Rare diseases are difficult to diagnose, research, and treat. Even though more than 7,000 rare diseases have been identified, only about 5% of those have indicated treatments. Our community was extremely fortunate to see not one, but two treatments receive FDA-approval in 2018 with an indication for Dravet syndrome (DS). When no treatment protocol works for every patient, it is invaluable to have options. The two drugs that recently received approval are Epidiolex, produced by Greenwich Biosciences, and Diacomit (Stiripentol), produced by Biocodex.

Epidiolex is the first prescription pharmaceutical formulation of highly purified, plant-derived cannabidiol (CBD), a cannabinoid lacking the high associated with marijuana, and the first in a new category of anti-epileptic drugs. It is used to treat DS and Lennox Gastaut syndrome in patients 2 years and older. Epidiolex is currently only distributed through five specialty pharmacies across the U.S. A specialty pharmacy differs from a traditional pharmacy in that they are accustomed to working with patients with complex and/or rare conditions and they can coordinate many aspects of patient care and disease management. You can learn more about Epidiolex and the patient and family support options Greenwich Biosciences offers at www.epidiolex.com.

If you are interested in obtaining Epidiolex:

  • Your physician should complete an enrollment form and submit your prescription (available on their website, in the section for Medical Professionals).
  • The specialty pharmacy will verify your insurance eligibility and other requirements in order to fill your prescription.
  • You will then be contacted by the specialty pharmacy.
  • After processing, you will receive your medicine shipped to your home, or in some cases, shipped to your local pharmacy for pick up.

Should you have issues with insurance coverage, Greenwich Biosciences has a support team in place to help you with insurance verification and approval. They also offer a co-pay assistance program for eligible patients.  

While Diacomit only recently received FDA approval, some patients have been receiving it under Expanded Access for many years. Expanded Access is a pathway for patients who have a life-threatening condition. It allows patients to try an investigational drug outside of a clinical trial, when there are no other comparable treatments available. The patient must meet the criteria under the law and FDA regulations, and then their physician and the Institutional Review Board (IRB) at the treating institution must agree that the investigational drug is appropriate for the patient before they can receive it.

One of the barriers that many of our patient families have faced in attempting to procure Diacomit for their child is the fact that the costs for a drug secured through Expanded Access are not necessarily covered by third-party payers such as private insurance or Medicare. For most families, the out-of-pocket cost in these situations are cost-prohibitive. Now that Diacomit has full FDA-approval with an indication for DS it will be much easier for families to obtain coverage. Biocodex has recently shared the process to obtain Diacomit in the US as of April 29, 2019. They will be utilizing a dedicated specialty pharmacy program administered by US Bioservices.

If you are interested in obtaining Diacomit (Stiripentol):

  • Patients will need a medical prescription for Diacomit that must be sent to US Bioservices, via fax at (833) 871-4137.
  • US Bioservices will coordinate with the patient and physician to determine the patient’s insurance eligibility and coverage.
  • In the event of no coverage or inadequate coverage, Biocodex offers a generous Patient Assistance Program to help patients gain access to Diacomit.
  • US Bioservices’ support team will keep the patient informed on the need for additional information and the outcomes of the application.
  • If you or your physician have any other questions on obtaining Diacomit, contact US Bioservices at (833) 248-0467.

As Thomas Edison said, When you have exhausted all possibilities, remember this: You haven’t. Our community is fortunate to not only have these two new treatment options, but also to have several other trials already underway or soon to be started, which could prove beneficial for patients with DS. There is hope on the horizon.