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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. ==European regulations== 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.〔(Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use )〕 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〔(Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency )〕 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. Despite these differences, the reporting of adverse events is expected in all EU member states (set forth in Article 83). The marketing authorization holder or manufacturer must report serious adverse reactions (SARs) occurring within the EU to the competent authority of the member state in which the incident occurred. 抄文引用元・出典: フリー百科事典『 ウィキペディア（Wikipedia）』 ■ウィキペディアで「Named patient programs」の詳細全文を読む スポンサード リンク
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