Listening to Patients’ Views on New Treatments for Obesity

By: Kathryn O’Callaghan and Jeffrey Shuren, M.D., J.D.

The world was a very different place in 1976, when the Food and Drug Administration launched its medical device program.

Kathryn O'Callaghan

Kathryn O’Callaghan, Associate Director for Science and Strategic Partnerships (Acting), FDA’s Center for Devices and Radiological Health

Since Steve Jobs and Steve Wozniak were just that year launching a computer company called Apple, doctors weren’t yet able to view X-ray images or look up drug prescribing information on their iPhones. Moreover, patients couldn’t Google treatments for heart disease, nor were they able to instantly find all open U.S. clinical trials for breast cancer. Not only was patients’ access to health care information much more limited, so was their role in making their own health care decisions.

Doctors diagnosed. Doctors made treatment decisions. Patients followed directions.

It’s different now.

Patients are more empowered today. Driven in part by a need to address emerging or neglected illnesses, such as HIV/AIDS and rare disorders, patients over the past three decades have increasingly banded together, creating organizations that advocated for their interests and generated public awareness of their diseases, their needs, and the lack of effective therapies. This activity produced legions of informed and empowered patients, who today urge us to take a more active role in our own health and urge clinicians to engage patients in shared health care decision-making. Patients are now not only partners in their health care but active consumers who make choices about their doctors, treatments, diagnostics, and health care experiences, an empowerment that is affecting the development of innovative therapies and new clinical solutions.

Today, there are no health care debates, discussions and decisions without considering the patient perspective.

Jeffrey Shuren

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

At FDA’s Center for Devices and Radiological Health (CDRH), we have been systematically involving patients in our regulatory decision making process. Since 1999, CDRH has included a patient representative on each of our advisory panels of outside experts, giving us a better understanding of patient concerns about particular technologies. And in 2012, we began focusing our medical device approval decisions on incorporating the patients’ perspective.

Under this benefit-risk framework for high-risk and innovative, lower-risk medical devices, CDRH’s health care professionals, scientists, and engineers consider the patients’ perspective on both a product’s benefits and their tolerance for any risks when weighing the evidence to determine whether or not to approve a product.

In the past, CDRH experts may have determined that a device should not be approved because its probable risks outweighed its probable benefits. However today, under a patient-centric assessment of risk, if adequate evidence indicates that a subset of well-informed patients with a particular illness or condition would value the product’s benefits more than its risks, CDRH may approve the device for that particular group. However, if we were to approve such a device we may require appropriate product labeling that clearly defines the patient sub-population and their benefit-risk preference. That information would be included in the product’s “Indications for Use” section of the label to ensure that patients and health care practitioners are able to make well-informed decisions.

Better tools are needed to more reliably and scientifically characterize patient preferences about benefit and risks, so we launched our Patient Preferences Initiative, to identify and develop methods for assessing patient valuations of benefit and risk related to specific device types and specific illnesses and conditions.

The goal is to ensure we have sufficient confidence in these methods to rely on them to inform product approval decisions.

Earlier this month, a team of FDA scientists led by Telba Irony, PhD, Chief of General and Surgical Devices Branch in the Division of Biostatistics, published an article in Surgical Endoscopy with leading behavioral economists at RTI Health Solutions, a business unit of RTI International, illustrating how this paradigm can inform medical device approval decisions. The authors successfully tested a new method for capturing patient sentiment and translated it into a decision-making tool for incorporating patient preferences into clinical trial design for obesity treatments. They were able to estimate the tradeoffs in risks that obese patients are willing to accept in exchange for a certain amount of weight loss, and the minimum number of pounds they would have to lose to tolerate the risks of a weight loss device.

Shortly after the study was published, FDA approved a new weight loss device – the Maestro Rechargeable System, an important therapeutic option for obese patients. The decision to approve the device was based in part on the data from Irony’s study that showed a substantial portion of obese patients would accept the risks associated with a surgically implanted device if they lost a sufficient number of pounds. Maestro is the first FDA-approved obesity device since 2007.

Our Patient Preferences Initiative is testing other ways to reach out to patients and capture their views through public workshops, websites, and a new patient-focused advisory committee. CDRH is also participating in related research as a member of the Medical Device Innovation Consortium (MDIC), a non-profit partnership between the FDA, National Institutes of Health, Centers for Medicare & Medicaid Services, and 43 medical device companies, patient groups and other non-profit organizations. MDIC is developing a framework for incorporating patient preferences into the device development and assessment process, and compiling a catalog of methods for collecting patient preference information that can be used to develop, design, and market devices that meet the needs of patients. Simultaneously, CDRH is developing draft guidance outlining how data from patient preference assessment tools can inform device approvals and other regulatory decision making.

As patient groups, industry sponsors, and others conduct more patient preference studies, we will better understand the tradeoffs that patients with medical device-treatable diseases and conditions are willing to make. This research, along with actions taken by CDRH, MDIC and others will drive more patient-centered device development and assessment. As a result, patients will play an influential role in determining which treatments and diagnostics are available in the U.S. market.

It may have taken more than 30 years, but patients are finally having their say.

We should take care to listen.

Kathryn O’Callaghan is Associate Director for Science and Strategic Partnerships (Acting), 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

A CDRH Priority: Clinical Trials in the U.S.

By: Owen Faris, Ph.D., and Jeffrey Shuren, M.D., J.D.

