Priority setting for orphan drugs: An international comparison

Rosenberg‐Yunger, Zahava R. S.; Daar, Abdallah S.; Thorsteinsdóttir, Halla; Martin, Douglas K.

doi:10.1016/j.healthpol.2010.09.008

Cited by 45 publications

(76 citation statements)

References 23 publications

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“…Difficulty arises because drug reimbursement decision-making includes competing equity and efficiency obligations. It results in different levels of funding and opposing interests of the stakeholders involved (14). The most apparent problem here before public health authorities is what reimbursement criteria to use and in which manner to implement them in realworld settings.…”

Section: Discussionmentioning

confidence: 99%

Criteria for Drug Reimbursement Decision-Making: An Emerging Public Health Challenge in Bulgaria

Искров

Стефанов

2016

Balkan Med J

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Background: During times of fiscal austerity, means of reimbursement decision-making are of particular interest for public health theory and practice. Introduction of advanced health technologies, growing health expenditures and increased public scrutiny over drug reimbursement decisions have pushed governments to consider mechanisms that promote the use of effective health technologies, while constraining costs. Aims: The study's aim was to explore the current rationale of the drug reimbursement decision-making framework in Bulgaria. Our pilot research focused on one particular component of this process -the criteria used -because of the critical role that criteria are known to have in setting budgets and priorities in the field of public health. The analysis pursued two objectives: to identify important criteria relevant to drug reimbursement decision-making and to unveil relationships between theory and practice. Study Design: Cross-sectional study. Methods: The study was realized through a closedended survey on reimbursement criteria among four major public health stakeholders -medical professionals, patients, health authorities, and industry. Empirical outcomes were then cross-compared with the theoretical framework, as defined by current Bulgarian public health legislation. Analysis outlined what is done and what needs to be done in the field of public health reimbursement decision-making. Results: Bulgarian public health stakeholders agreed on 15 criteria to form a tentative optimal framework for drug reimbursement decision-making. The most apparent gap between the empirically found preferences and the official legislation is the lack of consideration for the strength of evidence in reimbursement decisions. Conclusion: Bulgarian policy makers need to address specific gaps, such as formal consideration for strength of evidence, explicit role of efficiency criteria, and means to effectively empower patient and citizen involvement in public health decision-making. Drug reimbursement criteria have to be integrated into legitimate public health decision support tools that ensure the achievement of national public health objectives. These recommendations could be expanded to all Eastern European countries who share common public health problems.

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Section: Discussionmentioning

confidence: 99%

Criteria for Drug Reimbursement Decision-Making: An Emerging Public Health Challenge in Bulgaria

Искров

Стефанов

2016

Balkan Med J

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“…Reimbursement policies in various countries have been reported [19,22]. These were based on cost-effectiveness, rule of rescue, and equity, showing that an unfavorable recommendation was given in Canada based on insufficient evidence for costeffectiveness, although each province could formulate their own decision, thus affecting drug access depending on where in the country the patient lived.…”

Section: Treatment Of Fabry Diseasementioning

confidence: 99%

“…These were based on cost-effectiveness, rule of rescue, and equity, showing that an unfavorable recommendation was given in Canada based on insufficient evidence for costeffectiveness, although each province could formulate their own decision, thus affecting drug access depending on where in the country the patient lived. A favorable recommendation was made in Israel, France, Slovakia, and Hungary and in Australia funding was recommended through the Life Saving Drug Program for which there are no co-payments [19,22].…”

Section: Treatment Of Fabry Diseasementioning

confidence: 99%

Orphan drug policies and use in pediatric nephrology

Karpman

Höglund

2016

Pediatr Nephrol

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Orphan drugs designed to treat rare diseases are often overpriced per patient. Novel treatments are sometimes even more expensive for patients with ultra-rare diseases, in part due to the limited number of patients. Pharmaceutical companies that develop a patented life-saving drug are in a position to charge a very high price, which, at best, may enable these companies to further develop drugs for use in rare disease. However, is there a limit to how much a life-saving drug should cost annually per patient? Government interventions and regulations may opt to withhold a life-saving drug solely due to its high price and cost-effectiveness. Processes related to drug pricing, reimbursement, and thereby availability, vary between countries, thus having implications on patient care. These processes are discussed, with specific focus on three drugs used in pediatric nephrology: agalsidase beta (for Fabry disease), eculizumab (for atypical hemolytic uremic syndrome), and cysteamine bitartrate (for cystinosis). Access to and costs of orphan drugs have most profound implications for patients, but also for their physicians, hospitals, insurance policies, and society at large, particularly from financial and ethical standpoints.

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“…18,19 However, there is considerable debate about providing patients with access to ERTs, particularly within publicly funded drug programs. 20 An important characteristic of lysosomal storage disorders is a high degree of heterogeneity among patients with the same specific disease. This heterogeneity applies to age at symptom onset, clinical severity, speed of disease progression, and responsiveness to treatment.…”

Section: Outcomes: Defining Effectivenessmentioning

confidence: 99%