Having a rare, multi-system disease like a peroxisomal disorder often means taking multiple medications and supplements too. Nearly 95% of all rare diseases have no FDA approved treatment and hope can often be difficult to find. Rare patients and caregivers often desperately seek anything that may possibly help our loved one. Balancing hope and safety are a tight rope rare caregivers and rare patients know all too well.
Health care professionals should be aware of all medical issues, as well as all the medications and supplements a rare patient takes. Patients and caregivers spend hours and hours managing our healthcare while also navigating a busy and complex healthcare system, and making sure that communication is happening between multiple specialties.
A diagnosis of a peroxisomal disorder causes many symptoms that can change and increase at any time, resulting in medication management that can also frequently change. Patients and caregivers often feel like they are in a constant state of reprioritizing needs while also working with healthcare professionals and researchers to find and provide the best symptomatic treatments possible.
Peroxisomal disorders are multi-system diseases, with that comes concern of whether medications are safe and not causing more symptoms to the already lengthy list of medical problems connected to a rare diagnosis. What is not well understood is the role supplements can play. Supplements are not the same as drugs. Common supplements may include vitamins, minerals, herbs, compounds, amino acids and even live microbials, which many of us refer to as probiotics. Supplements are everywhere, so how do we know which ones are safe? Who is regulating them? Will they interact with prescription medications or other supplements? Do the benefits outweigh the risks and what are the risks?
U.S. Food and Drug Administration (FDA) Facts About Supplements:
- The FDA does NOT have the authority to approve dietary supplements for safety and effectiveness, or to approve their labeling, before the supplements are sold to the public.
- Under the FD&C Act, it is the responsibility of dietary supplement companies to ensure their products meet the safety standards for dietary supplements and are not otherwise in violation of the law.
- The FDA’s role in regulating supplements primarily begins after the product enters the marketplace.
It is important to understand the FDA process for drugs is not the same process for supplements. For drugs, there is a lengthy process involved to demonstrate safety and effectiveness in animals and humans. For supplements, the process is much more reactive compared to the proactive approach for drugs. Additionally, the process is not different whether the supplement is considered a natural product or a synthetic version. The bottom line: discuss supplements with your healthcare provider before taking supplements.
Questions When Considering Supplements
- Have you reached out to talk with your healthcare provider?
- Who is suggesting the supplement?
- Why is the supplement being suggested?
- Have appropriate safety studies occurred?
- Do you have the final formulation of the ingredients in the supplement?
- Are there additional healthcare professionals who should be consulted about the supplement?
- Is this part of research and if so, have all appropriate information and consents been provided and obtained by all parties?
- How will the correct dose of supplement be determined?
- What are the known and/or more “common” side effects?
- Have there been any reported adverse events with the use of this supplement?
The decision to take supplements should be a decision made between a patient and the healthcare team familiar with the patient, their diagnosis and medical history. We hope this article has given knowledge to empower you to ask questions and be involved in your healthcare decisions, alongside your healthcare team. To learn more, check out these additional resources listed below.
Written By: Katie Sacra and Melissa Bryce