ARCHIVED - Real World Drug Safety and Effectiveness

An important element of the National Pharmaceuticals Strategy (NPS) is the concept of stronger monitoring and evaluation of real world drug safety and effectiveness. The current system of drug approval in Canada emphasizes evaluation in the pre-market phase, where evidence is usually limited to clinical trials designed to demonstrate efficacy against placebo and to detect relatively frequent adverse events.

However, the safety and effectiveness of a drug post-market - in "real world" conditions - can be very different because the drug may be used in different populations, at different doses, for different indications and for longer periods of time. Ideally, performance of a drug should be assessed in real world settings using clinically meaningful outcomes and compared to other treatment options.

Closing the existing gaps in therapeutic effectiveness data and long-term safety information would have many implications. An improved evidence base could be used by Health Canada to better determine whether and how a drug is approved for the market and how similar new drugs will be evaluated in the future. Provincially, the information could be used to better determine if a drug should be - or continue to be - covered on public plans and under what conditions. It would allow researchers to more clearly identify patient groups who may have adverse outcomes, and help industry to better target drugs to the population in which they would be most safe and effective. Finally, improved pharmacosurveillance would support physicians and pharmacists in their prescribing and dispensing decisions, and provide patients with better information on the benefits and risks of a particular drug.

The rationale for strengthening the monitoring and evaluation of real world drug safety and effectiveness is compelling - determining the right model is complex. An integrated and comprehensive pharmacosurveillance system will require the collaboration of many stakeholders - regulators and policy makers, drug plan managers, health care providers, patients, the pharmaceutical industry, researchers, and private insurers - so that patients experience better health outcomes and fewer adverse events.

In support of this initiative, the Working Conference on Strengthening the Evaluation of Real World Drug Safety and Effectiveness took place in Ottawa, Ontario on September 13 to 15, 2005.

A second event, The Invitational Workshop on Priority Research Projects for a Drug Safety and Effectiveness Network was held in support of this initiative in Ottawa, Ontario on May 17 to 18, 2006.

In February 2007, a consultant's report, Medicines that Work for Canadians: Business Plan for a Drug Effectiveness and Safety Network, was completed under contract with Health Canada as part of the National Pharmaceuticals Strategy. The project was a collaboration among the National Pharmaceuticals Strategy, the Canadian Institutes of Health Research and the Canadian Drug Policy Development Coalition. The recommendations in this report will be considered in developing an integrated and comprehensive approach to support the evaluation of safety and effectiveness of a drug throughout its life cycle.

Related Reports

The following reports were commissioned under Health Canada's Health Policy Research Program and are related to drug safety and effectiveness.

The views expressed within the summaries do not necessarily represent the views of Health Canada

(2005) Evaluation of an Integrated Model and Iterative Loop for Assessment of Drug Effectiveness in the "Real World"

(2004) Effectiveness and Cost-Effectiveness of New Multiple Sclerosis Drugs in the 'Real World'

(2004) Development and Evaluation of a Framework for Incorporating Pharmacosurveillance in Provincial Formulary Decision Making

(2004) Production and Use of Evidence of Drug Effectiveness: Systematic Review, Evaluation and a Guidebook for Decision Makers

(2004) Evaluation of Data Sources to Support Pharmacosurveillance