近期朋友和小编，在《药物一致性评价》公共号平台，推出特别栏目，名字为【小白说】，既是小白的学习笔记，也是跟大家共同交流的话题。同时欢迎各位大牛，小白提供话题讨论。FDA四条“特别审批通道”小白整理自网络FDA有三条特别审批通道，即快速通道（Fast Track）、优先审评（Priority Review）、加速批准（Accelerated Approval）。
突破性药物（BreakthroughTherapies） 附FDA原文： Fast Track Fast track is a processdesigned to facilitate the development, and expedite the review of drugs totreat serious conditions and fill an unmet medical need. The purpose is to getimportant new drugs to the patient earlier. Fast Track addresses a broad rangeof serious conditions. Determining whether acondition is serious is a matter of judgment, but generally is based on whetherthe drug will have an impact on such factors as survival, day-to-dayfunctioning, or the likelihood that the condition, if left untreated, willprogress from a less severe condition to a more serious one. AIDS, Alzheimer’s,heart failure and cancer are obvious examples of serious conditions. However,diseases such as epilepsy, depression and diabetes are also considered to beserious conditions. Filling an unmetmedical need is defined as providing a therapy where none exists or providing atherapy which may be potentially better than available therapy. Any drug beingdeveloped to treat or prevent a condition with no current therapy obviously isdirected at an unmet need. If there are available therapies, a fast track drugmust show some advantage over available therapy, such as:
A drug that receives FastTrack designation is eligible for some or all of the following:
Fast Track designation must be requested by the drug company. The request canbe initiated at any time during the drug development process. FDA will reviewthe request and make a decision within sixty days based on whether the drugfills an unmet medical need in a serious condition. Once a drug receives FastTrack designation, early and frequent communication between the FDA and adrug company is encouraged throughout the entire drug development and reviewprocess. The frequency of communication assures that questions and issues areresolved quickly, often leading to earlier drug approval and access bypatients. Breakthrough Therapy Breakthrough Therapydesignation is a process designed to expedite the development and review ofdrugs that are intended to treat a serious condition and preliminary clinicalevidence indicates that the drug may demonstrate substantial improvement overavailable therapy on a clinically significant endpoint(s). To determine whetherthe improvement over available therapy is substantial is a matter of judgmentand depends on both the magnitude of the treatment effect, which could includeduration of the effect, and the importance of the observed clinical outcome. Ingeneral, the preliminary clinical evidence should show a clear advantage overavailable therapy. For purposes ofBreakthrough Therapy designation, clinically significant endpoint generallyrefers to an endpoint that measures an effect on irreversible morbidity ormortality (IMM) or on symptoms that represent serious consequences of thedisease. A clinically significant endpoint can also refer to findings thatsuggest an effect on IMM or serious symptoms, including:
A drug that receivesBreakthrough Therapy designation is eligible for the following:
Breakthrough Therapydesignation is requested by the drug company. If a sponsor has not requestedbreakthrough therapy designation, FDA may suggest that the sponsor considersubmitting a request if: (1) after reviewing submitted data and information(including preliminary clinical evidence), the Agency thinks the drugdevelopment program may meet the criteria for Breakthrough Therapy designationand (2) the remaining drug development program can benefit from thedesignation. Ideally, a BreakthroughTherapy designation request should be received by FDA no later than theend-of-phase-2 meetings if any of the features of the designation are to beobtained. Because the primary intent of Breakthrough Therapy designation is todevelop evidence needed to support approval as efficiently as possible, FDAdoes not anticipate that Breakthrough Therapy designation requests will be madeafter the submission of an original BLA or NDA or a supplement. FDA willrespond to Breakthrough Therapy designation requests within sixty days ofreceipt of the request. Accelerated Approval When studying a newdrug, it can sometimes take many years to learn whether a drug actuallyprovides a real effect on how a patient survives, feels, or functions. Apositive therapeutic effect that is clinically meaningful in the context of agiven disease is known as “clinical benefit”. Mindful of the fact that it maytake an extended period of time to measure a drug’s intended clinical benefit,in 1992 FDA instituted the Accelerated Approval regulations. Theseregulations allowed drugs for serious conditions that filled an unmet medicalneed to be approved based on a surrogate endpoint. Using a surrogate endpointenabled the FDA to approve these drugs faster. In 2012, Congresspassed the Food and Drug Administration Safety Innovations Act (FDASIA).Section 901 of FDASIA amends the Federal Food, Drug, and Cosmetic Act (FD&CAct) to allow the FDA to base accelerated approval for drugs for seriousconditions that fill an unmet medical need on whether the drug has an effect ona surrogate or an intermediate clinical endpoint. A surrogate endpointused for accelerated approval is a marker - a laboratory measurement,radiographic image, physical sign or other measure that is thought to predictclinical benefit, but is not itself a measure of clinical benefit. Likewise, anintermediate clinical endpoint is a measure of a therapeutic effect that isconsidered reasonably likely to predict the clinical benefit of a drug, such asan effect on irreversible morbidity and mortality (IMM). The FDA bases itsdecision on whether to accept the proposed surrogate or intermediate clinicalendpoint on the scientific support for that endpoint. Studies that demonstratea drug’s effect on a surrogate or intermediate clinical endpoint must be“adequate and well controlled” as required by the FD&C Act. Using surrogate orintermediate clinical endpoints can save valuable time in the drug approvalprocess. For example, instead of having to wait to learn if a drug actuallyextends survival for cancer patients, the FDA may approve a drug based onevidence that the drug shrinks tumors, because tumor shrinkage is considered reasonablylikely to predict a real clinical benefit. In this example, an approvalbased upon tumor shrinkage can occur far sooner than waiting to learn whetherpatients actually lived longer. The drug company will still need to conductstudies to confirm that tumor shrinkage actually predicts that patients willlive longer. These studies are known as phase 4 confirmatory trials. Where confirmatorytrials verify clinical benefit, FDA will generally terminate the requirement.Approval of a drug may be withdrawn or the labeled indication of the drugchangedif trials fail to verify clinical benefit or do not demonstratesufficient clinical benefit to justify the risks associated with the drug(e.g., show a significantly smaller magnitude or duration of benefit than wasanticipated based on the observed effect on the surrogate). Priority Review Prior to approval, eachdrug marketed in the United States must go through a detailed FDA reviewprocess. In 1992, under the Prescription Drug User Act (PDUFA), FDA agreed tospecific goals for improving the drug review time and created a two-tieredsystem of review times – Standard Review and Priority Review.A Priority Review designation means FDA’s goal is to take action on anapplication within 6 months (compared to 10 months under standard review). A Priority Review designationwill direct overall attention and resources to the evaluation of applicationsfor drugs that, if approved, would be significant improvements in the safety oreffectiveness of the treatment, diagnosis, or prevention of serious conditionswhen compared to standard applications. Significant improvementmay be demonstrated by the following examples:
FDA decides on thereview designation for every application. However, an applicant may expresslyrequest priority review as described in the Guidance for Industry ExpeditedPrograms for Serious Conditions – Drugs and Biologics. It does not affect thelength of the clinical trial period. FDA informs the applicant of a PriorityReview designation within 60 days of the receipt of the original BLA, NDA, orefficacy supplement. Designation of a drug as “Priority” does not alter thescientific/medical standard for approval or the quality of evidence necessary.
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来自： tina360tina > 《临床》
