U.S. flag An official website of the United States government
  1. Home
  2. News & Events
  3. Speeches by FDA Officials
  4. Remarks to NC BIO’s Annual Meeting 2018 - 10/04/2018
  1. Speeches by FDA Officials

Speech | In Person

Event Title
Remarks to NC BIO’s Annual Meeting 2018
October 4, 2018

Speech by
Anna Abram

Remarks by Anna Abram
Deputy Commissioner for Policy, Planning, Legislation and Analysis
NC BIO’s Annual Meeting 2018
Research Triangle, NC

(Remarks as prepared for delivery)

Thank you for that introduction, Chad [Cooper]. It is good to be back in North Carolina –and not just because of my previous work with Senator Burr on the Senate HELP Committee, but because your state is home to major biomedical research institutions and to a significant portion of the life sciences industry.

This is certainly a difficult time for your state. Our thoughts go out to the many North Carolinians affected by Hurricane Florence including 26 FDA employees in the state. Like many Americans, I have been thinking of those impacted by this devastating Hurricane, not just in the midst of the historic storm as it was occurring, but with the significant flooding that followed.  While I’m heartened by the progress made in the short time since Hurricane Florence’s landfall, and I understand that the Triangle region was not directly affected in the same way other communities were, I know that the impacts will be felt for many months, especially in low-lying communities like Wilmington.

I also closely followed this storm knowing that your state has a vibrant medical products sector. FDA’s Emergency Operations Center began preparing maps to identify and track FDA-regulated manufacturers, including those that make critical medical products, in the days before the Hurricane. Over 1,000 medical product facilities were identified in North Carolina, including several that produce critically important drugs, devices or biologics. Thankfully, we can report that there are no expected national level shortages of critical medical products from Hurricane Florence at this time. We continue to monitor the situation and to provide, as needed, technical assistance to NC firms. In fact, our drug shortages team was in contact with approximately 30 firms in both North and South Carolina before, during and after the storm.

I want to recognize the hard work that went into preparing and enacting preventive measures ahead of the storm. Preventing medical product shortages is a collaborative effort that benefits from planning, communication and information sharing. In fact, the cooperation and vigilance necessary to prevent shortages is a key aspect of protecting and promoting public health and this paradigm has many parallels to FDA’s partnership in medical product development.

When FDA talks about protecting and promoting public health in the context of product development, we often talk about addressing the full continuum of the development and use of new products. This comprehensive approach spans the entire life cycle of the product, from pre-market development to clinical trials to post-market safety surveillance, and for some products to generic competition. It includes providing sufficient guidance and helping to develop the tools that product developers need. Across this entire continuum, the FDA works to ensure that products are safe and effective for their intended use. And it’s an approach that remains important today amid such rapid biomedical discovery.

It’s often been said that we are at an important inflection point in biomedical discovery. Just consider how discovery has been accelerating in precision medicine, a focus of today’s meeting. The structure of the DNA double helix was discovered in 1953. The earliest versions of the advanced technology that we use today to sequence DNA was developed in the mid-90s. Less than a decade later, scientists were able to decode the human genome. In the 15 years since then, sequencing technology has advanced at an amazing pace, becoming faster, cheaper, and more capable of detecting many different types of genetic variations, large and small. And during this period of time we’ve also seen the development of innovative therapies that are targeted to specific individual characteristics of patients and diagnostics that can predict risk of disease or disease presence and seek to identify optimal drugs for treatment. In short, molecular biology, genetics and genomics, and information technology are converging to offer opportunities once unheard of for treatment of previously untreatable diseases. And I’m describing development in only one area of medicine – we’re also seeing rapid advances in areas like regenerative medicine, including cell and gene therapies, and 3D printing to name just a few.

We are fortunate to have Scott Gottlieb at the helm of FDA during such a significant moment for biomedical discovery because he brings a broad range of experience as a medical doctor, as a former official at both FDA and CMS, as a communicator, and from his role in venture finance, not to mention the perspectives he gained as a cancer survivor. Taken together these life and work experiences have given Dr. Gottlieb a broad understanding of the work that you do. Having served twice before at FDA, he was already highly familiar with the agency’s unique culture and was quick to mobilize the talent and knowledge of experts in the centers to help inform his thinking. He thus was able to hit the ground running with a robust list of thoughtful initiatives, some of which I’ll discuss today. Since he arrived at FDA in May of last year he has taken seriously FDA’s mission of protecting and promoting the public health. This means if you sell e-cigarettes to underage youth, he will take action. It means that he is prepared to support pulling an opioid drug from the market because of its abuse potential.  And it means that he is proactive in calling out branded drug companies for gaming the system to prevent generic competition.

