科学监管与对患者和家庭的同情心
出自识林
科学监管与对患者和家庭的同情心
笔记 2014-11-03 Lachman CONSULTANTS 科学监管与对患者和家庭的同情心 — FDA关于治疗杜式肌营养不良症药物的声明 FDA承认杜式肌营养不良症有未满足的医疗需求，疾病对于患者及其家庭的毁灭性影响，及对新治疗方法的紧迫性需求。并表示将继续致力于与所有企业合作，加快开发和批准用于治疗该疾病的安全有效的药物。在尽其所能确保不泄漏企业Sarepta Therapeutics新药申报机密信息的前提下，FDA讲述了已与企业进行的讨论，包括FDA给予的对话和建议，以及提交NDA时所需要的附加信息。FDA的全文声明在2014年10月27日申请人的新闻发布会后发出。 FDA还表示，愿意对企业的申请实施滚动审评（rolling review, 即新药申请书中的内容，企业完成一部分，就可递交一部分，FDA就审评这一部分，而不必像普通新药申请那样等所有部分都完成后再递交及审评），并同意该产品可能会获得优先审评。前提是企业必须根据FDA已给予企业的咨询，提供所需的数据和分析。尽管我在40多年的职业生涯中还没有看到FDA做出这样的声明，我很高兴看到FDA不仅仅是一个监管机构，并且对饱受这一疾病折磨的看护人和患者具有同情心。回忆起早年艾滋病的蔓延和肿瘤团体的争论以及给FDA带来的重大冲击（FDA的企业收费制度及近年来新药审批速度的明显加快，都与这些冲击密切相关），看来FDA在交流和公共关系方面已经很好地吸取了教训。 Lachman CONSULTANTS - Bob Pollock先生 2014-10-31 识林www.shilinx.com版权所有，未经许可不得转载。如需使用请联系zhzhao@shilinx.com In a Fairly Unusual Move, FDA Makes Statement on Duchenne Muscular Dystrophy Drug FDA usually does not make comments on pending new drug applications (NDAs) nor do they usually publicly discuss conversations with sponsors. However, in this instance, FDA has made a rather interesting disclosure relative to a pending NDA for a serious and devastating disease, Duchenne Muscular Dystrophy (DMD). See FDA's full statement here. However, FDA says it “is addressing questions the agency has received from DMD patients, their families, and others in the community who are concerned about the timing of the filing of an NDA for eteplirsen.” FDA notes that they recognize “the unmet medical need in Duchenne muscular dystrophy (DMD), the devastating nature of the disease for patients and their families, and the urgency to make new treatments available. We remain committed to working with all companies to expedite the development and approval of safe and effective drugs to treat this disease.” FDA goes on to describe (as best they can to assure nondisclosure of confidential information) the discussions it has had with the sponsor, Sarepta Therapeutics . The FDA statement came on the heels of an October 27, 2014 press release from the sponsor. In FDA's statement, they go on to describe (in general) the conversations and advice that they have provided to Sarepta over the course of their discussions and outlined (again, in general terms) the additional information that FDA notes that would be needed for filing of the NDA. The Agency also indicates its willingness to conduct a rolling review of the firm's application and agrees that the product will likely receive a priority review. But the sponsor must provide the required data and analysis upon which the Agency has counseled the firm. While I have not seen the Agency make many of these statements over my 40 year career, I am happy to see that the Agency is more than just a regulatory body and has compassion for both the caregivers and patients stricken with this disease. Remembering the early years of the AIDS epidemic and the controversy with the cancer community, it appears that FDA has learned a good communication and public relations lesson. 相关指南 Expedited Programs for Serious Conditions––Drugs and Biologics |