Lecture 5: Regulation of Pharmacoepidemiology Research Flashcards
What was the significance of the FDA amendment act (FDAAA) of 2007?
1) Increased authority to require postmarketing studies and/or clinical trials
2) authority to require changes to drug labeling
3) Mandate to develop guidance on best practices for epi studies
4) Mandate to develop a new post-market risk identification and analysis system
Types of observational epidemiological studies that FDA may require
1) Assess a serious risk associated with a drug exposure or to quantify risk or evaluate factors that affect the risk of serious toxicity (eg, drug dose, timing of exposure, or patient characteristics)
2) examine natural history of a disease or to estimate background rates for adverse events in a population not treated wth the drug
FDA postmarking study requirements
1) Develop study protocol (analytic study or descriptive study)
2) Must submit periodic reports on status, including any challenges to meeting study timelines
3) Non-compliance may result in financial penalties and/or drug distribution limitations
Define Comparative effectiveness research
- Research that provides evidence on the effectiveness, benefits and harms of different treatment options.
- Evidence is generated from studies that compare drugs, medical devices, tests, surgeries or ways to deliver health care
- Designed to inform health care decisions such as reimbursement.
US Pubic disclosure requirements
There is currently no US legal requirement to disclose non interventional studies
EU definition of non-interventional study
A study where: the medicinal products are prescribed in the usual manner in accordance with the terms of the marketing authorization
Note that the assignment of a patient to a particular therapeutic strategy is not decided in advance by a trial protocol, but falls within the current practice
EU definition of low interventional clinical trial
A study where: the investigational medicinal product is authorized. Assignment to a particular therapeutic strategy is decided in advance by a trial protocol and taken together with the decision to include the patient (ex. LST)
Post Authorization safety studies (PASS)
Imposed as a condition of or following marketing authorization
Includes non-interventional (observational) and interventional studies (involves randomization)
Post Authorization efficacy studies (PAES)
- Complements the data available at the time of authorization with additional information concerning the efficacy of a medicinal product.
- addresses concerns that could not be resolved prior to market authorization
- European commission empowered to require PAES
ENCePP Guidance
European Network of centers for pharmacoepi and pharmacovigilance: a collaborated scientific network coordinated by the EMA
ISPE guidance
International Society for Pharmacoepidemiology provides guidelines for pharmacoepi practices
