MDA MOVR Data Hub

With nine new drug approvals in the last five years and close to 300 therapeutics in development, the field of neuromuscular disease is expanding rapidly and there is a constant demand for patient data to inform drug development efforts by industry and to improve clinical management. 

Recognizing this need, MDA has leveraged its strong, historical relationships in the medical and scientific communities to efficiently capture clinical data from regular clinic visits happening already through its nationwide network of specialty Care Centers.  The MOVR Data Hub expanded on the US Neuromuscular Disease Registry (USNDR) pilot registry and has enrolled over 3,800 participants across 46 clinical sites to date.  Spanning seven neuromuscular diseases (NMDs) (ALS, BMD, DMD, SMA, LGMD, FSHD and Pompe Disease), MOVR captures real-world data sources through a state-of-the art technology platform, plus has integrated a custom Visualization and Reporting Platform (VRP) for user-friendly advanced data analysis.

Over 300,000 children and adults live with neuromuscular disease in the US. There are over 40 different types of neuromuscular disease, all of which are considered a rare disease. In the last five years, we have seen a rapid expansion in the field of neuromuscular disease with nine new drugs approvals and close to 300 therapeutics in development.  With such rapid expansion, there is a constant demand for patient data to inform drug development efforts by industry and to improve clinical management. Yet, there is a lack of unified data available. MDA's MOVR Data Hub aims to solve this problem by leveraging our nationwide network of Care Centers where over 60,000 patients living with neuromuscular disease are seen each year. MDA has a unique opportunity to grow the data set by efficiently capturing clinical data from patient visits that are already occurring. Additionally, we have the opportunity to collect data across multiple neuromuscular diseases, adding impact to the data collected as progress in one disease often translates to progress in another. 

MOVR data are captured through electronic Case Report Forms (eCRFs) and electronic health record (EHR) integration on a web-based portal developed by IQVIA. There are four eCRFs: (a) diagnosis, (b) demographics, (c) encounter, and (d) discontinuation. There are many core data elements that are captured across all indications, including: 

• Demographics (disease type, enrollment date, gender/DOB/race/ethnicity, insurance, education, and employment);
• Diagnosis (date and age at diagnosis, clinical diagnosis, muscle biopsy, body region affected, family history, molecular and DNA results, and gross and developmental motor milestones); 
• Encounter (encounter date, height and weight, clinical trial participation, surgical history, falls and hospitalizations, medications, mobility, assistive devices, disease progression, spinal conditions and neuroimaging, nutritional and GI therapies, pulmonary and cardiology care, and multidisciplinary care); and
• Discontinuation (cause and date, reason for study withdrawal, date of death, and cause of death).

The data are then transformed into the Clinical Data Interchange Standards Consortium (CDISC) standard format on a nightly basis.

MOVR data is shared through data use requests and approved agreements are executed based on the type of request.  MDA allows for both academic and industry (life sciences) requestors to obtain access to the data and offers a selection of customizable data deliverables.  

Neuromuscular diseases impair muscle function and can affect an individual’s ability to run, walk, speak, eat and even breathe. MDA currently serves the over 300,000 individuals living with muscular dystrophy, spinal muscular atrophy, ALS and related neuromuscular diseases across the United States. Individuals range in age from birth to natural death and represent a diverse demographic of multiple races and ethnic backgrounds. 75% of the individuals we serve are reliant on Medicaid, Medicare, or other state or locally funded insurance programs. Over 60% of those we serve come from low-to-moderate income households. 100% of the individuals MDA serves have a disability. 

Including those we serve in the goals and strategies of our programs is a critical component of our organization. We seek input and guidance from the neuromuscular disease community in numerous ways. A few members of our Board of Directors are individuals living with a neuromuscular disease or a family member of someone living with neuromuscular disease. We also have several staff employed on MDA’s fundraising and program teams that are living with neuromuscular disease or have a family member who is living with neuromuscular disease. MDA’s National Ambassadors also play a critical role in driving our mission as they interact directly with our sponsors and donors, sharing their personal stories and ideas. We also collect insights from those we serve through outreach calls and data collected by our National Resource Center, formal survey research, panel discussions and program-specific evaluations. This feedback drives our mission, helping us to understand the unmet needs and challenges facing the NMD community and MDA’s role in supporting those needs. More specific to MOVR, we are currently working on expanding our governance structure to include a Community Advisory Committee made up of active MOVR participants.  This committee will meet regularly to gain insights into registry best practices and continuous improvement around data collection and sharing channels. 

