首页 > 资讯 > GDUFA再授权提上日程，请听企业的声音

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2015-04-22 识林

FDA日前在联邦公报上发文，将于2015年6月15日在 White Oak 召开公开会，已在征求公众对GDUFA再授权的意见。为期五年的GDUFA项目将于2017财年结束时到期，现已进行过半。 联邦公报通告请见此处。

FDA意在征求各种对于GDUFA绩效目标函中现存内容的修改意见，行业已经积累了大量看法，当然也不全是积极的。比如目前为达成绩效目标，主要依赖于发出完全回应函而不是批准申请，这显然与 Hatch-Waxman 推动仿制药申请获批的精神是相背的。业界重申该法案旨在保障高质量的仿制药以可以接受的价格尽早上市，而这一点必须要在GDUFA第二轮方案中得以体现。面对质量参差不齐的各项申请，如何给出一个相对固定的审评时限，保质保量并兼顾进度，或许的确需再审慎权衡。比如在特定产品的申请中出现了此前从未涉及到的科学问题，必须在批准前进一步调查；或者虽然科学上没有问题，但是基于GMP视角发现了一些可能影响批准的合规问题。无论如何，业界还是希望尽早看到具体的目标，以及针对这些目标的讨论与考虑。

沟通问题是另一个关注的焦点。企业希望更好的跟进申请在审评与批准过程中的状态。或许在对GDUFA第二轮方案讨论中，有建议会提出FDA要进一步保障其IT平台的可靠性，缩短回应时间以及公开更多信息。FDA曾对此作出过回应，介绍了“审评中交流”以及“对暂缓批准的预先通告”等内容。可能企业暂时还不能很充分的使用这些工具，但势必会在这番讨论中，就此提出更多问题。

基于第一轮GDUFA前半程的执行经验，政府与企业双方都会进一步探讨下一个五年计划该如何推进。有人怀疑FDA能否在第一轮GDUFA剩下的时限内，证明自己可以兑现当初立下的承诺；而企业自己的建议，或许会直接关系到自己要缴的份儿。在政府不会大幅增加对仿制药项目拨款的前提下，讨论会持续发酵，而我们，也将持续保持关注。

最后，或许我们不应该总是作为旁观者。作为中国出口企业（也就是FDA眼中的海外企业），我们是否也应积极参与，发出自己的声音，以下观点可供参考：

• 1 应该以大致相同的比例在征收申请费和设施费的情况下对于制剂企业同时征收产品费。
• 2 如果尚未产品在美国实际上市（针对制剂尚在申报排队阶段），应延迟收费乃至不收费。
• 3 应争取将境外企业多交的那部分费用（foreign levy）降低至0-30,000美元，能够支付实际产生的海外花费即可。
• 4 应该在GDUFA框架下争取小企业费用豁免（small business fee exemption）以及首次提交费用豁免（first time fee exemption）。

- Bob Pollock, Garth Boehm 2015年4月20日
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English Version

In the Federal Register today, FDA announced a public meeting to be held on June 15, 2015 at the FDA’s White Oak campus. The purpose of the meeting is to get the public’s input on the potential reauthorization of the Generic Drug User Fee Act (GDUFA). GDUFA was a 5 year program which will expire at the end of FY 2017 (in government time – that is right around the corner).

FDA want input as to what has and what has not worked and is asking for suggestions as to how to change the essence of the GDUFA goals letter. There has been lots of industry buzz about GDUFA I and clearly not all positive. One of the biggest contentions relates to goal success being linked primarily to Complete Response Letters (CRL) rather than approvals. Industry points to the Hatch-Waxman Act as evidence that the Agency should be assuring that applications move towards approval rather than solely basing goals on CRLs. Industry point to the basic goal of Hatch-Waxman, to bring high quality generic products at an affordable price to the market at the earliest possible date as its mantra for how GDUFA must change in round II. While this is a lofty goal, there does need to be some balance as it is difficult to provide a goal of approving an application in a certain fixed period of time based on the disparity in the quality of many applications. In addition, there may be new or challenging scientific issues not previously evaluated by the Agency in any application or an application may present unique issues related to the complexity of a particular product that must be vetted prior to approval. There are also issues associated with the compliance of facilities from a cGMP standpoint that may delay the approval of an application while the issues are being worked through. None-the-less, industry is clearly looking to land the goals somewhere in the middle. What that might look like we don’t know, but we certainly expect to see significant discussion on this issue.

Another area that will likely be on the table relates to communication. The industry is looking for a better way to track the status of applications through the review and approval process. Certain suggestions of increased access to perhaps a secure IT platform that will allow sponsors to view where their applications stand in the queue or at least some improvement in response time and information available to industry will be a key issue in the negotiations for GDUFA II. The Agency is responding to these concerns now with the introduction of mid-cycle communication and advanced notice of pending approvals, but industry has not yet had sufficient experience with these new tools to judge their impact, but from what I hear industry may be asking for more.

Both sides have learned during the first half of the 5 year cycle and each side (FDA and industry) likely have wants and asks for the next 5-year cycle. The first questions are how much will industry’s asks cost and what the generic industry will be willing to pay. The next question is can FDA deliver on its promises. So far in GDUFA I there are skeptics on both sides and the second half of the first 5 year cycle will bear out FDA’s overall performance, which will also dictate whether the industry is willing to plop down additional revenues. With the potential of significant increase in government appropriations for the generic program not really in the cards, it seems almost a forgone conclusion that there will be a GDUFA II, but what it will look like is the substance of the occult.

To some supplement, what shall we do as a Chinese export company? Maybe we should consider more other opinions such as follow:
1. Revenue should be raised from product fees, application fees, and facility fees (for FDF) in approximately equal parts.
2. Facilities fees should only be charged when a facility actually manufactures commercial product for sale in the US.
3. The "foreign levy" should be $0 to $30,000 and ONLY enough to actually cover differential foreign costs should be charged.
4. There should be a first time fee exemption in addition to a small business fee exemption.

- by Bob Pollock, Garth Boehm, April 20, 2015