How You Can Report Serious Drug Side Effects to the FDA

When our children are hurting, we go to our medicine cabinets and take great care to select products that will ease their pain. Reduce the fever. Relieve the ache.

In Duchenne, we know that the products and supplements prescribed often come with side effects. But we carefully weigh those side effects against the risk of doing nothing. We consult with our providers. We learn all of our options. We make the best choices we can.

But over the last week, our community’s hearts have been broken as we have watched some unknowns creep in. Young men who are hurting because of unfamiliar side effects suggested or suspected to be related to Emflaza.

We all know that there is a benefit-risk equation we must consider before we make decisions about our child’s health. But some of the photos and stories being shared on social media of young men covered in blisters, rashes – these might not have been possibilities we ever thought we’d have to consider. Others have shared experiences of physical or behavioral changes. We need to understand what is causing these reactions. And we must sort out whether these experiences have a common thread that can be linked to a common product or manufacturer or dosing regimen.

What is MedWatch?

is an online form to report adverse events that you observe or suspect for human medical products, including serious drug side effects, medication errors/product use errors, product quality problems, and therapeutic failures in approved FDA therapies. 

FDA’s Mission Statement states that:

"FDA is responsible for protecting the public health by assuring the safety, efficacy and security of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation.

FDA is also responsible for advancing the public health by helping to speed innovations that make medicines more effective, safer, and more affordable and by helping the public get the accurate, science-based information they need to use medicines and foods to maintain and improve their health….”

To ensure that patient communities have a direct mechanism for reporting experiences with FDA approved products, FDA created the MedWatch form. The form which has versions for both clinical providers to report on behalf of a patient and patient community members to self-report, has recently been updated and can be accessed here.  Importantly, these events should also be reported to your treating physician immediately. This site should not take the place of the communication you have with your physician about the adverse event. 

PPMD has been in touch with the FDA. Key officials at the agency requested that community members who’ve experienced noteworthy concerns on any FDA approved product take the time to complete the MedWatch form — and specifically asked that the form be completed as fully as possible. FDA has assured us that they will diligently monitor the MedWatch reports. This information will help regulators regulate – providing the agency with information so that they can ensure the product they approved is being monitored for any ongoing safety concerns expressed directly by physicians and patients.

PPMD has also reached out to PTC Therapeutics to find out how the company is addressing these issues. As soon as we receive an update, we will share that with you.

Your Experience Matters

PPMD and our Duchenne community have long understood that we must partner with diverse stakeholders to ensure that all efforts to find treatments and cures for Duchenne are informed and driven by patients and patient experiences. To that end, just as we – as a community – have made it a priority to produce robust data around patient–preferences to inform trial designs and outcomes, to power our community’s DuchenneConnect registry, and to participate in clinical trials, we must also ensure that we provide a feedback loop to clinical providers and regulators about our community’s experiences with the therapies and approved products to which we are exposed. That’s why any adverse side effects that you or your child experience with an approved therapy should be reported to MedWatch – your experience matters.

PPMD will continue to work with regulators, industry, and you to help collect your experiences with any approved Duchenne therapy. This data will help industry improve current therapies, as well as inform the path forward for future therapies. We all want approvals for this community, approvals that will treat every single person living with Duchenne. But we don’t want those approvals to cause any of our loved ones to suffer needlessly.

Views: 442


You need to be a member of PPMD Community to add comments!

Join PPMD Community

Need help using this community site? Visit Ning's Help Page.



© 2023   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service