Executive Summary
The Standing Senate Committee on Social Affairs, Science and Technology’s report entitled “Prescription Pharmaceuticals in Canada: Off-label Use” is the third report in a four-phase study on prescription drugs that began in March 2012. As described in the previous reports for the first two phases of this study, drug approval by Health Canada is required before a drug can be marketed and is based on the information provided by the manufacturer and on the specifics that the manufacturer requests, such as population group, condition, dosage etc. Frequently, approved drugs are used in a broader population, for different indications or at different dosages than have been approved by Health Canada. This is referred to as off-label use and it is widespread. Off-label drug use is associated with both advantages and disadvantages. Off-label use is most prevalent in prescriptions for the elderly, children, and pregnant women – these groups are rarely included in clinical trials. Off-label use is often the only source of drugs in rare diseases and in treating cancers.
This report makes 18 recommendations that address several issues including: data collection; research and information sharing; vulnerable sub-groups of the population; assessments of off-label use; electronic formats; and, the Orphan Drug Framework.
In terms of data collection, the committee heard that the extent of off-label prescribing is not known. In fact, several witnesses emphasized that frequently physicians are unaware that they are prescribing off-label. Consequently, little is known about the true extent of off-label use and the committee would like to see this identified and quantified. Further it would like Health Canada to require that off-label use be identifiable within the department’s adverse drug reaction form.
While the committee acknowledges the clear benefits of some off-label prescription drug use, it cautions that in other instances a lack of evidence of effectiveness can mean that patients are consuming drugs that either are not helping or that can produce harm. The committee recommends the Drug Safety and Effectiveness Network at the Canadian Institutes for Health Research take an active role in assessing the effectiveness of off-label drug uses. Further it recommends an expanded role for the Canadian Agency for Drugs and Technologies in Health to conduct and communicate findings wherever possible on the safety and effectiveness of off-label use.
This report also emphasizes the need to implement recommendations made in this committee’s previous two reports on prescription pharmaceuticals. With respect to vulnerable sub-groups of the population including children, the elderly and pregnant and nursing women, the report reiterates recommendations previously made in Canada’s Clinical Trial Infrastructure: A Prescription for Improved Access to New Medicines and Prescription Pharmaceuticals in Canada: Post-Approval Monitoring of Safety and Effectiveness regarding the need for more drug research in order to evaluate safety and effectiveness in these groups. The report further reiterates recommendations made in the post-approval monitoring report about: the need to implement electronic medical records, electronic health records and the electronic system of dispensed prescription drugs; improved access for patients to the adverse drug reaction form; and the need for additional legislative authorities.
Other recommendations in this report relate to: expanding Health Canada’s recently announced Orphan Drug Framework to include older drugs; providing both negative and positive Summary Basis of Decision documents, including those for new indications for older drugs; and addressing enforcement of the prohibition on off-label drug promotion by drug manufacturers.