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"Reviewing FDA's Implementation of FDASIA"


Statement of

Janet Woodcock, M.D.
Director, Center for Drug Evaluation and Research
Food and Drug Administration
Department of Health and Human Services


Jeffrey Shuren, M.D., J.D.
Director, Center for Devices and Radiological Health
Food and Drug Administration
Department of Health and Human Services

Before the
Subcommittee on Health
Committee on Energy and Commerce
U.S. House of Representatives

November 15, 2013


Mr. Chairman and Members of the Subcommittee, we are Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research (CDER) and Dr. Jeffrey Shuren, Director of the Center for Devices and Radiological Health (CDRH), at the Food and Drug Administration (FDA or the Agency), which is part of the Department of Health and Human Services (HHS).  Thank you for the opportunity to be here today to discuss implementation of the Food and Drug Administration Safety and Innovation Act (FDASIA) (P.L. 112 144).  We appreciate the extensive bipartisan efforts of this Subcommittee in crafting the legislation and passing it well in advance of the statutory deadline for user fee reauthorization.

On July 9, 2012, President Obama signed FDASIA into law, reauthorizing user fee programs for innovator drugs and medical devices and establishing two new user fee programs for generic drugs and biosimilar biological products.  The law also gave FDA new authority to better protect the drug supply chain, which is critical in an increasingly global marketplace.  In addition, FDASIA provided the Agency with new authorities to combat drug shortages and stimulate antibacterial drug development, made permanent programs to enhance development of products used to treat pediatric populations, included provisions intended to encourage drug innovation, made a number of important changes to medical device regulation, and added a number of other important provisions.  As we discuss below, FDA has made significant progress in implementing FDASIA and is on track to meet most due dates.  We have established a three-year implementation plan, which we have been updating monthly to keep the public informed.

User Fee Program Implementation

FDASIA includes the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), which was first enacted in 1992, and the third authorization of the Medical Device User Fee Act (MDUFA III), which was first enacted in 2002.  Two new user fee programs—for generic drugs and for biosimilar biological products—build on the successes of these two established user fee programs.  Coming at a time of continuing budget restraints, this steady source of funding is essential to support and maintain FDA’s staff of experts who review the thousands of product submissions we receive every year, and do so in a timely and thoughtful manner.  Over the years, our user fee programs have ensured predictable, consistent, and streamlined premarket programs for industry and have helped speed patient access to safe and effective new products.  It is important to note that, due to across-the-board budget cuts imposed by sequestration, approximately $56 million in FY 2013 user fee funds from PDUFA, MDUFA, the Generic Drug User Fee Amendments (GDUFA), and the Biosimilar User Fee Act (BsUFA) are unavailable to FDA, which puts at risk key commitments negotiated under FDASIA.  For example, the reductions in PDUFA and MDUFA funds may result in delays in the availability of novel and important new drugs and devices for patients.  Sequestration also poses a challenge for FDA as we take the necessary steps to launch the new user fee programs for generic drugs and biosimilars.  These programs are designed to enable FDA to leverage user fee resources to provide many benefits to the public, including expediting the availability of high-quality, cost-effective generic drugs and biosimilars.


PDUFA V addressed many of the top priorities identified by public stakeholders, the top concerns identified by industry, and the most important challenges identified within FDA.  PDUFA V enhancements included increased interaction during regulatory review of New Molecular Entity New Drug Applications (NME NDAs) and original Biologics License Applications (BLAs); regulatory science enhancements to expedite drug development; the development of important new guidance for drug developers; a commitment to develop a structured framework for benefit-risk assessment; various enhancements to the drug safety system; and requirements for electronic submissions and standardization of electronic application data.  This additional work was funded by a modest 6 percent increase in PDUFA user fees.  In fiscal year 2013, however, the amount sequestered from PDUFA fees nearly cancelled out the 6 percent increase in fee funding.


