We’re driven to discover new treatment approaches in autoimmunity and fueled by the resilience of patients to urgently deliver them.
Patient Access to Investigational Therapies
argenx is committed to improving the lives of people suffering from rare diseases.
Jamilah, CIDP patient
In cases where a patient faces a life-threatening or serious condition, or a clinical trial isn’t an option, or the patient has exhausted all currently available treatment options, their licensed treating physician may request access to an investigational medicine. When reviewing an unsolicited request for treatment, argenx will consider providing access to investigational medicines to patients through an argenx Pre-Approval Access program if the request meets the argenx Pre-Approval Access Principles.
Pre-Approval Access Principles:
Criteria for Requests
a properly licensed treating physician requests the use of an argenx unapproved product to treat a patient who is seriously ill, has no alternative treatment options and cannot enroll in a clinical trial;
the requested product is in active clinical development and has sufficient scientific evidence to demonstrate the benefits outweigh the risks in the context of the disease or condition being treated;
the patient profile and requested treatment regimens are aligned with those from current registrational studies;
the request is neither tied to a commercial interest nor associated with any promotional activities;
providing the medicine for the requested use will not interfere with the initiation, conduct, or completion of clinical trials;
argenx will only consider a PAA request in a country where there is a Bonafide intent for long-term supply after a PAA;
if approved, argenx will provide medicine and adhere to all local laws and regulations.
To request Pre-Approval Access, the licensed treating physician should submit a request to PAATeam@argenx.com. Requests for treatment will be acknowledged and reviewed expeditiously by the argenx Medical Team. If a request for Pre-Approval Access is approved by argenx the physician may also need to obtain Health Authority approval in that country prior to access to the investigational therapy.
Information about argenx clinical trials can be found here or on clinicaltrials.gov. Ongoing argenx PAA programs are listed at https://reaganudall.org/. General inquiries about patient access may be made to PAATeam@argenx.com. argenx is committed to responding promptly to all inquiries received about Pre-Approval Access; inquiries will be addressed within 5 business days.
Submission of a request does not guarantee access to investigational medicinal products (IMP).
In accordance with global laws, including the US 21st Century Cures Act, argenx may revise the posted Pre-Approval Access principles at any time
Post-Trial Access
The Principal Investigator may request PTA for patients who were actively enrolled in an argenx clinical trial, immediately following the end of study, if the patients will experience a temporary treatment gap in treatment until the medication is approved and reimbursed in the patient’s country.
The clinical trial Physician must submit a written request for access for patients who are deriving benefit and do not have an alternative treatment option available to them in their country.
Post-Trial Access Principles
Criteria for Requests
- The participant has a serious or immediately life-threatening condition, has no other suitable treatment options available and does not qualify for another ongoing clinical trial;
- The medicine is not yet approved or commercially available for the indication or is not available by other means in the country where the trial was conducted;
- The request for Post-Trial Access was made by the clinical trial investigator in writing;
- The participant was part of a clinical trial in which the medicine was administered immediately following the end of active clinical trial participation;
- The risks and benefits of using the medicine in the particular disease or condition indicate a favourable risk-benefit assessment for the individual based on the investigator’s evaluation of the participant’s response to the intervention, in consultation with argenx;
- argenx has an adequate supply of product to provide to the patient and, if applicable, to support ongoing clinical trials that are critical to getting the new treatment approved and thereby reach the broadest possible patient population;
- argenx will only consider a PTA request in a country where there is a Bonafide intent for commercialization after a PTA;
- A request for PTA to a product is in compliance with all local laws and regulations and access within the PTA is temporary until the product is commercially available.
To request Post-Trial Access, the licensed treating physician should submit a request to PAATeam@argenx.com. Requests for treatment will be acknowledged and reviewed expeditiously by the argenx Medical Team. argenx is committed to responding promptly to all inquiries received about Post-Trial Access; inquiries will be addressed within 5 business days.
If a request for Pre-Approval Access is approved by argenx the physician may also need to obtain Health Authority approval in that country prior to access to the investigational therapy.
If new information becomes known about the investigational product, the ongoing health of the individual and/or market availability of the product or alternative therapies, Post-Trial Access may end with clear communication to investigator and patient.
