We know that many of you have questions regarding Marathon Pharmaceuticals and their expanded access program for deflazacort in the US. PPMD has reached out to the company to get answers to some of the questions. The FAQs below explain what we have learned so far. As we learn more about Marathon, deflazacort, and their expanded access program, we will share all information with you.
Please remember: Participation in any clinical trial, research, or expanded access program requires a conversation between you and your medical providers. We encourage you to discuss any questions or concerns that you may have with your neuromuscular team.
Q: What is the difference between the Marathon deflazacort and other deflazacort that I/my child has been using?
A: Marathon’s deflazacort should be no different than the deflazacort your child is currently taking. However, unlike the deflazacort you are currently importing, Marathon’s deflazacort is manufactured in the US under GMP (Good Manufacturing Practices) conditions. You can be ensured that a 6 mg tablet of deflazacort contains 6 mg of deflazacort. Deflazacort is not approved in any country for the treatment of Duchenne. Marathon has conducted a full development program to ensure the FDA that it is safe and effective for the treatment of Duchenne. The EAP and the deflazacort used in the EAP has been allowed by the FDA. Thus, Marathon will be submitting an NDA (as a New Drug Application) to the FDA and not an ANDA (which is to the Office of Generic Drugs).
Q: Why does deflazacort need to be tested? We know that it is safe – we have used it for years.
A: Deflazacort is not currently approved in the United States for any conditions. As with any new drug, Marathon needs to run a full development program for deflazacort to ensure the FDA that it is safe and effective for the treatment of DMD. Marathon will be presenting data from two phase three studies in DMD patients, pharmacokinetic data for deflazacort as well as preclinical animal data to support the approval of deflazacort. This is the same process for any new drug seeking approval in the US. Marathon plans, to submit a New Drug Application (NDA) to the FDA by May 2016. Marathon’s hope is to have their deflazacort approved for use in the US by January 2017.
Q: Why would the FDA allow me/my child to take a drug that they have not yet approved?
A: Because deflazacort is being used for the treatment of a rare disease for which they feel there is no comparable therapy, the FDA is allowing Marathon to make deflazacort available through an “expanded access program” (EAP) while going through the necessary approvals. By making deflazacort available, the FDA is not assuring that this drug is safe or will work; the risks and benefits of using this investigational drug will need to be discussed by patients and medical providers.
Q: Why would my medical provider choose not to participate in the EAP?
A: One reason may be due to decisions of the hospital IRB. By participating in an EAP (or any other clinical trial), your medical provider is giving patients access to an investigational new drug. Every hospital has an Institutional Review Board (IRB) that maintains control of research and experimentation within the institution. If the IRB at your medical provider’s hospital feels that the risks for patients and providers to participate in the EAP outweigh the benefits, they will not allow their institution to participate in the EAP. This is not the decision of your provider, but of their institution. Another reason is that your medial provider has not yet submitted to the IRB or is in the process of garnering approval from the IRB. You may want to speak with your medial provider to find out more about the IRB status of the EAP.
Some providers may want to wait until the FDA has approved Marathon’s deflazacort before prescribing it to you.
Q: If I/my child takes Marathon’s deflazacort, will this impact participation in current or future clinical trials?
A: This will need be determined by each trial’s inclusion/exclusion criteria. A trial’s inclusion/exclusion criteria is up to the company or investigator of that study. If you/your child is participating in a clinical trial now, or anticipates that they will be participating in a trial in the near future, the best advice is to contact that trial coordinator and ask whether taking Marathon’s deflazacort will exclude participation. That way there will be no surprises. However, if your child is participating in another clinical trial, Marathon will allow him to participate in the EAP.
Q: If my provider does enroll in this program, how long will it take to get Marathon’s deflazacort?
A: This is a new program, so how long it will take to get the drug is not known for sure. The first phase is to enroll physicians to the EAP. Marathon states that it will take at least a few weeks or even a couple of months for your provider to enroll in the EAP program. Regarding the second phase (process to register a patient), there is no definite information about how long it will take to get the deflazacort. Your provider, once they are enrolled, will present you an Informed Consent Form with details about the expanded access program, your rights as a participant, potential side effects and other information about deflazacort. Your provider will then will be able to set clear expectations on how long it will take to get Marathon’s deflazacort. If you are already receiving deflazacort by other means (not through the EAP) you should consult with your physician first before stopping shipments in order to ensure there is no interruption in your treatment. The drug will also be shipped to families, so it is recommended that families have enough drug on hand to wait out the shipment. Marathon will work directly with families, and should keep families informed regarding their shipments.