One of FDA’s major undertakings since last July has been putting in place the infrastructure for a new generic drug user fee program that will expedite the availability of low-cost, high-quality generic drugs. The program has already achieved several milestones, including making significant strides in reducing the backlog of pre-GDUFA applications and enhancing review efficiencies.  FDA has completed scientific review of approximately 40 percent of GDUFA backlog applications since the program launch.  In addition, FDA has conducted completeness assessments for over 900 drug master files[1] and has launched the creation of a public list of drug master files available for reference to expedite review of applications containing referenced active pharmaceutical ingredients (API).  Further, FDA held a public meeting on June 21, 2013, to discuss regulatory science priorities to expand the availability and quality of generic drugs and solicit input from stakeholders.  The Agency streamlined the hiring process to recruit new scientific reviewers, project managers, investigators, and support staff, and met its ambitious year-one GDUFA hiring goal by bringing on board at least 25 percent of GDUFA program hires by October 1.  Lastly, FDA has facilitated development of the most comprehensive list of generic drug industry participants:   as of October 1, 2013, more than 2,200 manufacturing and testing facilities have submitted self-identification information to FDA, enhancing the quality and transparency of our knowledge of the generics industry.


Reauthorization of the medical device user fee program has helped to expedite the availability of innovative new products to market by boosting the medical devices regulatory review capacity through hiring new staff.  MDUFA III represented a commitment between the U.S. medical device industry and FDA to increase the efficiency of regulatory processes in order to reduce the total time it takes to make decisions on safe and effective medical devices.  It was the result of more than a year of public input, negotiations with industry representatives, and discussions with patient and consumer representatives.

As we have previously testified, prior to MDUFA III, beginning in 2010, we put in place a series of reforms designed to improve predictability, consistency, and clarity in the device review process.[2]  We were seeing results from these reforms before enactment of MDUFA III, but the additional user fee funding authorized under FDASIA enhances our ability to implement positive changes for patients and industry.  Under MDUFA III, FDA is authorized to collect user fees that will total approximately $595 million over five years.  With this additional funding, plus stable appropriated funding, FDA intends to hire more than 200 full-time equivalent (FTE) workers over the course of MDUFA III.  As of October 1, 2013, we have hired more than 90 new employees in support of the medical device review process.

In exchange for the additional user fees, FDA committed to meet much-enhanced performance goals for the device review process.  Preliminary data indicate that FDA has the potential to meet all of its FY 2013 MDUFA III performance goals and expects to see a 25 percent decrease in the backlog of 510(k) submissions.  In addition, FDA expects to see a decrease in average total time for review of 510(k) submissions and Premarket Approval (PMA) Applications.

FDA is providing substantially more detailed quarterly reporting on our progress in implementing those performance goals, and our quarterly performance reports are online and available to the public.  These reports are also presented and discussed at FDA-conducted, quarterly meetings with representatives from medical device member organizations.

In addition, FDA and the medical device industry agreed in MDUFA III to have an independent contractor conduct a two-phase assessment for performing technical analysis, a management assessment, and program evaluation required to objectively assess FDA’s premarket review processes.  This assessment is currently in process.  High-priority findings are expected to be published by the end of this calendar year.


The Biologics Price Competition and Innovation Act (BPCI Act), which was enacted as part of the Affordable Care Act, established a new abbreviated approval pathway for biological products shown to be “biosimilar to” or “interchangeable with” an FDA-licensed biological product.  Approved biosimilars are expected to be less expensive than the reference products, providing clinicians and their patients access to more affordable treatments that are biosimilar or interchangeable.

BsUFA addresses many of the top priorities identified by public and industry stakeholders and the most important challenges identified by FDA in bringing biosimilar products to market.  The biosimilars user fee program is similar to the PDUFA program in that it includes fees associated with marketing applications, manufacturing establishments, and products.  However, there are some differences between BsUFA and PDUFA because of the nascent state of the biosimilars industry in the United States.  For example, there are currently no FDA-approved biosimilar biological products; accordingly, the biosimilars user fee program includes fees for products that are in the development phase to generate fee revenue in the near-term and to enable sponsors to have meetings with FDA early in the development of biosimilar biological product candidates.

In March 2013, FDA published a draft guidance for industry entitled “Formal Meetings Between the FDA and Biosimilar Biological Product Sponsors or Applicants.”[3]  This draft guidance provides recommendations to industry on formal meetings between FDA and sponsors or applicants relating to the development and review of biosimilar biological products regulated by the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).  The guidance assists sponsors and applicants in generating and submitting a meeting request and the associated meeting package to FDA for biosimilar biological products.