But it also means he is equally serious about promoting public health by taking steps to help facilitate the efficient access to beneficial, safe, effective, and innovative products that can address existing, novel and emerging health issues. He puts a premium on transparency – communicating with the public – and on processes that are as modern, efficient, effective, and as evidence- and risk-based as possible. And here’s why - when the costs of developing medical products are high, sometimes a drug or medical device targeted to uncommon conditions might not be developed at all. This can happen if the costs associated with demonstrating safety and efficacy are greater than the potential economic gain to the innovator. Making sure that the regulatory process is predictable, transparent and scientifically modern may help reduce these costs while improving our necessary assurances of safety and efficacy. The new technologies I mentioned before offer transformative opportunities, but they also challenge FDA to modernize its approach to evaluating innovations. Or more simply stated, to keep up.  This has meant, in many cases, creating new approaches that are better suited to the efficient evaluation of these advances without compromising FDA’s science-based standards.

Today I’m going to talk about some of the steps being taken to make FDA’s processes more modern, efficient, predictable and evidence- and risk-based that are of interest to the biomedical industry.

Medical Innovation Access Plan

Many of these steps fall under the umbrella of FDA’s Medical Innovation Access Plan – which strives to create pathways that allow beneficial technologies to efficiently reach patients by cultivating new policies and guidance for product regulation around innovative medical science. A key feature of this plan is modernizing clinical trials.

While prospectively-randomized, placebo-controlled clinical trials are the most powerful tool that we have for answering fundamental questions about the safety and efficacy of new medical products, we have an opportunity to capitalize on innovations that make complex trials more efficient. The FDA is working across its medical product centers in collaboration with the North Carolina-based Clinical Trials Transformation Initiative and the Medical Device Innovation Consortium to facilitate innovative designs and patient-centered endpoints for drugs, biologics and medical devices. This includes the use of master clinical trial protocols, such as basket, umbrella and platform trials, which involve a common clinical trial infrastructure to study one or more interventions in multiple diseases or a single disease with multiple interventions, each targeting a biomarker-defined population or disease subtype. This also includes adaptive approaches to clinical development such as a seamless trial design used in oncology that compresses the traditional trial phases into one large, continuous trial.  Such approaches can reduce the number of patients needed for clinical development and the time it takes to complete necessary studies—thus reducing costs, while also enabling investigators to learn more about a product’s efficacy and safety and help regulators and sponsors detect efficacy and safety signals earlier in the development process.

We know how important guidance is for predictability and clarity. Last week we issued draft guidance for drug and biologic sponsors detailing FDA’s current thinking on principles for designing, conducting and reporting the results from an adaptive clinical trial as well as draft guidance on master protocols, specifically simultaneous evaluation of more than one investigational drug or more than one cancer type within the same overall trial structure in adult and pediatric cancers. Additional guidance is coming on the use of innovative endpoints. To help solidify the science used to support these novel approaches and promote their adoption, we’re also launching a new pilot program in which drug and biologic companies can meet with agency staff to discuss the use of novel complex innovative trial designs for their clinical development programs.

For devices, clinical trial modernization has meant improving efficiency in trial site contracting, in so-called first in patient studies and in Institutional Review Board or IRB approval – three of the costliest factors in device trials. And through the FDA’s Breakthrough Devices Draft Guidance we have proposed a concept called sprints, in which the sponsor of a breakthrough device identifies a question or regulatory challenge to solve.  FDA then works interactively with the sponsor to provide feedback on a path forward, sometimes in just a few weeks.   These types of early interactions have helped facilitate flexible clinical study designs.

