[Webinar Takeaways] Preparing for Access to Approved Therapies

Last week, PPMD began a webinar series on access – an exciting new chapter for our Duchenne community. But we are just at the beginning of this chapter and there is lots to explore.

We knew this day was coming – we knew that one day we would have our first approval in Duchenne, an approval we believe will beget many more approvals to come. To that end, PPMD has been working with experts and partners in the access environment for more than two years in anticipation of this new chapter – and our efforts have just begun.

Over the coming weeks and months, PPMD will provide informational webinars with experts in our community and the rare disease community, as we dive into the myriad of questions that approved therapies will have us asking. To determine the topics we will be discussing, we will look at other disease spaces and see what they’ve learned, we will convene thought leaders in our community, and we will turn to you, asking you to share your experiences and ask your questions.

The purpose of last week’s kickoff was to provide several useful takeaways, including:

  • Information about how to know what approved or emerging Duchenne therapies you may be eligible for;
  • Concrete items to discuss with your physician to prepare for access to therapies;
  • Items to query your health insurer to better understand your policy in the context of the benefits you may soon be seeking;
  • A sense of the timelines that may be involved and why;
  • Resources for who to turn to for support for all of this;
  • And an understanding of what PPMD is doing to work towards the broadest access and coverage for every eligible person with Duchenne.

We also wanted to make sure we dove into some of the unanswered questions you may have about Exondys 51, what exactly an ‘accelerated approval’ means in terms of our patient community, the post-market commitments now required of Sarepta, and the follow-on studies being conducted. Dr. Tim Franson, Chief Medical Officer for Your Encore and long-time friend and consultant to PPMD and our community joined us on today’s call and discussed the AA of Exondys 51 in the context of the post-approval space and the conditions for approval.

For those who were unable to join us on our first webinar, an archived recording is available:

Overview of the the process discussed: 

When the FDA approves a product to be marketed to the public, it does so within a specific set of rules that the company must follow to make the medication available to patients.


Because of the profound unmet medical need in the Duchenne community, many of the products being developed for us can take advantage of expedited FDA development and review programs such as “accelerated approval.”


When the FDA clears a new medication through an accelerated approval, the company must market the product consistent with the label and conduct any required post-approval studies to verify the anticipated clinical benefit for patients.


If the post-approval studies are successful, the product can remain on the market following relevant label and other FDA protocols.  If the post-approval studies do not verify the clinical benefit, the FDA can order the product to be withdrawn.


Once a new therapy is FDA-approved, a physician may prescribe the medication to appropriate patients.   Patients may be either those who are indicated in the drug’s label or others who, in the professional judgment of the physician, are also appropriate for the therapy (“off-label”).


Duchenne patients prescribed a new therapy will likely be covered by one of several different health plans that fall into two general types – private health insurance (primarily employer-sponsored or individual) or public programs (such as Medicaid, Medicare, and military).


All health plans have specific rules about how they cover medications and it is often important to understand these rules to obtain access, especially to newly approved therapies.


When a newly approved drug comes to market, the company must negotiate the terms of making the drug available to and through specific health plans.  This can require some time to resolve depending on a variety of factors.


Commercial or private health insurance may cover the cost of drug therapies either through the medical or drug benefit portions of the plan, each of which have distinct rules.  Oral drugs are primarily covered in the drug benefit; infused or injected therapies, mostly under the medical benefit; and biologic therapies span both sides.


Medicaid, a joint federal-state health insurance program administered under each state’s rules, is an important source of coverage for eligible members of the Duchenne community, including through the “Katie Beckett waiver” which provides coverage for significantly disabled children regardless of family income.


If the health plan refuses to cover a specific therapy, you have the right to request an exception to those rules and, if that fails, to appeal the decision to a plan administrator.  Your physician is a key partner in this process and there are supporting resources to pursue these options both within the plan administrator and across the community.


Even when a health plan, private or public, has agreed to cover a new medication, its specific rules can create barriers to access such as through higher costs to the patients (co-pays and co-insurance) or other tactics such as prior authorization, quantity limits, and step therapy.


The Affordable Care Act both expands health insurance coverage and prohibits certain coverage limitations in private health insurance such as those on pre-existing conditions and lifetime/annual benefits.


In summary, FDA approval is only one of the key steps in bringing a new medicine to the patients in need.  Private and public health insurance is an essential component of gaining access where the patient and caregivers must be informed and engaged.

Looking forward:

Recognizing that the access and health insurer landscape is a highly dynamic and variable environment, we will continue to expand our Access Webinar series to ensure that you are equipped with comprehensive and up-to-date information. We also know that the patient voice will have a central role in the new access environment — just as we do in the drug development regulatory environment. PPMD is engaging and will continue to innovate as we work to ensure that our voices and our patient community’s considerations are applied to the regulatory frameworks that impact our access to care. 


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