University of Paris V, Creativ-Ceutical, 215 rue du Faubourg St Honoré, 75008 Paris, France.
Expert Rev Pharmacoecon Outcomes Res. 2012 Feb;12(1):23-9. doi: 10.1586/erp.11.95.
Orphan drugs target small populations of patients. In order to make the field more attractive to pharmaceutical companies and encourage R&D in rare diseases, incentives were put forward by the EU, which are discussed in this article. Because they often are the only available option to treat a disease, some orphan drugs are considered to have high value and as such benefit from high prices on national markets. This has made orphan drugs an attractive market for pharmaceutical companies, with approximately 40 approved orphan drugs generating over $200 million each in yearly sales. The resulting burden this puts on national health insurances may lead to a change in regulation and will certainly lead to new national pricing and reimbursement strategies. They will need to be coherent, fair, effective and sustainable so as to be predictable for companies. Reflection on the subject needs to be initiated.
孤儿药针对的是患者人群较小的疾病。为了使制药公司对该领域更具吸引力并鼓励对罕见病进行研发，欧盟提出了激励措施，本文对此进行了讨论。由于孤儿药通常是治疗疾病的唯一选择，因此一些孤儿药被认为具有高价值，因此在国家市场上享有高价。这使得孤儿药成为制药公司的一个有吸引力的市场，大约有 40 种已批准的孤儿药每年的销售额超过 2 亿美元。这给国家健康保险带来的负担可能导致监管的改变，也肯定会导致新的国家定价和报销策略。这些策略需要具有连贯性、公平性、有效性和可持续性，以便对公司具有可预测性。有必要对此问题进行反思。
