Peer-Reviewed Studies in the PMPRB´s Price Review Process

In September, the leading medical journals represented on the International Committee of Medical Journal Editors announced tougher ethical standards regarding the publication of peer-reviewed studies.

This development provides a good opportunity to remind patentees and others of the standards of evidence relied on by the PMPRB in reviewing the prices of new patented medicines.

As set out in the Compendium of Guidelines, Policies and Procedures, Board Staff and the Human Drug Advisory Panel (HDAP) rely on information provided by the patentee, publicly available scientific literature, and their own expertise in making recommendations for the categorization of new drug products and selection of comparable medicines, dosage forms and dosage regimens. The HDAP reviews and evaluates scientific information available, including submissions by patentees, and advice from other experts when deemed necessary. Its recommendations are significant in determining the appropriate price test to be applied for purposes of the Excessive Price Guidelines.

The Scientific Review Procedures (chapter 3 of the Compendium) request patentees to provide Board Staff with the product monograph for the new drug and references supporting any submissions they choose to make regarding the categorization of the new drug, its primary use, comparable drugs and comparable dosage regimens. In addition, where the patentee is proposing that a drug be reviewed as a category 2 drug, i.e., breakthrough or substantial improvement, it should submit, among other things, up to five references (if available), including:

• a minimum of two well-controlled doubleblind statistically sound clinical trials which compare the new drug product to standard medicines whose value in the treatment of the disease is well recognized; generally, trials should be published in recognized peer-reviewed journals; and
  
  
• published reviews in recognized journals of the performance of the drug product or of the class of drug.

More details and examples are provided in sections 6 and 7 of the Scientific Review Procedures.

The Board considers that these provisions guide the HDAP to put the greatest weight on original reports in recognized journals, such as the journals represented on the International Committee; where the complete trial is not available, the HDAP and the Board have to rely on the highest level of evidence that is available and determine the appropriate weight to put on it.

Under the Board´s Transparency Initiative reported elsewhere in this NEWSletter, the PMPRB will soon begin publishing reports on the review of new patented medicines for purposes of applying the Guidelines. These reports will ordinarily include the HDAP´s assessment and references to the clinical studies and other evidence on which the HDAP relied to make recommendations.

See, for example, “Sponsorship, Authorship, and Accountability,” New England Journal of Medicine, 2001; Vol. 345, No. 11, pp. 825-827. www.nejm.org.

The Compendium is available on the website under Legislation, Regulations, Guidelines.

The PMPRB expects patentees to submit the best scientific evidence available, such as studies that meet the “Uniform Requirements” of the International Committee of Medical Journal Editors. It also recognizes that where such evidence is limited or is not available, the PMPRB must rely on the best information available and will strive to ensure its reports reflect such limitations.