FDA’s Role in Medical Device Cybersecurity

By: Suzanne Schwartz, M.D., M.B.A.

Virtually every aspect of our lives – including our health – has gone digital. Medical devices from insulin pumps to implantable cardiac pacemakers are becoming more interconnected and, like computers and the networks they operate in, can be vulnerable to security breaches.

Suzanne SchwartzA computer virus or hack resulting in the loss of or unauthorized use of data is one thing. A breach that potentially impacts the safety and effectiveness of a medical device can threaten the health and safety of an individual or patients using the device.

Global cyber-attacks in 2017, including WannaCry and Petya/NotPetya, have had a significant impact on our nation’s critical infrastructure, including the health care and public health sector. Hospitals, pharmaceutical companies, and even the Kiev airport were among organizations affected by cybercriminals who unleashed copies of the ransomware earlier this year, with demands of payment to restore access to computer networks and crucial files.

Because cybersecurity threats are a constant, manufacturers, hospitals, and other facilities must work to prevent them. There is a need to balance protecting patient safety and promoting the development of innovative technologies and improved device performance.

It is the goal of FDA’s Center for Devices and Radiological Health to encourage a coordinated approach of vigilance, responsiveness, resilience, and recovery that fits our culture of continuous quality improvement.

This means taking a total product lifecycle approach, starting at the product design phase when we build in security to help foil potential risks, followed by having a plan in place for managing any risks that might emerge, and planning for how to reduce the likelihood of future risks.

Specifically, FDA encourages medical device manufacturers to proactively update and patch devices in a safe and timely manner. The concept of updates and patches, while not new to traditional information technologies, is complex when it comes to critical safety systems and requires a collaborative approach to finding solutions.

FDA has published guidances – recommendations for manufacturers and others – that contain recommendations for comprehensive management of medical device cybersecurity risks throughout the total product life cycle. This includes closely monitoring devices already on the market for cybersecurity issues. And because we want to enable more expedient actions, our overall approach incentivizes industry to make changes to marketed and distributed medical devices to reduce risk.

FDA continues to work closely with manufacturers and the public to dispel myths about medical device cybersecurity. On our web site is a printer-friendly fact sheet where we address some of the more prevalent myths concerning FDA and our role in helping to maintain the security of medical devices.

With so many devices dependent on software and internet access today, having a plan in place to address cybersecurity risks is as essential to the device development process as coming up with a novel new product. Working with the medical device industry and other federal agencies, FDA will continue its work to ensure the safety and effectiveness of medical devices at all stages of their lifecycles against potential cyber threats.

Suzanne B. Schwartz, M.D., M.B.A., is FDA’s Associate Director for Science and Strategic Partnerships, at the Center for Devices and Radiological Health

The Mammography Quality Standards Act – a 25-year Public Health Success Story

By: Helen Barr, M.D.

Mammography has been widely used as a screening tool to detect early stage breast cancer since the mid 1960s.

As use of the technology expanded it became clear that there were wide variations in the quality of the mammograms and the dose being delivered to patients.

Helen BarrFollowing a series of hearings, Congress stepped in and enacted the Mammography Quality Standards Act of 1992 (MQSA).

This week we celebrate the 25th anniversary of this important law that gave the FDA authority to improve the quality of mammography services by setting standards for image quality, radiation dose, personnel qualifications, and record retention and reporting requirements, among others. The MQSA also requires that each facility be accredited by an FDA-approved body and hold an active MQSA certificate to legally practice mammography.

The goal of the MQSA was to ensure that mammography facilities – regardless of their location – adhere to uniform standards that consistently ensure high quality breast images, which are critical to the diagnosis of breast cancer. The program also ensures that patients receive their mammogram results within 30 days (sooner if there are problems) and in plain language they can understand.

Today the nation’s 8,700 -plus mammography facilities are inspected every year, an immense task that is accomplished by both FDA inspectors and our state contracting partners. We recently enhanced the inspection process by creating our Enhancing Quality Using the Inspection Program (EQUIP) initiative, which adds inspection questions related to already existing regulations aimed at image quality.  In this way, we can ensure that facilities have processes in place which help them continuously maintain  image quality and spot issues early so that they can be rapidly corrected, benefiting both patients and facilities.

