Ординатура / Хирургия / Библиотека им академика М.И. Перельмана / Книга_5389_Библиотеки_им_академика_М_И_Перельмана

This chapter aims to establish understanding of the drug regulatory activities that take place to ensure that a drug’s benets outweigh its risks and appreciate how drug regulatory sys­tems support safe and effective use of drugs in clinical practice. First, it discusses the series of events that led to the current drug regulatory systems. Specically, the emergence of con­temporary drug regulation and pharmacovigi­lance in response to drug safety crises and European and global drug regulatory harmoni­sation are addressed. Second, it discusses cur­rent principles of pharmacovigilance and drug regulation. Important aspects comprise phar­macovigilance as a drug lifecycle activity, risk management planning, routine pharmacovigi­lance activities such as periodic safety updates and additional pharmacovigilance activities such as post- authorisation safety stud­ies(PASS), as well as post-authorisation ef­cacy studies (PAES) and the concept of expedited regulatory pathways. Third, it dis­cusses advanced pharmacoepidemiological approaches in drug regulatory decision- making, including contemporary approaches to signal detection, the use of multi-database observa­tional studies, the use of external control groups to contextualise, for example, single-arm stud­ies, and the target trial emulation approach to facilitate causal inference from observational studies. Although some sections of this chapter focus mostly on the European drug regulatory system, connections to important global activi­ties and developments are made to put the con­tents in a broad perspective.

15]. The Council’s tests comprised quality,

1925 set standards for (testing of) quality, purity, and potency and required a licence to manufacture drugs “of which the purity or potency cannot be adequately tested by chemical means” [20, 24, 25]. Furthermore, Norway (from 1928) and Sweden (from 1934) required that drugs were “medically justied” prior to marketing [19, 26, 27]. France followed in 1946 by installing a committee that evaluated the thera­peutic aim and safety of each drug before authorisa­tion for marketing was granted by the Minister of Public Health [28]. Interestingly, the Norwegian regulatory agency only authorised drugs for which there was an unmet need, in order to restrict the number of authorised drugs [27].

However, it would require another major safety crisis before the contours of current regulatory systems were established in most countries. In 1956, thalidomide was rst marketed in the

17, 18, 29]. Importantly, physicians that shared

In the late 1960s and early 1970s, the World Health Organization (WHO) contributed substan­tially to the formation of and collaboration between national and international systems for the spontane­ous reporting of adverse events, through its Programme for International Drug Monitoring [40]. The thalidomide tragedy was an important driver for this programme since the lack of a worldwide system to share adverse event information had pre­vented the early recognition of thalidomide-induced phocomelia [41]. Over the years, the programme has expanded extensively, from ten member coun­tries at its inception to over 175 full and associate members in 2023 [42]. Importantly, around the inception of the programme, the use of the term “pharmacovigilance” seems to have started [43].