Factors associated with positive and negative recommendations for cancer and non-cancer drugs for rare diseases in Canada

Fernanda Naomi Inagaki Nagase,Devidas Menon,Tania Stafinski,Gian Jhangri,Jian Sun

doi:10.1186/s13023-019-1104-7

Fernanda Naomi Inagaki Nagase, Devidas Menon + Show 3 more

Open Access

https://doi.org/10.1186/s13023-019-1104-7

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Abstract

BackgroundIn Canada, reimbursement recommendations on drugs for common and rare diseases are overseen by the Canadian Agency for Drugs and Technologies in Health (CADTH) and made through the pan-Canadian Oncology Drug Review (pCODR) and the Common Drug Review (CDR). While the agency specifies information requirements for the review of drug submissions, how that information is used by each process to formulate final reimbursement recommendations, particularly on drugs for rare diseases (DRDs) in which per patient treatment costs are often high, is unclear. The purpose of this study was to determine which factors contribute to recommendation type for DRDs.MethodsInformation was extracted from CDR and pCODR recommendations on drugs for diseases with a prevalence < 1 in 2000 from January 2012 to April 2018. Data were tabulated and multiple logistic regression was applied to explore the association between recommendation type and the following factors: condition/review process (cancer vs non-cancer), year, prevalence, clinical effectiveness (improvements in surrogate, clinical and patient reported outcomes), safety, quality of evidence (availability of comparative data, consistency between population in trial and indication, and bias), clinical need, treatment cost, and incremental cost-effective ratio (ICER). Two-way interactions were also explored.ResultsA total of 103 recommendations were included. Eleven were resubmissions, all of which received a positive recommendation. Among new submissions (n = 92), DRDs that were safe or offered improvements in clinical or patient reported outcomes were more likely to receive positive reimbursement recommendations. No associations between recommendation type and daily treatment cost, cost-effectiveness, or condition (cancer or non-cancer) were found.ConclusionsClinical effectiveness, as opposed to economic considerations or whether the drug is indicated for cancer or non-cancer, determine the type of reimbursement recommendation.

Highlights

In Canada, reimbursement recommendations on drugs for common and rare diseases are overseen by the Canadian Agency for Drugs and Technologies in Health (CADTH) and made through the pan-Canadian Oncology Drug Review and the Common Drug Review (CDR)
This study aimed to address the following questions: 1. Are certain factors associated with positive or negative reimbursement recommendations for drugs submitted to CDR and pan-Canadian Oncology Drug Review (pCODR)?
After 2011, the interim Joint Oncology Drug Review (iJODR) was formalized to a permanent body known as pCODR managed by CADTH and the first recommendation was published in 2012

Summary

Introduction

In Canada, reimbursement recommendations on drugs for common and rare diseases are overseen by the Canadian Agency for Drugs and Technologies in Health (CADTH) and made through the pan-Canadian Oncology Drug Review (pCODR) and the Common Drug Review (CDR). In addition to high per patient treatment costs, DRDs typically come with a paucity of outcomes data due in part to the small number of patients available for studies. This creates significant uncertainty around their value proposition. In 2011, a parallel process, the pan-Canadian Oncology Drug Review (pCODR), was established for new cancer drugs Both of these centralized processes are overseen by the Canadian Agency for Drugs and Technologies in Health (CADTH) [3, 4]. CADTH broadly specifies factors considered when formulating recommendations (clinical benefit, cost, cost-effectiveness, and patient preferences), how they are weighed relative to one another and whether it varies under certain conditions remain unclear [5]

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