Right now, there are more than 10-12,000 young people diagnosed and living in the United States with Duchenne. These young people are being cared for by clinics both near and far. Every one of them deserves the best care and treatment possible. Families want care and treatment to be transparent; they want to know who is delivering care to their child, what comprehensive care looks like, and which centers are delivering that care. For this reason, a group of experts agreed to give families the information that they deserve and to develop a methodology and process by which to certify clinics delivering comprehensive care, and then measure results.

PPMD’s Transforming Duchenne Care Initiative (TDCI) set out to reduce discrepancies in care. The goal – to make comprehensive care, aligned with the recommendations of the DMD Care Considerations Working Group (DMDWG, which published the CDC Care Considerations standards of Duchenne care, made available in 2009 and published in 2010), available and accessible to as many patients across the US as possible.

Transforming Duchenne Care Initiative

In 2012, just prior to the Connect Conference, PPMD hosted the first Transforming Duchenne Care meeting. This meeting included health care providers and administrators from 17 institutions, parents, and representatives from MDA, CDC, NIH, and industry. Key themes emerged from this meeting:

• Currently in the US, there are significant discrepancies in care for patients with Duchenne.
• Parents would like to have transparent, accurate information regarding providers and services available at each clinic easily accessible, preferably online.
• Parents would like to know whether clinics are providing care in agreement with the CDC Care Considerations.
• Industry is having difficulty conducting clinical trials and research due to discrepancies both in care and models of care across the country.

The Transforming Duchenne Care Initiative (TDCI) was developed in order to address the above key themes.

The TDCI was divided into four phases.

Phase I: Transparency of clinical services

The goal of Phase I is to gather information regarding subspecialty care and services available to people with Duchenne in the United States, and to make this information transparent and easily accessible to patients and families using online and written materials, enabling families living with Duchenne to make informed decisions regarding access to specialty care.

In collaboration with the TDCI, the MDA enhanced their clinical pages. The MDA Clinic Pages now include clinic specific information (address and phone number, contact person, appointment scheduling information, clinic directors name NMD specialty, other sub specialties available at the clinic). In addition to the MDA information, families have communicated the need to have Duchenne-specific information, as well as information regarding non-MDA affiliated clinics/providers, available to them. In an effort to collect that information, the Clinical and Sub-specialty Services Survey was developed. This survey elicits Duchenne specific information such as, the number of patients with Duchenne seen, the length of time to obtain an appointment for a new diagnosis, the members and board certifications of the health care team, clinical services available and the structure of appointments, etc. This survey has been piloted by five neuromuscular programs and will be posted on the PPMD site. This phase is ongoing, with the goal of collecting and posting information online for all neuromuscular providers and clinics across the country that wish to have this information publicly shared.

Phase II: Transparency of clinical Care

In 2013, members of the TDCI Core Team met to discuss developing a method by which to evaluate Duchenne care. Members of the Core Team included experts in sub-specialty Duchenne care from across the United States. Measures for each sub-specialty were developed in agreement with the CDC Care Considerations. These measures were combined to create the Duchenne Care Survey. Development of this tool hopes to encourage clinics to adopt comprehensive care as described in the Care Consideration, improving care for all people living with Duchenne.

Phase III: PPMD’s Certified Duchenne Care Center Program 

The Duchenne Care Center Certification Program (CDCC Program) is the natural evolution of the TDCI, and has resulted in two additional phases. The mission of TDCI Phase III is to develop the CDCC Program. The CDCC Program Advisory Committee has developed Standards, which includes a complete description of the program, the process by which a center may become certified and the responsibilities of both certified centers and PPMD in this unique partnership. The current CDCC Program Advisory Committee (two year term – myself, Lawrence Charnas (Shire Pharmaceuticals), Jim Dowling (Sick Kids Hospital, Toronto), and Ellen Wagner (Parent, Chicago) have also developed Metrics for certified centers, which are “process measures,” that will demonstrate the continued efforts of centers to improve the care and services to people living with Duchenne muscular dystrophy.

Phase IV: Certified Duchenne Care Centers

Phase IV of the TDCI will certify Certified Duchenne Care Centers around the United States who are providing care in alignment with the Standards of the Certified Duchenne Care Center Program and in agreement with the CDC Care Considerations. The current certification committee (two year term – myself, Linda Cripe (Nationwide Children’s Hospital), Richard Finkel (Nemour’s Hospital), Dennis Matthews (Children’s Hospital of Colorado) and Susan Apkon (Seattle Children’s Hospital)) will review the center’s application, the Clinical and Subspecialty Services and Duchenne Care Surveys, participate in/review the summary from the center site visit, discuss findings and make recommendations regarding certification. If a center is granted Certification, they will remain a Certified Duchenne Care Center for 5 year and receive an annual stipend. This use of this stipend can only be used for Duchenne-specific program development and quality improvement projects, to further improve the care and services provided by that center. Centers will encourage/enable their families to register and annually update their information in DuchenneConnect. The information collected in DuchenneConnect will serve to validate the care provided, and to collect patient reported outcomes resultant of that care.

Centers that, for some reason, are unable to attain certification will be given suggestions and guidance from the Certification Committee. They will be given a year to implement those suggestions and the opportunity to reapply without beginning the application process again.

Certification of the First Duchenne Care Center

Jerry Mendell, M.D., director for the Center of Gene Therapy at Nationwide Children’s Hospital, has been involved since the beginning and has been integral to the development of the TDCI. For this reason, it was felt that Nationwide Children’s Hospital was the perfect hospital at which to pilot the inaugural certification process.

The surveys, site visit, and review by the Certification Committee are complete, and we are very proud and happy to announce that the first Certified Duchenne Care Center is at Nationwide Children’s Hospital.

PPMD’s plan is for this to be the first of several Certified Duchenne Care Centers across the country. We would like the program to grow slowly (5-6 centers/year). Certified Duchenne Care Centers will be spread across the country, making comprehensive care available and accessible to a great number of families. The certification of clinics is expected to ensure clinics maintain the highest standards of care and services, rapidly apply new evidence based knowledge and comply with standards for care that were established by the CDC Care Considerations. With this new program, we hope to Transform Duchenne Care.

Proposed Centers for Certification

We hope to certify five to six additional centers in 2014, with hopes of adding another five to six clinics in 2015. The proposed centers for certification in 2014-2015 include:

• Denver Children's
• Seattle Children's
• Cincinnati Children's
• Dallas Children's
• UC Davis Children's Hospital
• University of Iowa Children's Hospital

To learn more about PPMD’s Certified Duchenne Care Center Program, please contact Kathi Kinnett at Kathi@ParentProjectMD.org.

Read the press release.