In 1995, 30 percent of mammography facilities in the United States were operating in violation of the law. Today I’m happy to report that 99 percent of facilities have no serious violations.

Over the last 25 years, breast imaging technology has moved from screen film technology, to full field digital mammography (first cleared by FDA for marketing in 2000), to digital breast tomosynthesis, a new technology that takes x-ray “slices” to improve visualization of breast tissue, for which the first unit received FDA’s marketing approval in 2011.

The MQSA program has continued to adapt and evolve along with the technology aided by the dedicated work of my staff.  This involves coordinating expertise with our colleagues in other parts of our Center for Devices and Radiological Health and in FDA’s Oncology Center for Excellence.

The advances in mammography technology, combined with FDA’s scientific expertise and regulatory authority in this area, have served the public well. Thanks in large part to early detection of the disease through quality mammography under the MQSA, as well as improved therapies, the five-year survival rate for all women diagnosed with breast cancer in the United States between 2006 to 2012 was 90.8 percent compared to 74.8 percent between 1975 to 1977, the Centers for Disease Control and Prevention reported earlier this year. Moreover, today’s mammograms require very small doses of radiation, far less than was required decades ago.

Helen Barr, M.D., is Director, Division of Mammography Quality Standards, at FDA’s Center for Devices and Radiological Health

For more information go to: Mammography: What You Need to Know

Medical Device Development Tools: Helping to Speed Medical Device Evaluation and Approval

By: Hilda F. Scharen, M. Sc., Capt. USPHS, and Jeffrey Shuren, M.D., J.D.

FDA relies on sound science in its decision-making, which provides medical device innovators with a strong base for their product development. Part of good science is relying on tools to efficiently and accurately measure the product’s performance at all points in the process.

Hilda Scharen

Hilda F. Scharen, M.Sc., Capt. USPHS

Finding those accurate, efficient, and reliable tools can be challenging, especially given the diversity of technology and of medical devices. That’s where FDA’s voluntary Medical Device Development Tools (MDDT) program can help. FDA can evaluate a tool and decide whether to “qualify” it—which means we can determine if the tool measures what it’s supposed to measure, and does so reliably. FDA only intends to qualify tools where it can make certain high level information about the tools publicly available, so that the device development community can benefit from using tools that they know work.

Qualified tools should help lead to more efficient and robust medical device development, for example, by minimizing the use of animals, reducing testing duration or sample sizes, or by optimizing patient selection for a device clinical study.

In a final guidance issued in August, FDA defined three categories of MDDT:

  • Clinical Outcome Assessment: tools that measure how a patient feels or functions – including patient-reported or clinician/observer-reported measures-  or performance outcome measures, such as walking speed or memory recall;
  • Biomarker Test: a tool (test) used to provide diagnostic, predictive, or safety biomarkers for patient selection or as clinical study endpoints. Examples include blood pressure or an assay that detects a hormone level to determine whether a patient is a safe and appropriate candidate for a clinical trial; and
  • Nonclinical Assessment Model: a tool (model or method) that measures or predicts a device function such as a computer model or a material phantom of the heart.

It is important to understand that FDA qualification of an MDDT is not the same as FDA clearance or approval of a medical device, and that qualification relies on different types of evidence. MDDT qualification helps streamline the FDA review process. It can help address questions about data validity and reduce the time and other resources needed for new product development while maintaining the same level of patient safety.

Jeff Shuren

Jeffrey Shuren, M.D., J.D.

This month, we qualified our first MDDT tool— the 23-item Kansas City Cardiomyopathy Questionnaire (KCCQ), a Clinical Outcome Assessment, that measures patient-reported outcomes (health information reported directly by the patient) from patients with congestive heart failure or weakened heart muscle due to prior heart attacks, heart valve problems, viral infections, or other causes. Patients are asked about their symptoms, physical and social limitations and their views on whether these symptoms have an impact on their quality of life.

By measuring and evaluating this information, the KCCQ can assess whether a patient perceives that their heart failure symptoms have improved following an intervention. These results can then be used as part of the benefit/risk assessment to help determine a device’s safety and effectiveness.