Many individuals living with neuromuscular disease have a shortened life expectancy due to the complications of their disease. For example, individuals living with ALS often only live 3-5 years after their diagnosis and individuals living with Duchenne muscular dystrophy often pass away before they reach their 40s. All individuals living with a neuromuscular disease experience a lower quality of life due to their disability. For example, many individuals must use a power wheelchair to get around or they are unable to work or live independently. According to a 2012 cost of illness study conducted by MDA and The Lewin Group, the estimated costs of illness to the nation for just three of the neuromuscular diseases (ALS, Duchenne muscular dystrophy, and myotonic dystrophy) are over $1 billion annually. While clinical practices and research has come a long way in increasing life expectancy and quality of life, approved treatments and therapies are still limited.  The data collected by MOVR will drive understanding into the course of the disease, help predict disease progression, and speed up recruitment for clinical trials, ultimately leading to additional treatments, therapies, and possibly even cures. In addition to accelerating therapies and improving clinical practices, MOVR will help to address health disparities as it identifies gaps in treatment and clinical practices for other minority populations such as Blacks, Hispanics and Native Americans. 

MDA's MOVR Data Hub technology aligns with the Horizon Prize Challenge as its main goal is to accelerate the development and approval of treatments for individuals living with a rare disease. The data collected by MOVR will assist scientists and healthcare providers in understanding the course of the disease, identifying trends and gaps in treatment and areas needing further research. It can also quickly identify and mobilize clinical trial participants.  MOVR is a collaboration that brings together both patient and provider-reported data and then makes that data accessible to scientists and clinicians to drive innovation.

The MOVR Data Hub has expanded on the US Neuromuscular Disease Registry (USNDR) pilot registry and currently has over 3,800 participants at 46 active sites. We are currently in a phase of growth focused on expanding the MOVR Data Hub to all 150 of our MDA Care Center sites by 2023 and growing the number of participants by 1,000 or more each year. MOVR has also grown from a pilot with data on just four diseases, to now collecting data on seven diseases and a goal of nine diseases in 2022.

While there are other disease registries, MOVR's integration into our extensive Care Center network sets it apart by providing an unmatched level of stability and scalability. With access to and support from a network of healthcare providers treating the diseases of interest, data can be entered accurately and efficiently for thousands of patients.

MDA's MOVR is also innovative in its robust and representative coverage of diseases, patients, sites, physicians and clinical practices. It offers credible data types and collection methodologies that provide scientists and clinicians the information they need to make discoveries and enhance patient care. MOVR can enrich, enhance, and add to existing data by modifying the electronic Case Report Form (eCRF). The customizable Visualization and Reporting Platform (VRP) allows data sets to be manipulated into Projects with advanced data analysis tools.

MOVR is already making a significant impact in the neuromuscular disease space as well as in broader life science research. The data captured by MOVR offers clinicians and researchers insight into:

• Development/refinement of standards of care
• Quality improvement
• Clinical best practices
• Characterization of genotype phenotype correlation
• Combination of patient perspectives & clinical data sets 
• Identification of pre symptomatic infants via newborn screening
• Characterization of patient experience 
• Tracking natural history of disease 
• Real world data (RWD) 
• How therapies are changing the course of a disease

On a broader scale, the use of MOVR Data Hub is providing value for life science research by enabling:

• Study/Trial feasibility and design
• Descriptive analysis
• Comparative analysis and 
• Market Reporting

MDA's MOVR Data Hub is powered by a state-of-the-art web-based portal developed by IQVIA. MOVR captures real-world data sources through this platform. In 2020 MDA partnered with DNAnexus, the leading cloud-based SaaS company serving the life science community, to unleash the power of its MOVR Data Hub and develop the MOVR Visualization and Reporting Platform (VRP). The VRP offers user-friendly advanced data analysis that MDA Care Center clinicians and researchers can use to analyze and visualize data across multiple Care Center sites in order to better understand disease progression and compare outcomes across therapeutic categories.

The security, compliance and collaboration components of the platform make MOVR data increasingly accessible to the wider neuromuscular clinical and research communities. Targeted efforts include training, fulfilling data requests, reviewing publications, encouraging peer-to-peer collaboration and publishing, as well as sharing learnings across multiple channels to drive awareness. MOVR data now complies with privacy and HIPAA regulations and conforms with the FDA-required format for observational clinical studies.The MOVR VRP is HIPAA, FAIR data principals compliant and has the ability to transform the data to CDISC SDTM (FDA submission data standard).  MOVR also has the ability to interface with other data registries and data sets and utilizes Global Unique Identifiers and data use policies to ensure appropriate protections for patient privacy. The portal also has the ability to extract clinical information from Electronic Health Records (EHR). In the future, MOVR will also capture data directly from the patient through apps, wearable devices and digital devices.

