Pharmacoepidemiology, Drug Safety & Pharmaceutical Policy
Full course description
When a new medicine is granted a marketing authorization, its clinical safety profile has been assessed on the basis of results from randomised clinical trials (RCTs). The number of patients recruited for these pre-marketing (Phase-III) trials (in general up to 3,000), is able to detect adverse events that occur with frequencies of up to 1:1000 patient-years. Therefore, it is difficult to adequately assess the risk/benefit profile of a drug for regulatory authorities, such as the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA). The authorities will ultimately decide whether a drug can remain on the market, whether its use will be restricted to certain subgroups of patients or whether it will be entirely pulled off the market. This problem is further enhanced by exclusion criteria for patients enrolled in RCTs, and their short duration of follow-up (generally several months up to 2-3 years). The intake of other medications or inclusion of children, elderly or pregnant women- such as in a real life setting - is often not allowed in RCTs. As a result the EMA and FDA usually request pharmaceutical companies to conduct so called post-authorisation safety (PASS) studies. Similar studies are also conducted by other stakeholders such as academia or drug regulators such as the FDA.
This course will give an overview of the lifecycle of drug development, with a strong emphasis on pharmacoepidemiology in Phase IV research. It will evaluate stakeholders, legislation scientific methods and commonly used data sources to assess the risk-benefit profile of drugs after market authorisation.
Students will be able to understand:
- the latest developments of the regulatory process of drug development (Phase I-IV);
- common and novel pharmacoepidemiological methods for the conduct of post-authorisation safefty studies (PASS). These include meta-analysis, case-control studies, cohort studies, and case-only methods;
- commonly used datasources for the conduct of Phase IV research, inlcuding their strengths and limitations;
- risk/benefit assessments by regulatory agencies; pharmacovigilance procedures;
- the interactions between patients, prescribers, and payers (health insurance companies and governments).