Individual Access to Investigational Medicines Intended to Treat Serious Diseases
As it relates to individuals, expanded access, sometimes called “compassionate use”, is the individual patient use of an investigational medicinal product outside of a clinical trial that is intended to treat a serious or life-threatening condition. Wherever possible, use of an investigational medicinal product by a patient as part of a clinical trial is preferable because clinical trials generate data regarding the efficacy and safety of the investigational medicinal product. Those data help inform whether the benefits of the investigational medicinal products outweigh the risks, whether it should be approved by the regulatory authorities leading to wider availability and, if approved, when and how it should be used.
When it is not possible for a patient to enroll in a clinical trial, Orpha Labs AG will strive to provide access to the investigational medicinal product for individual patient use, or through an expanded access protocol when appropriate.
Various regulatory mechanisms exist in different countries to provide expanded access to investigational medicinal products and, as a result, regional variations in expanded access will occur.
It is important to remember that investigational medicinal products have not yet received regulatory approval; thus, their potential risks and benefits are not yet established. Physicians and patients should consider all possible benefits and risks when seeking access to an investigational medicinal product.
Expanded Access Criteria
Orpha Labs AG uses the following criteria for considering requests to provide access to individual patients for expanded access: The patient is suffering from a serious/life-threatening condition, and:
There is a strong biological rationale or clinical data that support the possibility that the potential benefits of administration of the investigational medicinal product to the patient for that serious/life-threatening condition could outweigh the potential risks.
The patient’s physician has determined that treating the patient with the investigational product is in the patient’s best interests.
The investigational medicinal product will be administered in accordance with applicable laws and regulatory requirements of the country where the patient is treated, including importation requirements.
The patient is not eligible or able to participate in a clinical trial or similar sponsored access program.
No therapeutic alternative is available.
Treating physicians submit expanded access requests on behalf of patients to Orpha Labs AG at in addition to obtaining the local regulatory approval for the proposed use of the investigational medicinal product.
Medical professionals at Orpha Labs AG who are familiar with the data collected on the investigational medicinal product evaluate each request and respond based on the scientific evidence available at the time of the request and the criteria listed above.
All individual patient use requests submitted by a treating physician must include the following information to be considered:
Date of request
Requesting physician’s name, contact information, address (including country), and professional designation (i.e., MD) or qualifications
Name of the requested investigational medicinal product along with physician’s intended treatment plan, including therapeutic indication and expected duration of treatment
Medical rationale for request including an explanation for why alternative therapy cannot be used, why the patient does not qualify for a clinical trial, and why use of the investigational drug is in the patient’s best interest.
A response for expanded access is typically received by the requesting physician within 30 days with either: approval, a request for additional information, or a rationale for non-approval.
Additional information regarding our pipeline of investigational medicinal products can be found on our pipeline page and information regarding specific Orpha Labs AG clinical studies can be found at www.clinicaltrials.gov.
Compassionate Use for Cell Therapy
Our Expanded Access Program for cell therapy varies regionally as a result of the unique regulatory mechanisms in different countries worldwide. Since investigational medicinal products have not yet received regulatory approval, their potential risks and benefits are not yet established. It is important for physicians and patients to consider all possible benefits and risks when seeking access to an investigational medicinal product.
Treating physicians may request information about Expanded Access for cell therapy by contacting 1-844-ORL-LABS.
Orpha Labs AG will evaluate these requests individually. We may provide physician requested Expanded Access for cell therapy for patients with no other available therapies and for patients who are not eligible to participate in a current Orpha Labs AG clinical trial. We will acknowledge receipt of the request via telephone call or email within 5 business days of receipt of the request.
Information on all Orpha Labs AG trials can be found on clinicaltrials.gov.
Pursuant to the 21st Century Cures Act: the posting of policies by manufacturers and distributors shall not serve as a guarantee of access to any specific investigational therapy by any individual patient.
Some of the content on this page is not intended for users outside the US.