RARE-X BPAN PLATFORM: WORKING TOGETHER WITH OUR GLOBAL BPAN/NBIA COMMUNITY TO ACCELERATE BPAN RESEARCH

 

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RARE-X is a 501(c)(3) nonprofit created by leaders in the fields of patient advocacy, medical research, biopharma, and technology.

RARE- X is piloting a series of demonstration projects, in partnership with rare disease communities, to apply technology proven in other large-scale public health and genomic data-sharing initiatives to support researchers developing treatments for rare disease patients.

RARE-X is leveraging existing technology powered by the Broad Institute of MIT and Harvard, which will support patients (in data collection, structuring and responsible sharing), clinicians (in accelerating diagnosis and improving and tracking health outcomes), researchers and biopharma (with the data they need to identify, develop and track the impact of breakthrough treatments and cures).

Since RARE-X is a nonprofit, there is no cost to you or the BPAN community. RARE-X’s mission is to serve patients, and drive research towards therapeutic development.

Our goal is to make the process as easy as possible for you. You can learn more about this one-of-a-kind Data Collection Program and how it will help to serve our BPAN patient community.

Megan O'Boyle, RARE-X, Patient Engagement Lead

NBIA Disorders Family Conference 2021

I'm Confused! Why more data collection?​

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Collaboration is key to our success!

Our BPAN/NBIA Community Partners

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RARE-X: TRANFORMING BPAN PATIENT DATA INTO REAL WORLD TREATMENT OPPORTUNITIES
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BPAN WARRIORS PARTNERSHIP WITH RARE-X

BPAN Warriors and the BPAN community is partnering with RARE-X [a 501(c)(3) nonprofit] to build a Data Collection Program for BPAN families.  When you participate in the BPAN Data Collection Program, you’ll help accelerate research and the development of new drugs, devices, and other therapies. 

The BPAN Community is building the BPAN Data Collection Program to …

  • Inform researchers how BPAN changes over time

  • Enable better data to use in clinical trials

  • Give patients the opportunity to participate in clinical trials

  • Reduce the time it takes to study new medicines

  • Speed up the time to get treatments to patients

  • Enable the use of data as a placebo (instead of actual patients) in a clinical trial

 

The BPAN Data Collection Program will be patient-owned but enabled by RARE-X technology. All data governance, consent support, and data security will be put in place by RARE-X.  BPAN families will benefit from a streamlined process for collecting research-ready data in a phased approach.

 

PATIENTS' DATA POWERING PROGRESS

Through RARE-X we hope to affect positive change in the cost and the lack of access to reliable and comprehensive patient data that are critical factors influencing the speed and productivity of innovation:

  • The data exists, but is captive within silos, institutional or company owned, because of institutional / technological constraints, or is being held for exclusive commercial purpose

  • The data may exist, but not in a structure/form where it is available, visible or useful to researchers or patient communities.

  • The data doesn’t yet exist (because patients can’t access genomic or other testing, there is not a way to effectively collect patient-reported information, etc.).​

RARE-X IS…

  • RARE-X is a 501(c)(3) nonprofit created by leaders in the fields of patient advocacy, medical research, biopharma, and technology.

  • RARE- X is piloting a series of demonstration projects, in partnership rare disease communities, to apply technology proven in other large-scale public health and genomic data-sharing initiatives to support  researchers developing treatments for rare disease patients.

  • RARE-X is leveraging existing technology powered by the Broad Institute of MIT and Harvard, which will support patients (in data collection, structuring and responsible sharing), clinicians (in accelerating diagnosis and improving and tracking health outcomes), researchers and biopharma (with the data they need to identify, develop and track the impact of breakthrough treatments and cures).

  • Since RARE-X is a nonprofit, there is no cost to you or the BPAN community.  

  • Our goal is to make the process as easy as possible for you. By clicking on the link below, you can learn more about this one-of-a-kind Data Collection Program and begin the first step in making your patient information available to researchers.

 

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RARE-X was founded on these core principles:

  • Patients’ data is critical to rare disease innovation — but does little to help advance progress if it is not widely accessible for research

  • Researchers need access to more and better data to transform rare disease drug development. Providing a way to silo-break these data sets is essential to progress.

  • Rare patient communities want their data shared, securely but openly — where it can drive better, faster progress

  • Meaningful, equitable advancement in rare disease innovation requires global collaboration and sharing of data

  • If patients are empowered as data stewards, it benefits all involved in rare disease diagnosis, research, discovery, drug development and treatment

  • Patients’ data should only be shared with their consent and for their primary benefit

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“The measure of greatness in a scientific idea is the extent to which it stimulates thought and opens up new lines of research.”

Paul Dirac