Compassionate Use Program (CUP)

Often a Compassionate Use Program (CUP) is for a group of patients defined by a life-threatening or chronic serious condition requiring the use of an unlicensed medicine for patients without treatment options.

A CUP program is typically applied for by the pharmaceutical company to the respective country Health Authority (HA), at the time when no specific (named) patient is known, but there is anticipated need for such a program to serve a patient community. This is in contrast to a Named Patient, where the physician is the applicant and submits the application to the local health authority.

A CUP is applied for by the pharmaceutical company to the respective country HA at the time when no specific patient is known.

In contrast, in a Named Patient program the physician has direct personal responsibility for a patient under their care requiring the unlicensed medicine and submits the application to the local HA.

Planning for a CUP requires early strategic planning addressing multiple disciplines, including:

Internal pharma company alignment to support the program
Well defined criteria for patient eligibility often in a protocol or guidance document
The medical condition is life-threatening, seriously debilitating, or chronic
No licensed therapeutic treatment option available
The anticipated positive risk-benefit ratio for the treatment of a patient participating
Submission of program to country HA, and appropriate ethics body
A contract between the respective medical professional and the pharma company
Patients undergo informed consent procedure that meets the local requirements
Drug supply labeling meets the planned country regulatory requirements

In most cases the approval is issued by a national HA, and in some countries an Ethics Committee needs approval as well. In all cases, CUP programs require planning to ensure the required components for submission to HAs are satisfied. Once a CUP is approved by a specific country HA, the CUP is ‘active’ in that country and ready to receive requests from physicians.

End of Program

In CUPs, the criteria is that a properly licensed treatment alternative is not available. This is certainly not the case when the unlicensed medicine becomes available commercially in the local country. This usually marks the end of the program and is outlined in an exit strategy. However, a pharma company needs to consider when to stop the inclusion of new patients and when to stop treatment of existing patients.

In a clinical trial, the ‘end’ of the program is at the final visit of the last patient, focused on regulatory submission.

In a CUP, the ‘end’ of the program will vary by country, depending on your launch strategy and availability of the product in the respective country.

Early Access Care Can Help You With Your Strategic Needs For A CUP, From Planning Through to Local Market Exit

According to article 83 of Regulation (EC) no 726/2004, medicinal products without a Marketing Authorisation may be made available for compassionate reasons to a group of patients with a chronically or seriously debilitating disease or whose disease is considered to be lifethreatening and who can not be treated satisfactorily by an authorized medicinal product.

Compassionate use programs fall under national jurisdiction and in most Member States under the remit of National Competent Authorities (NCA). The individual NCA decides whether or not to approve the use of medicinal products without a market authorization.

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