As of September 17, 2013, FDA had received 56 meeting requests for an initial meeting to discuss biosimilar development programs related to 13 different reference products and had held 47 initial meetings with sponsors.  FDA is actively engaging with sponsors, including holding development-phase meetings and providing written advice for ongoing development programs for proposed biosimilar products.  As of September 17, 2013, CDER had received 17 Investigational New Drug (IND) Applications for biosimilar development programs, although additional development programs are proceeding under pre-INDs.

Implementation of the Additional FDASIA Provisions

User fees are by no means the only focus of the law.  FDASIA also includes provisions to strengthen the drug supply chain; enhance patient engagement with FDA; address the problem of drug shortages; promote innovation; promote the development of antibacterial drugs; encourage the development of drugs and devices for use in pediatric populations; and enhance FDA’s medical device premarket review program, among others.

Global Drug Supply Chain

Title VII of FDASIA strengthens drug safety by giving FDA new authorities to protect the integrity of an increasingly global drug supply chain in which nearly 40 percent of finished drugs and 80 percent of APIs are imported.  Title VII allows FDA to protect the global drug supply chain by:  (1) increasing FDA’s ability to collect and analyze data to enable risk-informed decision-making, (2) advancing risk-based approaches to facility inspection, (3) partnering with foreign regulatory authorities, and (4) driving safety and quality throughout the supply chain through strengthened enforcement tools.

Since enactment of FDASIA, FDA has been working diligently to implement the Title VII supply chain authorities in a meaningful way that strives to maximize its public health impact.  For example, FDA issued a proposed rule to extend the Agency’s administrative detention authority to include drugs intended for human or animal use, in addition to the authority that is already in place for foods, tobacco, and devices; issued draft guidance defining conduct that the Agency considers delaying, denying, limiting, or refusing inspection, resulting in a drug being deemed adulterated; issued draft guidance addressing specification of the unique facility identifier system for drug establishment registration; and successfully worked with the U.S. Sentencing Commission on higher penalties relating to adulterated and counterfeit drugs.

The Agency had already taken steps toward development of a risk-based inspection schedule, prior to FDASIA.  However, the enhancements provided by FDASIA will further assist the Agency in responding to the complexities of an increasingly globalized supply chain.  For example, provisions in FDASIA that permit FDA to request records in advance or in lieu of an inspection and that require firms to submit a unique facility identifier will allow FDA to increase its inspectional efficiency and its knowledge base.

In addition, FDA hosted a public meeting in July 2013 to solicit comments from the public about implementation of Title VII generally, and to specifically address the provisions related to standards for admission of imported drugs and commercial drug importers, including registration requirements and good importer practices.

Title VII implementation requires not only the development of new regulations, guidance, and reports, but also major changes in FDA information systems, processes and policy—a challenging task given that Title VII was not additionally funded through user fee support or otherwise.  However, FDA has worked to make progress in each of these areas, prioritizing the Agency’s efforts to achieve the greatest public health impact and deploy its limited resources most effectively.

Patient Engagement

In accordance with our commitments in PDUFA V, FDA has initiated the Patient-Focused Drug Development Program. The objective of this five-year effort is to more systematically obtain the patient’s perspective on a disease and its impact on patients’ daily lives, the types of treatment benefit that matter most to patients, and the adequacy of the available therapies for the disease. We have already held patient meetings on several major diseases.

CDRH launched a comprehensive Patient Preference Initiative earlier this year.  This Initiative builds upon our 2012 Benefit-Risk Guidance entitled “Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications,”[4] which outlines the principal factors FDA considers when making benefit-risk determinations during the premarket review process for certain medical devices, including patient perspectives on meaningful benefits and acceptable risks.  However, while this guidance outlines a strategy for how patient preference results should be compared to other sections of a submission, it does not outline which methods, tools, and approaches can be used to collect patient views or provide guidance on how to establish and evaluate the validity of the data, nor does it describe how patient preference data may be used in a broader context of the total product cycle of medical devices.

Therefore, CDRH established the Patient Preference Initiative to provide the information, guidance, and framework necessary to incorporate patient preferences on the benefit-risk tradeoffs of medical devices into the full spectrum of CDRH regulatory processes and to inform medical device innovation by the larger medical device community.  CDRH held a two-day public workshop in September 2013 to engage and solicit information on patient preference from stakeholders, including patients, providers, industry, and academic thought leaders.  CDRH has also recently completed an obesity pilot study that has developed new tools that can be used to measure patient preferences.  Finally, CDRH is working to expand both the number of patient Special Government Employees (SGEs) and the ways in which FDA uses these expert patients throughout the Agency.

