NAMED PATIENT ACCESS PROGRAMS (NPAP)

Patients with serious or life-threatening diseases may seek medical products that, while potentially approved by a stringent regulatory authority such as the US FDA, have not yet been approved or made available in their respective country. ‘Named Patient Access Programs’ help address this need by providing eligible patients access to certain unapproved or investigational treatments. Below you’ll find additional details about this program for pretomanid.

WHAT IS PRETOMANID’s NPAP ACCESS PROGRAM?

Tuberculosis (TB) is the world’s leading cause of death from a single infectious disease. In 2018, an estimated 10 million people developed TB. There were an estimated 1.2 million TB deaths among HIV-negative people in 2018 and an additional 251,000 deaths among HIV-positive people. Overall, there were an estimated 484,000 (range, 417,000–556,000) incident cases of MDR/RR-TB (Multi Drug Resistant/Rifampicin Resistant Tuberculosis) in 2018, 6.2% of which had XDR-TB (Extensively Drug Resistant Tuberculosis).1 

Patients with XDR-TB are infected with strains of M. tb (Mycobacterium tuberculosis) that are resistant to rifampicin, isoniazid, at least one fluoroquinolone, and at least one second-line injectable agent (amikacin, capreomycin, or kanamycin). MDR-TB is caused by strains of M. tb resistant to at least isoniazid and rifampicin, and patients with Treatment-intolerant or Non-Responsive (TI/NR) MDR-TB are intolerant of, or do not respond to, the treatment prescribed for already difficult-to-treat MDR TB.2 

On August 14, 2019, the US FDA approved a drug called 'pretomanid' to be used as part of a combination regimen with bedaquiline and linezolid for the treatment of adults with pulmonary Extensively Drug Resistant (XDR), Treatment-Intolerant or Non-Responsive Multidrug-Resistant (MDR) tuberculosis (TB). Treatment Intolerant (TI) MDR TB patients are those who are unable to continue second-line drug regimen due to a documented intolerance. Non-Responsive (NR) MDR-TB patients are those with documented non-response to MDR-TB treatment for 6 months or more.2

The pretomanid Named Patient Access Program (NPAP) allows the treating physician to submit a request for pretomanid prior to regulatory approval in their respective country on a named-patient basis, provided it is permitted by applicable local laws.

Are you Eligible?

The following criteria* must be met by a patient in order to participate in Mylan’s NPAP:

  • The patient seeking treatment has highly drug resistant TB (adults with pulmonary XDR or TI / NR MDR TB#) and there is no satisfactory alternative therapy available to treat the disease
  • The patient is not eligible to enroll in a clinical trial
  • The potential benefit of treatment use outweighs the potential risk in the context of the disease to be treated as confirmed by the treating physician at the time of request
  • The product is not yet approved by the local regulatory body in the treating physician’s respective country
  • Pretomanid should be used as part of a combination regimen with bedaquiline and linezolid.

* All above criteria are subject to local laws and regulations

#Based on written request from country regulatory authority, MDR TB patients outside of TI/NR category can be considered for access to pretomanid

How is your Request Evaluated?

Mylan

As a part of the global access initiative, Mylan is working with TB Consilium – an initiative offered by the Global TB Network4 - to provide access to pretomanid on a named-patient basis in those countries where the product is not yet approved by the local regulatory body.

Each request will be immediately acknowledged and carefully reviewed by the TB Consilium. During this review period, the treating physician may be asked questions by the TB Consilium to ensure the request is properly evaluated. After the request is reviewed, the TB Consilium will make a recommendation to the Mylan ‘Pretomanid NPAP’ Committee which will review and either approve or reject the request. The physician will then be immediately notified of the decision and informed on next steps by Mylan within 7 business days of the original request.

Please note that your request will be assessed in consideration of applicable local laws and regulations. For pretomanid NPAP-related questions, please contact the below email id.
accesspretomanid@mylan.com.

SUBMIT A NPAP REQUEST

A request must be submitted by the treating physician on behalf of the patient. Request forms can be found below:

Please fill the request form below.

** = Required

Physician Information

Pretomanid NPAP request

#Based on written request from country regulatory authority, MDR TB patients outside of TI/NR category can be considered for access to pretomanid

*Please provide specific information to aid decision-making. However, do not include any patient identifiers.

I confirm that:

  • I am the treating physician for the patient
  • The patient seeking treatment has highly drug resistant TB and there is no satisfactory alternative therapy available to treat the disease
  • The patient is not eligible to enroll in any clinical trial for a medical product
  • The potential benefit of treatment use outweighs the potential risk in the context of the disease
  • The product is not yet approved by the local regulatory body in my respective country
  • Pretomanid would be used as part of a combination regimen with bedaquiline and linezolid.

https://app.globaltbconsilium.com


Next Steps: How it works
  1. Register your account on TB Consilium
  2. Submit the TB Case through TB Consilium
  3. Receive a written decision from Mylan within 7 business days


FAQs

  • If my request is approved, what happens next?