Other creative efforts are also underway. FDA is exploring the development of predictive models based on natural history data, which may obviate the need for placebo arms in some clinical trials for rare diseases by allowing researchers to estimate what a subject’s outcome would have been if they had not received an experimental therapy. And we’re encouraging more widespread use of modeling and simulation in both new and generic drug product development that can be used to organize diverse data sets, explore alternate study design strategies, and predict performance. In our device center, scientists and engineers are building in silico regulatory models for product design and evaluation, including the development of a digital library of models and a family of virtual patients for device testing. To assist sponsors in incorporating modeling and simulation FDA is currently updating guidance on this topic.

Clinical trials are often conducted in specialized and controlled research settings and can still leave critical questions unanswered, particularly about the effects of a medical product after it is used by a broader population over an extended period. Over the past decade, FDA has begun to harness real world data to help us answer some of these questions. Our work with the Sentinel System was initially focused on postmarket surveillance but FDA is actively evaluating the potential uses of real-world evidence to support pre-market regulatory decisions.  And FDA’s Center for Devices and Radiological Health recently issued guidance that provides recommendations that will enable clinical investigators to understand how FDA will evaluate data collected from routine medical care and how FDA will determine whether the collected evidence is appropriate to support regulatory decision making.

Patients have much to tell us about medical product development and so FDA is actively working to elevate patient voices in developing new medical products to treat their diseases. Through the Patient Focused Drug Development initiative, started as part of the commitments under PDUFA V and continued under PDUFA VI, FDA has been addressing the need to better enable patients to provide meaningful input into drug and biologic development. To date, FDA has led more than 20 patient focused drug development meetings to learn from patients impacted by diseases including autism, HIV, and Parkinson’s disease. This has given the FDA’s professional staff a deeper understanding of patient and caregiver experiences. We concluded that patient input can inform the clinical context of the disease and provide not only insights to help frame the assessment of benefits and risks but also a direct source of evidence if methodologically-sound data collection tools could be developed and used within clinical studies of an investigational therapy.  We recently issued draft guidance on how to collect comprehensive and representative patient input. This is the first in a planned series of guidance documents that are intended to facilitate the advancement and use of systematic approaches to collect and use robust and meaningful patient and caregiver input. Our Center for Devices is also partnering with patients, incorporating patient preference information into regulatory decision-making including by championing patient reported outcomes.

How FDA is organized is also key to regulatory efficiency that will promote medical innovation. Over a year ago, FDA launched its Oncology Center of Excellence (or OCE) to leverage the combined skills of regulatory scientists and reviewers with expertise in drugs, biologics, and devices (including diagnostics). Authorized by the 21st Century Cures Act, OCE is FDA’s first inter-center institute that focuses on a specific disease area rather than type of product.

OCE’s interdisciplinary work is yielding significant advances. For example, last May, FDA approved for adult and pediatric patients the first cancer treatment based on a tumor’s biomarker rather than the tumor’s site or cell type. The product was granted accelerated approval and demonstrated efficacy in treating certain solid tumors that progressed following treatment for colorectal cancer and other cancer types. Testing was permitted using a single therapeutic approach for patients with different tumor types rather than requiring separate development programs for each disease site.

In November, using a coordinated, cross-center approach, our Center for Devices approved the first breakthrough-designated, next generation sequencing-based in-vitro diagnostic test that can identify patients with any of five tumor types who may benefit from 15 different FDA-approved targeted cancer treatment options. OCE supported CDRH’s review team in evaluating this innovative testing approach which provides patients and health care professionals with access to critical information in one test report, which may avoid the need for duplicative biopsies.

We intend to apply many of the lessons we have learned from creating and operationalizing OCE elsewhere within FDA as we strive to flatten our review process and break down traditional silos in the development of treatments for other diseases and conditions. Our goal is to be more disease focused, and more integrated across the disciplines involved in drug review and better able to evaluate and analyze data. Our Center for Drug Evaluation and Research is currently planning to reorganize its Office of New Drugs in this way.

One area where we’re already putting this new organization to the test is through guidance development. Beginning with a series of guidances on neurological diseases, we’ve piloted a new, streamlined process for writing science-based, practical, guidance documents and getting them out more quickly. Guidances in the pilot are intended to be concise and advance the goals of providing clear scientific feedback.

I talked earlier about how FDA recognizes that we must take advantage of innovative approaches that are better suited to the efficient evaluation of new technology but to do so without comprising FDA’s science-based and regulatory standards.  A prime example is what FDA is doing regarding cell-based regenerative medicine.

