Common drug review recommendations for orphan drugs in Canada: basis of recommendations and comparison with similar reviews in Quebec, Australia, Scotland and New Zealand

John I Mccormick,Nabil Tadros,L Diana Berescu

doi:10.1186/s13023-018-0759-9

Abstract

BackgroundPublic payer reimbursement for non-oncology drugs in Canada, including orphan drugs, is based on recommendations by the Common Drug Review (CDR) (with the exception of Quebec). CDR has been criticized for negative recommendations for orphan drugs and contributing to delays in patient access to these drugs. However, it is unclear how CDR makes recommendations for orphan drugs and the role clinical and economic factors play in decision making. The objective of the present study was to analyze the basis for CDR orphan drug recommendations and to compare recommendations to those in other jurisdictions.MethodsA list of orphan drugs reviewed by CDR (between 2004 and 2017) was compiled and final recommendations (list/do not list) assessed. The basis of each recommendation was categorized as clinical only, price only or combined clinical and price factors, based on the ranking of clinical and price parameters in recommendation summaries. The reimbursement status of the same drugs was determined in Quebec and other jurisdictions and level of agreement with CDR decisions assessed using a kappa analysis.ResultsSixty eight orphan drug submissions were identified in the CDR database. Clinical, clinical and price and price parameters were the basis of 48.5%, 44.1% and 7.4% of the reviews, respectively, and corresponding positive recommendation rates were 45.5%, 86.7% and 40.0% (p = 0.0008); overall positive recommendation rate was 63.2%. Positive recommendation rate increased from 50.0% for drugs reviewed between 2004 and 2009 to 86.7% in 2016; however, 84.6% of the latter were conditional on a price reduction. Of the drugs reviewed by CDR, 80.9%, 88.2%, 80.9% and 58.8% were reviewed for the same indications by health technology assessment agencies in Quebec, Scotland, Australia and New Zealand, respectively, with positive listing rates ranging from 60.0% (Quebec) to 92.7% (Australia) with fair (kappa coefficient 0.3307) to poor (kappa coefficient 0.0611) agreement with CDR in listing decisions, respectively.ConclusionsThe positive CDR recommendation rate for orphan drugs was highest when clinical and price parameters supported the assessment. Over time there has been an increase in CDR positive recommendation rates for orphan drugs, although most are conditional on a price reduction. It is unclear if this change in CDR recommendations will impact equitable and timely access to orphan drugs across Canada.

Highlights

Public payer reimbursement for non-oncology drugs in Canada, including orphan drugs, is based on recommendations by the Common Drug Review (CDR)
In the current study we have reviewed CDR recommendations for orphan drugs, defined the parameters involved in decision making, and compared recommendations with those made in Scotland, Australia and New Zealand
Data collection: CDR recommendations Publicly available CDR recommendations were reviewed for each identified orphan drug and the following information was extracted from the Recommendation and Reasons file [29]: date of CDR recommendation, generic and brand name of drug, drug indication, type of recommendation, basis of decision for recommendation, incremental cost-utility ratio (ICUR) and whether or not recommendation was based on a request for reconsideration [29]

Summary

Introduction

Public payer reimbursement for non-oncology drugs in Canada, including orphan drugs, is based on recommendations by the Common Drug Review (CDR) (with the exception of Quebec). The limited number of available patients means small clinical trial populations making it difficult to obtain valid comparative efficacy data [4, 5]. This creates issues for reimbursement authorities who may have to make decisions under conditions of uncertainty, at the time of market authorization. For pharmaceutical manufacturers the small number of patients makes it difficult to recover drug development costs, unless the drugs are premium priced This high cost in turn creates further issues for payers where application of traditional measures of efficacy and cost defining acceptable criteria for reimbursement may disqualify drugs for orphan diseases [4,5,6]. Health Canada is working on an overall regulatory review of drugs and devices, including drugs for rare diseases, few details of the impending changes to regulatory policies have been released [16]

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