Transforming Duchenne Care Initiative Core Team Meeting 4/11-12, 2013

Meeting Summary

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

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


As a result of this meeting, the Transforming Duchenne Care Initiative was started in order to address the above key themes.  A smaller working group (the “Core Team”) was formed and began working together in the fall of 2012 to address these key themes.


The TDCI is divided into 2 phases


Phase I: Transparency of clinical services


The goal of Phase I is to make information regarding access to specialty and subspecialty care available to people with Duchenne in North America, to make this information transparent and easily accessible to patients and families via an online website and written materials, enabling families living with Duchenne to make informed decisions regarding access to specialty care at individual clinics.


In collaboration with the TDCI, the MDA agreed to enhance their clinical pages.  Each clinic page now includes clinic specific information (address and phone number, contact person, appointment scheduling information, clinic directors name NMD specialty, other sub specialties available at the clinic).  Clinics are encouraged to view their page and alert the MDA if there are errors or updates.  The MDA plans to add additional information that they will collect with their new patient registry.  The comprehensive registry will include clinician entered patient specific information, as well as, clinical care data.  Currently, the registry is in the process of beginning piloting in 25 clinics.  The MDA estimates that they should have data to share with the community in 3-5 years; they will wait to decide what information will be shared with whom when the data collection begins.


There was extensive discussion regarding the sharing of information pertaining to clinics that are not MDA affiliated. Through DuchenneConnect we have learned that a number of clinics are not affiliated with the MDA.  It was decided that these clinics/providers should be available online.  Information will be collected and posted online by summer 2013.  Information on the MDA supported clinics will be posted as well. 


Phase II: Transparency of clinical Care


The goal of Phase II is to encourage clinics to adopt baseline levels of care as described in the CDC Care Considerations and/or more current guidelines, thereby helping to ensure that people living with Duchenne and Becker receive consistent, optimal baseline care in accordance with these guidelines. Care in five sub-specialty areas will be reviewed.  These areas include: neurology, cardiology, pulmonary, physical therapy/physical medicine and rehabilitation, and coordination/communication. Patient outcome data will be collected, which will inform quality improvement efforts.  


It was decided to add an additional subspecialty – endocrine/bone health.  Each subspecialty discussion leader gave a brief presentation followed by discussion.  The subspecialty groups include (leaders are in bold):


Physical Medicine & Rehabilitation/Physical Therapy​​​​                                  

Nanette Joyce

Mike Dichiaro

Craig McDonald

Laura Case

Jackie Smith​​​​

Helen Posselt

Dennis Matthews​​​                                                     

Sue Apkon


Coordination and Communication

​​​Theresa Miller

​​​​ Andrea Manning

Jennifer Olson

Lisa Dellefave-Castillo

Susan Hernandez

Alissa Clark

Stacy Zamora

​​​​​Christina Trout

Ben Salinas

Kim Creamer

Cynthia George

Cardiology​​​​                                          ​​​    

Linda Cripe​​​​                                       ​​                                   

Larry Markham

Beth McNally​​​​                                 

Nancy Halnon​​​​                                

Jeff Towbin​​​​

J. Lynn Jefferies

Stephanie Killian

Donna Saccamanno



Jonathan Finder​​​                         

Richard Shell

Jodi Wolff

Joe Lorio

Brian Denger


Endocrine and Bone Health

Garey Noritz

Ellen Wagner

Paula Clemens

Annie Kennedy

Alison Willis



Jim Dowling

Brenda Wong​​​

Michele Yang​​​​

Jerry Mendell​​​​

Kevin Flanigan​​​​​​

Pery Shieh​​​​

Mathew Wicklund​​​

Susan Iannacone

Mena Scavina

Barry Burnett

Yaacov Anziska

Jen McNarry

Saunder Bernes

Larry Charnas


Each subspecialty group is tasked with identifying two to three variables that could serve as metrics by which care in that subspecialty could be evaluated.  May 10, 2013 was set as the deadline for variables to be identified. 


The next step will be to develop a survey using the variables. The survey will collect aggregate data specific to each clinic, but will not be patient specific. A patient reported survey will also be developed, either on an independent website or through DuchenneConnect that will collect patient specific data for the same variables.  When collated, the patient reported data will be de-identified and only identified by their clinic.  The surveys will be piloted in the 17 clinics associated with the TDCI.  After the surveys are refined, they will be piloted in 5 clinics not associated with the TDCI.  We hope to get clinician and patient responses, compare the answers, and identify whether care is being provided in agreement with the Care Considerations.  We will work with experts in Quality Assurance to be certain that the data collected is meaningful and usable.  The information, per clinic, will be publicly available, most likely on DuchenneConnect.


We are only in the beginning phases of this very complex project.  We will do our best to keep everyone informed of our progress. 

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