Policy alternatives for treatments for rare diseases

Panju, Abbas H.; Bell, Chaim M.

doi:10.1503/cmaj.081429

Cited by 24 publications

(26 citation statements)

References 29 publications

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“…Variations in access may also be related to media coverage of individual patients seeking reimbursement for a particular DRD in a specific jurisdiction. A national approach may address some of these inter-jurisdictional disparities, as well as other issues related to rare diseases, such as an inconsistency in the definition of a rare disease, and challenges related to Devidas Menon et al establishing patient registries with small numbers of patients in each jurisdiction (Panju and Bell 2010).…”

Section: Discussionmentioning

confidence: 99%

Reimbursement of Drugs for Rare Diseases through the Public Healthcare System in Canada: Where Are We Now?

Menon

Clark

Stafinski

2015

hcpol

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Introduction: Over the past 20 years, the number of therapies developed for rare diseases has rapidly increased. Often, these therapies represent the only active treatment for debilitating and/or life-threatening conditions. However, they create significant challenges for public and private payers. Because they target small patient populations, clinical evidence of efficacy/ effectiveness is typically limited, while the cost per patient is high. In Canada, each province/territory establishes its own mechanisms for determining which drugs for rare diseases (DRDs) to provide. Objectives: To compare current mechanisms across provinces and territories, and explore their impact on access. Methods: A systematic review of relevant published and unpublished documents was performed. Electronic bibliographic databases, the internet, and government websites were scanned using structured search strategies. Information was extracted independently by two researchers, and included aspects such as program type, condition/patient/therapy eligibility criteria, role of health technology assessment (HTA), decision options, ethical assumptions, and stakeholder input. It was validated through member-checking with provincial/territorial policy experts and tabulated to facilitate qualitative analyses. Impact on access was assessed through a cross-province/territory comparison of the coverage status of all non-cancer therapies reviewed by the Common Drug Review for indications affecting <1/2,000 Canadians using the Kappa statistic. Reasons for variations were explored using qualitative techniques. Results: Each province/territory has formal and informal mechanisms through which such therapies may be accessed. In most cases, formal mechanisms constitute the centralized HTA processes that also apply to common therapies. While several provinces have established dedicated processes/programs, whether they have affected access is not clear. Despite broadly comparable approaches, there is less than perfect agreement on publicly funded DRDs across jurisdictions. Conclusions: Individual jurisdictions have developed different approaches to providing access to these therapies. However, as the number increases, a more systematic approach to decisionmaking may be needed.

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Section: Discussionmentioning

confidence: 99%

Reimbursement of Drugs for Rare Diseases through the Public Healthcare System in Canada: Where Are We Now?

Menon

Clark

Stafinski

2015

hcpol

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“…Recently, authors have suggested an alternative economic evaluation to ensure the availability of public funding for orphan drugs. Panju and Bell advice a system based on the Rule of Rescue [46]. The suggestion of an alternative funding mechanism may address this tension, however further investigation is required to determine the exact implication of such a mechanism, particularly for different arrangement health systems.…”

Section: Discussionmentioning

confidence: 99%

Priority setting for orphan drugs: An international comparison

Rosenberg‐Yunger¹,

Daar²,

Thorsteinsdóttir³

et al. 2011

Health Policy

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“…As a result, investments in research and development (R&D) for rare diseases are borne by a small number of potential consumers, resulting in prohibitive prices for patients and healthcare systems, which resist supplying the drugs free of cost to the population. policymakers contend that the technologies supplied to patients with rare conditions fail to meet all the requirements for incorporation and reimbursement by health systems, that is 3,4,5,6 : (i) the evidence is insufficient on their safety and efficacy; (ii) the incremental cost-effectiveness ratios that signal efficiency in allocating scarce resources are generally higher than the thresholds commonly accepted by health systems; and (iii) there is a high opportunity cost to provide these technologies to patients with rare diseases, since the same budget funds could treat more patients with common diseases, or those that affect a large contingent of the population (high prevalence).…”

Section: Introductionmentioning

confidence: 99%