Remarks of Bernard Murphy, Chair, Juvenile Arthritis Leadership Group, Arthritis Foundation
Public Workshop on Developing a Consolidated Pediatric Rheumatology Observational Registry
May 12-13, 2009

Thank you for inviting me and the Arthritis Foundation here today to give voice to the 300,000 children in the United States diagnosed with a form of juvenile arthritis.  My name is Bernie Murphy and my daughter Kate is one of those 300,000 children afflicted with some form of rheumatic disease. The fact that we’re here having this discussion is exciting to me, and encouraging for all those parents who help their children cope with chronic, painful, and often debilitating illnesses.

The story of Kate’s illness with psoriatic arthritis demonstrates how valuable a registry would be for children afflicted with rheumatic disease.  Kate was three when, after having chicken pox, she awoke one morning with one of her knees swollen to four times its normal size.   After an ER visit where the doctors opined on everything from an insect bite to flesh eating bacterial infection, the condition resolved on its own within 24 hours.  Her disease made its persistent appearance at three years later with swollen ankles and knees.    Kate’s pediatric rheumatologist explained that little girls presenting with such aggressive symptoms but with a seronegative profile was a bit unusual and that Kate’s long-term prognosis was really unknown. After a few years of getting little relief from a variety of DMARDS and the temporary effects of cortisone joint injections, Enbrel became available, and we breathed a hopeful sigh of relief.  Unfortunately, Kate turned out to be an Enbrel non-responder and while on the typical methotrexate/Enbrel combo the synovitis in her right ankle escalated to the point of requiring surgery. The true nature of her disease would remain a mystery for another few years until she manifested dermatologic symptoms of psoriasis as an early teen.   At 17, having switched to Remicade as the biologic, her disease is thankfully being held at bay, but she clearly still has active disease.  We feel fortunate to have gone through this at a time when the first biologics became available, which probably prevented complete debilitation in her ankles.  At the same time, as parents we can’t help but want something better for Kate than a life of continued pain, monthly infusions, and the fear of unforeseen consequences of a lifetime of anti-TNF therapy. 

Wouldn’t it be helpful to use a registry to learn more about the course of the disease and successful therapeutic approaches in other little girls that presented like Kate did?  Wouldn’t it be helpful to have a registry to enable scientists to study the genetics of the disease, to discover specific markers to facilitate diagnosis, to study the pharmacogenomics of Biologic responders and non-responders?  It seems that we should be past an age when an afflicted child’s physician’s must rely primarily on personal experience and best guesses as to what therapeutic approaches might work best.  The need for standardization of care is great, and a registry is an integral part of establishing best practices.  And ultimately, wouldn’t it be great to use this database to facilitate studies that might cure rather than just quiet (?) the disease?

I am proud to be here with you today to let the FDA know we are extremely pleased and grateful that the FDA has recognized and elevated the public discussion of the value of establishing a Consolidated Pediatric Rheumatology Observational Registry for our children.  The Arthritis Foundation has been advocating for such a registry for many years and has sought the support of Congress with the introduction of the Arthritis Prevention Control and Cure Act.  Since its initial introduction a number of years ago, the bill has called for a juvenile arthritis or pediatric rheumatology registry.

The funding and initiation of a Consolidated Registry of children with JA is a prime example of what I am fighting for as a parent of a child with JA and as a champion for the future generation of children who will be diagnosed with JA.  We were lucky in that our pediatrician had the educational exposure to childhood rheumatologic disease and hence Kate’s diagnosis was relatively quick.  I regularly hear the anguish of so many parents where their child’s diagnosis took many years while the damage to their child’s young body went unchecked, or was made worse by inappropriate treatment.  We must find ways of better educating clinicians to recognize and appropriately treat these diseases.

The Arthritis Foundation wholeheartedly supports the creation of a fully funded, Consolidated Pediatric Rheumatology Observational Registry for the following reasons:

  • A consolidated registry will help establish treatment protocols for specialists treating children with rheumatic disease.  With an adequate database including tissue samples, complete medication histories, side effect histories, and efficacy data, physicians will be able to pinpoint which medication has the highest likelihood of benefiting each individual child with the least likelihood of negative side effects.
  • We need to ensure, however, that all clinicians who treat children have access to the Registry.  As we are all well aware, the United States has a desperate shortage of pediatric rheumatologists. Many, many children with JA across the US are treated by general practice pediatricians or adult rheumatologists or adult internists. Those physicians simply do not have the equivalent personal experience that pediatric rheumatologists do have when choosing a treatment plan for a child with JA.  A consolidated registry will eventually lead to better treatment guidelines and protocols.  For these protocols to be meaningful, the Registry needs to be national in scope and include all products in a class. This is what the patient community is seeking.  As a parent of a child with JA, I want to know both the efficacy and the long term side effects of these powerful therapeutics my daughter is taking. 
  • The Arthritis Foundation also requests that as a part of the Consolidated Registry a representative from the patient community be included in future meetings and efforts to establish the Registry. Additionally, the Arthritis Foundation recommends the FDA give serious consideration to future communications about this Registry with the affected patient community.  
  • Most importantly, the Arthritis Foundation requests that the FDA provide consistent, long-term support and funding for such a Registry and/or work with the industry to establish user fees to help maintain the Registry long term. 

We are at a crossroads today. We have fought for and have been given this rare opportunity of helping to change the future for our children with JA. The Arthritis Foundation commends the FDA and the Childhood Arthritis & Rheumatology Research Alliance (CARRA) for organizing this workshop today and for your continued hard work and efforts so that we can improve the lives of hundreds of thousands of children with juvenile arthritis.  

 The Arthritis Foundation looks forward to working with the FDA and parent support groups such as the Friends of CARRA as we move forward to turn the discussions of today into tomorrow’s reality.  Thank you very much for the opportunity to join you today. 

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