Mallinckrodt Policy on Expanded Access Program for Investigational Drugs
The purpose of this policy is to describe an Expanded Access Program for the use of Mallinckrodt’s non-licensed drugs outside conventional clinical studies.
It is Mallinckrodt’s policy to be able to provide appropriate and ethical access to its investigational medicines to patients under certain defined circumstances prior to marketing authorization, but outside of the planned clinical research program. Such Expanded Access Programs, or EAPs (often called “Compassionate Use” programs), are for exceptional circumstances only.
Expanded Access Program
An EAP is a program of treatment with a Mallinckrodt unlicensed product alone or in conjunction with licensed products in a patient population identical to that expected to be treated with the product when approved. An EAP corresponds to an investigational treatment and is generally not a randomized clinical trial, but it requires notification to and/or approval from regulatory authorities and investigational review boards.
EAPs are 1) limited to patients with a serious, life-threatening or chronically debilitating condition 2) for patients for whom there is no approved therapeutic alternative; and 3) for access outside of a conventional clinical trial. This policy does not apply to the use of a commercially available Mallinckrodt product for an unapproved indication. All access programs must be conducted in agreement with applicable local, regional, and federal laws and regulations.
Mallinckrodt EAP Criteria
Mallinckrodt reviews all requests on a case-by-case basis against the following criteria:
- Sufficient evidence of efficacy that a clinically meaningful benefit may be expected;
- Sufficient evidence of safety indicating that patients would not be exposed to unreasonable risk if prescribed the product;
- The patients have a serious, life-threatening or chronically debilitating disease;
- There is no comparable or satisfactory alternative therapy to diagnose, monitor or treat the disease or condition; and
- The patients are ineligible for, or otherwise unable to participate in, a clinical trial for the product.
A physician or institution may apply for access to a Mallinckrodt investigational product in the pre-approval stage of development by contacting this email: CompassionateUse@mallinckrodt.com. The requestor may expect to receive acknowledgment of the request within two business days. Any physicians or institutions participating under an EAP must agree to comply with any reporting requirements, Good Clinical Practice guidelines, and other procedures as outlined by Mallinckrodt.