Last November, FDA released a comprehensive framework for the oversight of regenerative medicine products that include cell therapies, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products, as well as gene therapies that lead to a durable modification of cells or tissues (including genetically-modified cells). The framework includes two final and two draft guidance documents that, among other things, more clearly describe the distinction between products that require premarket authorization and those that do not.  In addition, we articulated a risk-based compliance and enforcement policy so that, for the first 36 months following issuance of the final guidance, developers of certain human cell and tissue products will have time to determine if they need to comply with the premarket approval requirements and if such preapproval is needed, to prepare an investigational new drug application or a marketing application. One of the two draft guidance documents proposes a novel and efficient clinical development model by which promising cell-based products could pursue review and approval by FDA.

Regulatory Strategy for Development and Implementation of Advanced Manufacturing

Let me turn to another initiative of significance to the biomedical industry, the work we are doing to promote advanced manufacturing, including continuous manufacturing, which integrates a series of manufacturing steps into one single continuous process that utilizes modern monitoring and controls. Under this closed-loop system, there is a steady output of finished drug products even as raw materials are continuously added. The process allows for more nimble control, requires smaller footprints and is far more efficient than conventional manufacturing.

We’re actively trying to promote this promising technology through guidance and have approved several New Drug Applications and supplements that utilize continuous manufacturing technology. In addition, we established an Emerging Technology Team in 2014. This multidisciplinary team works collaboratively and closely with companies for both new and currently marketed drugs to support the use of advanced manufacturing, novel product design, and new product testing methods even before the drug candidate is identified. To further drive a broader adoption of these technologies, the FDA supports establishment of clear principles for how these new platforms will be evaluated and approved and we will need to invest in the regulatory science to develop policies to support these innovations. Specifically, the International Conference on Harmonisation has recently adopted the proposal for a new guidance for continuous manufacturing, and FDA will play a pivotal role in developing this important guidance.

Drug Competition Action Plan

The high price of prescription drugs has certainly been one of the most controversial issues confronting the biopharmaceutical industry in recent years. Drug pricing is a major concern for the public and for the Administration. The fact is, new medical innovations will not benefit patients and consumers if people cannot afford to access these innovations. To the extent that the FDA can ensure that our regulatory requirements are streamlined, predictable, and science-based, we can help reduce the time, uncertainty, and cost of drug development for branded drugs as well as generic and biosimilar alternatives.

At the same time, we are working to help prevent certain practices across the drug supply chain that can push the cost of effective treatments out of the reach of patients and prevent the public from realizing the full benefits of innovation. And by lowering the direct costs of drug product development, as well as the time and risk embedded in these important endeavors, we also reduce the cost of the capital needed to underwrite new discovery, which in turn can translate to lower costs and greater opportunities for patients to afford and get the treatments they need.

As many of you know, last year the Commissioner introduced the three-pronged Drug Competition Action Plan (also called DCAP), that addresses some of the issues that make it difficult for sponsors to bring generic copies of novel drugs to market after all of the legal barriers to approval, such as patents and statutory exclusivities, have lapsed or otherwise been addressed. First, we’ve worked on further streamlining the generic drug review process to increase efficiency and effectiveness with the ultimate goal of more approvals. To accomplish this we’re making both internal and external changes - issuing an internal policy guide on good Abbreviated New Drug Application assessment practices designed to improve the quality and consistency of assessments and issuing a draft guidance that describes common deficiencies that we continue to see in generic drug applications that result in a low percentage of first cycle approvals. We are also implementing a new statutory provision to incentivize and expedite competitive generic therapies, which are drug products with inadequate generic competition.

Second, we’re advancing new guidance to make the development of generic versions of complex products more efficient and predictable and we’re prioritizing review of many applications for complex generic drug products.  Third, we’re looking for places where branded companies may be taking advantage of FDA’s policies to prevent generic competition in ways that were not anticipated.

We have heard that some brand manufacturers use tactics to prevent generic developers from obtaining the samples of the branded drug product that are typically needed to conduct required testing to demonstrate, among other things, that a generic medicine is bioequivalent to its brand reference drug.  The FDA has received more than 150 inquiries from generic drug developers seeking assistance with problems getting access to product samples. Such tactics raise questions about whether the current system for distributing prescription drugs serves the best interests of patients and the public health.