The KCCQ has been used by researchers, clinicians, and medical device developers for around 20 years. Qualification of the KCCQ means that FDA review staff can rely on it without having to reconfirm that the tool is suitable for measuring response for the intended use, saving time and money.

MDDTs qualified by FDA can be used in clinical trials by the medical device industry to support both device submissions and post-approval studies. But the program’s impact goes much further. It offers developers increased opportunities to discuss early concepts of tool development, foster collaboration, and increase the potential that tools will be used and adopted.

Any tool developer, medical device manufacturer, health care provider, researcher, or scholar may voluntarily submit a proposal for their tool to be considered by FDA for qualification. Once an MDDT is qualified, FDA intends to publicly list it, along with a summary of evidence and the basis for qualifying the tool. FDA will not publicly disclose a tool developer’s proprietary information, unless and until we obtain written permission from the developer.

MDDT is an exciting way that FDA can help support innovation, and get better medical devices safely to the patients who need them most.

Hilda F. Scharen, M. Sc., Capt. USPHS, is Director, Medical Device Development Tools, at FDA’s Center for Devices and Radiological Health

Jeffrey Shuren, M.D., J.D., is Director of FDA’s Center for Devices and Radiological Health

FDA Taking New Steps to Better Inform Physicians about Biosimilars Through Education about these Potentially Cost-Saving Options

By: Scott Gottlieb, M.D., and Leah Christl, Ph.D.

Dr. Scott Gottlieb

Scott Gottlieb, M.D., Commissioner of the U.S. Food and Drug Administration

The cost of prescription drugs is an ongoing concern, however, a growing market for potentially lower-cost biological products called biosimilars can offer more competition and options for patients.

Biosimilars can potentially reduce costs for consumers by creating price competition for products that previously faced few market competitors. FDA wants to ensure that health care providers have the information they need when considering prescribing biosimilars when these products are available.

An FDA-approved biosimilar is highly similar to — and has no clinically meaningful differences in terms of safety, purity and potency (safety and effectiveness) from — an already FDA-approved biological product, called the “reference product.” In general, biological products are highly complex and are often used to treat patients with serious and life-threatening conditions.

FDA’s Center for Drug Evaluation and Research (CDER) has approved seven biosimilars to date.  As more biosimilars are approved by FDA, we want health care providers to understand what these drugs are, and how they can help patients. So we are taking new steps to make sure providers are properly informed about biosimilars by launching an educational campaign today.

Leah Christl

Leah Christl, Ph.D., Associate Director for Therapeutic Biologics in the Office of New Drugs, at FDA’s Center for Drug Evaluation and Research

We planned and researched extensively prior to developing the materials for this education and outreach effort. Through this process, we learned the specific areas that health care providers had questions about. These include questions about the data and information FDA reviews when it’s making decisions to approve a biosimilar. Based on that feedback, FDA has developed educational materials  to help health care providers gain a better understanding of these important products and the approval process they undergo. This includes fact sheets and graphics for health care professionals, as well as materials for organizations to use in disseminating this information to their interested members. These resources:

  • Provide the basic definitions of terms like: biological product, reference product, biosimilar, interchangeable; and other terms to facilitate understanding of the relationship between biosimilars and their reference products;
  • Describe the rigorous standards any biosimilar must meet prior to approval and explain how the FDA approval pathway works for these products; and,
  • Provide easily accessible information about the data and information FDA reviews to determine biosimilarity, and how to find more resources.

The new website also highlights information about an important reference for biosimilars known as the Purple Book. This reference can help prescribers and patients learn which biological reference products currently have one or more approved biosimilar or interchangeable product approved by FDA.

Next, FDA plans to embark on additional research with health care professionals to learn more about the types of information prescribers need to properly communicate with their patients about biosimilars. An increase in market competition, offered by a growing complement of biosimilars, may lead to meaningfully reduced costs for both patients and our healthcare system. As with the significant savings that we’ve seen through the introduction of generic drugs in the United States, biosimilars could also lead to substantial savings, thereby potentially improving access and promoting better public health outcomes. Understanding the rigorous process FDA uses to evaluate biosimilars can help prescribers and patients maximize the benefits from these products.

