FDA Public Workshop Report – Our Message Is Having an Impact

Last week, Pat Furlong and I were invited by the FDA to their meeting on Complex Issues in Developing Drugs and Biological Products for Rare Diseases. I was asked to participate on the Tolerating Risk and Uncertainty in Pediatric Clinical Trials Panel, in recognition of the work PPMD has done to:


  1. Systematically collect data about risk tolerance;
  2. Obtain parent stories for the FDA; and
  3. Understand family experiences in clinical trials.

Following are a few highlights. Information about the meeting is available on FDA.gov. 


Tolerating Risk and Uncertainty in Pediatric Clinical Trials panel

We started the Tolerating Risk and Uncertainty in Pediatric Clinical Trials panel by discussing what benefits families are expecting in pediatric clinical trials. I shared perspectives gained from parents of children in clinical trials through our Clinical Trials Expectations project

Several FDA and bio/pharma individuals approached me after the panel, urging us to get more information on this topic. So:


The conversation moved to how much risk families would tolerate. We heard compelling stories from parents on the panel and in the audience about their willingness to accept risk. I encouraged the FDA to create or endorse a model for collecting benefit/risk information from a broad range of patients and parents (which could be used by any advocacy group interested in collecting such information), as well as developing a plan for how this data would be used in the review process. Dr. Andrew Mulberg (Deputy Director, DGIEP, FDA) reinforced the need for this model, and suggested that the audience look to PPMD’s benefit/risk program as a model (with a copy of our program summary in his hand!).

Trial Design

Of course, risk tolerance came up often throughout the meeting. Dr. Unger’s (Director, Office of Drug Evaluation, FDA) introduction to the panel on trial design referenced the FDA’s growing appreciation for significant tolerance for risk in rare disease populations. He put up a slide that read, “We can accept risks that would not be feasible to accept for non-rare conditions, as long as the labeling is adequate!” 

By “labeling is adequate,” Dr. Unger was referring to giving patients/families and their clinicians the information they need (that is, how much expected risk for how much expected benefit) to make their own decisions about taking the drug. “Adequate labeling” can only happen if clinical trials show a benefit that can be meaningfully described on the patient level, and provide data about risks. It’s showing a shift to an FDA that is willing to approve drugs with considerable risk as long as they also have equally considerable benefits, and allow patients and families to make decisions.

Safety & Dosiing

On the panel about Safety and Dosing, there were interesting discussions about how difficult it may be for patients and families to make informed decisions about risk when the safety profiles of the drugs are often not well known. There was a discussion about how risk tolerance may change over time as the disease progression changes, the patient gets older, and/or new treatments become available. Panel participant Dr. Emil Kakis referenced the PPMD Benefit/Risk study as a model for understanding risk tolerance in light of uncertainty. In her summary of the panel, Dr. Kashoki (Associate Director for Safety, FDA) described the possibility of the FDA developing a more structured method to understand risk tolerance in the FDA’s upcoming community meetings. Finally, in her wrap-up remarks Dr. Dianna Murphy (Director, OPT, FDA) referenced the need for the FDA to give guidance on how they would like to receive benefit/risk data, and their plans to use standardized data on risk tolerance that reflects the views of many patients and families.


Keeping Duchenne Front & Center

In light of all the rare diseases that exist and the fact that PPMD very recently hosted a policy forum for the FDA, we were honored to be invited to represent the Duchenne community. Understanding risk tolerance is a hot topic among government agencies and clinical trial sponsors. PPMD is pleased that our project is being held up as a model.

To have not just one, but several participants at the meeting reference our work is a further reminder that our information, our message, and our urgency is having an impact. There is still work to do to understand what additional information the FDA needs, and exactly how the FDA will use this information in their decision making. We’re keeping Duchenne front and center in these discussions.

Help Us Collect More Information for the FDA

Several FDA and bio/pharma individuals approached me after the panel, urging us to get more information on the perspectives of parents of children in clinical trials. If your child has participated in a clinical trial in the US/Canada in the past 3 years, please complete this survey so we can keep sharing this important information with regulators and sponsors.

Complete PPMD's Survey on Expectations for Clinical Trials

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