We’ve seen these kinds of access problems where brand products are subject to limited distribution, whether the company has voluntarily adopted limitations on distribution, or the limitations have been imposed in connection with a Risk Evaluation and Mitigation Strategy or REMS.
Whether there is a REMS program in place or not, generic drug developers should still be able to secure samples of the product to conduct necessary testing. To facilitate the transfer of samples in cases where there is a REMS impacting distribution in place, the FDA has a voluntary process through which generic companies can submit their bioequivalence testing protocols to the agency, and we will evaluate these protocols to assess whether they contain safety protections comparable to those in the brand product’s REMS program.

If the safety protections are comparable, then the generic drug developer can ask FDA to inform the brand company in writing that providing sufficient quantity of the brand drug for testing to support an ANDA application would not be considered a violation of the REMS.

To facilitate transparency about access issues, we’ve also recently made public a list of all the drug products for which we’ve received inquiries from generic developers experiencing difficulties obtaining samples for development, along with the number of inquiries we’ve received from developers about each product. We will also refer access inquiries to the Federal Trade Commission or FTC, the agency responsible for addressing anticompetitive business practices, upon FTC’s request and encourage generic drug developers to submit these inquiries to FTC as well. We’ve also formed an interagency working group to explore areas where the FDA can more closely collaborate with the FTC to address anticompetitive activities.

In addition, we’ve issued draft guidance to help generic firms navigate the development of single, shared-system REMS with brand drug makers, which can be a lengthy and complex process. Bringing together multiple products under one REMS program can have real benefits for the health care system, including for providers. But the process of developing a shared-system REMS shouldn’t become a tool that drug companies can use to delay or block competition from generic products or hinder their ability to enter the market. When final, our draft guidance will make this process more efficient. A second draft guidance, Waivers of the Single, Shared-System REMS Requirement, describes when and how the FDA will consider waiving the single, shared-system requirement, and how generic applicants can request a waiver.

We are also looking at the use of citizen petitions that are subject to section 505(q) of the Federal Food Drug & Cosmetics Act.  It is sometimes argued that these petitions block generic entry. While the record shows that citizen petitions have rarely delayed specific generic drug approvals, there is no doubt that the process requirements associated with 505(q) petitions can add to resource burdens on the generic review process and the FDA’s regulatory decision making. On Tuesday, the FDA issued a revised draft guidance that is intended to make the process surrounding 505(q) petitions more efficient. When the guidance is finalized, we think the increased efficiency in handling these petitions will permit reviewers to focus more of their time on the scientific aspects of ANDA reviews.

Finally, our Fiscal Year 2019 Budget proposal includes an important legislative fix that would make the eligibility for tentative approval of a subsequent generic drug applicant that is blocked solely by a first applicant’s 180-day exclusivity, where certain other conditions are met, a trigger of the first applicant’s 180-day exclusivity. This means that the period of exclusivity would begin sooner for the first filer in certain circumstances and the barrier to approval of subsequent applicants would be lifted more quickly. This proposal will enhance competition, facilitate more timely access to generic drugs, and is expected to create meaningful savings.

Our focus and work on generics is already bearing fruit. Last year, we approved a record number of new generic drug applications, including 80 first generic drugs. In July we saw the highest number of approval actions for a single month in the history of the generic drugs program with 126 total approvals (96 final approvals + 30 tentative approvals). In August, for the first time, we approved several strengths of a generic drug product with a Competitive Generic Therapy designation and did so in the first cycle of review. And we recently approved a significant complex generic, the first generic version of a drug-device combination product, EpiPen, the most widely prescribed epinephrine auto-injector in the U.S. The approval makes possible a potentially lower-cost option for patients living with severe allergies who require constant access to life-saving epinephrine, and by providing an FDA-approved alternative, it will also help protect against potential drug shortages.

Biosimilar Action Plan

Biologics play a critical role in the treatment of many serious illnesses but are also responsible for a significant portion of rising drug costs. About one out of every three new drugs approved by the FDA is a biologic but biologics accounted for 70 percent of the growth in drug spending from 2010 to 2015, according to a RAND study.

