Transforming Duchenne Care Update: Part II - Meeting with the Cystic Fibrosis Foundation

It’s been a little while since our last update, but I wanted to wait until I had definite news. It’s been an exciting month for Transforming Duchenne Care (TDC), and I will do my best to get everyone up to speed. We had two important and informative meetings. Below is an update on our recent meeting with the Cystic Fibrosis Foundation, as well as PPMD’s contribution to Duchenne care. Click here to read about our meeting with the MDA.


The Cystic Fibrosis Foundation (CFF) is revered as the model of clinical registries, systematically collecting data, making that data transparent and accessible to patients and families, and utilizing registry data to improve care. Holly Peay and I were very fortunate to be able to spend a day with the CFF, where eleven members of their staff spent over three hours with us, discussing the principal components of their Care Centers, registry, clinic accreditation, and family advisory councils. The CFF began accrediting Care Centers in the 1960’s, when it was discovered that children who received multidisciplinary care lived healthier, longer lives. The CFF began to aggregate their data, and then gathered together key healthcare providers in order to begin to develop standards of care. Aggregated data evolved into the CFF registry. Today, the registry gathers key data elements, plus additional data, at each quarterly patient visit. The function of the registry is to pool knowledge in order to continuously improve the standard of care. 


The CFF also has developed a system of accreditation for their CF clinics, which are called CF Care Centers. Care Centers that are accredited have access to their clinic specific patient outcome and practice data, as well as aggregate data from all CF Care Centers, demonstrating how their clinic fares compared with the other clinics. All Care Center information is publically available and shared annually by report and on the CFF website, fostering a sense of community and sharing.  The CF community is then able to utilize registry data to review patient outcomes and evaluate practice patterns, which drive improvements in care.


Contributing to the registry is one requirement for clinic accreditation.  In order to become accredited Care Centers, clinical programs submit an application, which is reviewed by the accreditation committee, who decides if the applying site is worthy of a site visit.  If a site visit is granted, two members of the accreditation committee review the clinic. Their findings are then presented to the entire accreditation committee, who either grant accreditation or suggest improvements for the clinical program.  Care Centers may be accredited as either “core” or “affiliate” centers, core centers being larger regional centers that offer multidisciplinary care and affiliate centers being smaller local programs. Most clinics serving the CF population have applied for, and been granted, accreditation and there are CF accredited Care Centers in 48 states.  Accredited clinics are generally reviewed every 3-5 years.


Accredited Care Centers are granted funds that may be used as the center determines best, but must be accounted for annually.  Care Centers are also able to apply for additional grant funding that may be used for quality improvement projects.  To date, 97 of 168 accredited centers have received funding for quality improvement projects. Quality improvement projects began as any projects that would hope to enhance pulmonary or nutritional care. As the quality improvement projects have evolved, they have generated specific collaboratives for adult and pulmonary care.  Family advisory boards, which involve patients and families living with CF, develop at the center or hospital level, and have helped to inform quality improvement projects.


PPMD has already made generous contributions to the areas of patient and family involvement and patient reported outcomes.  The FACES (Families Advocating, Connecting, Educating and Supporting) groups are supported by PPMD and work to mentor newly diagnosed families, support national awareness and advocacy campaigns and raise funds to support PPMD research, advocacy, care and education. To date, there are FACES groups scattered across North America, giving families a local DBMD communities that are able to provide education, guidance and support. Groups meet regularly and are always available to DBMD families. 


The PPMD Annual Connect Conference prominently features patients and families bringing them together with clinicians, research and industry in an effort to create dialog between the entire community. The PPMD conference is a model that many organizations are attempting to replicate. 


DuchenneConnect, the PPMD Patient Registry, has been collecting patient-reported outcomes since 1997. The data in DuchenneConnect is used by industry to understand feasibility of clinical trials and recruit for clinical trials; by academic collaborators to answer questions about Duchenne; and to share information with the community through articles, reports, newsletters and emails. It is our hope that the combination of the information from MDA’s clinical registry and DuchenneConnect’s patient registry will paint a more complete picture of natural progression with current care and inform future decisions regarding standards of care.


So now you are all up to date!  We have made extensive progress in meeting our goals. Through continued collaboration, we will continue to move forward.

Kathi Kinnett, Director of Clinical Care
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