• Corpus ID: 41323464

An Orphan Drug Framework (ODF) for Canada.

@article{Lee2014AnOD,
  title={An Orphan Drug Framework (ODF) for Canada.},
  author={David K. Lee and Barbara Wong},
  journal={Journal of population therapeutics and clinical pharmacology = Journal de la therapeutique des populations et de la pharamcologie clinique},
  year={2014},
  volume={21 1},
  pages={
          e42-6
        }
}
  • David K. Lee, Barbara Wong
  • Published 2014
  • Medicine, Political Science
  • Journal of population therapeutics and clinical pharmacology = Journal de la therapeutique des populations et de la pharamcologie clinique
An orphan drug is a pharmaceutical or biological drug that is used to treat a rare disease. The development of a Canadian orphan drug framework is intended to help translate scientific discoveries into meaningful results for Canadian rare disease patients. 
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21 Citations
Background Citations
12
Results Citations
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21 Citations

Citation Type

Citation Type

We need a “made in Canada” orphan drug framework
TLDR
Canada is one of the few developed countries without a regulatory framework for orphan drugs, and medications used for the treatment of rare or orphan diseases are defined by their low prevalence and serious, life-limiting nature.
The study of drugs for rare disorders: harnessing research contributions by Canadian academic institutions.
  • S. Macleod, P. Lasko, A. MacKenzie
  • Medicine, Political Science
    Journal of population therapeutics and clinical pharmacology = Journal de la therapeutique des populations et de la pharamcologie clinique
  • 2014
The development of an orphan drug policy with the focus on essential research that may be undertaken in Canada's universities.
An international comparative analysis of public reimbursement of orphan drugs in Canadian provinces compared to European countries
Background The Canadian government has committed to developing a national strategy for drugs for rare diseases starting in 2022. Considering this announcement, we conducted a comparative analysis to…
Pharmaceutical expenditure on drugs for rare diseases in Canada: a historical (2007–13) and prospective (2014–18) MIDAS sales data analysis
TLDR
Overall spending on orphan drugs is lower relative to the number of patients affected with an orphan disease in Canada, and concerns that growth in orphan drug expenditure may lead to unsustainable drug expenditure do not appear to be justified.
Towards a New Way of Evaluating Orphan Drugs at CADTH
  • Medicine, Political Science
  • 2016
TLDR
Although individual rare diseases have a low prevalence, collectively they have a large societal, clinical, and economic burden.
Alignment of health technology assessments and price negotiations for new drugs for rare disorders in Canada: Does it lead to improved patient access?
  • N. Rawson
  • Medicine, Political Science
    Journal of population therapeutics and clinical pharmacology = Journal de la therapeutique des populations et de la pharmacologie clinique
  • 2020
TLDR
The CADTH-pCPA alignment is working for the governments who own and fund public drug programs but has yet to lead to coverage for all appropriate patients in all provinces.
Biologic medicine inclusion in 138 national essential medicines lists
TLDR
It is hypothesized that biologic disease-modifying antirheumatic drugs (DMARDs) are underrepresented relative to conventional DMARDs in existing national EMLs, and they are excluded from most national E MLs.
Common drug review recommendations for orphan drugs in Canada: basis of recommendations and comparison with similar reviews in Quebec, Australia, Scotland and New Zealand
TLDR
The positive CDR recommendation rate for orphan drugs was highest when clinical and price parameters supported the assessment, and it is unclear if this change in CDR recommendations will impact equitable and timely access to orphan drugs across Canada.
Health technology assessment of new drugs for rare disorders in Canada: impact of disease prevalence and cost
  • N. Rawson
  • Medicine, Political Science
    Orphanet Journal of Rare Diseases
  • 2017
TLDR
The aggregated analysis of CDR submissions for drugs for disorders with wide ranging prevalence rates concealed information of concern to patients because the negative reimbursement recommendation rate and the significance of cost in the CDR assessments increased as the prevalence of the treated disorder decreased.
Factors associated with positive and negative recommendations for cancer and non-cancer drugs for rare diseases in Canada
TLDR
Clinical effectiveness, as opposed to economic considerations or whether the drug is indicated for cancer or non-cancer, determine the type of reimbursement recommendation.
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