Q: How will this affect the costs of deflazacort now for our family? In the future?
A: Deflazacort provided through the EAP is of no costs to families. If Marathon’s deflazacort is approved and available in the US in the future, it is unclear how this will affect pricing. If deflazacort is approved and available in the US, it is thought that you will most likely not be able to import the drug from outside the US (i.e., UK, Mexico, Switzerland, etc.).
Q: Are any additional laboratory studies/procedures part of the EAP program? Are any extra visits to clinic needed?
Q: Are there specific places/providers that already are enrolled in the EAP and where do I find that out?
A: Marathon has developed a website (AccessDMD.com) with all pertinent information, including providers who are currently participating in the program. If you see a city listed on the “participating physician” map with no doctor listed, this means that there is a site that is going through the process of enrolling but not yet complete. The website also has a “frequently ask questions” section and “resources” section that can answer some of your additional questions.
Additionally, if your doctor is not listed on the website and you would like to participate in the EAP, please contact them directly and ask him or her to enroll in the program.
UPDATE 1/15/16: Addendum to Marathon FAQ
We know that there have been questions about whether taking Marathon’s deflazacort would impact involvement in currently ongoing and/or future clinical trials. We have had the opportunity to talk both with Marathon Pharmaceuticals, as well as several other industry partners, about the use of Marathon’s deflazacort. This is what we have been told:
Q: If I/my child elects to participate in Marathon’s extended access program (EAP), and switch from our current supplier of deflazacort to the Marathon deflazacort, is there a “wash out” period? Will I need to wean off the current deflazacort, stay “steroid free” for a period of time, then wean on to Marathon’s deflazacort?
A: No. Marathon’s deflazacort is essentially the same as the deflazacort that you are currently using. You may switch immediately from one to the other, keeping the dose the same.
Q: Do other industry partners consider Marathon’s deflazacort to be a “study drug”?
A: We did contact several industry partners with this question. Their answer was that no, they do not consider Marathon’s deflazacort to be a “study drug.” It is considered to be another form of deflazacort. We recommend that you contact the specific industry sponsor of the trial that you are involved in, or considering, to confirm.
Q: I/my child is involved in a clinical trial and taking deflazacort. Will my participation in Marathon’s EAP, taking Marathon’s deflazacort, change whether I/my child can stay in that trial?
A: Again, we recommend that you contact the specific industry sponsoring your trial, or the trial that you are considering. The industry partners that we contacted stated that they would actually prefer that patients in their trials be on Marathon’s deflazacort. They felt more comfortable with this supply of deflazacort than other global supplies.
Q: Is this drug safe? Has it been reviewed by the FDA?
A: The FDA has reviewed summaries of two completed phase 3 studies, pharmacokinetic data from healthy volunteers, data from the literature, preclinical animal data, manufacturing data and has allowed for Marathon to provide deflazacort through the EAP while the new drug application is under preparation and review at the FDA. The FDA will completely Marathon’s NDA submission in 2016, with a US approval for deflazacort expected first quarter of 2017. Marathon intends to file the new drug application for deflazacort in May of next year and the FDA will then review the complete package and determine if the drug can be approved in the U.S.
Q: Right now my deflazacort prescription is $1500/year/patient. How much will patients pay for deflazacort after US FDA approval?
A: Currently for 2015, 2016 and first part of 2017, people participating in the Marathon EAP will receive deflazacort at no cost. With the FDA approval of deflazacort, patients in the Marathon EAP program will be transitioned from the EAP program to a deflazacort prescription. Marathon is committed to ensure that all patients will have a successful transition without experiencing any gaps in deflazacort supply. The marathon call center representatives will assist patients with the transition to insurance coverage, Medicaid coverage, and potential patient assistance programs. Marathon’s goal is to have 100% access to deflazacort with zero out of pocket expense through a large offering of patient assistance programs.
Q: My clinical trial states that I cannot participate in any other clinical trial while I am participating in this current/future clinical trial. Is Marathon’s EAP considered to be a clinical trial?
A: No, Marathon’s EAP is only considered to be access to deflazacort and is not considered to be a clinical trial. However, we still recommend that you contact the specific industry sponsoring your trial, or the trial that you are considering