Scott Gottlieb, M.D., is Commissioner of the U.S. Food and Drug Administration

Leah Christl, Ph.D., is the Associate Director for Therapeutic Biologics in the Office of New Drugs, at FDA’s Center for Drug Evaluation and Research

Follow Commissioner Gottlieb on Twitter @SGottliebFDA

Generic Drug User Fees Reauthorization: A Victory for Public Health

By: Kathleen Uhl, M.D., and Michael Kopcha, Ph.D., R.Ph. 

We marked an important milestone in the U.S. generic drug program on Oct. 1, 2017 – the start of the first reauthorization of the Generic Drug User Fee Amendments (GDUFA). These fees from industry provide FDA with vital funding that advances the Agency’s regulatory work to review and approve applications for generic drugs.

Kathleen "Cook" Uhl

Kathleen Uhl, M.D., is Director, Office of Generic Drugs at FDA’s Center for Drug Evaluation and Research

This wasn’t just a milestone for FDA – it was a victory for public health. Increasing consumer access to safe, high-quality, and affordable generic drugs is a top priority at FDA. Generic drugs often cost a fraction of the price of the brand-name version and almost 9 out of 10 prescriptions are filled using generic drugs. During the past decade, generic medicines have saved the American health care system almost $1.7 trillion.

While FDA does not have a direct role in establishing drug pricing, when more than one generic version of a drug is approved, it spurs competition and facilitates affordable options for consumers.

Congress first approved the collection of user fees from industry to help fund FDA’s generic drug program in 2012 – for a five year period.  Having another source of funding beyond our traditional budget appropriations from Congress for an extended period of time allowed us to hire additional staff and to better allocate our review activities. As a result, FDA approved record numbers of generic drug applications.

In fact, 25 percent of all generic drugs currently approved were approved during the first five years of GDUFA. In the last two years of GDUFA, the FDA approved more generic drugs each year than in any other year in the history of the generic drug program. The reauthorization of GDUFA will help us build on this success.

Michael Kopcha

Michael Kopcha, Ph.D., R.Ph., is Director, Office of Pharmaceutical Quality at FDA’s Center for Drug Evaluation and Research

User fees support the dedicated FDA staff who review generic drug applications and inspect the facilities that make generic drugs. User fees provide funds for important IT infrastructure that helps FDA manage its increasing workload, improves efficiency, and increases transparency. They also fund important regulatory research that helps clarify and improve the scientific and clinical understanding that serves as the foundation for generic drug product assessment.

The goals and commitments we agreed to in the reauthorization of GDUFA support public health. We now have shorter review goals available for applications that are public health priorities. In addition, these goals promote two major objectives: reducing the time it takes to approve generic drug applications and increasing the number of approved generic drugs. We have increased communication with industry to help ensure that the Agency receives complete, high-quality, and scientifically sound applications. Another new feature is a flexible user fee structure to address the needs of small businesses. This encourages competition that in turn can result in lower drug prices for consumers.

Developing the generic version of complex drug products – such as inhaled medicines or some types of injectable medicines – has unique challenges. FDA will meet earlier and more frequently with an applicant to anticipate the challenges that might arise in the development of these products.

Our experience in GDUFA allowed us to improve review processes and implement best practices for communicating with industry and consumers. The reauthorization of GDUFA builds on the successes during the first five years. We look forward to the next five years of helping the American public gain even greater access to affordable, high-quality generic drugs.

Please visit fda.gov/genericdrugs for more information on FDA’s generic drug program and to track progress on the reauthorized GDUFA.

Kathleen Uhl, M.D., is Director, Office of Generic Drugs at FDA’s Center for Drug Evaluation and Research 

Michael Kopcha, Ph.D., R.Ph., is Director, Office of Pharmaceutical Quality at FDA’s Center for Drug Evaluation and Research

How FDA ‘Triers’ and Food Sampling Have Prevented Tragedy

By: Suzanne Junod, Ph.D.

Suzanne Junod holding a grain trier

Suzanne Junod, Ph.D., holding two grain triers.