Drug Shortages

Preventing drug shortages is, and will continue to be, a priority for FDA.  Our performance in this area has improved over the past two years, including in response to the President’s Executive Order in 2011 and the passage of FDASIA.  In 2012, there were 117 new drug shortages, a decrease of more than 50 percent from the previous year.  Overall, significant progress is being made to prevent and mitigate drug shortages as a result of collaboration between FDA, industry, and other stakeholders.  FDA has worked hard to prevent more shortages:  in 2012, 282 drug shortages were prevented, a 45-percent increase from the previous year.

FDASIA has been extremely helpful in assisting FDA in addressing drug shortages by requiring that manufacturers notify the Agency early if they identify issues that could lead to a potential shortage or a manufacturing disruption and to report the reasons for these issues.  Notifications to FDA from manufacturers increased six-fold after the Executive Order was issued by President Obama on October 31, 2011, and they have continued at this heightened rate since enactment of FDASIA.  Early notifications from the manufacturer to FDA are a key step in helping to prevent drug shortages.  With the benefit of extra time, FDA can use mechanisms within its power to work with sponsors to resolve manufacturing and quality issues, expedite inspections and reviews of product submissions, determine if other manufacturers are willing and able to increase production, help sponsors qualify new sources of raw material, and in appropriate cases, review possible risk mitigation measures for remaining inventory.

For example, a company recently notified FDA of the risk of particulate matter in an injectable drug product needed for IV nutrition therapy.  The particles were found to be foreign material from the manufacturing line, and, through ongoing communications with FDA, the manufacturer was able to show that a filter successfully removed the particulate.   FDA exercised discretion to allow distribution of the product along with a letter included in the drug’s packaging, warning health care professionals to use a filter when administering the drug.  The drug was available while the firm addressed the root cause of the problem, so that it could resume producing a drug that did not need the work-around involving the filter.  FDA has exercised this type of regulatory flexibility while maintaining safety for important drugs several times over the past two years, including lifesaving components of IV nutrition for newborns, children, and other patients who are unable to eat and drink by mouth.

FDA is implementing the drug shortages provisions of FDASIA, including the publication of a Strategic Plan for Preventing and Mitigating Drug Shortages (the Strategic Plan), a plan that outlines our current actions as well as potential future directions for FDA and industry.  Because it is clear that FDA cannot solve this public health threat alone, as a part of this work, FDA’s Drug Shortages Task Force has sought input from a variety of stakeholders to help find solutions.  The Strategic Plan includes two overarching strategies to address drug shortages: improving FDA’s response to notifications received from industry to prevent a shortage (or where a shortage is unavoidable, to mitigate its impact on patients); and preventing the issues that are the root cause of most shortages, including efforts to promote and sustain quality manufacturing. The Strategic Plan is posted on FDA’s website.[5]  FDA also issued a proposed rule implementing the expanded early notification requirements included in FDASIA, and established a docket for the public to provide comment on the proposed rule.[6]


FDASIA gave FDA another powerful expedited development tool known as the “breakthrough therapy” designation.  This tool is designed to expedite the development of new drugs based on preliminary clinical evidence that indicates the drug may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases.  The sponsor of a product with “breakthrough therapy” designation receives, among other things, the benefits of “fast-track” designation, as well as intensive guidance from the Agency on efficient drug development, beginning as early as the Phase 1 period.  Breakthrough therapy drugs go through the same process for approval as other drugs; the program does not change approval standards.  We have been very active on this subject, meeting with companies and discussing ways to expedite the drug development process for drugs that show striking early results.

FDA reviews requests for breakthrough designations within 60 days of receipt of the request.  As of August 31, 2013, FDA had received 85 requests for breakthrough therapy designation, and FDA has already granted the breakthrough therapy designation to 27 potential innovative new drugs that have shown encouraging early clinical results in treating conditions, such as cystic fibrosis, epidermolysis bullosa, hepatitis C infection, breast cancer, Waldenstrom’s macroglobulinemia, and Duchenne muscular dystrophy, to name a few.  Many of the breakthrough therapy designations granted so far have been for rare disease indications.  FDA has not yet approved any products for which a breakthrough therapy designation has been granted.