At the 2021 MDA Virtual Clinical & Scientific Conference, two researchers presented first use cases of MOVR data. One of those researchers was Susan Apkon, MD, Chief of Pediatric Rehabilitation at Children’s Hospital Colorado. Her team at Children’s Colorado set out to understand the care they are providing to boys with Duchenne muscular dystrophy (DMD) and to assure equitable care is delivered. This quality improvement work utilized the MOVR database and the VRP to compare their hospital’s use of corticosteroids and cardiac medications in non-Hispanic and Hispanic boys.

The results showed that 90% of non-Hispanic boys compared to 61% of Hispanic boys received corticosteroids at some time while under their care. A similar difference was found when looking at cardiac medication use, with 57% of non-Hispanic boys on these medications compared to 31% of Hispanic boys under their care. 

“These results are a starting point for our team,” Dr. Apkon said. “They showed we have work we need to do.”

For example, while members of her team believe they discuss corticosteroid and cardiac medication use with all patients, they need to confirm this. In addition, if they find that members do discuss those medications with all families, they need to better understand why some families choose to start the medications while others do not.

Use of MOVR allowed Dr. Apkon and her team to get a glimpse into that aspect of their care, and now the team has clear marching orders to ensure that they provide all patients with the highest level of care regardless of race or ethnicity.

As with all patient-related data, privacy and security are at risk. To mitigate these risks the MOVR EDC system is hosted on a secure registry platform compliant with the Health Insurance Portability and Accountability Act (HIPAA) of 1996 and its implementation requirements, which govern the privacy of protected health information (PHI). The registry platform is also compliant with the FDA 21 Code of Federal Regulations Part 11 standards, which governs the proper use of electronic records and electronic signatures.

 The MOVR platform undergoes an annual independent HIPAA risk assessment and International Organization for Standardization (ISO) 27001 certification, an international standard for information security. MOVR uses industry technology standards to implement administrative, physical, and technical safeguards to protect the accessibility confidentiality, and integrity of PHI and any other confidential data. All such safeguards are in accordance with applicable federal and state laws.

Only authorized site personnel have access to the fully identified information for participants at their site. Personnel from one site are not able to view identifiable data about participants enrolled at another site. Third-party vendors contracted by MDA to build tools and services associated with the MOVR database may also have access to identified data if necessary, assuming such data are stored and accessed within HIPAA-compliant systems.

The MOVR Data Hub currently has just over 3,800 participants. By the end of 2021, we project 4,372 participants and by the end of 2022 there will be 5,428 participants. By the end of 2023, we project just over 6,800 participants.

To understand the full impact of the MOVR Data Hub though, we have to look at the number of lives that will be impacted by the data collected.  There are over 300,000 individuals living with a neuromuscular disease in the US. Potentially all of these individuals could be served by this solution as it propels new therapies and treatments and enhances clinical practices for all those impacted by these diseases.

The long-term outcome of MDA's MOVR Data Hub is to improve individual health outcomes and accelerate drug development for neuromuscular diseases.

Over the next year, MOVR is focused on expansion and will:

• Activate 24 new MDA Care Center sites
• Enroll 1,000+ participants
• Implement direct to patient capabilities (Patient Related Outcomes captured through surveys and/or scales via smartphone technology)
• Providing longitudinal data analysis, refreshed quarterly
• Implementing GUID

Over the next five years, MOVR will continue focusing on expansion while also building an innovation ecosystem. This includes:

• Expanding to 100+ MDA Care Center sites
• Doubling the number of participants enrolled
• Capturing data on 11+ neuromuscular diseases
• Partnering with other accredited data sources to grow the breadth and depth of information available
• Building genomic sequencing/linkage capabilities

Through these short-term objectives, the MOVR Data Hub will provide researchers and clinicians with insights into how drugs and other treatments affect outcomes, how clinical trials could be better designed, and how neuromuscular disease affects people the same or differently. It will also ensure that doctors can quickly identify patients who may benefit from new therapies or who may want to participate in a clinical trial. On a broader scale, MOVR will also assist life science researchers in study/trial feasibility and design and can be used as a model for other rare diseases looking to compile similar data to improve clinical outcomes and accelerate treatments.