Throughout history, one of the most important tools used by FDA inspectors to protect the food supply is also one of the simplest – a trier – which allows an inspector to collect a representative sample of a product.

Triers vary in design, depending on the product being sampled. But this straightforward, efficient tool makes it possible for FDA inspectors to quickly gather samples to test for contamination and other signs of adulteration. For decades, triers have prevented potential tragedies involving grains, cheese, frozen eggs, olives, and many other products. During the 1950s, for instance, many consumers were unknowingly put at risk by the smallest of food products – the seeds used by many American farmers to grow wheat. The public health threat stemmed from the fact that some farmers used mercury-treated seeds to grow their wheat, and a number of these seeds found their way into the food supply. Thanks to the sharp eyes of FDA inspectors, this threat was removed.

We hope you enjoy your visit to the FDA History Vault.

Suzanne Junod, Ph.D., is an FDA Historian.

Expanded Access: FDA Describes Efforts to Ease Application Process

By: Scott Gottlieb, M.D.

FDA has a long history of supporting patient access to investigational new treatments. This includes working with drug and device companies through the clinical trial process that may lead to FDA approval of the treatment. We also offer expanded access programs that provide investigational drugs and devices to patients with serious conditions (generally prior to product approval), when there is no therapeutic alternative.

Dr. Scott GottliebEach year, FDA receives over 1,000 applications for the treatment of patients through expanded access, also known as compassionate use, and the agency authorizes the vast majority (about 99%). FDA recognizes that time is critical for these seriously ill patients who do not have  alternative therapies, and who cannot take part in a clinical trial of an investigational therapy.

Submissions are authorized quickly, often in a matter of days. In the case of emergencies, FDA will typically provide authorization over the phone in a matter of hours. In an effort to eliminate potential hurdles that might delay or even discourage applications, FDA streamlined the expanded access process by introducing a new application form which a physician may use to request expanded access for their patient. Form FDA 3926 reduced the number of required information fields and attachments, and is estimated to take only 45 minutes for a physician to complete. Before expanded access can occur, the drug company must decide whether or not to provide the product. FDA cannot require a manufacturer to provide a product.

Today, we’re lifting another potential burden for physicians who apply to FDA to use an investigational drug to treat their patient.

Prior to treating a patient under expanded access, the physician must obtain approval from the Institutional Review Board (IRB) at their facility. This is an important step to protect the rights, safety and well-being of human subjects in clinical research – but assembling the full board may cause delays because it may not routinely meet.

As part of a plan to simplify the process for physicians seeking access to an investigational product to treat their patient, I’m announcing today that just one IRB member – the chair or another appropriate person – can now approve the treatment. I believe the simplified IRB process will facilitate access while still protecting patients.

More simplifications and clarifications are in the pipeline.

We’ve seen some reluctance among companies to provide investigational drugs for expanded access. This may have been due, in part, to uncertainty about how data for adverse events that occur during treatment under expanded access are viewed by FDA. Companies have voiced concerns that any apparent negative effects might jeopardize the product’s development.

We recognize that patients receiving expanded access are usually treated outside of a controlled clinical trial setting. As a result, they may have more advanced disease than clinical trial participants, be receiving other drugs at the same time, and have other diseases. FDA recognizes that these factors make it more difficult to determine the cause an adverse reaction.

To clarify how adverse event data in these circumstances are viewed, we’ve updated the guidance for industry entitled, ‘Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers’ (questions 25 and 26). The guidance clarifies that suspected adverse reactions must be reported “only if there is evidence to suggest a causal relationship between the drug and the adverse event.”

I’m confident these changes will help to address recent issues raised by the Government Accountability Office (GAO), which said that FDA “should further clarify how adverse event data are used.” We are still evaluating the GAO recommendations to identify other possible ways to respond to their concerns.

We’re committed to helping patients and physicians fully understand the expanded access process. Dedicated staff in the Office of Health and Constituent Affairs and CDER’s Office of Communications, Division of Drug Information, already assist physicians and patients in navigating this system. We issued three final guidance documents last year to clarify and explain the process. This past July, we collaborated with the Reagan-Udall Foundation, patient advocacy groups, the pharmaceutical industry, and other federal agencies to launch a new online tool called the Expanded Access Navigator. This includes a directory where companies can submit public links to their expanded access policies, the criteria used by companies to determine whether to make a drug available through expanded access, and contact information. The directory offers patients and physicians a helpful starting point for researching available investigational therapies.