It is FDA’s view that drug developers should have a clear understanding of all of FDA’s expedited development and review tools, including breakthrough therapy designation, accelerated approval, the fast-track program, and priority review.  To help industry better understand each of these tools, including when the tool can be used and the features of each, in June 2013, we published a draft guidance for industry entitled “Expedited Programs for Serious Conditions—Drugs and Biologics.”[7]  Among other important information, the draft guidance describes FDA’s policies and the threshold criteria and features for each expedited program, defines and discusses important concepts (including “serious condition,” “unmet medical need,” and “available therapy”), and provides general considerations for products utilizing an expedited program, such as manufacturing and product quality, non-clinical considerations, and clinical inspection considerations.

Development of Antibacterial Drugs

Recognizing the need to stimulate investments in antibacterial drugs, Congress enacted the Generating Antibiotic Incentives Now (GAIN) title of FDASIA to create an incentive system. The primary framework for encouraging antibacterial development authorizes FDA to designate human antibacterial or antifungal drugs that are intended to treat “serious or life-threatening infections” as “qualified infectious disease products” (QIDP).  With certain limitations set forth in the statute, a sponsor of an application for an antibacterial or antifungal drug that receives a QIDP designation gains an additional five years of exclusivity to be added to certain exclusivity periods for that product.  A drug that receives a QIDP designation is also eligible for designation as a fast-track product, and the application for that drug is eligible for priority review.  Between July 9, 2012 (when the GAIN title of FDASIA went into effect) and September 17, 2013, FDA granted 24 QIDP designations.  On June 12, 2013, FDA issued a proposed rule to establish a Congressionally mandated list of “qualifying pathogens” that have the potential to pose a serious threat to public health and made public the methodology for developing the list, as required by FDASIA.


FDASIA strengthened and made permanent provisions to improve the safety and effectiveness of drugs, biological products, and medical devices intended for use in pediatric populations.  It made permanent the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), and authorized certain funding associated with pediatric device development.  As we mark the 16-year anniversary of BPCA and the 10-year anniversary of PREA, we are pleased to report that, since passage of those important pieces of legislation, more than 500 drug labels have been revised to contain information about use of products in pediatric populations.

Under FDASIA, PREA was amended to require the submission of pediatric study plans, typically at the end of Phase 2.  This provision provides an opportunity to improve the pace of pediatric drug development by requiring sponsors to submit pediatric study plans early in a product’s development program; it is consistent with FDA’s stated regulatory objectives and facilitates alignment with European efforts in the arena of pediatric product development.  FDA implemented this provision in early January 2013.  In addition, FDA has published a draft guidance to industry, “Pediatric Study Plans:  Content of and Process for Submitting Initial Pediatric Study Plans and Amended Pediatric Study Plans.”

Congress also provided FDA with increased flexibility and mechanisms to ensure the completion of pediatric studies required under PREA.  Under FDASIA, FDA was given the authority to grant extensions of deferred pediatric studies under PREA.  FDA has successfully implemented a process for review and granting of deferral extensions.   As of October 1, 2013, 99 deferral extensions have been requested and 84 deferral extensions have been granted.  FDA was also given authority to issue non-compliance letters for failure to submit required studies under PREA.  These non-compliance letters must be publicly posted.  FDA began posting non-compliance letters for failure to complete PREA-required studies in August 2013.  As of October 1, 2013, nine non-compliance letters were posted.

FDA is also working to address the lack of sufficient labeling information for neonates, by establishing a Neonatal Subcommittee of the Pediatric Advisory Committee and by entering into a two-year contract for a part-time neonatologist to assist FDA in establishing priorities and scientific pathways for product development for neonates.

FDASIA reauthorized grant funding for non-profit consortia to stimulate pediatric device development.  The Pediatric Device Consortia (PDC) Grant Program funds consortia, which provide advice and device development resources to innovators in developing medical and surgical devices designed for the unique needs of the pediatric population—needs that often go unmet by devices currently available on the market.  In FY 2013, FDA awarded PDC grants to seven consortia[8] supporting innovators in the pediatric device development space.

