Health technology assessment of drugs for rare diseases: insights, trends, and reasons for negative recommendations from the CADTH common drug review.

Ghayath Janoudi,Trevor Richter,Chander Sehgal,Brendan Mcintosh,William Amegatse

doi:10.1186/s13023-016-0539-3

Ghayath Janoudi, Trevor Richter + Show 3 more

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https://doi.org/10.1186/s13023-016-0539-3

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Abstract

BackgroundA shift in biochemical research towards drugs for rare diseases has created new challenges for the pharmaceutical industry, government regulators, health technology assessment agencies, and public and private payers. In this article, we aim to comprehensively review, characterize, identify possible trends, and explore reasons for negative reimbursement recommendations in submissions made to the Common Drug Review (CDR) for drugs for rare diseases (DRD) at the Canadian Agency for Drugs and Technologies in Health (CADTH), a publicly funded pan-Canadian health technology assessment agency. A public database (cadth.ca) was screened to identify DRD submissions to CDR. A diseases prevalence of ≤50 per 100,000 people was considered a rare disease. We calculated descriptive statistics for prevalence, study design, study size, treatment cost, reimbursement recommendation types, and reasons for negative reimbursement recommendations.ResultsFrom 2004 to 2015, 63 of 434 submissions to the CDR were for DRD (range: 1 submission in 2005 to 10 submissions in 2013). Most (74.6%) submissions included at least one double-blind randomized controlled trial (RCT). The average study size was 190 patients (range: 20 to 742). The average annual treatment cost was C$215,631 (range: $9,706 to $940,084). Reimbursement recommendations were positive for 54% of the submissions. Negative reimbursement recommendations were made due to a lack of clinical effectiveness (38.5%), insufficient evidence (30.8%), multiple reasons (23.1%), or lack of cost effectiveness/high cost (7.7%).ConclusionThe number of DRD submissions to CDR increased since 2013; from 4 to 5 per year between 2004 and 2012, to 10, 9, and 8 in 2013, 2014, and 2015 respectively. More than half of DRD submissions received positive reimbursement recommendation. Poor quality evidence and/or lack of supportive clinical evidence was at least partly responsible for a negative reimbursement recommendation in all cases. Although the average cost of DRD treatments was high, high cost was a reason for a negative reimbursement recommendation in only two (7.7%) of negative reimbursement recommendations.

Highlights

A shift in biochemical research towards drugs for rare diseases has created new challenges for the pharmaceutical industry, government regulators, health technology assessment agencies, and public and private payers
One reviewer (G.J.) extracted the following data from the published Common Drug Review (CDR) reports: CADTH project ID, project status, date of submission, type of submission, disease under review, prevalence of disease under review, Health Canada indication, manufacturer name, drug brand name, drug non-proprietary name, number of studies considered within the CDR review, type of studies considered in each submission, comparisons used in the studies, size of the largest study, highest evidence level, per patient annual treatment cost, cost per quality-adjusted life year (QALY), reimbursement recommendation, and reason(s) for a negative reimbursement recommendation
Assessment of the number of drugs for rare diseases (DRD) submissions made to the CADTH CDR revealed two distinct, contrasting periods: the first period was between 2004 and 2012, within which the number of submissions was generally between 4 and 5 per year, and ranged from a minimum of 1 submission in 2005 to a maximum of 5 submissions in each of the years 2004, 2009, 2010, and 2012; the second period was between 2013 and 2015, which was distinct from the preceding period in that the number of submissions increased, up to 10, 9, and 8 submissions for the years 2013, 2014, and 2015, respectively

Summary

Introduction

A shift in biochemical research towards drugs for rare diseases has created new challenges for the pharmaceutical industry, government regulators, health technology assessment agencies, and public and private payers. We aim to comprehensively review, characterize, identify possible trends, and explore reasons for negative reimbursement recommendations in submissions made to the Common Drug Review (CDR) for drugs for rare diseases (DRD) at the Canadian Agency for Drugs and Technologies in Health (CADTH), a publicly funded pan-Canadian health technology assessment agency. Janoudi et al Orphanet Journal of Rare Diseases (2016) 11:164 technology assessment (HTA) organization that evaluates drugs for reimbursement purposes through its Common Drug Review (CDR) and pan-Canadian Oncology Drug Review [pCODR] processes [4]. The Canadian Drug Expert Committee (CDEC), CADTH offers non-binding reimbursement recommendations to all public drug plans in Canada except for those in Quebec, which has a separate review process. CADTH has no formal definition that would make a distinction between DRDs and non-DRDs

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