ArticleOrphanet journal of rare diseases2017
Health technology assessment of new drugs for rare disorders in Canada: impact of disease prevalence and cost.
Article in Orphanet journal of rare diseases, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed, 26 citations in OpenAlex.
- RAPID framework for improved access to precision oncology for lethal disease: Results from a modified multi-round delphi study.Frontiers in health services · 2023Article
- Health technology assessment and price negotiation alignment for rare disorder drugs in Canada: Who benefits?Orphanet journal of rare diseases · 2022Article
- Associations between uncertainties identified by the European Medicines Agency and national decision making on reimbursement by HTA agencies.Clinical and translational science · 2021Article
- National Pharmacare in Canada: Equality or Equity, Accessibility or Affordability Comment on "Universal Pharmacare in Canada: A Prescription for Equity in Healthcare".International journal of health policy and management · 2020Article
- The work, goals, challenges, achievements, and recommendations of orphan medicinal product organizations in India: an interview-based study.Orphanet journal of rare diseases · 2019Article
- Factors associated with positive and negative recommendations for cancer and non-cancer drugs for rare diseases in Canada.Orphanet journal of rare diseases · 2019Article
- Access to new drugs for rare disorders in Canada.CMAJ : Canadian Medical Association journal = journal de l'Association medicale canadienne · 2018Article
- Characteristics of drugs for ultra-rare diseases versus drugs for other rare diseases in HTA submissions made to the CADTH CDR.Orphanet journal of rare diseases · 2018Review
- Common drug review recommendations for orphan drugs in Canada: basis of recommendations and comparison with similar reviews in Quebec, Australia, Scotland and New Zealand.Orphanet journal of rare diseases · 2018Article
- Do reimbursement recommendation processes used by government drug plans in Canada adhere to good governance principles?ClinicoEconomics and outcomes research : CEOR · 2017Article
- Access to drugs for rare diseases (DRD) in Canada: a comprehensive review of the provincial DRD-specific programs.Therapeutic advances in rare diseaseReview
- From care to cure: a patient engagement framework for rare disease and orphan drug research.Therapeutic advances in rare diseaseArticle
Corrections and comments
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Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundAuthors from the Canadian Agency for Drugs and Technologies in Health (CADTH) presented an analysis of submissions to the Common Drug Review (CDR) between 2004 and February 3, 2016 for drugs for rare disorders (disorders with a prevalence of <50 per 100,000).
objectiveThe aim of this analysis was to examine the same CDR submissions to evaluate whether the negative reimbursement recommendation rate, clinical evidence of efficacy and statements concerning the drug's cost in the CDR reports varied with the prevalence of the disorder treated by the drug grouped into three decreasing categories: <50 to >10, ≤10 to >1, and ≤1 per 100,000.
resultsAs the prevalence of the treated disorder decreased, the median daily cost of the drug, the negative recommendation rate and the proportion of submissions with statements in the CDR reports highlighting the cost of the drug increased, while the proportion of submissions with acceptable evidence of clinical efficacy decreased. Moreover, although the CADTH authors reported that only two submissions received a negative recommendation due to a "lack of cost-effectiveness/high cost," high cost was mentioned in the CDR reports of 15 drugs with negative recommendations, all for disorders with a prevalence of ≤10 per 100,000.
conclusionsThe aggregated analysis of CDR submissions for drugs for disorders with wide ranging prevalence rates concealed information of concern to patients. The negative reimbursement recommendation rate and the significance of cost in the CDR assessments increased as the prevalence of the treated disorder decreased. Since 2012, the manner in which high cost drugs for rare disorders have been dealt with by the CDR has changed. Cost has ceased to be a factor in negative recommendations but is included in criteria accompanying positive recommendations. This trend is associated with the integration of the CDR process with the system for price negotiation between public drug plans and pharmaceutical companies.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.