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FDA Approval Granted, Access Denied – Now What?

By Stephanie Trafton

Head of US Value & Access

10/4/24

The United States of America is famous for medical innovation. Our country leads the world in the number of new drugs and medical devices that are given the green light by our government. Unfortunately, this does not mean that every approved new therapy makes it easily to the patients who need it. The complexity of our health care system can create a frustrating and even intimidating process for patients and their families to navigate. That’s where people like me come in — as Head of US Value & Access at ITF Therapeutics, my job is to support people who are prescribed our medication so they can get that prescription filled, begin treatment, and continue on therapy as long as medically appropriate.

The first thing we do is make sure that our therapy, DUVYZAT™ (givinostat), makes it to the US from the manufacturing facility in Madrid, Spain. My team also works with insurance companies to help them understand the value that DUVYZAT brings to patients and its role in the evolving DMD treatment paradigm. Speaking for all of ITF Therapeutics, and my team in particular, we are committed to appropriately supporting patients and their caregivers as they navigate the often confusing insurance process. This includes supporting communication with insurance companies, providing educational resources and financial assistance, and – when appropriate – supplying drug without charge to eligible patients facing insurance challenges.

After more than 20 years working in patient access, I know it may feel discouraging when a treatment is available and prescribed by your physician, yet instead of ordering it up for immediate delivery you have to wait and even fight for access. But do not despair. The DMD community is incredibly strong and supportive, and lots of help is available.

Here are three resources that can support you through the process:

  1. ITF ARC is a program we created to help patients start and stay on treatment with DUVYZAT. Through ITF ARC – which stands for Access, Resources, and Care – you’ll find assistance with navigating insurance benefits, financial support to help cover out-of-pocket costs, and useful aids once treatment begins such as refill reminders. To get the access process started, your prescribing physician will fill out the patient start form. In order for us to work with the physician and insurance company on your behalf, the patient (or guardian if the patient is under 18) has to provide consent. If your doctor has prescribed DUVYZAT, keep an ear out for a phone call from ITF ARC as we need your consent to keep the ball rolling. The sooner you provide consent, the sooner we can take action to get the treatment delivered to your door.
  2. Little Hercules Foundation is a nonprofit organization dedicated to helping families access FDA-approved therapies. To quote Founder Kelly Maynard, whose son Jackson has Duchenne, “Sick kids should not be getting denied an approved treatment.” The foundation has an incredible team working to help educate payors and assist families in getting access. Contact the foundation if you’ve gotten an initial denial from your insurance company or if you just want to learn more about the process of obtaining access to treatment.
  3. This recorded webinar explains what DUVYZAT is and how you can go about getting access. In the 50-minute session, representatives from ITF Therapeutics (including me), Parent Project Muscular Dystrophy (PPMD), and Little Hercules Foundation explain what support services are available and answer questions from the community.

As often happens with newly approved treatments, many patients get denied coverage on their first try. A denial does not mean the insurance company does not believe the treatment may help you, or that they ultimately will not cover it for you. Nor does it mean that you will not get access to DUVYZAT. It’s just the way our system works – there are often multiple steps involved to getting an individual on therapy. Stay strong and seek help – remember that you have an empowered community at the ready to help guide you through the process.

And now we're going to share some Important Safety Information, often called "ISI." Whenever a company mentions the brand name of a specific medication they market, as well as the condition it treats, they are legally required to provide ISI. This typically includes the FDA-approved uses of the drug and the potential risks and side effects. Reviewing the ISI is crucial to support the safe and informed use of a medication. Here is the ISI for DUVYZAT:

Indication and Important Safety Information

What is DUVYZAT?

DUVYZAT is a prescription medicine that is used for the treatment of Duchenne muscular dystrophy (DMD) in people 6 years of age and older.

It is not known if DUVYZAT is safe and effective in children under 6 years of age.

Important Safety Information

What is the most important information I should know about DUVYZAT?

  • Low platelet counts in your blood (thrombocytopenia). Platelets are important for blood clotting, and a decrease in their numbers can lead to an increased risk of bleeding or bruising. Your healthcare provider will check your blood count before you start DUVYZAT and regularly during treatment for any signs of thrombocytopenia. Call your healthcare provider right away if you notice any unusual bleeding or small red or purple spots on the skin called petechiae. Your healthcare provider may change your dose of DUVYZAT if your blood platelet counts continue to be low or may stop your treatment with DUVYZAT.
  • Increased levels of fat (triglycerides) in your blood. You may not have any symptoms, so your healthcare provider will do blood tests before you start DUVYZAT and regularly during treatment to check your triglyceride levels. Your healthcare provider may change your dose of DUVYZAT if your triglyceride levels continue to be high or may stop your treatment with DUVYZAT.
  • Frequent watery loose stools (diarrhea) and vomiting. DUVYZAT can cause vomiting and moderate to severe diarrhea. If diarrhea occurs, you should keep track of the frequency and severity of your diarrhea symptoms, drink plenty of fluids, and contact your healthcare provider. Your healthcare provider may change your dose of DUVYZAT if the diarrhea cannot be managed or does not go away. Your healthcare provider may also stop your treatment with DUVYZAT.

Before Taking DUVYZAT, tell your healthcare provider about all of your medical conditions, including if you:

  • have any heart problems or if you take any medicines that could increase your chance for irregular heart rhythms.
  • have any bleeding problems.

Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Taking DUVYZAT with certain other medicines may affect each other. Taking DUVYZAT with other medicines can cause serious side effects. Do not start or stop other medicines without talking to your healthcare provider.

DUVYZAT can cause serious side effects, including:

  • See “What is the most important information I should know about DUVYZAT?”
  • Changes in the electrical activity of your heart called QT Prolongation. QT Prolongation can increase the risk of developing a type of irregular heart rhythm known as Torsades de Pointes. Call your healthcare provider right away if you feel faint, have an irregular heartbeat, feel dizzy, or lose consciousness.

The most common side effects of DUVYZAT included diarrhea, nausea, vomiting, stomach pain, low platelet counts in the blood, increased fat level in the blood and fever.

These are not all of the possible side effects of DUVYZAT. For more information, ask your healthcare provider or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please see full Prescribing Information and Medication Guide.

C-DUV-US-0088 09/2024