A comparative study of European rare disease and orphan drug markets

Denis, A.; Mergaert, Lut; Fostier, Christel; Cleemput, Irina; Simoens, Steven

doi:10.1016/j.healthpol.2010.05.017

Cited by 99 publications

(95 citation statements)

References 3 publications

(3 reference statements)

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“…Regulations and policies regarding the use of a novel drug are typically imposed based on the above-mentioned ICERs, usually with a higher threshold for orphan drugs (increased willingness to reimburse) and a degree of leniency if the treatment is lifesaving [1,3]. Certain countries also have legislation regarding off-label use of orphan drugs as well as procedures for compassionate use [1]. A few countries disregard costeffectiveness ratios, to a certain extent, in favor of moral aspects, the so-called Brule of rescue^, whereby saving a human life is valued more than the price of the intervention [3].…”

Section: Regulations Imposed By Societymentioning

confidence: 99%

“…Studies have compared health policies regarding orphan drugs in various countries and on different continents [1,3,19]. These policies take into account incentives to promote the development of orphan drugs, market authorization, pricing and price regulation, budget impact, profit control, and reimbursement coverage, and show considerable variation between countries.…”

Section: Regulations Imposed By Societymentioning

confidence: 99%

“…Registries collect data from larger groups of patients, globally or nationally, and should ideally function independently of the pharmaceutical industry [1,2].…”

Section: Regulations Imposed By Societymentioning

confidence: 99%

“…There is an estimate of close to 7000 rare diseases altogether [1,2], and the cumulative prevalence is estimated to affect up to 6-8 % of the world's population [3]. Ultra-rare diseases have a prevalence of 1 in 50,000 individuals or less in Europe (EU regulation 536/2014).…”

Section: Introductionmentioning

confidence: 99%

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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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Section: Regulations Imposed By Societymentioning

confidence: 99%

Section: Regulations Imposed By Societymentioning

confidence: 99%

“…Registries collect data from larger groups of patients, globally or nationally, and should ideally function independently of the pharmaceutical industry [1,2].…”

Section: Regulations Imposed By Societymentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Orphan drug policies and use in pediatric nephrology

Karpman

Höglund

2016

Pediatr Nephrol

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“…человек [4]. В Соединенном Королевстве введены тер-мины «ультраорфанные лекарства» и «ультраредкие ВОПРОСЫ СОВРЕМЕННОЙ ПЕДИАТРИИ /2016/ ТОМ 15/ № 4 заболевания», к которым относят нозологии, поражаю-щие менее 1000 жителей по всей Великобритании [5]. Всего в мире под определение орфанных попадает около 7000 заболеваний [6].…”

Section: актуальностьunclassified

Current Problems in Provision of Orphan Drugs and Ways to Solve Them

Колбин¹,

Гапешин²,

Малышев³

2016

Vopr. sovr. pediatr.

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Unintended effects of orphan product designation for rare neurological diseases

Murphy

Puwanant

Griggs

2012

Annals of Neurology

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Since the introduction of the Orphan Drug Act in 1983, designed to promote development of treatments for rare diseases, at least 378 orphan drugs have been approved. Incentives include financial support, tax credits and, perhaps most importantly, extended market exclusivity. These incentives have encouraged industry interest and accelerated research on rare diseases, allowing patients with orphan diseases access to treatments. However, extended market exclusivity has been associated with unacceptably high drug costs; both for newly developed drugs and even for drugs which were previously widely available. We suggest that a paradoxical effect of orphan product exclusivity can be reduced patient access to existing drugs. In addition, the costs of each new drug are arguably unsustainable for patients and for the American health care system. Of all the specialties, neurology has the third highest number of orphan product designations, and neurological diseases account for at least one fifth of rare diseases. Citing the use of tetrabenazine for chorea in Huntington’s disease, adrenocorticotropic hormone for infantile spasms and enzyme replacement therapy with alglucosidase alpha for Pompe’s disease we highlight these paradoxical effects.

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A comparative study of European rare disease and orphan drug markets

Cited by 99 publications

References 3 publications

Orphan drug policies and use in pediatric nephrology

Orphan drug policies and use in pediatric nephrology

Current Problems in Provision of Orphan Drugs and Ways to Solve Them

Unintended effects of orphan product designation for rare neurological diseases

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