a single disease registry which can support the data requirements of different stakeholders, negating the need for multiple registries for Niemann-Pick diseases.​​

The INPDR can serve as a data source to support pharmaceutical industry requirements, including post-authorisation studies which may be required by regulatory agencies such as the European Medicines Agency and the US Food and Drug Administration, as well as opportunities to support the development of new therapies.​

Any work undertaken with pharmaceutical organisations is governed by agreements and managed with oversight from our Scientific Advisory Committee and board of trustees. 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. No access to direct patient-identifiable raw data is provided.