It’s been over eight years since Congress paved the way for more competition in the biologics marketplace by establishing an abbreviated pathway to approval for certain biologics, called biosimilar and interchangeable products, once any exclusivity periods for the reference product have lapsed.

Since then, the FDA has approved 12 biosimilars, including the first biosimilars for cancer, and interest remains high in these products with nearly 70 development programs enrolled in FDA’s Biosimilar Biological Product Development Program. While that’s good news, so far, only a fraction of the biosimilars approved have actually gone to market due in part to obstacles in the marketplace and uncertainty by health care prescribers to prescribe a newer type of medication.

FDA is committed to supporting biosimilar development. In July we announced a 4- pronged Biosimilars Action Plan that strives to enhance regulatory predictability and encourage market acceptance of these products.

First, we’re improving the efficiency of our biosimilar and interchangeable product development and approval process. We’re doing that by transitioning a new office of therapeutic biologics and biosimilars to improve coordination with review divisions and creating standardized review templates to improve review efficiency. And our scientists are developing and validating pharmacodynamic biomarkers and in silico modeling and developing an index of critical quality attributes with the goal of allowing development programs to be more efficient.

Second, we’re maximizing greater scientific and regulatory clarity for the biosimilar product development community, including through guidance. We recently issued guidance on labeling, which we expect will help health care practitioners make informed prescribing decisions for their patients, and we have additional guidance in the works on such topics as reference product exclusivity, statistical approaches to evaluate analytical data, post-approval manufacturing changes and those cases when a biosimilar manufacturer seeks approval for some but not all indications of a reference product branded biologic.  We’re also actively exploring the potential for data sharing agreements with foreign regulators to facilitate increased use of non-U.S. licensed comparator products in certain studies conducted to support approval of a biosimilar in the US. And yes, we’ll meet our BsUFA goal of finalizing a guidance on interchangeability by next May, although sponsors don’t need to wait until then to consult with us on their interchangeable development programs.

Third, since education is crucial for the adoption of biosimilars, we’re continuing to build on the momentum created with the education and outreach campaign we launched last October - with webinars, videos and additional materials for providers and patients. We are actively engaging with stakeholders to identify knowledge gaps and develop appropriately tailored materials.

Finally, fourth, we’re supporting market competition by reducing gaming of FDA requirements or other attempts to unfairly delay market competition by follow-on products. We held a public meeting in September on FDA's approach to enhancing competition and innovation in the biological products marketplace and we are weighing the public feedback.

Taken together, we think the Biosimilars Action Plan will lay the groundwork for achieving a robust biosimilars program in the coming years.


One of the highest policy priorities of the Department of Health and Human Services and FDA is advancing efforts to address the crisis of misuse and abuse of opioid drugs harming families. The opioid epidemic is responsible for more than 600,000 deaths over the last decade and is killing more than 115 Americans every day. The FDA has taken significant steps to reduce the risk of opioid addiction and death, including requiring generic and branded sponsors of extended-release and long-acting opioids to make education available to prescribers. And for the first time, this requirement is being expanded to immediate-release formulations. We’re also using our existing drug safety surveillance resources to explore how we can create benchmarks for more rational opioid prescribing through product labeling or REMS. Together, with the application of modern tools, we believe it’s possible to work with prescribers to create rational opioid prescribing standards for acute pain that can lower the risk of opioid addiction and drug diversion, while keeping these powerful medicines available for patients with severe pain such as from metastatic cancer.

In addition, we’re doing what we can to encourage the development of opioids that are harder to manipulate and abuse. While not abuse-proof, these formulations can make certain types of abuse, such as crushing a tablet to snort or dissolving a capsule to inject more difficult or less rewarding. Several abuse-deterrent products have been approved, but their uptake has been slow in part because they are only available as more expensive, brand-name products, many of which are not covered by payors. Last year we issued a final guidance to assist industry in the development of generic versions of approved abuse-deterrent formulations and in July we posted three revised product-specific guidances.


