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: age 2 and up

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.
- Sample Letter of Medical Necessity
- PPMD convened expert clinicians in the creation of a consensus statement addressing access to approved therapies in Duchenne, published in Annals of the Child Neurology Society. This statement is a helpful resource in advocating for positive coverage policies for approved therapies and can be a helpful piece of supporting documentation in the prior authorization and appeals process. View the full-text version of the publication: Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians
- 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.
- Sample Letter of Medical Necessity
- 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: all ages

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.
- PPMD convened expert clinicians in the creation of a consensus statement addressing access to approved therapies in Duchenne, published in Annals of the Child Neurology Society. This statement is a helpful resource in advocating for positive coverage policies for approved therapies and can be a helpful piece of supporting documentation in the prior authorization and appeals process. View the full-text version of the publication: Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians

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
- Relevant Publications
  - Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial

Duvyzat: age 6 and up

On July 25, 2024, ITF Therapeutics announced the U.S. commercial availability of DUVYZAT™(givinostat), a histone deacetylase (HDAC) inhibitor indicated for individuals diagnosed with Duchenne from six years of age and older that was granted FDA approval in March 2024.

Stage 1 Resources
- DUVYZAT™ (givinostat), a histone deacetylase (HDAC) inhibitor indicated for individuals diagnosed with Duchenne muscular dystrophy from six years of age and older. View the Medication Guide.
- Read the full FDA Label here.
- For more information on DUVYZAT™, visit our FAQ.
- Sample Letter of Medical Necessity (for clinicians/doctors)
- To help enable access to treatment with DUVYZAT for appropriate patients in the U.S., the ITF ARC program from ITF Therapeutics includes services to help patients and families navigate through insurance challenges, personalized pharmacist support, financial and access assistance for eligible patients, educational materials, and other resources. For more information about patient support services offered through ITF ARC, visit www.DUVYZAT.com or call 1-855-448-3272.
- PPMD convened expert clinicians in the creation of a consensus statement addressing access to approved therapies in Duchenne, published in Annals of the Child Neurology Society. This statement is a helpful resource in advocating for positive coverage policies for approved therapies and can be a helpful piece of supporting documentation in the prior authorization and appeals process. View the full-text version of the publication: Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians
Stage 2 Resources
- The Little Hercules Foundation provides assistance navigating insurance denials, appeals, and more.
- Sample Letter of Medical Necessity (for clinicians/doctors)
- Relevant Publications:
  - Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
  - P129 Givinostat in DMD: results of the Epidys study with particular attention to MR measures of muscle fat fraction

Elevidys: age 4 and up

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.orgPhone: (888) 520-8675
- PPMD convened expert clinicians in the creation of a consensus statement addressing access to approved therapies in Duchenne, published in Annals of the Child Neurology Society. This statement is a helpful resource in advocating for positive coverage policies for approved therapies and can be a helpful piece of supporting documentation in the prior authorization and appeals process. View the full-text version of the publication: Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians

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) – Ambulatory Patients
- 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 / Deflazacort: age 2 and up

Deflazacort is a corticosteroid that demonstrates anti-inflammatory and immunosuppressant effects indicated for the treatment of Duchenne muscular dystrophy (DMD). There is currently a generic form—deflazacort—for individuals ages 5 and up, and brand name—Emflaza (PTC Therapetuics)—for individuals ages 2 and up.

EMFLAZA — PTC Therapeutics (BRAND NAME)

Stage 1 Resources
- Emflaza is a corticosteroid that demonstrates anti-inflammatory and immunosuppressant effects for individuals with Duchenne muscular dystrophy ages 2 years and older. 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
- PPMD convened expert clinicians in the creation of a consensus statement addressing access to approved therapies in Duchenne, published in Annals of the Child Neurology Society. This statement is a helpful resource in advocating for positive coverage policies for approved therapies and can be a helpful piece of supporting documentation in the prior authorization and appeals process. View the full-text version of the publication: Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians

Stage 2 Resources
- Full approval of Emflaza allows for Emflaza cares to provide resources for the appeals process. Please contact PTCCares for more information.

DEFLAZACORT (GENERIC)

Stage 1 Resources
- Deflazacort is a corticosteroid that demonstrates anti-inflammatory and immunosuppressant effects indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older. Drug label.
- Manufacturers:
  - Cranbury Pharmaceuticals (Tris Pharma) – oral suspension (liquid) only
  - Aurobindo Pharma -6 mg, 18 mg, 30 mg, and 36 mg tablets only
- Cranbury Connects supports families in the process of gaining access to deflazacort (oral suspension) through case management & financial assistance programs including copay assistance, bridge program and patient assistance for uninsured or underinsured patients.

Viltepso: all ages

Stage 1 Resources

- VILTEPSO 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
- 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
- PPMD convened expert clinicians in the creation of a consensus statement addressing access to approved therapies in Duchenne, published in Annals of the Child Neurology Society. This statement is a helpful resource in advocating for positive coverage policies for approved therapies and can be a helpful piece of supporting documentation in the prior authorization and appeals process. View the full-text version of the publication: Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians

For U.S. Residents:

- For those amenable to exon 53 skipping please contact NS Support to be connected with a case manager who can help you begin the process for access.
- NS Support Patient Services Overview [PPMD Webinar Recording]
- Sample Letter of Medical Necessity (for Clinicians/Doctors)

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:
  - Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
  - Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

Vyondys 53: all ages

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
- PPMD convened expert clinicians in the creation of a consensus statement addressing access to approved therapies in Duchenne, published in Annals of the Child Neurology Society. This statement is a helpful resource in advocating for positive coverage policies for approved therapies and can be a helpful piece of supporting documentation in the prior authorization and appeals process. View the full-text version of the publication: Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians

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:

Innovative Access Efforts

Exondys 51: all ages

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