International Patient Registry

WHAT IS THE INTERNATIONAL PATIENT REGISTRY?

Chiari malformation (CM), syringomyelia (SM) and related disorders (RD) affect millions of people worldwide. Starting on the path to improved care is daunting, to say the least.

An important early step is to establish the CSF International Patient Registry, which will be a collection of patient-reported data that can serve as an important tool for improving the lives of people with CM/SM/RD.

Physician-reported data will be added to supplement all patient information through the implementation of EHR integration set to be completed late this year or early next. Registries like this are essential for tracking the identification and diagnosis of people with these disorders and monitoring their health. They also help experts set priorities for health care.

AN INTERNATIONAL REGISTRY WILL HELP PATIENTS IN THE US AND ABROAD

Having an international patient registry means being able to answer the most fundamental questions about the disorders that are not well understood, even by the experts.

    • What is the actual incidence and prevalence of Chiari malformation, syringomyelia and related disorders?
    • What are the causes and risk factors associated with Chiari malformation? Are they strictly genetic or also environmental?
    • What is the pathophysiology of syrinx development?
    • What other clinically-relevant disorders are complicating the symptoms and treatment methodology?
    • What is the best treatment for these disorders?
    • When is treatment really necessary?
    • What is the public health and health utility burden of Chiari, syringomyelia and related disorders?
    • Etc.

Information like this is absolutely essential for lobbying government and advocating for improved care within the healthcare sector.

The registry will help international patients, too. Having an international patient registry is also useful, globally. Having standardized metrics will ensure that the global data that is collected is accurate and meaningful.

Being able to compare information among different countries can be helpful when lobbying for improved care with huge organizations like the World Health Organization and similar powerhouses.

 

IT WILL IMPROVE HEALTHCARE QUALITY AND REDUCE SPENDING

Quantifiable results contained in a national registry facilitate the measurement of the effectiveness of healthcare programs.

Basic data, such as increases in the number of patients diagnosed or the life expectancy of people with these disorders are useful in evaluating the worth and success of various development programs to improve care. This means, that current and future treatment options can be measured to see if they are working in their goals to help patients– and if they are not, they can be edited or terminated.  This will also keep costs down as more and more patients improve in quality of life and will need less frequent surgeries, hospitalizations and other care options that send medical bills skyrocketing.

The truth is, research takes many years. Sadly, most adults that are affected by CM/SM/RD must accept the fact that we may never have the answers for them. However, the projects that are started today will yield answers for children who are not yet born and the people who love them.

Bobby Jones CSF has already started the process to create the standards necessary for all this data to do its job! Since 2013, a collection of over 50 physicians from institutions all around the world have agreed to collaborate and create what are known as Common Data Elements. These are the data points that researchers will use in this registry, and with them safely in place, all of this work will improve the lives and outcomes for patients!

 

HOW THE REGISTRY IS OF USE TO INTRA AND EXTRAMURAL RESEARCHERS

We are hoping to recruit as many patients as possible, particularly those who may have positive outcomes after treatment and therefore may never interact with something such as a patient registry (contributing to selection bias in current datasets and the literature, overall).

A bit about the registry…

Broadly speaking, the goals of the ambitious dataset that we are creating are to:

  1. Normalize the use of Common Data Elements (CDEs) and better standardize measurements/scales in the literature related to Chiari, syringomyelia and related disorders;
  2. Provide an as-of-yet unprecedented dataset (and, therefore, sample size) for Chiari, syringomyelia and related disorder populations that will be accessible to researchers around the globe for prospective and cross-sectional research;
  3. Include international perspectives and treatment methodologies into analysis in a more systematic manner;
  4. Build the evidence base that will be able to more definitively link and/or distinguish Chiari to/from other medical conditions;
  5. Understand natural history and follow patients who have undergone successful treatments who are frequently lost-to-follow-up in conventional studies;
  6. And incorporate the patient and caregiver voice into clinical research.

Additionally—and importantly— one longer-term goal is to develop methodology for the incorporation of EHR data to all self-reported registry modules. This inclusion of the EHR will improve the scientific rigor of this dataset and create an international repository for investigators interested in these conditions, the likes of which has not been possible, until now.

 

HOW WILL THE PROCESS WORK FOR EXTRAMURAL RESEARCHERS?

We are int he process of developing a formal process by which researchers can reach out to access the dataset as it currently builds. If you have any questions in the meantime, please reach out to Kaitlyn Esposito by phone or email: 718-966-2593 | kesposito@bobbyjonescsf.org.

 

 

 

Revised: 9/2019