Last week, PPMD was joined by Kathy Mathews, MD (Neurologist), Christina Trout, RN, MSN (Clinic Coordinator), and Rachel Kinn, PharmD, BCPPS (Pharmacist) from the University of Iowa to discuss decision making regarding steroids. In recent months we have learned there will soon be more steroid options clinically available to people living with Duchenne. We invited the University of Iowa team to walk us through considerations for initiating a new steroid drug and/or regimen, and the different processes for obtaining access to these medications.

Below are some key takeaways from the webinar:

Steroid Drug, Dose, and Regimen Decision Making

1. What are the current steroids approved by the FDA that may be prescribed for the treatment of Duchenne?
   • Prednisone/Prednisolone is a widely utilized steroid for many medical conditions, and may be prescribed for patients with Duchenne. 
   • EMFLAZA, also called deflazacort in its generic form, is approved and commercially available for treatment of Duchenne. 
   • AGAMREE (vamorolone) has been approved by the FDA for treatment of Duchenne.
2. What should I consider when deciding which steroid drug and regimen to initiate for my child, or if I am considering switching from one steroid drug or regimen to another?
   • These decisions require an in-depth conversation between you and your neuromuscular team. Dr. Kathy Mathews developed this summary snapshot of steroids used in Duchenne that may help guide your discussion.
3. Why are individuals living with Duchenne prescribed different drugs and regimens than other patients with the same diagnosis?
   • There is not one best steroid choice that applies to every situation. It is important to work in partnership with your neuromuscular team to make the best choice for you/your child and revisit this decision based on side effects.

Steps Towards Starting a New Medication

1. I have chosen a specialty medication (EMFLAZA, generic deflazacort, AGAMREE); how do I initiate the process to access the drug?
   • You will be asked to complete an enrollment, or “start,” form, which is specific to the brand drug and manufacturer where you will consent to provide information such as demographics, insurance information, etc. 
2. How do I know whether my steroid choice is covered by insurance?
   • There will be a benefits investigation (BI) to review your medical and pharmacy benefits provided by your insurance, whether private or government funded. Your neuromuscular team and/or the drug manufacturer will work with your insurer on a prior authorization process to determine criteria for eligibility for coverage and whether it is approved or not.
3. Can I receive specialty medications through any pharmacy?
   • Typically, specialty medications are dispensed through a limited distribution specialty pharmacy, which is licensed and accredited to dispense specific drugs. Medications will typically need to be mailed to you from one or more locations and would not typically be available to pick up at physical location. 

Brand vs. Generic Drugs – What Does This Mean for Me/My Child?

1. I heard EMFLAZA is losing patent exclusivity, what does this mean and why is it happening?
   • The patent and exclusivity period for the brand medication (EMFLAZA) recently expired. This means other companies can make a generic version of the medication deflazacort. The FDA determines the amount of time a particular drug receives exclusivity, which expired on February 9th, 2024.
2. How are generic medications different from brand medications?
   • Generic and brand medications have the same active ingredient, but are made by different companies. 
3. Is it safe to switch from a brand medication to a generic?
   • Yes, it is safe to switch! Generic manufacturers have to perform extensive testing to prove to the FDA that their generic drug shows therapeutic equivalence, meaning it has equal effect and no difference when substituted for the brand. 
4. Does generic deflazacort work the same as brand?
   • Yes! It has the same medication in the same amounts so will have the same effect on the body.
5. Will there be different side effects with generic deflazacort?
   • No, the side effects should be the same since it contains the same active ingredient
6. Will the generic medication look the same?
   • Generic deflazacort may look slightly different because it will be made by a different company. The tablets may be a different color or shape. The liquid may have a different flavor, color, or bottle 
7. How do I switch to generic deflazacort?
   • You will need to discuss this change with your neuromuscular team, and if making a decision to switch to generic deflazacort, a neuromuscular provider will need to write a new prescription and have it sent to your pharmacy. It is possible that a specialty pharmacy will still be required for medication distribution.
8. Can we choose to stay on EMFLAZA and not switch to generic deflazacort?
   • At this time, PTC Therapeutics will continue to produce the brand drug EMFLAZA. If you and your neuromuscular team decide remaining on EMFLAZA is the best choice for you/your child, they will include a “Dispense as Written” (DAW) code on the prescription so it may not be substituted for a generic.
9. Will my insurance coverage of deflazacort change when generic deflazacort becomes available?
   • This will depend on your individual insurance plan.
   • Insurance may require the brand or generic.
   • Typically, insurance coverage of a medication improves when a generic becomes available. Insurance may also cover generic deflazacort if they had not previously covered brand Emflaza.
   • Insurance cannot make you change the medication. The decision to switch medications is between you and your provider. However, your insurance may choose not to cover brand EMFLAZA or may increase your cost-sharing. 
10. Will my cost responsibility change?
    • This is dependent on your individual insurance plan. Typically when medications are available as generic, patient and societal costs decrease. 
    • If you switch to generic deflazacort, you may not have the same access to the manufacturer copay assistance programs. This may not be necessary if insurance is covering the medication or cost-sharing responsibility is lower. 
11. Who is the best person to talk to about my questions and concerns?
    • Your neuromuscular provider!

Watch the webinar recording