    If your request is approved by Mylan, you will be directed to WEP Clinical who will work directly with you to obtain all consents, waivers and permits required under your country’s applicable laws to import pretomanid for your patient.

  • What will pretomanid cost?

    Mylan is making pretomanid available for this program. Our partner, WEP Clinical will work with you to obtain all necessary approvals to enable pretomanid to be provided in your country. As soon as your request is approved, they will provide relevant logistic details including costs that may be applicable.  Special considerations have been made to ensure affordability for low-income and lower middle-income countries as defined by the World Bank; these countries will be offered a price on par with the Global Drug Facility price.

  • What happens if pretomanid is already approved in my country?

    If pretomanid is approved in your country, the patient would not be eligible for the NPAP.  Please reach out to your country’s national TB program or directly to Mylan (accesspretomanid@mylan.com)

  • How long will this process take from start to finish?

    Your request will be reviewed within 7 business days from when the original request is made.  Following this, timelines will depend on your country’s regulatory requirements and processes for importing unapproved product for patients on a named basis. WEP Clinical will help guide you on timelines once your request is approved.

  • Can you help me obtain the other drugs in the regimen?

    NPAPs are designed to provide access to new drugs in countries that have not yet issued regulatory approval. Please contact your national TB program for information regarding accessing bedaquiline and linezolid, which must be used along with pretomanid per the US FDA approved label.

For any other questions on the Mylan NPAP, please reach out to accesspretomanid@mylan.com

References:

  1. Global tuberculosis report [Internet]. WHO. 2019 [cited 23 Oct 2019]. Available from: https://apps.who.int/iris/bitstream/handle/10665/329368/9789241565714-eng.pdf?ua=1
  2. Pretomanid FDA Briefing Document [Internet]. 2019 [cited: February 2020]. Available from: https://www.fda.gov/media/127593/download
  3. U.S. Food and Drug Administration. (2019). FDA approves new drug for treatment-resistant forms of tuberculosis that affects the lungs. [online] Available at: https://www.fda.gov/news-events/press-announcements/fda-approves-new-drug-treatment-resistant-forms-tuberculosis-affects-lungs [Accessed 20 Aug. 2019].
  4. Tiberi S, Pontali E, Tadolini M, D'Ambrosio L, Migliori GB. Challenging MDR-TB clinical problems - The case for a new Global TB Consilium supporting the compassionate use of new anti-TB drugs. Int J Infect Dis. 2019 Mar;80S:S68-S72. doi: 10.1016/j.ijid.2019.01.040.

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Copyright Notice – Except as otherwise noted in this website, the contents of this website are copyrighted by Mylan. All rights are reserved. NO part of the information on this website, including text, graphics and html code, may be reproduced or transmitted in any form by any means without the express written consent of Mylan.

Website Disclaimer

General Information – The material and information contained in this website is provided for general information only and should not be used as a basis for making business decisions. Any advice or information received via this website should not be relied upon without consulting primary source of information or specific professional advice. Warranty and Guarantee - Mylan does not, expressly or impliedly, warrant, guarantee or make any representations concerning the use, results of use or inability to use or access the information or the contents of the website, in terms of accuracy, reliability completeness, functionality continuity, timeliness, fitness for a particular purpose and/or non-infringement.

Mylan does not warrant that the website or functions thereof will be uninterrupted or free of any error or defect or that such information will satisfy any intended purpose or requirement of the user. Mylan does not endorse and shall not be responsible for any linked sites or their contents available in the website.

Limitation of Liability - Mylan specifically disclaims any and all liability, whether based in contract, tort, strict liability or otherwise, for any direct, indirect, incidental, consequential or special damages arising out of or in any way connected with access to or use of the website(including any bulletin boards contained herein), even if Mylan has been advised of the possibility of such damages including but not limited to reliance by any user on any content obtained through use of this website, or that arises in connection with mistakes or omissions in, or delays in transmission of, information to or from the user, interruptions in telecommunications, connections to the website or viruses, whether caused in whole or in party by negligence, acts of god, telecommunication failure, theft or destruction of , or unauthorized access to the website or related information. Mylan does not guarantee response to any emails received through its website. Mylan does not warrant the privacy and/or security of emails during internet transmission.

Mylan specifically disclaims any and all liability arising from any content posted by a third party on any bulletin board contained in this Website, including any content which may be offensive, profane or obscene. However, Mylan reserves the right to remove the content that is deemed unacceptable. Jurisdiction - Mylan generates and maintains this website from India and Indian laws will have the jurisdiction over this website and courts of Bangalore shall have jurisdiction to deal with any disputes arising out of or in connection with this website.

Copyright Notice – Except as otherwise noted in this website, the contents of this website are copyrighted by Mylan. All rights are reserved. NO part of the information on this website, including text, graphics and html code, may be reproduced or transmitted in any form by any means without the express written consent of Mylan.