FDA has also issued a proposed rule,[9] as required under FDASIA, relating to the tracking of pediatric uses of devices, along with companion draft guidance.[10]  This proposed rule would require applicants to include in certain premarket submissions readily available information about pediatric patients who suffer from the disease or condition that the device is intended to treat, diagnose, or cure.  The information submitted will be used to help FDA better track the number of approved devices for which there is a pediatric subpopulation that suffers from the disease or condition that the device is intended to treat, diagnose, or cure.  FDA would like to use this data to identify unmet pediatric needs in medical device development.

Rare Pediatric Disease Initiatives and Other Rare Disease Programs

FDASIA added a number of new provisions for rare diseases, including the rare pediatric disease priority review voucher program, consultation with external experts on rare diseases, and a pediatric rare diseases public meeting.  Under PDUFA V, a rare diseases program in CDER and a rare diseases liaison in CBER were added.

The rare pediatric disease priority review voucher provision is intended to encourage development of new drugs and biologics for prevention and treatment of rare pediatric diseases.  FDA has already designated one drug product as a drug for a rare pediatric disease and is reviewing several other designation requests.  Designation is only one part of the voucher program.  It does not guarantee that the sponsor will receive a priority-review voucher upon approval because voucher eligibility depends on the contents of the marketing application.  FDA is working on a draft guidance that will explain the voucher eligibility criteria and the processes for requesting designation and a voucher.

FDA has already scheduled a public meeting, as required by FDASIA (specifically, PDUFA V), to discuss ways to encourage and accelerate the development of new therapies for pediatric rare diseases and to discuss complex issues in rare-disease drug development.  This meeting is scheduled for January 2014.  FDA is seeking input from academicians, clinical practitioners, patients and advocacy groups, industry, and governmental agencies on issues associated with drugs, biological products, and medical devices used for the diagnosis and treatment of pediatric patients affected by rare diseases and on complex issues in rare-disease clinical trials.  The input from this public workshop will help in developing a strategic plan to encourage and accelerate the development of new and improved therapies for pediatric patients affected by rare diseases.

For rare diseases generally, FDASIA reauthorized grant funding for the Orphan Products Grants Program.  This program funds clinical trials involving rare diseases; i.e., diseases that affect less than 200,000 people in the United States.  In FY 2013, FDA awarded 15 grant awards to boost development of rare disease therapies.[11]  This year’s funded studies have an emphasis on vulnerable populations, populations that are extremely difficult to treat, and populations that have no available options. 

Unique Device Identification (UDI) System

On September 20, 2013, FDA announced the final rule for a UDI system, which, once implemented, will provide a consistent, standardized, unambiguous way to identify medical devices.  The UDI system will be phased in over several years, focusing first on the highest-risk medical devices.  Once fully implemented, the UDI system rule is expected to have many benefits for patients, the health care system, and the device industry.  It will provide improved visibility as devices move through the distribution chain, enhancing the ability to quickly and efficiently identify marketed devices when recalled and improve the accuracy and specificity of adverse event reports; it will also offer a clear way of documenting device use in electronic health records and clinical information systems.

Investigational Device Exemptions

FDA is also implementing the provisions of FDASIA relating to investigational device exemptions (IDEs).  FDA approval of an IDE is required for U.S. human study of a significant-risk device that is not approved for the indication being studied.  CDRH has made changes to its IDE decision letters to align them with FDASIA’s requirement that FDA may not disapprove a clinical investigation on the basis of its conclusion that the investigation may not support future premarket clearance or approval of a device, or that a different investigation may be necessary to support a premarket submission.  In addition, on June 14, 2013, FDA re-issued a draft IDE guidance,[12] which incorporates the FDASIA requirements related to IDEs.  In this guidance, the Agency proposes a voluntary process, called a “Pre-Decisional IDE,” intended to enable a sponsor to obtain timely feedback from FDA on its IDE prior to a formal submission to FDA, in order to facilitate faster approval times for IDE submissions and to help address commonly reported challenges in the initiation of clinical trials.  Also in the guidance, in an effort to promote timely initiation of clinical investigation enrollment that protects study subjects, FDA has developed methods to allow a clinical investigation of a device to begin under certain circumstances, even when there are outstanding issues regarding the IDE submission.

