What You Need to Know about FDA and Children’s Drugs
When my 15 year old son, Gary, was screaming in pain from complications from Crohn’s Disease in the ER, a young doctor injected him with something that instantly calmed my son’s out of control pain. I didn’t ask what it was because I was grateful that he finally seemed comfortable. An hour later, my son asked the nurse for another “hit.” His choice of words shocked me. He already had enough medical issues, the possibility of addiction as a by-product of medical care scared me to my core. Fortunately, the NG (naso-gastric) tube, that carries food and medicine to the stomach through the nose, that was causing his discomfort was removed and he coped, without drugs, with his residual pain.
To this day, I do not know what the hospital gave my son. Deep down, I just trusted it was FDA approved and safe for my child. In the throes of a medical crisis, parents can’t question everything. Most parents can’t even comprehend all of the medical jargon tossed around. At the moment a child needs us to be strong, we feel scared. Our trust in the doctor, trust in the health care system, trust in drug regulations become imperative for us to be able to just hold our child’s hand to comfort them. It was on my son’s healthcare journey, that I discovered gaps in my expectations and the reality of providing care. It became my mission to help other families thrive with a healthcare crisis.
Today, I am the only voting Consumer Representative on the FDA’s (Food and Drug Administration) 12-person Pediatric Advisory Committee. My voice needs to be a megaphone for all the parents that expect the drugs their child receives are safe and effective. The other 11 members are doctors, many of whom are parents as well.
On September 25th, 2019 our panel convened to review FDA approved OxyContin Extended-Release tables and an OxyContin derivative called Opana Immediate-Release tablets for use with children. As usual, the FDA presented drug data to the panel and asked us to vote on the need for labeling changes. All medicines have “labels” which are not what lay people think of as labels. These are the FDA instructions for prescribing and warnings of side effects of medicines and they are published online and you’ll get a copy if you buy the drug (as compared to getting it in the hospital). The rolled up paper inside of drug packaging covered in tiny type is the official drug label, the information that your doctor receives.
Once the Pediatric Advisory Committee meets, the comments and votes are compiled to be used to guide the recommendations by the FDA to direct the drug manufacturers. In our recent meeting for the first vote, the drug manufacturer wanted to add the information about dosing for children to the label. The FDA position was that no new safety signals were identified in their post-market review (after Oxycontin was approved for adult use) for Oxycontin extended release tables in the current pediatric safety review. The recommendation was to continue ongoing, routine, post-market safety monitoring. The second vote was to approve pediatric labeling for Opana IR.
Here are the three things parents need to understand about the drug labeling process.
1. Children can be prescribed drugs “off label,” without additional FDA testing.
Most drugs, Oxycontin included, were tested only on adults when the FDA approved them. Doctors can prescribe any drug for any person, even if that drug has not been approved or tested in that group. Typically, doctors who want to prescribe a drug like Oxycontin to children have to use their best judgment as to how much to prescribe and if it will be safe. They use their clinical experience because the research hasn’t been done.
2. Drug testing sample size for children is small
Research “post FDA approval” continues on drugs. Finding children to test pain medication is difficult. Most children’s pain is an emergency and there isn’t time to review and discuss joining a research study. Therefore, the sample sizes used for recommendations are typically under 100. For example, in a study presented to the Pediatric Advisory Committee to compare single doses of immediate-release oxycodone and control-release oxycodone in children, the study planned for 24 subjects. It enrolled 13 and only 11 children completed the study. Out of the 13 subjects 69% experienced 1 or more adverse reactions.
From my background in aerospace, I was trained to design products to have at most 3.4 defects per million occurrences. While I understand that testing to this confidence level is expensive and time consuming, the data presented to our panel simply didn’t have enough research subjects to make a fully informed intelligent decision. It was not a rigorous study.
3. Post-market safety monitoring -
Drugs safety warnings are labeled based on the results of initial studies. Once marketed to the public, the drugs are then monitored for adverse events. The FDA collects the data from the public in the MedWatch database. https://www.accessdata.fda.gov/scripts/medwatch/index.cfm The manufacturers are also required to report known adverse events to the FDA. This data is compiled and used for labeling updates. With an estimated 5% of the adverse events reported from the public to MedWatch, it is clear that many doctors and patients are unaware of how and when to report side effects and adverse events. For Oxycontin the number of adverse events such as drug abuse, drug dependence and overdose or depressed respiratory rates for 11- to 17-year-olds from 2015 to 2018 were 67.
Clearly, with the opioid epidemic in the newspapers, the data used from the FDA was underreported.
At the end of the day, I voted NO for both drug labeling approvals. In both cases, the data size was too small. As the Consumer Representative, I believe that families expect a drug that may cause addiction has been rigorously tested prior to release in the market. However, for the first vote, I was not in the majority and the panel voted “yes” that no new safety signals were identified for OxyContin Extended Release and that the FDA should continue post-market safety reviews. That means we the consumer need to post adverse events in MedWatch. If your child is given this drug and they experience an adverse event, only if you report it can the FDA know how many people are truly affected.
The second question to approve pediatric labeling for Opana IR the majority voted NO. Consensus was that the data was inadequate for pediatric approval.
The FDA will use the panel’s input to advise changes for the drug labels.
Here are three things parents need to do every time your child, loved one or you are prescribed a medicine:
1. Ask for the names of drugs given in writing. Take a photo of the drug bottle or label. Look up the drug at the national library of medicine. https://dailymed.nlm.nih.gov/dailymed/index.cfm Read the label. If there is a black box warning (the FDA’s most serious warning) or a side effect that concerns you, call the doctor and ask why this medication was prescribed over other choices. Don’t assume the doctors know there is a black box warning, some are unaware. Review the side effects and warning signs. If a side effect is labeled, it is a known risk which means a possibility for your child. Call your doctor if you think your child is experiencing any of them.
2. Ask about the side effects of transitioning off the medication. Sometimes a quick fix may cause new issues that are more difficult to deal with in the long run. Ask the doctor about their experience with their patients who discontinued medications and challenges faced. Also, look up on the internet if there are any forums about the drug and user experience. This can help guide you to know what questions and concerns to share with your doctor.
3. Report adverse events into MedWatch. Your experience matters. It is the process for the FDA to monitor drugs once released into the market for adverse events. When the number of adverse events are significant the FDA reviews and determines if drug labeling changes are needed. Without your input, the FDA lacks the data needed to determine risks with a medication. Please submit your adverse reactions to the FDA. It is easy to do. https://www.accessdata.fda.gov/scripts/medwatch/index.cfm
I remember when Gary was in pain it felt like a double-edged knife stabbing my heart. At the time, I did not know that drugs are approved with relatively small sample sizes. I never heard of MedWatch and the process the FDA uses to monitor drugs once in the public domain. Now, I know that our experience does matter. It is actually part of the process. We are not alone as long as we share any adverse events on MedWatch. The pediatric panel will review the collective data and advise the FDA to make labeling changes. Your experience may help others. Your voice counts.
Randi Redmond Oster is the award-winning author of Questioning Protocol and CEO of Help Me Health in Fairfield, CT. She serves as consumer representative on the FDA Pediatric Advisory Committee.
This essay was first published as a First Person blog on Medshadow.org.