Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products

Dear, James W.; Lilitkarntakul, Pajaree; Webb, David J.

doi:10.1111/j.1365-2125.2006.02654.x

Cited by 79 publications

(94 citation statements)

References 16 publications

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“…But perhaps the most common objection to setting higher costeffectiveness thresholds for orphan drugs derive from the principle of maximizing the health gain achieved with society's limited health care resources (Schlander, 2008). The opportunity cost of such a policy is larger health losses among those who suffer from common, highly prevalent diseases (Dear et al, 2006;Hughes et al, 2005;McCabe et al, 2006). This debate has proceeded in a virtual vacuum of evidence regarding the views of the public regarding such a special status for drugs used to treat orphan diseases.…”

Section: Introductionmentioning

confidence: 99%

A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study

Mentzakis

Stefanowska

Hurley

2010

HEPL

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Policy debate about funding criteria for drugs used to treat rare, orphan diseases is gaining prominence. This study presents evidence from a discrete choice experiment using a convenience sample of university students to investigate individual preferences regarding public funding for drugs used to treat rare diseases and common diseases. This pilot study finds that: other things equal, the respondents do not prefer to have the government spend more for drugs used to treat rare diseases; that respondents are not willing to pay more per life year gained for a rare disease than a common disease; and that respondents weigh relevant attributes of the coverage decisions (e.g., costs, disease severity, treatment effectiveness) similarly for both rare and common diseases. The results confirm the importance of severity and treatment effectiveness in preferences for public funding. Though the first study of its kind, the results send a cautionary message regarding the special treatment of orphan drugs in coverage decision making.

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Section: Introductionmentioning

confidence: 99%

A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study

Mentzakis

Stefanowska

Hurley

2010

HEPL

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“…In the USA, these include exclusive licensing to market such drugs for 7 years, faster assessment procedures, and tax incentives. 1 In Europe, incentives include exclusive licensing for 10 years, reduction in the fees paid for regulatory activities, and provision of scientific advice by drug regulatory bodies. 1 5 Despite these incentives, the costs of orphan drugs are still high, especially when generic versions of the same agent are available.…”

Section: Introductionmentioning

confidence: 99%

The Effectiveness, Safety and Costs of orphan Drugs: An Evidence-Based Review

Onakpoya

Spencer

Thompson

et al. 2015

Clinical Therapeutics

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“…For the patients, the illness not only has a biological reality but, above all, it presents a psychological and social burden, which is integral part of the morbid course and from which can depend the degree of acceptance and adaptation to it [11]. Almost all rare illnesses are also chronic and invalidating and the patients must cohabit with the symptoms and the difficulties of the illness for the whole life, often since the birth [22]. Thus, the subjects with hereditary angioedema develop higher anxiety, depression and sensitivity to the stress.…”

Section: Discussionmentioning

confidence: 99%

Psychological Correlates in Subjects with Hereditary Angioedema (HAE)

Bella¹

2014

J Psychol Psychother

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Objectives: Hereditary Angioedema (HAE) is a rare serious medical condition caused by a deficiency of C1-inhibitor, due to mutations in its structural gene. The disease appears clinically as cutaneous swelling of the extremities, face, genitals, and trunk, painful swelling of the gastrointestinal mucosa and life threatening laryngeal edema. In this study we evaluated in HAE patients and in the relative controls the psychological status and gender differences to verify if there is a link between disease and mental status. Methods:We studied "psychological stress", using the Cognitive Behavioural Assessment 2.0 (CBA-2.0), in a total of 70 patients with confirmed HAE from different Italian Hospitals (41 women, 29 men; aged 17 to 78). Results:The analysis of the majority factors of CBA-2.0 tests indicates a clear difference between male and female patients; in fact, women perceive more intensively than men the different signs of the disease (have more consciousness of the consequences that this illness provokes in their life). Conclusion:Our data indicate that HAE is associated with emotional factors that can also complicate the clinical status of patients.

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Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products

Cited by 79 publications

References 16 publications

A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study

A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study

The Effectiveness, Safety and Costs of orphan Drugs: An Evidence-Based Review

Psychological Correlates in Subjects with Hereditary Angioedema (HAE)

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