The unknowns. Some of the hardest beasts to conquer in life.
The unpredictable variables that make life impossible to plan.
The not being able to commit to vacations and holiday plans because – well, you just don’t know what curve balls life will toss at you between now and then.
The not knowing whether you’ve selected the correct intervention or course of treatment. And truthfully – is there really a ‘right one’?
And just when you thought things might get easier, when the clinical trial landscape showed promise and our community’s first therapies were approved – you learn that those amazing wins also come with big unknowns.
New paths to navigate. New worlds to learn and worries to take on. This was all supposed to feel so much easier. Right?
The PPMD team has worked tirelessly since the approval of EXONDYS 51 to help families navigate access. That same commitment carries over to EMFLAZA, including seeking answers to your top concerns.
I think we all thought the fact that it was deflazacort – a steroid we understood – would make it simpler. But as the last few days have proven, we were mistaken.
We’ve been faced with new unknowns such as "weight-based" pricing and prior-authorization. And while we wish we had all of the answers, we will navigate this access environment together and work to provide clarity when we can.
We are eager for PTC to publish the data they have from previous studies that may support the efficacy of deflazacort over prednisone. For some, families and providers may determine that prednisone is the preferred option. We do not believe that a child or family should have to endure weight gain or behavior struggles to justify the need for deflazacort. That is not fair or ethical.
PPMD will continue to work to make existing data to support access available in our Access Resource Center. We believe that the decision to use steroids, and which steroid to use, should be in the hands of the family and provider who are together able to make optimal medication choices.
This term as applied to the commercial launch of EMFLAZA has certainly introduced a whole new world of unknowns. But perhaps it’s because it wasn’t properly defined. Following the release of the information about EMFLAZA’s pricing, we of course began doing calculations according to the “weight-based” pricing model that had been described.
But here’s what gets lost with that term when applied to Duchenne and deflazacort.
When my boys were diagnosed, they were prescribed an original dose of steroids based on their weights. But then that dose was rarely adjusted – and if it was, it was only a small dose adjustment up until a ceiling or maximum threshold was reached. No matter how old they got, or how much they weighed, they stayed on that maximum dose. In fact, as early teens, they were on only a very low dose of steroids.
The truth is, this isn’t a standard “weight-based” calculation, where the dose continues to increase with weight. Regardless of the patient’s weight, the prescription is filled based upon the dose and frequency that the physician prescribes and the price is based upon that.
While this doesn’t by any means alleviate the concerns regarding the price point and much still is left unanswered, we need to take this a step at a time. We encourage you to have a conversation with our provider to understand what the price of EMFLAZA might be for you/your child, based on his current steroid dose – and then continue that conversation to determine whether pursuing access to EMFLAZA is the right treatment course for you/your child.
If it is, we’ll be here helping to navigate the rest of these unknowns. Together – as a community.
We look forward to updated communications from PTC that address these and other outstanding issues.
Life is filled with so much uncertainty, but here’s what I do know: We will figure access out…for every single one.