2014年10月21日发布

对于准备进入儿童用药市场的企业来说，政策释放的信号是产业界是否“投入”的晴雨表。

国家食品药品监督管理总局（CFDA）药品审评中心在网站发布了《儿科人群药代动力学技术指导原则》（征求意见稿）（下称《指导原则》），意见征集工作将于本月28日结束。

科学合理设定注册政策

原药品审评中心主任张象麟认为，儿童药研发技术上的特点和困难给注册政策提出了两个方面的考量：其一，已上市药品如何“转化”为儿童用药，包括大量临床数据的利用和判读，也涉及对改剂型、变规格、改包装的处置；其二，儿童药临床试验指导原则和审评要求科学合理的设定，在众多的期待下，获益和风险的权衡策略也是个不小的挑战。

随着业界对儿童用药的关注度持续升高，我国儿童用药所面临的问题令利益相关者开始思考如何从实践层面推动儿童药的研发、注册和市场的良性发展。

《指导原则》系统地对儿科人群药代动力学特点进行阐述，并以设计和方法学为重点，就如何安全、有效并符合医学伦理地在儿科人群中进行药代动力学的关键技术要点进行分析和说明，重点阐明了儿科人群的特殊关注之处。

药品审评中心化药临床一部杨焕指出，新药在开展儿科临床之前一般考虑5点：第一，了解儿科人群发育特点及与成人生理差异；第二，学习成人的临床数据；第三，通过成人的药代特征分析儿童可能的药代变化；第四，分析幼年动物数据；第五，确定最为合适的剂型。

中国医师协会儿科医师分会会长朱宗涵认为，如果《指导原则》能够顺利出台，将有助于全面认识儿科人群与药物间的相互作用，为临床制定合理用药方案提供依据。

儿童试验伦理学挑战

截至2013年9月，CFDA共发布36份公告，其中446家公示的医疗机构中含儿科专业的临床机构共51家（儿童专科医院及妇幼医院共计9家），儿科专业共134个。

首都医科大学附属北京儿童医院副院长申昆玲坦言，虽然近年来能够开展儿童药临床试验的医疗机构越来越多，但大部分机构专业数量较少，且没有统一的操作规范，很难满足多中心儿童药临床试验的基本要求，更远远不能满足约占患病总人口20%的患病儿童用药需求。

早前CFDA副局长尹力在接受《人民日报》专访时特别谈到我国儿童药研发和用药所面临的问题，认为儿童的生理状况决定了临床试验面临更多的伦理障碍，这些问题需要在政策上加以引导和鼓励。

“我国现行GCP要求，儿童作为药物的受试者，必须征得其法定监护人的知情同意并签署知情同意书，当儿童能做出决定时，还必须征得其本人的同意。”申昆玲指出，现阶段临床试验对儿童的伦理学要求越来越高，试验规范性要求趋严。

但实践证明，8岁以下儿童认知能力较差，家长引导因素多，基本不能作为本人意愿；8岁以上儿童稍有认知能力，但意向多变，即便当时同意，也不能算作长期稳定意愿，目前来看只有12岁以上的儿童能够较完整的理解参与一项药物临床的意涵。

-- New test will help increase access for earlyinfant HIV diagnosis

PLEASANTON, California, Feb. 18, 2014 /PRNewswire/-- Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that its dual-targetHIV-1 qualitative test, v2.0[1] has received CE Mark certifications, allowingit to be sold for clinical use in the European Union and those countriesaccepting CE Marked products. The test simultaneously amplifies anddetects two separate regions of the HIV-1 genome, which are not subject toselective drug pressure. This unique, dual target design allows for morereliable results to confidently and effectively diagnose HIV-1 infection.

The new dual-target HIV-1 qualitative test, v2.0works with both plasma and dried blood spot (DBS) collection cards tofacilitate PCR testing, and more importantly make sample collection andtransportation easy, even from the smallest infant in the most rural area.

The DBS collection card and the need for moresensitive and accurate information to diagnose babies early in their life andpatients across many African countries is instrumental to facilitate the stateof the art healthcare required for the region. The HIV-1 dual targetqualitative test and DBS cards eliminates need for refrigeration, drasticallyreduces the volume of blood to be stored or transported, and the stress for themother and the child is dramatically improved.

"The ability to determine the HIV status of aninfant as early as six weeks is crucial to determining the appropriate care andsupport for HIV-positive babies," stated Paul Brown, President of RocheMolecular Diagnostics. "With this new solution from Roche, we arereiterating our commitment to healthcare solutions that make a real differencefor patients."

About Early HIV Infant Diagnosis

Diagnosing HIV in infants and young children is amajor challenge. Antibody tests are ineffective in children because themother's antibodies remain in the child's system following birth. Infact, antibody tests in infants may yield false positive results for up to 15months. There are a number of alternate methods available for early diagnosisof HIV infected infants. Many approaches use molecular techniques, specificallydetecting the viral DNA and/or RNA, which enables HIV diagnosis as early as sixweeks.

Early diagnosis is critical for children infectedwith HIV. If found HIV positive, they can receive appropriate medical carebefore they develop significant illness, and can remain healthy despite theirinfection. If found HIV negative, the mother can get appropriate counseling toensure their child maintains a HIV-free status.

About the test

The COBAS? AmpliPrep/COBAS? TaqMan? HIV-1Qualitative Test, version 2.0 is an in vitro diagnostic, total nucleic acidamplification test for the qualitative detection of Human ImmunodeficiencyVirus Type 1 (HIV-1) DNA and RNA (or total nucleic acid, TNA) in human plasmaor dried blood spots using the COBAS? AmpliPrep Instrument for automatedspecimen processing and the COBAS? TaqMan? Analyzer or COBAS? TaqMan? 48Analyzer for automated amplification and detection.

The test is a diagnostic test, indicated forindividuals who are suspected to be actively infected with HIV-1. Detection ofHIV-1 TNA is indicative of active HIV infection. Infants born to mothersinfected with HIV-1 may have maternal antibodies to HIV-1, and the presence ofHIV-1 nucleic acid in the infant indicates active HIV-1 infection. In adults,the test may be used as an aid in the diagnosis of HIV-1 infection.

About HIV-1

Accordingly to the World Health Organization (WHO),there were 35 million people living with HIV around the world in 2012. Thatsame year 2.5 million people became newly infected with HIV worldwide.[2] For the most impacted region of Sub-Saharan Africa nearly 1 in every 20 adultsor 69% of all people living in this region are with HIV positive.

The transmission of HIV from an HIV-positive motherto her child during pregnancy, labor, delivery or breastfeeding is called verticalor mother-to-child transmission (MTCT). In the absence of any interventions HIVtransmission rates are between 15-45%.

Diagnosis, highly active antiretroviral treatment(HAART) and viral load tests, a test to determine the amount of circulatingHIV, have contributed to a steady increase in life expectancy for HIV infectedpeople of 13 years.