De Novo Device Classification Pathway

FDASIA also included provisions to streamline the de novo classification pathway for novel devices of low-to-moderate risk.  Sponsors of these devices may now submit a de novo request for classification without being required to first submit a 510(k) premarket notification to FDA and receiving a “not substantially equivalent” determination.  FDA is using its authority under this new provision to review “direct” de novo petitions from sponsors and, as of September 19, 2013, has initiated review of 33 such “direct” de novo device submissions.  FDA has also implemented process improvements to increase the efficiency, transparency, and accountability of the Agency’s de novo review process.  For example, we are encouraging utilization of the pre-submission process for sponsors to engage in active dialogue with FDA to further encourage efficiencies related to de novo review.  In addition, decision summaries for de novos granted marketing authority through the de novo process are available on FDA’s website.

Health Information Technology (Health IT)

Pursuant to section 618 of FDASIA, FDA is currently working with the Federal Communications Commission (FCC) and the HHS Office of the National Coordinator for Health IT (ONC) to develop a report that contains a proposed strategy and recommendations on an appropriate, risk-based regulatory framework for health IT that promotes innovation, protects patient safety, and avoids duplicative regulation.  FDA, in collaboration with ONC and FCC, set up a working group under ONC’s Health IT Policy Committee (HITPC) in early 2013 to gather input from a variety of stakeholders and experts to inform the HITPC’s recommendations to FDA on an appropriate, risk-based regulatory framework pertaining to health IT; that working group held numerous, productive meetings on this topic.  On September 7, 2013, the working group provided its final recommendations to the HITPC.[13]  FDA, ONC, and FCC intend to use the input from the HITPC in the agencies’ development of the report.

On September 25, 2013, FDA published its final guidance on mobile medical applications (mobile medical apps).[14]  FDA issued the mobile medical apps guidance to provide clarity and predictability for manufacturers of mobile apps.  This guidance informs manufacturers, distributors, and other entities about how FDA intends to apply its regulatory authorities to software applications intended for use on mobile devices that perform the same functions as traditional medical devices.

Consistent with FDA’s existing oversight approach, which considers functionality rather than platform, the Agency intends a tailored approach and to apply its regulatory oversight to only those mobile apps that are medical devices whose functionality is of a kind that FDA already regulates and that present a greater risk to patients if they do not work as intended.  FDA has already cleared more than 75 such mobile apps since the late 1990s.

Device Modifications

On June 13, 2013, FDA held a full-day public meeting to obtain input from external stakeholders on when a modification made to a 510(k)-cleared device requires a new 510(k) submission.  FDA will consider all comments and input from interested stakeholders, including comments received during the public meeting and submitted to the docket for the Federal Register notice announcing the meeting, when formulating FDA’s Modifications Report to Congress and any future guidance on this topic.  FDA also plans to seek public comment on the contents of the report prior to issuing any future guidance. 

Class III “Preamendments” Devices

FDASIA changed the process for reclassifying devices and requiring premarket approval for Class III preamendments devices[15] from rulemaking to an administrative order process.  FDA is using its authority under this new provision and has taken significant steps toward completing the Agency’s ongoing efforts to finalize the classification process for Class III preamendments device types.  We have issued proposed orders to finalize the classification of 13 Class III preamendments device types using the new FDASIA authority.  As of October 1, 2013, FDA has either issued a proposed order, or finished classifying the device type through the rulemaking process, for all but six Class III preamendments device types.  FDA continually updates our progress on the Class III preamendments devices through our publicly accessible “515 Project Status” webpage.[16]

Humanitarian Device Exemptions (HDE)

To encourage the development of medical devices intended to benefit patients in the treatment and diagnosis of rare diseases, certain devices for rare diseases or conditions may be granted an HDE, which allows the sponsor to seek FDA approval for the device by demonstrating only a reasonable assurance of safety and not a reasonable assurance of effectiveness.  Previously, only devices approved under an HDE that were intended and labeled for use in pediatric patients after the date of the enactment of the Pediatric Medical Device Safety and Improvement Act of 2007 could seek to make a profit on their device.  FDASIA broadened the circumstances under which a sponsor of an HDE-approved device could make a profit, in order to further encourage the development of medical devices for rare diseases and conditions without undermining the incentive for sponsors to develop these devices for pediatric populations.  As of October 1, 2013, FDA has approved five HDE supplements for HDE device sponsors, taking advantage of this modified provision.