Diagnostics play a critical role in all aspects of healthcare. They can help identify the right therapy for the right patient at the right time. FDA has an obligation to regulate diagnostics, but just as I’ve discussed about our approach to other medical product areas, we must be flexible and risk-based about where we focus our resources and expertise. That’s FDA’s approach to diagnostics in the feedback we’re providing to Congress as it works on proposed diagnostics legislation

Currently our device program is constrained by a framework that’s existed since 1976, which hasn’t been enforced consistently with respect to all in vitro clinical tests and isn’t well suited to the modern challenges presented by the new technologies we’re seeing.

FDA believes that the approach needs to be the same whether the test developer is a traditional manufacturer or a clinical laboratory. FDA should focus its premarket review on evaluating the analytical and clinical validity of a subset of tests that are higher risk, or highly novel and used in circumstances where they can present greater uncertainties that can impact patients. Iterative development is a constant with these products, and we don’t want to discourage such improvements and don’t believe they should always require premarket review.

Thus, what we have articulated to Congress is to grandfather all currently marketed laboratory developed tests and exempt many new in vitro clinical tests from premarket review - including manual tests, tests for rare disease and low-risk and low-volume diagnostics. Many of these diagnostic tests are already well-understood. They’ve become a part of the standard delivery of healthcare. These established tests may not need the same level of oversight as other newer and less well-understood tests. About 50% of new tests would fall into this category of exempt from premarket review.

Another approximately 40% of new tests would qualify for a precertification pathway that builds on an approach we recently applied to direct-to- consumer genetic health risk tests. Pre-certification would be technology-based and involve review of a single test and the procedures used to develop that test and tests within the requested scope, to serve as an umbrella for clearance of a suite of related tests. Developers would be able to add new tests and modify existing tests within the scope of their precertification.

Through this modernized approach, we expect that fewer than 10 percent of all new tests would require individual premarket review and only about one-half of the test modifications that are currently subject to FDA premarket review would still be reviewed premarket.


Finally, a brief word on preparedness. I mentioned FDA’s role in natural disasters, such as hurricanes. We also play a critical role in facilitating our Nation’s preparedness for and response to public health emergencies involving chemical, biological, radiological, and nuclear threats as well as emerging infectious diseases, such as pandemic influenza and the Zika and Ebola viruses. North Carolina is no stranger to this work, as host of one of three HHS Centers for Innovation in Advanced Development and Manufacturing, at the Novartis Vaccines facility in Holly Springs. These centers were put in place to ensure a sustainable domestic medical countermeasure infrastructure with unprecedented ability to accelerate development and manufacture of these important countermeasures in time of need, and to explore emerging and innovative advanced manufacturing technologies that could reduce risk, increase yield, and ultimately reduce total life-cycle costs.

FDA’s work largely focuses on facilitating the development and availability of medical countermeasures such as vaccines, therapeutics, and diagnostic tests. We review investigational and marketing applications; provide regulatory advice to sponsors and government partners; enable access to medical countermeasures that are not yet approved, when necessary, through appropriate regulatory mechanisms such as FDA’s Emergency Use Authorization authority; and support regulatory science research in this area.

Like OCE, FDA’s Medical Countermeasure Initiative –started in 2010-- is another example or model of successful efforts to break down traditional silos to provide an integrated approach across the disciplines involved in the review, evaluation, and analysis of data to support the development and availability of medical products necessary to counter the most pressing public health threats.  FDA’s approach is well-recognized for its contributions toward advances in medical countermeasures, regulatory science developments, and public health response efforts.   In January we launched a joint program with the Department of Defense to prioritize the efficient development of safe and effective medical products intended for American military personnel.

These efforts continue to be highly successful. For example, in the past year the FDA has enabled access to medical products in support of response efforts to the Ebola outbreak in the Democratic Republic of Congo and the Zika virus outbreak in the Americas, approved a new antimalarial drug, granted an Emergency Use Authorization to enable access to a freeze-dried plasma product, and approved the first drug with an indication for treatment of smallpox, based on the animal rule, the first product to be awarded a Material Threat Medical Countermeasure priority review voucher.

Whether it’s a cell-based gene therapy or a cancer diagnostic, a generic version of the Epi-Pen or freeze-dried plasma for our troops, the hallmark of FDA’s work today is to make sure that our organization and policies are as modern as the products we’re being asked to evaluate. Achieving that goal requires regulatory processes that are predictable, flexible, efficient, and risk-based, and guided by the best science available, to benefit patients and protect public health.

Thank you.

Back to Top