Access & Coverage Resources for New Therapies

With the first FDA approvals of Duchenne therapies, the Duchenne community has entered in a new environment for access and reimbursement. With this brave new world at play, PPMD has been leading the effort to educate and guide the community through these unchartered waters.

Roadmap for Navigating the Path to Access

We have all had frustrations getting healthcare paid for, whether it is getting access and coverage for appointments, procedures, equipment, and/or medications. Coverage is especially difficult when new medicines or procedures are recommended. It can be a complicated and often an overwhelming process, but what is important to remember is that this is a process and policy determinations are not always final.

To make this process easier, PPMD has assembled resources that will help families and medical providers at each stage of the healthcare access process. Included below is a road map aimed at guiding you through this complex process – complete with sample letters and links to relevant publications and other resources.

We are working closely with health insurance payers (public and private) to increase their understanding of Duchenne and how access to available and emerging interventions, therapies, and equipment can improve health outcomes for our community.

As our engagement continues and we learn more, we will update and produce additional tools and resources for utilization by our community. Please contact PPMD’s Brian Denger with any questions.

Understanding the Path to Access

The process for access to new medicines is not always linear. If your plan does cover the medicine, there are programs available to limit your out of pocket costs. Take a look at PPMD’s Overview of the Fundamentals of Access for more information on the process.

Below are resources outlined for each stage of the process, including if a denial occurs.

How do I start the process for access?

To begin the process for access, most drug companies have a start form that can be filled out by you or your physician.

Some insurers will require what’s called Prior Authorization. This may involve confirmation of genetic test results and other medical records to be submitted with the prescription from your doctor.

Your clinician will be a key advocate for this process. He/she should consider submitting a Letter of Medical Necessity (sample letter below), which provides background information about the patient, disease, and drug.

What happens if access is denied by my insurer?

It is at this stage a complication could occur.

If you receive a denial, there are steps you can take in order to appeal the decision. Your clinician will be a key advocate in this process. The denial letter should have specific information about the appeals process.

The clinician or doctor can request what is called a “peer to peer” where your clinician will interact directly with the insurer one-on-one to provide justification for access and coverage. Please be sure you are in touch with the drug company’s case managers who will also provide you with guidance during the appeals process.

Things to Consider:

Ask your clinician and the insurer about whether a “peer to peer” can occur and how to initiate.
Write your own Patient Appeal Letter (sample below)
Has your clinician submitted a Letter of Medical Necessity? (sample below)

Tips for Advocating for Access:

Track all communications with the drug company, health insurer, and clinician/doctor. Save all letters and emails and write down who you speak with and when. Request letters sent by your physician.
Consider contacting your state Consumer Assistance Program (CAP) or Department of Insurance during an appeal process. Visit localhelp.healthcare.gov.
Get familiar with your health insurer’s policy. Read your policy and call a representative to better understand your coverage. If you have private insurance through your employer, contact Human Resources and have them explain the policy’s provisions.
You have the right for an Appeal. Your insurer must outline what that appeals process entails and specifically why you were turned down.
You CAN request an urgent or expedited appeal if your medical provider believes a delay in treatment could jeopardize your overall health.

Therapy Specific Resources

AGAMREE® (Catalyst Pharmaceuticals)

Stage 1 Resources

- AGAMREE® (vamorolone) is a dissociative steroid indicated for individuals diagnosed with Duchenne muscular dystrophy from age 2 years and older. FDA label
- AGAMREE is available as a liquid oral suspension at 40mg/ml. It is dosed at 6mg/kg once daily up to a maximum daily dose of 300mg.
- You can read the full FDA package insert here.
- Please contact Your Catalyst Pathways, Catalyst’s patient support program, to be connected with the Patient Services team to access resources and support, and begin the Enrollment Form.
- View PPMD’s FAQ document for additional information.

Stage 2 Resources

- With a full FDA approval, Your Catalyst Pathways team of Patient Access Liaisons and care coordinators are available to help navigate the appeals process.
- Additionally, the Little Hercules Foundation provides assistance navigating insurance denials, appeals, and more.
- Relevant publications:
  - Efficacy and Safety of Vamorolone Over 48 Week in Boys With Duchenne Muscular Dystrophy
  - Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy

AMONDYS 45 (Sarepta Therapeutics)

Stage 1 Resources

- AMONDYS 45 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (Duchenne) in patients who have a confirmed mutation of the Duchenne gene that is amenable to exon 45 skipping. FDA label document
- Unsure if you are amenable to this therapy?
  Contact one of PPMD’s Genetic Counselors for guidance:
  Email: coordinator@duchenneregistry.org
  Phone: (888) 520-8675
  Or learn more about mutations that are amenable to exon skipping.

For U.S. Residents:

- For those amenable to exon 45 skipping please contact SareptAssist to be connected with a case manager who can help you begin the process for access. SareptAssist will contact your physician for the start form and the prescription.
  - Learn about the AssistanceFund

Stage 2 Resources
- Sample Letter of Medical Necessity (for Clinicians/Doctors)
- Sample Patient Appeal Letters (For Families)
  - Ambulatory
  - Non Ambulatory
- Sample Physical Appeal Letter (for Clinicians/Doctors)
- FDA Label Document

DUVYZAT™ (ITF Therapeutics)

On March 21, 2024, FDA granted approval to DUVYZAT™ (givinostat), a histone deacetylase (HDAC) inhibitor indicated for individuals diagnosed with Duchenne muscular dystrophy from six years of age and older. View PPMD’s FAQ document for additional information. We will begin to our resources in the coming weeks as more information becomes available.