Other FDASIA Provisions

Pursuant to section 907 of FDASIA, on August 20, 2013, FDA released a report entitled “Collection, Analysis, and Availability of Demographic Subgroup Data for FDA-Approved Medical Products.”[17]  To inform the Agency's follow-up action plan on this issue, FDA has opened a 90-day docket soliciting public comment on the report.

In accordance with Subtitle B of Title XI, on December 18, 2012, FDA issued draft guidance titled “Certification Process for Designated Medical Gases.”  FDA explained the certification process for designated medical gases in January 2013; this process allows certain medical gases listed in the statute for specific indications to be approved efficiently.

With regard to FDA advisory committees, FDA has continued to reduce the percentage of standing-member vacancies on the Agency’s advisory committees.  Between October 2012 and August 2013, the rate of such vacancies decreased from 22 percent to 16 percent.  At the same time, the percentage of conflict-of-interest waivers granted to advisory committee members has remained low and is currently less than 1 percent.


Implementing FDASIA is a considerable undertaking, requiring detailed planning to integrate these tasks with the rest of FDA’s workload.  All told, the 140-page law called for multiple deliverables of all types, including more than 30 proposed and final rules, more than 40 draft and final guidance documents, more than 20 reports to Congress, and many other additional reports, assessments, public meetings, and plans.  FDA continues to meet most of its FDASIA milestones and is on track to implement more provisions very soon.  To help the public keep track of our progress on these and other provisions, we have established a FDASIA web portal[18] that includes a link to our three-year implementation plan, which we update regularly.

FDA is committed to implementing the requirements of FDASIA in a way that provides lasting improvements to public health, and we will meet these objectives as quickly as resources allow. We are happy to answer any questions you may have.

[1]  Drug Master Files are widely used to provide FDA with information about the drug substance, also known as the API.
[2]  See CDRH, “Medical Device Pre-Market Programs: An Overview of FDA Actions” (Oct. 19, 2011) and “Accomplishments: CDRH Plan of Action for 510(k) and Science” (February 2013), available at http://www.fda.gov/aboutfda/centersoffices/officeofmedicalproductsand-tobacco/cdrh/cdrhreports/ucm239448.htm.
[3]  http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryin-formation/guidances/ucm345649.pdf.
[4]  http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationand-Guidance/GuidanceDocuments/UCM296379.pdf.
[5]  http://www.fda.gov/downloads/Drugs/DrugSafety/DrugShortages/UCM372566.pdf.
[6]  https://federalregister.gov/a/2013-25956.
[7]  http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatory-Information/Guidances/UCM358301.pdf.
[8]  The recipients of the PDC Grant Program Awards for FY13 were:  University of Michigan Pediatric Device Consortium, Atlantic Pediatric Device Consortium, National Capital Consortium for Pediatric Device Innovation, New England Pediatric Device Consortium, Southern California Center for Technology and Innovation in Pediatrics, Philadelphia Regional Pediatric Medical Device Consortium, and Boston Pediatric Device Consortium.
[9]  http://www.gpo.gov/fdsys/pkg/FR-2013-02-19/html/2013-03647.htm.
[10] http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm339162.htm.
[11] The recipients of these grants include:  Alkeus Pharmaceuticals, Inc., Children’s Hospital Medical Center Cincinnati, Children’s Hospital of Philadelphia, Duke University, Emory University, PNA Center for Neurological Research, Spineform LLC, Tufts Medical Center, University of Colorado Denver, University of Texas MD Anderson Cancer Center, University of Texas Medical Branch Galveston, University of Washington, and Vanderbilt University.
[12] http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationand-Guidance/GuidanceDocuments/UCM279107.pdf.
[13] http://www.healthit.gov/facas/calendar/2013/09/04/hit-policy-committee.
[14] http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationand-Guidance/GuidanceDocuments/UCM263366.pdf.
[15] “Preamendments” devices are those devices that were in commercial distribution prior to passage of the Medical Device Amendments of 1976.
[16] http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsand-Tobacco/CDRH/CDRHTransparency/ucm240310.htm.
[17] http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatory-Information/Guidances/UCM332136.pdf.
[18] www.fda.gov/fdasia.