The Duchenne community has waited 20 years to be where we are. We are reminded daily of how far we have come, how the landscape has changed, how there are close to 20 clinical trials to choose from. While these changes within are landscape bring us to a hopeful time in Duchenne, the journey to this point has not been painless for the families involved.

As we have watched clinical trials in Duchenne expand, we have witnessed the impact of participating in these trials on children and families. Trials with complex protocols that may extend for lengthy periods of time exert significant pressures that families feel, and exert on each other. Examples of children and parents rebelling against the pressure, trying their very best to cope or, more often, not coping at all, creep onto social media and into our hearts. 

Last fall, PPMD had a discussion with Dr. Robert “Skip” Nelson (Deputy Director and Senior Pediatric Ethicist in the Office of Pediatric Therapeutics (OPT) in the Office of the Commissioner) that illuminated concerns that PPMD, industry, and the FDA all shared – concerns of the burdens being placed on participants and families involved in pediatric research. It became clear that it was time to examine and address the needs of our community more closely.

Photo from today's "Unique Burdens of Pediatric Clinical Trials" meeting

Today and tomorrow, PPMD will convene the "Unique Burdens of Pediatric Clinical Trials" meeting. This meeting will include presentations of the perspectives of parents/patients, industry, and primary investigators, on four main areas that have been identified as areas of burden in clinical trial participation.

These four areas include:

• Protocol and technical burden
  • How does a protocol that attempts to address the complexities of Duchenne and assess the safety and efficacy of a drug, translate to the life of a child and family? 
  • Are we asking children to do what is needed and appropriate, and no more? 
  • Or are there tasks that could be dropped from the study, procedures that could be taken out, or parts of the trial that could be completed closer to home or maybe via telehealth? 
• Consent/assent
  • Are families fully able to comprehend what they are signing on to when they give consent/assent? Or are there tools that we can develop or use that will assure that families and children are aware of the impact that trial participation will have on their lives?  
  • Does an initial assent given as a child still count 5 or 10 years later? Or should the assent be re-visited regularly? 
  • Are there procedures that we might use that reduce the possible pressure that families feel to participate, and empower children to have an equal voice in whether to continue in a trial?
• Psychological burden
  • Are families able to adapt to the impact of living life around a clinical trial? Or should there be considerations given to including counseling in clinical trial protocols? 
  • Do families feel comfortable expressing the stresses that trial participation causes? 
  • Do they possibly feel “survivor’s guilt” that they/their child has the appropriate deletion and will participate in the trial and other children do not qualify? Or are there tools that we could use to better evaluate the family’s psychosocial health? 
• Financial burden
  • The outward costs of clinical trial participation (costs of travel, food, housing) are obvious, but what are the hidden costs? What are the costs of the loss of time at work, lost vacation days that are spent for trial visits, childcare for siblings, to name only a few? 
  • Are there ways, as a community, that we can reduce these costs for families?

A published summary of this meeting will be available to the community.

We appreciate the FDA’s participation in this meeting and their openness to discussion. The burden of trial participation for children appears to be a priority for the agency. In fact, later this month we expect the FDA to announce that they will be holding a Pediatric Advisory Committee Meeting soon. This meeting will take a closer look at trial protocols, in particular the use of ports.

In all areas of life we have heroes – firemen who rush into burning buildings to pull out stranded victims, athletes that dig a little deeper to find that shred of spirit needed to push them across the finish line, workers who sacrifice whatever is needed to help just one more person. 

In Duchenne, we don't have to look farther than our clinical trial sites to find our heroes. Every day, trial participants dig deep into their spirit to bear the burdens and make the sacrifices needed to make sure that new therapies are safe, that they work, that they make the tumultuous journey from bench to bedside – that these new therapies are available to every patient that wants and needs them. 

PPMD heralds these families, and recognizes the tremendous burdens that they endure. We are hopeful that, together, we can help make being a hero a little less painful.