ELEVIDYS (Sarepta Therapeutics)

Stage 1 Resources
- ELEVIDYS is a gene transfer therapy designed for targeted expression of ELEVIDYS micro-dystrophin protein, a shortened dystrophin retaining key functional domains of the wild-type protein for the treatment of Duchenne muscular dystrophy. This gene addition therapy uses adeno-associated virus (AAV) as a vector to deliver a cDNA copy of functional DMD to affected tissues.
- Currently, patients with deletions in exons 8 and 9 are not eligible for treatments. Additionally, some insurance policies only cover individuals with mutations between exons 18 and 58. FDA Label document.
- Unsure if you are amenable to this therapy? Contact one of PPMD’s Genetic Counselors for guidance:

Email: coordinator@duchenneregistry.org

Phone: (888) 520-8675

For U.S. Residents:

- For those eligible for ELEVIDYS please contact SareptAssist to be connected with a case manager who can help you begin the process for access. SareptAssist will contact your physician for the start form and the prescription.
- Sample Letter of Medical Necessity (for Clinicians/Doctors)
- Learn about the AssistanceFund

If you live outside of the U.S.:

- Sarepta supports a Managed Access Program (Expanded Access Program) for those residing in numerous countries, including: Argentina, Brazil, Canada, Colombia, France, Germany, Greece, Iceland, Italy, Mexico, Spain, Turkey, and the United Kingdom.
- For more information about eligibility and access, visit Sarepta’s Managed Access Program.

Stage 2 Resources

EMFLAZA (PTC Therapeutics)

Stage 1 Resources

- Emflaza is a corticosteroid that demonstrates anti-inflammatory and immunosuppressant effects. Emflaza is available in an immediate-release tablet formulation at multiple dosages (6 mg, 18 mg, 30 mg, 36 mg) as well as in an oral suspension formulation (22.75 mg/mL). Emflaza label and prescribing information
- Please contact PTC Cares to be connected with a case manager who can help you begin the process for access. PTC Cares will contact your physician for the start form and the prescription.
  - Emflaza FAQs
  - Learn about the AssistanceFund
  - PTC Cares 2021 Insurance Changes and Open Enrollment Notice: Available in English & Spanish

Stage 2 Resources

- Full approval of Emflaza allows for Emflaza cares to provide resources for the appeals process. Please contact PTCCares for more information.

EXONDYS 51 (Sarepta Therapeutics)

Stage 1 Resources

- EXONDYS 51 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (Duchenne) in patients who have a confirmed mutation of the Duchenne gene that is amenable to exon 51 skipping.
  FDA label document
- Unsure if you are amenable to this therapy?
  Contact one of PPMD’s Genetic Counselors for guidance:
  Email: coordinator@duchenneregistry.org
  Phone: (888) 520-8675
  Or learn more about mutations that are amenable to exon skipping

For U.S. Residents:

- For those amenable to exon 51 skipping please contact SareptAssist to be connected with a case manager who can help you begin the process for access. SareptAssist will contact your physician for the start form and the prescription.

If you live outside of the U.S.:

- - Sarepta supports a Managed Access Program (Expanded Access Program) for those residing in numerous countries, including: Argentina, Brazil, Canada, Colombia, France, Germany, Greece, Iceland, Italy, Mexico, Spain, Turkey, and the United Kingdom.
  - For more information about eligibility and access, visit Sarepta’s Managed Access Program.

Stage 2 Resources

- Sample Letter of Medical Necessity (for Clinicians/Doctors)
- Sample Patient Appeal Letters (for Families):
  - Ambulatory
  - Non-ambulatory
- Sample Physician Appeal Letter (for Clinicians/Doctors)
- FDA label document
- Relevant Publications:

VYONDYS 53 (Sarepta Therapeutics)

Stage 1 Resources

- VYONDYS 53 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (Duchenne) in patients who have a confirmed mutation of the Duchenne gene that is amenable to exon 53 skipping. FDA label document
- Unsure if you are amenable to this therapy?
  Contact one of PPMD’s Genetic Counselors for guidance:
  Email: coordinator@duchenneregistry.org
  Phone: (888) 520-8675
  Or learn more about mutations that are amenable to exon skipping

For US Residents:

- For those amenable to exon 53 skipping please contact SareptAssist to be connected with a case manager who can help you begin the process for access. SareptAssist will contact your physician for the start form and the prescription.
- Sample Letter of Medical Necessity (for Clinicians/Doctors)
- Learn about the AssistanceFund

Stage 2 Resources

- Sample Letter of Medical Necessity (for Clinicians/Doctors)
- Sample Physician Appeal Letter (for Clinicians/Doctors)
- Sample Patient Appeal Letters (for Families):
  - Ambulatory
  - Non-ambulatory
- FDA label document
- Relevant Publications:
  - Publication on golodirsen (VYONDYS 53) dystrophin expression
  - Publication on golodirsen (VYONDYS 53) dystrophin production

Innovative Access Efforts

EXONDYS 51 (Sarepta Therapeutics)

With an accelerated approval for EXONDYS 51, comes the requirement for the company to perform what is called a “confirmatory study” – this means while the product is approved for use, definitive clinical benefit will need to be established through the study. Given these requirements, some payers are denying coverage of EXONDYS 51, stating that more data is needed prior to access. Since the approval, there has been an effort by clinicians to provide a rationale to payers for treatment across the spectrum of the disease. Below is one example of such efforts, Dr. McDonald has graciously shared the data and presentation that was compiled for their successful engagement with MediCal (California Medicaid) and recorded an explanation of the data and the context of the engagement, as well as details as to how he and his team have been engaging with private insurance companies and empowering families to work with insurers. Special thanks to Dr. John Day, Dr. Perry Shieh, and Dr. Chamindra Konersman, for their contributions to this effort.

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Access & Coverage Resources for New Therapies