In addition, we’re working with the Reagan-Udall Foundation to expand this new tool. I’m pleased to announce that Reagan-Udall will expand its portfolio to include FDA’s Rare Disease Program, with the goal of promoting more expanded access to treatments for rare disorders.

Looking ahead, there will be more options for expanded access. FDA will continue our efforts to educate stakeholders on these opportunities and further simplify the process in order to improve the program.

Scott Gottlieb, M.D., is Commissioner of the U.S. Food and Drug Administration

Follow Commissioner Gottlieb on Twitter @SGottliebFDA

Reducing the Hurdles for Complex Generic Drug Development

By: Scott Gottlieb, M.D.

Earlier this year, I announced our Drug Competition Action Plan to advance new policies aimed at bringing more competition to the drug market. My goal was to improve access consumers have to the medicines that they need. I consider access to medicine a matter of public health. If consumers are priced out of the drugs they need, that’s a public health concern that FDA should address, within the scope of its mandate and authorities.

Dr. Scott GottliebWhile FDA doesn’t control drug pricing, our policies do affect competition in the market. This is the nexus of our current efforts on drug pricing.

Our plan has a number of different domains. Among them is a compilation of efforts to improve the efficiency of the generic drug approval process; and another is a group of policies aimed at closing loopholes that allow branded drug companies to game our rules in ways that forestall the generic competition that Congress intended. One important group of policies is aimed at making it easier to bring generic competition to a category of branded drugs known as complex drugs. Today we’re announcing a major new set of policies to advance these goals.

Complex drugs comprise high cost medicines like metered dose inhalers used to treat asthma, as well as some costly injectable drugs. These medicines generally have at least one feature that makes them harder to “genericize” under our traditional approaches. As a consequence, these drugs can face less competition. In some cases, costly, branded drugs that are complex drugs have lost their exclusivity, but are subject to no generic competition.

The new policies we’re announcing today are aimed at ensuring that we provide as much scientific and regulatory clarity as possible with respect to complex generic drugs. This focus is critical because, first and foremost, these drug products provide important therapies to patients. They are also becoming increasingly significant to the economic health of the generic drug industry. Being able to “genericize” a complex drug can be a high-value opportunity for a generic drug maker that helps underwrite the costs of other generic applications. In other words, because brand-name versions of complex drug products are often higher-priced than many other brand name drugs, any steps we can take to encourage the development of generic competitors to complex drugs will have an outsized impact on access, and prices.

When considering the scope of complex drugs, people often first think of drug products where the active ingredient itself is complex. Glatiramer acetate injection, a drug used in the treatment of multiple sclerosis, is a good example. However, the terms “complex drug product” and “complex generic drug” are used to refer to a much larger and diverse group of drug products. In addition to drug products with complex active ingredients, or sites of action, complex drug products also include complex drug-device combination products.

Together, this diverse collection of drug products has one or more elements that are more complex than an average drug product. This complexity, in turn, means that the scientific and regulatory pathways for approval of generic versions of these drug products are not as well traveled by generic drug developers. In some cases, use of another established regulatory pathway may be appropriate to streamline development.

We’re undertaking a number of efforts to ensure that the pathways for approval for generic versions of complex drug products are as efficient as possible, including a number of new steps that I’m announcing today and others that we’ll be working on in the coming months.

We know that our regulatory requirements impact both the direct and indirect costs of drug development. These include costs associated with the time it takes to develop a drug and gain its regulatory approval, as well as those associated with the research and development of experimental products that ultimately do not make it to market.

Manufacturers of complex generic drugs face a number of challenges in developing their products and demonstrating that their products meet the approval requirements for generic drug applications (abbreviated new drug applications or ANDAs), including establishing that they are bioequivalent to and have the same active ingredient as the brand-name drug.

