EXPANDED ACCESS PROGRAMS

In particular cases, a patient group in a certain country suffers from a serious or life-threatening illness who have no viable treatments options available to them, meaning that:

• There are no approved treatments available

• There are approved treatments available, and they have been ineffective after the patient has tried them

• There is no possibility for the patient to enroll in an active clinical trial

This unmet clinical need can be satisfied through an Expanded Access Program, which allows physicians and patients access to pre-approval, investigantional drugs outside of clinical trial setting. Managed Access Program, Early Acess Program and Compassionate Use Program are other names for an EAP.

The unmet clinical need should be justified by the physician treating the patient and the access to the drug can also be granted by the drug manufacturer (sponsor).

Generally, in an EAP, physician have following responsibilities:

• Decision for the use of an investigational drug or medical product

• Requesting access to the drugby the drug manufacturer and / or Tecrapharm

• Completing the necessary documentation for the regulatory body in the target country as well as contacting the local ethics committee to get approval for import and administration of the investigational product

• Data protection around the patients and their treatments

Drug manufacturers (sponsors) assess the request of the physicians and decide for granting access to the investigational drug in alignment of the regulatory regulations in the target country in compliance with the data protection about the patient. The benefits of an EAP for the drug manufacturer are mainly the additional insight for the safety and efficacy of the drug within the experience of the real life clinical use. If the drug manufacturer decide to grant access to the medicine at no cost, then the term Compassionate Use will be used. Setting up and maintaining an Expanded Access Program is a complex regulatory organization, which needs at least, if not more, know how as much as for a drug registered and marketed in the typical way in a target country.