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:
a properly licensed treating physician requests the use of an argenx unapproved product to treat a patient that is seriously ill, has no alternative treatment options and cannot enroll in a clinical trial;
the requested product should be in active clinical development, and it is being requested to treat an indication for which there is positive data from a pivotal or registrational study;
there is sufficient scientific evidence to demonstrate that the benefits of the investigational medicine outweigh the risks;
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 IMP for the requested use will not interfere with the initiation, conduct, or completion of clinical trials;
if approved, argenx will provide IMP 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
Patients who are actively enrolled in an argenx clinical trial may qualify for Post-Trial Access if they will experience a temporary gap in treatment until the medication is approved and reimbursed in their 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
- 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 investigational product is not yet approved/authorized 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 investigational product was administered.
- The risks and benefits of using the investigational product in the particular disease or condition of the product resulted in clear favourable risk-benefit to the individual based on the investigator’s assessment of the participant’s response to the intervention, in consultation with argenx.
- argenx has an adequate supply of investigational product to provide to the patient requesting access and, if applicable, to support on-going clinical trials that are critical to getting the new treatment approved and thereby reach the broadest possible patient population.
- A request for PTA to an investigational product will be in compliance with all local laws and regulations and will be temporary until the product is commercially available. argenx will only consider a PTA request in a country where there is a possibility of longterm supply after the PTA.
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.