Measurable indicators to measure MOVR's progress include:

• The number of actively participating sites 
• The number of participants enrolled at each site and overall
• Site-specific metrics including
  • Adherence to activation timelines
  • Time to first participant enrollment and
  • Indication expansion
• The number, variety, and quality of research collaborations (industry and academic partnerships)
• The number of scientific publications resulting from MOVR data
• Quality improvement plans for the MDA Care Center Network as a result of MOVR data

Full-time staff: 5

Research Advisory Committee: 7 (volunteer)

MOVR Publication Subcommittee: 6 (volunteer)

Participant Leadership Committee (currently identifying): To be determined depending on interest. (volunteer)

Under the direction of MDA's Chief Research Officer, Dr. Sharon Hesterlee, the MOVR team is led by Rayne Rodgers, MPH. Rayne is responsible for MOVR operations, governance, strategy, performance, communications, data use and business development.  With a deep commitment to patient advocacy and years of experience in drug development and clinical trials, Rayne believes that MOVR will serve as a valuable tool for clinicians and researchers looking to improve care and develop new therapies for neuromuscular diseases.  In addition to experience with both large pharmaceutical and small biotechnology drug development, she has also successfully developed and launched patient registries in rare patient populations.  Rayne holds a BS in Finance from the University of Florida and a Master’s Degree in Public Health from Emory University.

MOVR has recruited team members from other departments within MDA, such as Innovations in Care and Research Grants, in order to bring patient- and research-facing perspectives into every day MOVR decision-making and operations.  MOVR is guided by a dedicated Research Advisory Committee, made up of research leaders in the neuromuscular disease space across top academic clinics. MOVR is also currently developing a Participant Leadership Committee, through which MOVR participants (patients) will directly advise on the purpose, direction and priorities of the data hub.

At MDA, a diverse, dynamic and inclusive culture underlies the success of our association. As an organization that fundamentally supports individuals and families who are affected by the structural inequities related to disability, MDA understands that diversity and inclusion widens our vision and mission. It helps our employees achieve their professional objectives to support the people we serve.  Different backgrounds and characteristics, such as race, ethnicity, gender, disability, culture and sexual orientation bring innovative viewpoints and skills to a company. Integrating diversity and inclusion initiatives across the organization is essential to our business strategy and long-term success. 

Therefore, MDA has established resources and diversity objectives, that are measured through annual reporting, to help ensure we recruit and maintain a diverse talent pool. Objectives include:

• Expanding and embracing diversity across our organization.
• Strengthening our partnerships with diverse national and local community organizations.

To successfully achieve these objectives, we also offer internal staff training and development opportunities that will help drive our organization’s culture of inclusion. Training includes:

• Diversity 
• Affirmative Action
• Working with People with Disabilities
• Leadership/Manager Training Workshops

Individuals living with neuromuscular diseases are involved with MDA on many levels, from participation in the National Board of Directors, to staff employed on MDA’s fundraising and program teams, to volunteer speakers, advocates and our National Ambassadors. We frequently gather insights from the neuromuscular disease community through surveys, panel discussion, and advisory committees.

No, however we are currently creating a Participant Leadership Committee for MOVR that will be comprised of individuals living with a neuromuscular disease.  This committee will provide valuable input into the strategy, growth, and impact of MOVR.

The Horizon Prize Challenge asks how technology can help people with rare diseases get the right care faster and more accurately.  MDA's MOVR Data Hub provides this solution, more specifically for individuals living with neuromuscular diseases, but also broadly as a model for all rare diseases.

MOVR will help individuals living with a rare disease, such as Duchenne muscular dystrophy or spinal muscular atrophy, have access to better care and treatments by providing academic and industry researchers with insights into how drugs and other treatments affect outcomes, how clinical trials could be better designed, and how neuromuscular disease affects people the same or differently. It will also ensure that doctors can quickly identify patients who may benefit from new therapies or who may want to participate in a clinical trial.

If awarded, funds from The Horizon Prize Challenge will make a significant impact in expanding the reach and capabilities of MOVR. Additionally, the opportunity to collaborate with and learn from other Solvers and from MIT faculty will be incredibly beneficial to the scope and impact of MOVR and will help us achieve our goals faster, bringing urgently needed solutions to the individuals and families affected by neuromuscular disease.

Our biggest barrier to reaching our goals for MOVR is budgetary constraints. We need to be able to increase funding in order to expand MOVR and fully implement its capabilities. To increase funding, we could benefit from collaborative opportunities to better understand product-market fit and to develop a stronger pitch as we meet with investors. 

We are always looking for ways to strengthen the impact of MOVR and could benefit from additional external evaluations on MOVR's capabilities, data usage and impact. This would include evaluations around the technology and ways we can improve upon or advance MOVR's design.

As we expand MOVR to include Patient Reported Outcomes (PROs), learning from other Solver Team such as BreathResearch or Digital Cognition Technologies would be beneficial. One of the goals of MOVR is to integrate smartphone apps and wearable technology to collect data straight from the patient.

MOVR could also benefit from collaborating with Solvers and MIT faculty skilled at identifying product-market fit and developing a strong pitch that will help us secure additional funding from various revenue streams.