Frequently Asked Questions

1. 1 What is the purpose of an expanded access program (EAP)?

   • The intent of an EAP is to provide patients with access to investigational treatments for life-threatening, severe and/or debilitating diseases.
   • If you would like more information on EAPs, please visit the Expanded Access Program Information for Physicians.

2. 2 What is the purpose of the PEACE program?

   • The purpose of the PEACE program is to provide access to pitolisant* to adult patients in the United States who suffer from narcolepsy with or without cataplexy while a New Drug Application is being prepared and submitted to the FDA for review for marketing approval.

3. 3 What is the difference between an expanded access program (EAP) and a clinical trial?

   • Expanded access is used to refer to a variety of regulatory mechanisms available to provide patients with life threating and/or severely debilitating diseases access to investigational treatments.
   • By definition compassionate use or EAPs are intended to address unmet medical need and, as such, no two EAPs are alike beyond the regulatory requirements.
   • The distinction between an EAP and a clinical trial is that the use of an investigational product in an EAP is intended for treatment, not to answer questions about safety and efficacy. The use of an investigational product in a clinical trial is intended for research (systematic collection of data with the intent to analyse it to learn about the product, especially as it relates to safety and effectiveness).

4. 4 Are patients diagnosed with other sleep disorders eligible to participate in the PEACE program?

   • All patients interested in participating in the program MUST be diagnosed with narcolepsy type 1 or type 2 based on ICSD-3 criteria.
   • Patients with other sleep disorders who are also diagnosed with narcolepsy may be eligible to participate.

5. 5 Is everyone who has been diagnosed with narcolepsy eligible for this expanded access program?

   • No, please refer to the full eligibility criteria posted on clinicaltrials.gov
   • All patients and/or their caregivers interested in taking part in the program are encouraged to speak to their physician about this program.

6. 6 What is pitolisant*?

   • Pitolisant* is novel, selective, orally active histamine H3 receptor antagonist/inverse agonist
   • Harmony Biosciences, LLC, in partnership with Bioprojet, will be submitting a New Drug Application to the FDA for the evaluation of the safety and efficacy of pitolisant.  
   • Pitolisant* is not approved in the U.S. but was approved for use in Europe in March 2016 for the treatment of adult patients with narcolepsy with or without cataplexy.

7. 7 How is pitolisant* administered?

   • Patients enrolled in the PEACE program will receive pitolisant* in an open-label fashion.
   • The recommended dose of pitolisant* is 35.6 mg, administered orally as a single, daily dose of two 17.8 mg tablets in the morning (upon wakening), before breakfast.
   • Patients should be titrated to 35.6 mg per day (or the highest tolerable dose) over a 3-week period:
     • Week 1: Initial dose of 8.90 mg (two 4.45 mg tablets) per day each morning (upon wakening) before breakfast.
     • Week 2: Increase dose to 17.8 mg (one 17.8 mg tablet) per day each morning (upon wakening) before breakfast.
     • Week 3: Increase dose to 35.6 mg (two 17.8 mg tablets) per day each morning (upon wakening) before breakfast.
   • A specialty pharmacy will deliver a sufficient supply of pitolisant*, based on the participating physician’s order of dose and regimen, directly to the patient, a caregiver, or to the treating physician themselves.

8. 8 Where can I learn more about pitolisant*?

   • Registered physicians can access the pitolisant* expanded access program Investigator’s Brochure and protocol through the online portal.
   • If you have further questions about pitolisant* please call: +1-844-246-5126 or email: PEACE-Program_Questions@parexel.com

9. 9 Will I still be responsible for my patient’s ongoing care if they he/she takes part in the program?

   • Yes, while participating in the PEACE program you will still retain the same responsibilities for your patient’s ongoing and usual standard of care.
   • You will also be subject to additional regulatory responsibilities as an investigator (see FAQ, “What are the regulatory requirements for an EAP?”).

10. 10 Can my patient participate in other clinical studies while enrolled 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.

11. 11 If my patient is eligible, but I do not want to participate in the program, is there a way for my patient to still take part in the program?

    • Yes, you and your patient can review the list of participating physicians in your area and decide if it is a good option for you to transfer your patient’s care to an alternate physician who is currently participating in the PEACE program.
    • Check the list of participating physicians to see who is currently participating in the PEACE program.

12. 12 How long will my patient take part in the program?

    • Patients may continue participating in the PEACE program until one of the following occurs:

    1. Pitolisant* is approved in the U.S. (if approved) and made commercially available
    2. The PEACE program is terminated
    3. The treating physician determines that the patient should stop taking part in the program
    4. The patient decides that he/she no longer wants to participate.

    • If pitolisant* obtains FDA approval, the sponsor or the sponsor’s representatives will begin working with you and your patient(s) to ensure a seamless transition of your patient(s) to the approved product.

13. 13 What are the expectations for data collection in this program?

    • The PEACE program will only collect information related to symptoms of narcolepsy, concomitant medications, and safety/tolerability data during the program in accordance with the protocol and applicable regulatory requirements.

14. 14 What is the program visit schedule?

    • There are no program-required visits following enrollment. Participating physicians will follow standard of care for the management of their patients.

15. 15 What are the regulatory requirements for an expanded access program (EAP)?

    • As a participating physician under an EAP, you are subject to the same regulatory responsibilities as other investigators in clinical studies, and as outlined on the Form FDA 1572. For example, you will be required to obtain informed consent from your participants and you are subject to adverse event reporting requirements and record keeping obligations.
    • All specific responsibilities will be outlined in the EAP protocol. Institutional Review Board (IRB) approval and oversight is also required for EAPs. You may either submit your regulatory package to your local IRB or to the identified central IRB.

16. 16 What is the role of the specialty pharmacy in this program?

    • For the purposes of this program, the specialty pharmacy will be involved in the delivery and return of pitolisant*. In addition, the specialty pharmacy will serve as a call center to address questions for patients and treating physicians.

17. 17 Who will have access to my patient’s 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 expanded access program 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 individual participant information will be deidentified.

18. 18 What happens if pitolisant* is approved and becomes commercially available?

    • If pitolisant* obtains FDA approval, the sponsor or the sponsor’s representatives will begin working with you and your patient(s) to ensure a seamless transition of your patient(s) to the approved product.

19. 19 How can I obtain a copy of the protocol and consent forms for review?

    • Registered physicians will have access to the protocol, informed consent documents, and other program-specific documentation through PAREXEL’s MyAccess Portal.

20. 20 Who can I speak to if I still have questions?

    • If you have further questions about this program, please call: +1-844-246-5126 or email PEACE-Program_Questions@parexel.com