Named patient programs

Governments worldwide have created provisions for granting access to drugs prior to approval for patients who have exhausted all alternative treatment options and do not match clinical trial entry criteria. Often grouped under the labels of compassionate use, expanded access, or named patient supply, these programs are governed by rules which vary by country defining access criteria, data collection, promotion, and control of drug distribution.

Within the United States, pre-approval demand is generally met through treatment IND (investigational new drug) applications (INDs), or single-patient INDs. These mechanisms, which fall under the label of expanded access programs, provide access to drugs for groups of patients or individuals residing in the US.

Outside the US, named patient programs provide controlled, pre-approval access to drugs in response to requests by physicians on behalf of specific, or “named”, patients before those medicines are licensed in the patient’s home country.

Through these programs, patients are able to access drugs in late-stage clinical trials or approved in other countries for a genuine, unmet medical need, before those drugs have been licensed in the patient’s home country.

A 1989 European Union (EU) Council Directive laid out the framework for the supply of unregulated medicines in response to unsolicited requests for use by individual patients “on his personal responsibility”. The current legal basis for access to pre-launched medicines in the EU is Article 5 of Directive 2001/83/EC. This legislation offered the possibility of pre-launch use as an exception to the rule that medicines must be authorized before use or used within the context of an approved clinical trial.

With the expansion of the EU in 2004, Regulation 726/2004 evolved compassionate use programmes in order to better meet the expectations of patients.

While pre-launch use is permitted by EU legislation, these programs are governed by the individual member states; each of the 30 member states of the European Economic Area (EEA) has its own nationalized regulations regarding the import of pre-launch medicines. Additionally, even after a drug receives approval, authorities in each country can take several months to make reimbursement decisions, which can delay the ability of patients to access these drugs.

In a majority of EU member states, an authorization from a competent authority is required to initiate named patient supply; in other countries it may be retrospective. For example, the French regulatory agency (AFSSAPS) is primarily concerned with the science supporting a patient’s request for access to a pre-launch drug and has in place a proactive approval system. In contrast to the French system, in Ireland, the prime concern is transparency of supply and patient safety, so retrospective notification is required.

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Wikipedia