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The 1983 US Orphan Drug Act (ODA) provided incentives to stimulate treatment product development for patients with rare disease. This article highlights a decade of ODA contributions to this goal for children with RDs.An internal US Food and Drug Administration database was the information source for orphan designations, marketing approvals, and prevalence numbers for 2000 […]
…In Europe Rare Disease (RD) is the one which affects less than 1/2000, in the USA 1/1250, while in Japan RD is the one that affects fewer than 1/50,000 patients. EU estimates that 5–8000 distinct rare diseases affect 6–8% of the Population. The impact of rare diseases in the health systems is impressive: at least […]
…The rule of rescue describes the moral impulse to save identifiable lives in immediate danger at any expense. Think of the extremes taken to rescue a small child who has fallen down a well, a woman pinned beneath the rubble of an earthquake, or a submarine crew trapped on the ocean floor. No effort is […]
…Patients with rare diseases are awaiting an answer to their needs. Traditionally, however, research on rare diseases has been limited by the idea that it was too difficult to do and too little rewarding in terms of return of profit. This attitude has actually changed during the last decade, because it was realized that research […]
…The present paper is focusing on rare diseases manifesting in late childhood or adulthood. A part of these syndromes are not of genetic origin, such as relatively or absolutely rare infections, autoimmune diseases, tumours, or diseases due to rare environmental toxic agents. In addition, even a large proportion of genetic disorders may develop in adulthood […]
…The term rare disease is often used to justify why special study designs and, in particular, randomisation cannot be implemented and therefore cannot be requested. Definitions of rare diseases are not uniform, and absolute numbers of common definitions are large enough to perform clinical trials. Due to smaller patient numbers in common clinical trials patients […]
…The care of patients with rare diseases is associated with many challenges, in particular those associated with high coordination complexity. In an explorative case study in a health-care network for patients with respiratory insufficiency, we have investigated the types and causes of coordination problems.We conducted semi-structured, qualitative interviews with 3 patients and their health-care providers. […]
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