1 What is the purpose of an expanded access program (EAP)?
- Expanded access refers to a variety of programs that provide patients with access to an investigational treatment for life threatening, severe and/or debilitating diseases.
- To learn more about EAPs, please click here
2 What is the purpose of the PEACE program?
The purpose of the PEACE program is to provide adult patients in the United States who suffer from narcolepsy with or without cataplexy access to pitolisant* while a New Drug Application is being prepared and submitted to the Food and Drug Administration for review for marketing approval.
3 What is the difference between an expanded access program (EAP) and a clinical trial?
The difference between an EAP and a clinical trial is that the use of an investigational medical product in an EAP is intended for treatment, and the use of an investigational medical product in a clinical trial is intended to answer questions about the safety and effectiveness of the investigational medical product.
4 Are patients diagnosed with other sleep disorders eligible to participate in the PEACE program?
- All patients interested in participating in the PEACE program MUST be diagnosed by a physician as having narcolepsy type 1 or type 2.
- Patients with other sleep disorders who are also diagnosed with narcolepsy may be eligible to participate.
5 Is everyone who has been diagnosed with narcolepsy type 1 or type 2 eligible for this expanded access program?
- Not all patients with narcolepsy will be eligible to participate in the PEACE program. You are encouraged to speak to your treating physician about the possibility of taking part in the program.
- You can also browse a list of physicians already participating in the PEACE program using the following link.
6 What is pitolisant*?
- Pitolisant* is an investigational medication in the U.S. being developed to treat the symptoms of excessive daytime sleepiness and cataplexy in patients with narcolepsy (type 1 and 2).
- Harmony Biosciences, LLC, in partnership with Bioprojet, will be submitting a New Drug Application to the Food and Drug Administration (FDA) for the evaluation of the safety and effectiveness of pitolisant.
- Although pitolisant* has not been approved by the FDA for any indication in the U.S., since March 2016 it has been approved for use in Europe for the treatment of adult patients with narcolepsy with or without cataplexy.
7 Do I need to ask my treating physician if I can participate in this expanded access program (EAP)?
- Yes, you will need to speak to the physician who treats your condition about the possibility of taking part in this EAP.
- Your treating physician will need to be registered with the PEACE program in order for you to receive pitolisant*.
- You will only be able to participate and receive pitolisant* through the PEACE program once your physician determines that you are eligible, and once you complete the enrollment process.
8 Can I participate in other clinical studies while in this program?
- Patients who are participating in clinical studies for other investigational treatments/therapies may still be eligible for participation in the PEACE program.
9 If my treating physician feels I am eligible, but does not want to participate, is there a way for me to still take part in the program?
Yes, you and the physician who treats your condition can review the list of participating physicians in your area and decide if it is a good option for you to visit a different physician who is currently participating in the PEACE program.
10 My next appointment is several months away. Can I sign up now or do I have to wait until my next appointment?
You should talk to your physician to determine what is best for you.
11 What will I be asked to do if I take part in this program?
- If you agree to enter this program, your physician may ask you to visit his/her clinic to confirm whether you are eligible for participation.
- You will be required to sign an Informed Consent Form before participation. Your treating physician will then decide if you should take pitolisant*.
- You can talk to your physician about this process.
12 How long will I take part in the program?
- If you participate in the PEACE program, you may continue participating until one of the following occurs:
- Pitolisant* is approved in the U.S. (if approved) and made commercially available
- The PEACE program is terminated
- Your treating physician determines that you should stop taking part in the program
- You decide you no longer want to participate.
- If pitolisant* obtains approval by the Food and Drug Administration, the sponsor or the sponsor’s representatives will begin working with you and your physician to ensure a seamless transition to the approved product.
13 How will I be compensated for my participation in this program?
While participating in the PEACE program, pitolisant* will be provided to participants at no cost. You will not be paid to take part in the PEACE program.
14 What is the role of the specialty pharmacy in this program?
For the purposes of this program, the specialty pharmacy will deliver pitolisant* and help with returns if necessary.
15 Who will have access to my information collected during participation?
- Any personally identifiable information (for example, contact details required for shipping and distributing the investigational medication) will not be accessible to anyone who is not directly associated with the PEACE program, except with the patient or caregiver’s permission or as required by law.
- Any information that is gained from this EAP may be used by the sponsor, Harmony Biosciences LLC, for publishing outcomes and for regulatory purposes. However, this information will be combined with other participant data and will not identify any individual participant.
16 Who can I speak to if I still have questions?
- If you have any questions about this program, talk to your physician. Alternatively, you may contact us at: +1-844-246-5126.
*Pitolisant is an investigational medication in the U.S that is not currently approved by the Food and Drug Administration (FDA). Harmony Biosciences, LLC, in partnership with Bioprojet, will be submitting a New Drug Application to the FDA for the evaluation of the safety and effectiveness of pitolisant. Pitolisant was approved in Europe in March 2016 for the treatment of adult patients with narcolepsy with or without cataplexy (type 1 and type 2).