Understanding the Path to Access – Resources for the Community

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. Parents, patients, and medical providers spend hours putting together documents and speaking on the phone trying to convince payers that this is what is needed to keep us, our children, or our patients, as healthy as possible.

It's a complicated and often an overwhelming process that makes you just want to throw in the towel. But we can’t. What is important to know 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.  


Access to Medicine Process 

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.

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.


Stage 1 Resources:

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.


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.


Stage 2 Resources (If you receive a denial letter):

    • Coming soon! PPMD will be adding resources for appeals as we work through the access space with the community.

New Resource for Clinicians and Patients on Exondys51: 

Dr. McDonald has graciously shared the data and presentation that was compiled for their successful engagement with MediCal 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. 


Share your experience with access:

In an effort to better understand where patients and physicians are with access to Exondys 51 and Emflaza we have created an intake form to collect data about the process. If you are willing to share information about your experience to date, please consider filling out one of the following surveys:


Exondys51 - Caregiver or patient survey for those trying to access drug or who currently have access to drug

Emflaza - Caregiver or patient survey for those trying to access drug or who currently have access to drug

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Comment by pam moodaly on December 19, 2016 at 3:30pm

Thank you for this very useful information. Will follow through

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