EMFLAZA Launch Plan

Today, PTC Therapeutics hosted a call for the Duchenne community to announce pricing and services for their launch of EMFLAZA, the commercial name for deflazacort.  The announced price will be  $35,000/year for a 25kg patient. This is the net pricing to payors.  PTC is committed to provide access and availability of EMFLAZA at no or minimal cost for eligible U.S. patients.

PTC has partnered with a national pharmacy with experience in delivering drugs to rare disease populations.  EMFLAZACares will assign a dedicated Case Manager to each patient to help them navigate reimbursement with insurance companies and other reimbursement issues.

PTC is providing a variety of programs to provide access to EMFLAZA through the EMFLAZACares program such as a Bridge Program and specialized financial assistance and support for Medicaid and Medicare patients through The Assistance Fund and NORD. The vast majority of states will have coverage.

In order to benefit from these services please follow these steps:

1. Fill out Prescription Start Form  www.EMFLAZA.com
2. Once the Prescription Start Form is received a dedicated case manager will contact you
3. They will work with you to identify the appropriate program
4. If you have submitted a Prescription Start Form there is no need to resubmit
5. If you have participated in the Expanded Access Program you will be transferred to the Bridge Program so there will continuity of care and supply
6. Case managers will work with specialty pharmacy for refill and delivery
7. Case managers will work with insurance companies for benefits and verification and prior authorization

For those with children under 5 or with Becker’s, a physician can prescribe EMFLAZA and case managers will work on a case by case basis to help provide access.

PTC has also committed to continue the additional studies which will seek to generate evidence of the benefits of deflazacort over other steroids, help expand access for all patients, and provide financial assistance to families in need. They have new data from their placebo controlled studies. Steroids are the standard care for Duchenne patients for muscle integrity.

CureDuchenne has had a long-standing relationship with PTC and we appreciate their dedication to the Duchenne community over the last two decades.  We are encouraged that they have additional data that should satisfy the insurance companies’ need for differentiation between EMFLAZA and prednisone, and we look forward to sharing this data with the community when it becomes available.

PTC’s pricing for EMFLAZA is consistent with other weight based drug pricing that have been approved.  PTC’s pricing is determined by a combination of factors such as access, improved standard of care, education, continued investment in Duchenne therapies and financial return to shareholders.

My son, Hawken, has taken daily deflazacort from the time he was diagnosed at age five.  Although we have used a lot of discipline, he has not gained excess weight nor had behavioral issues.  Not all those with Duchenne have the same experience, but I am a huge believer in this drug and I will fight to keep him on it.  I am hopeful that many more boys will be able to have this positive experience through PTC’s program, but we will watch it closely to make sure that the Duchenne community benefits from this drug.

The following is more specific information on your access and next steps in receiving EMFLAZA:

In order to ensure ease of EMFLAZA access and delivery to Duchenne patients, PTC has partnered with a national pharmacy that specializes in distributing medicines to small patient populations.  They will provide customized, personalized support to each patient through EMFLAZACares by offering everyone enrolled a dedicated Case Manager who will help navigate upfront and ongoing reimbursement requirements and schedule shipments of EMFLAZA.

The Case Manager will automatically enroll a patient with commercial insurance coverage to a program that will help to offset out-of-pocket costs for co-pay, co-insurance and deductibles for pharmacy benefits.  Patients with Medicaid and Medicare coverage will be directed to a foundation where they may get specialized financial assistance and support.  The two foundations that have programs for Duchenne are The Assistance Fund and NORD.  Last but not least, for patients that do not have insurance, they may qualify for EMFLAZA free of charge based on their family’s financial status.

Once you and your physician determine that EMFLAZA is right for you or your child, getting started is easy.  First, a Prescription Start Form is filled out and signed by both you and the physician and faxed into EMFLAZACares.  This serves as the prescription and your signature allows the Case Managers to provide the benefits investigation and discuss your case with your doctor.  These forms can be found on the website www.EMFLAZA.com.

Within a couple of days of receiving the form, the Case Manager will contact you to confirm your information and describe the process to you.  At this time you can share with him/her how you would like to be communicated with.  From this point forward, the interactions with the insurance company will be handled by the Specialty Pharmacist and Case Manager specifically assigned to you and they will keep you updated on the progress.  They will also determine which program may be appropriate for you to help offset the out-of-pocket costs depending on the insurance provider.  The amount of time that it will take to get insurance approval will vary from commercial plan or State Medicaid formularies as well as from patient to patient and may take a week to several months.

PTC currently has over 900 prescription start forms that have been submitted to EMFLAZACares.  If you have a start form submitted, you do not have to resubmit a form.  A Case Manager will be following up with you to discuss next steps.

PTC is committed to getting patients onto EMFLAZA as quickly as possible, without interruption of therapy.  For those that are currently accessing EMFLAZA through the Expanded Access Program or Master Pharmacy importation from outside of the US, you may have immediate access to a “Bridge Program” that will provide EMFLAZA free of charge for up to three months while insurance benefits are being investigated.  An EMFLAZA Prescription Start Form is required and your physician will need to sign the “Bridging Supply” section in the form and submit the completed form to EMFLAZACares.  The EMFLAZACares team will contact the patient to enroll them in the program and assess their need for “Bridge” to ensure continuity of care.  Once your insurance has approved your first prescription, the pharmacy will call you to schedule the delivery of EMFLAZA.  You can arrange for this to come to your home, office or another location of your choice.

When it comes time for a refill, EMFLAZACares and the pharmacy will handle any reauthorizations and call you to plan the next delivery based upon your schedule and location.

For more information contact patientinfo@PTCbio.com or 844-EMFLAZA.