کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
5553811 1403016 2017 7 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
New Drugs for Rare Diseases in Children
ترجمه فارسی عنوان
داروهای جدید برای بیماری های نادر در کودکان
موضوعات مرتبط
علوم پزشکی و سلامت پزشکی و دندانپزشکی پزشکی و دندانپزشکی (عمومی)
چکیده انگلیسی

PurposeUnited States (US) Pediatric Legislation (PL) was introduced in 1997 to improve children's health. The European Union PL (EUPL) has been in force since 2007. Both PLs facilitate additional pediatric research on primarily adult drugs. The EUPL declares that the forces of the market are not sufficient for children. Without a pediatric investigation plan, new drugs can no longer be registered with the European Union. New ways on how to facilitate drug development for rare pediatric diseases are being proposed.MethodsUS PL, EUPL, and implications of modern labels for medical decision making are discussed.FindingsModern drug labels constituted a step from eminence-based towards data-based medical decision making. However, approval by regulatory authorities did not replace knowledge transfer in medicine, which continued in university education, through conferences, consensus papers, and so on. Children were successfully treated with off-label drugs in pediatric oncology and in many other diseases. Describing children as “therapeutic orphans” reflected an overestimation of drug labels and an underestimation of nonregulatory systematic clinical testing. Therapeutic breakthroughs have occurred, for example, in acute myelogenous leukemia and cystic fibrosis. Rare diseases need new innovative drugs and therapeutic concepts for further breakthroughs.ImplicationsThe focus of PL on additional pediatric measures for predominantly adult new drugs reflects a tunnel view. Similar to the introduction of modern pharmaceutical legislation that triggered comparable laws in most countries worldwide after 1962, we currently need new worldwide steps to reward innovative treatment concepts for rare diseases-not against, but through the market. Created by philanthropy, parents, and other supporters, new therapeutic concepts should be rewarded upon meeting regulatory milestones. This market is limited today. It needs not only a boost by pioneers, but also acceptance, welcome, and re-thinking about drug development in academia, politics, and by the general public.

ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Clinical Therapeutics - Volume 39, Issue 2, February 2017, Pages 246-252
نویسندگان
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