Bioequivalence for complex generic drugs can be challenging with complex drug products that can’t be easily measured in the blood, or when the drug’s therapeutic effect is delivered locally to a particular organ, rather than systemically, through the bloodstream.  In other instances, showing active ingredient sameness can be challenging when the drug product contains an active mixture of components and not a single active molecule.

These challenges – and resulting regulatory uncertainties – may deter generic manufacturers from beginning development. It can mean these ANDAs undergo more review cycles than other generic drugs. These hurdles, in turn, may result in limited competition and higher prices.

We recognize these problems and are taking a number of new steps to support the development of high quality ANDAs for complex generic drugs.

First, FDA is issuing a draft guidance to assist ANDA applicants and prospective ANDA applicants in creating and submitting pre-ANDA meeting requests, including meeting package materials, so FDA can give better advice to sponsors looking to develop complex generic drugs.

The guidance provides information on requesting and conducting product development meetings, pre-submission meetings, and mid-review cycle meetings with FDA. These meetings will allow for enhanced communication between generic drug applicants and FDA early in the generic drug development process, allowing for more efficient generic drug development, review, and approval pathways. We’ve found from analyzing our new drug program, that early and better meetings between FDA and sponsors can improve development timelines. We want to bring these same types of opportunities to developers of complex generics.

Second, we’re issuing a draft guidance  to help applicants determine when submission of ANDAs for certain complex products, known as peptides, would be appropriate. Peptides are compounds made up of 40 or fewer amino acids, the building blocks of proteins. There are a number of branded medicines that are peptides, where exclusivity has lapsed, but these drugs face little or no competition. This new guidance applies to ANDAs for certain specific synthetic peptides, namely, glucagon, liraglutide, nesiritide, teriparatide, and teduglutide, that reference brand-name versions of these peptides manufactured using recombinant DNA technology.

This guidance represents how advances in regulatory science — when coupled with careful policy considerations — can enable generic drug development that was previously infeasible.

In this case, advances in technology for peptide synthesis and characterization allow an ANDA applicant for one of these products to demonstrate that its product meets the “sameness” requirements for generic drug approval. The recommendations in the new guidance will help ensure that the risk of an immune response from the generic due to differences in impurities will not differ from that of the reference drug.

We’re doing all of this without sacrificing the scientific rigor of the process one bit. A central aspect of our approach, and our efforts to spur innovation and generic competition, is focused on adopting more rigorous and sophisticated science, including sophisticated quantitative methods and computational modeling, in drug development, evaluation, and review.

We’ll soon release other important policies aimed at spurring competition to complex drugs. But we know that better guidance isn’t the only answer. Some drugs lack generic competition because they cannot be measured through traditional in vivo bioequivalence methods and there’s no efficient and convincing bioequivalence test method available.

In these instances, an applicant needs to conduct more extensive clinical endpoint testing to show bioequivalence of a generic drug to a brand-name drug. This can be burdensome and discourage generic product development. A further barrier to generic competition for certain complex drug products is the lack of established methods for showing the sameness of the active ingredient of a proposed generic drug to a brand-name drug for certain complex drugs.

Over the next year, FDA’s generic drug regulatory science program will work to identify gaps in the science and develop more tools, methods, and efficient alternatives to clinical endpoint testing, where feasible. To help with this task, we’re holding a series of important scientific workshops, beginning today, that will identify opportunities for complex generic drug development, discuss quantitative modeling approaches and principles and aid product-specific guidance development. The workshops will also help in the development of new analytical tools that will help overcome the unique development and regulatory challenges for demonstrating active ingredient sameness in complex products. We intend for these efforts to speed product development, reduce development costs, and improve access to these products.

Our announcements today are part of a broader effort by the administration to address the high and rising cost of drugs and in the coming months, we’ll advance other policies aimed at enabling generic competition to complex drugs. Some of these will be product specific guidance documents; others will deal with more crosscutting aspects of our process. And we’ll advance more new policies to help bring more competition to other aspects of the drug market. We’re just getting started. Drug access is a matter of public health concern. We know that enabling more generic competition, where Congress intended, helps reduce prices, enable more access, and improve public health.

Scott Gottlieb, M.D., is Commissioner of the U.S. Food and Drug Administration

Follow Commissioner Gottlieb on Twitter @SGottliebFDA