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FDASIA: Three Years of Success

Jul 07, 2015

It has been three years since the Food and Drug Administration Safety and Innovation Act (FDASIA) was enacted. This Act expands the U.S. FDA’s authorities and strengthens its ability to safeguard and advance public health by giving it the power to collect user fees from industry to fund reviews of innovator drugs, medical devices, generic drugs and biosimilar biological products; promote innovation to speed patient access to safe and effective products; increase stakeholder involvement in FDA processes; and more. FDA has established a three-year implementation plan to help the public track the progress of these, and other provisions, established under FDASIA. As the three-year anniversary approaches, it presents a critical milestone to evaluating the success of this multifaceted law. It is difficult to cover the law’s entirety, however, some highlights are provided herein to summarize progress of some of its provisions.

New User Fees for Generics, Biosimilars

FDASIA authorized continued enactment of the fifth Prescription Drug User Fee Act (PDUFA V), which provides FDA with resources to maintain a predictable and efficient review process for human drug and biological products. The third authorization of the Medical Device User Fee Act (MDUFA III)—also part of FDASIA—represents a similar commitment between FDA and the medical device industry to increase efficiency of regulatory processes for devices. The Act also introduced two additional user fees: the Generic Drug User Fee Act (GDUFA) and the Biosimilar User Fee Act (BsUFA). Both GDUFA and BsUFA promote access to generics and biosimilars as part of an effort to generate cost savings for patients (1).

Development of biosimilars is challenging as they can be difficult to characterize since they are composed of highly complex molecules; hence, there are currently few therapeutic alternatives to biologics available. As of this date, only one biosimilar has been approved; however, approximately 17 products are at the IND stage and approximately 51 products are under the biosimilar development program (2).

Encouraging Innovation

New user fees are not the only provisions encouraging innovation in FDASIA. The Act also introduced the “breakthrough therapy” designation. This serves as a powerful expedited drug development tool designed to assist the development and review of new drugs with preliminary clinical evidence that indicates that the drug may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. Simultaneously, FDASIA amended other expedited programs such as fast track and accelerated approval to ensure availability of medications to prevent life-threatening diseases.

For some insight into the impact of the breakthrough therapy designation, Figures 1 and 2 represent the number of breakthrough therapy designation application received, granted, denied and approved by CDER and CBER respectively after implementation of FDASIA. Similarly, Figure 3 signifies the number of applications granted fast track designation, accelerated approval, orphan drug designation and status of first generic. [Editor’s Note: See story on p. 20 for two FDA regulators’ perspectives on breakthrough therapies.]

FDASIA also features provisions for initiatives supporting drug products for rare pediatric diseases, such as the rare pediatric disease priority review voucher program. In November 2014, FDA published a draft guidance which describes criteria for the process of requesting the designation. To date, three companies have received the voucher.

Figure 1 Number of breakthrough therapy designation applications received (a), granted (b), denied (c) and approved (d) by CDER after implementation of the FDASIA (3)
Figure 2 Number of breakthrough therapy designation applications received (a), granted (b), denied (c) and approved (d) by CBER after implementation of the FDASIA (3)
Figure 3 Number of applications granted for fast track designation (a), accelerated approval (b), orphan drug for rare disease designation (c) and first generic (d) (4)

GAIN Act Plus Pediatrics Provisions

The Generating Antibiotic Incentives Now (GAIN) Act was included in FDASIA as an attempt to entice the development of new antibiotics, particularly treatments for “serious or life-threatening infections.” Antibiotic drugs for these conditions are designated as Qualified Infectious Disease Products (QIDPs) under the GAIN Act, and eligible for priority review under the expedited review program for fast track products. Upon approval, the designated products are qualified for five years of marketing exclusivity. To date, FDA has granted the QIDP designation to approximately 50 antibiotics under development and approved three products.

These numbers are encouraging; however, there is still a long way to go in terms of qualifying pathogens, streamlining clinical trial requirements, prescribing information upon approval and more.

Recognizing public support and interest for products specifically targeted for pediatric patients, FDASIA renews and strengthens three essential laws to improve the safety and effectiveness of pediatric drugs, biological products and medical devices used in children. These are the Best Pharmaceuticals for Children Act (BPCA), the Pediatric Research Equity Act (PREA) and the Pediatric Medical Device Safety and Improvement Act. Under FDASIA, PREA was amended to require the submission of a pediatric study plan, typically at the end of phase 2. FDA implemented this provision in early 2013.

As of May 2015, FDA has granted pediatric exclusivity for pediatric studies for 211 total approved drugs (5).

Abuse-deterrent Formulation

Opioids can be abused in a number of ways. Abuse-deterrent formulations target the known or expected routes of abuse for the specific opioid drug substance in that formulation. The science of abuse deterrence is relatively new but methods for evaluating those technologies are rapidly progressing. The final guidance, published in April 2015, explains FDA’s current thinking about the studies that should be conducted to demonstrate that a given formulation has abuse-deterrent properties, how those studies will be evaluated by the agency, and what labeling claims may be approved based on study results.

Stakeholder Involvement

FDASIA initiated a patient-focused drug development program with the goal of obtaining a patient perspective on certain disease areas during the five-year period of PDUFA V. Patient-focused drug development assessment of a product’s benefits and risks involves an analysis of the severity of the condition and the current treatment options available for the given disease. More than 15 public meetings have been held on various disease areas from various cancers, central nervous system disorders, gastrointestinal diseases, human immunodeficiency virus (HIV), and more.


FDASIA is a commitment between the FDA, industry and patients; thus, success its success is not only an achievement of the FDA, but a combined effort among all three. On the third anniversary of the passing of this law, it is clear that progress has been made to achieve this success and will continue to be made.

(The information presented here is the personal view of the author and does not reflect that of Banner Life Sciences. The data provided in the figures are approximate numbers with the sole purpose of summarizing the progress of FDASIA and are taken from the publicly available sources listed under reference.)


  1. U.S. FDA. “Fact Sheet: New User Fees for Generic Drugs Will Enhance Americans’ Access to Less Expensive Drugs and Generate Major Cost Savings.” (accessed June 1, 2015).
  2. U.S. FDA. “Cumulative number of biosimilar development programs in the BPD Program in the month.” (accessed June 1, 2015).
  3. U.S. FDA. “Frequently Asked Questions: Breakthrough Therapies.” (accessed June 1, 2015)
  4. U.S. FDA. “Number of Fast Track designation requests granted.” (accessed June 1, 2015)
  5. U.S. FDA. “Pediatric Exclusivity Granted.” (accessed June 1, 2015).

About the Author

Rensi Sutaria has expertise on preapproval and postapproval regulatory submission for U.S. and global markets for IND, NDAs and ANDAs.


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