At the Center for Devices and Radiological Health (CDRH), clinical trials are the foundation for our decisions to approve the most important medical devices—products that have the potential to save or sustain life, but that also present the greatest risk to patients.

Owen Faris

Owen Faris, Ph.D., Clinical Trials Director (acting), Office of Device Evaluation in FDA’s
Center for Devices and Radiological Health

Over the past year, we saw several exciting new medical devices reach U.S. patients, including devices to treat heart disease and diabetes and diagnose cancer. Just last week, we approved a new device to treat obesity. None of these products would have come to market without clinical trials.

CDRH is committed to improving U.S. patient access to new devices by strengthening and streamlining the process of testing complex medical devices so that their clinical trials are conducted in the U.S. in a safe, efficient and cost-effective manner. In fact, this is so important for us that we made it one of our three 2014-2015 Center Strategic Priorities, along with striking the right balance between premarket and postmarket data collection and improving our customer service. Please visit our website for an update on our Strategic Priorities.

Innovative medical products begin with clinical trials – and before a clinical trial of a significant risk device begins in the U.S., a researcher, among other things, must apply for and receive FDA’s approval through the Investigational Device Exemption (IDE) process.  The FDA reviews IDE applications to determine whether the sponsor has provided enough information to be sure that the study does not present an unreasonable risk to its participants. FDA takes into account the qualifications of the clinical investigators, information about the device, the design of the clinical investigation, the condition for which the device is to be investigated, and the health status of the participating patients.

Jeffrey Shuren

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

FDA reviews an IDE submission within 30 days, but the review often results in questions which the study sponsor needs to answer, or changes that are needed before the study can be approved. Just a few years ago, it was therefore not uncommon for a year or more to pass before FDA could grant approval to a medical device developer to begin the trial. This type of delay was one factor that led developers to seek approval in other countries.

Over the past year, CDRH has taken a number of actions to expedite the safe initiation of clinical trials in the U.S., and we believe these policies will result in conducting clinical studies in the U.S. earlier in the device development process than was the case in the past.

Our improvements started with establishing a formal Clinical Trials Program within the Office of Device Evaluation. This program provides consistency in decision-making and encourages more interaction between FDA and the device industry during the IDE process. We also provided extensive training to CDRH review staff and the device industry. In addition, we issued numerous guidance documents, including one explaining IDE Decisions and one introducing CDRH’s new Early Feasibility Study program.

We’re excited to report that these changes have greatly shortened the time for an IDE to reach approval, so that a clinical trial can begin. From 2011 to 2014, the median number of days to full IDE approval has decreased from 442 to only 101. This cuts the time it takes to bring a new medical device to market by nearly a full year.

To learn more about CDRH’s clinical trials program, please join us for a webinar on January 22, 2105, where we will discuss the implementation of the IDE processes, our 2015 performance goals, early feasibility studies and our future plans. More information, including how to attend, is on the CDRH Webinar webpage.

The FDA is charged with the enormous task of protecting and promoting the health of the American public. To do this, we must ensure that the medical products on which Americans rely every day have been rigorously tested and are safe and effective. We are committed to making U.S. patients the first in the world to have access to safe and effective medical devices. And we’ve taken the first step to that, by helping ensure that clinical trials take place here, in the U.S.

Owen Faris, Ph.D., is Clinical Trials Director (acting), Office of Device Evaluation in 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

Australia, Brazil, Canada, Japan, and the US: Safeguarding Medical Devices

By: Kim Trautman, M.S.

The FDA and its regulatory counterparts abroad have the weighty responsibility of ensuring the safety of the thousands of regulated medical devices imported in their countries each year. To make this task more manageable, FDA and regulatory agencies in Australia, Brazil, Canada, and Japan embarked in 2014 on a pilot called the Medical Device Single Audit Program (MDSAP). Its goal is to develop a process that allows a single audit, or inspection to ensure the medical device regulatory requirements for all five countries are satisfied, in an efficient yet thorough manner.

Kim TrautmanOn January 1, 2015 the MDSAP pilot reached a major milestone – manufacturers around the globe interested in marketing medical devices in Australia, Brazil, Canada, and the U.S. were invited to participate in the program. This summer, when Japan enters the MDSAP as a full member, the same invitation will be issued also to medical device manufacturers interested in marketing in Japan.

Under this pilot, audits will be conducted by recognized third-party organizations, and medical device regulators in the participating countries will be able to use these inspection reports when making their regulatory decisions. Not only does this program reduce the participating regulators’ need to individually perform routine inspections; it allows them all to have the same reliable information about inspectional findings.

Manufacturers, too, can benefit from the MDSAP pilot by cutting down on the number of regulatory audits they have to host, thereby minimizing manufacturing plant and personnel disruptions. This form of international and standardized oversight lessens the burden on manufacturers by bringing more consistency and transparency to the regulatory process.

The MDSAP pilot does not increase regulatory requirements for medical device manufacturers – the audits cover only existing requirements of the regulatory authorities participating. In many cases, these requirements are already harmonized or very similar to one another, such as the international standard for medical devices quality management systems (ISO 13485:2003), the Brazilian Good Manufacturing Practices (RDC ANVISA 16/2013), the U.S. Quality System Regulation (21 CFR Part 820), and other specific pre- and post-market regulatory requirements of the authorities participating in the MDSAP pilot.

The FDA will accept MDSAP audits as a substitute for routine FDA inspections, typically done every two years for all classes of medical devices and including in vitro diagnostic devices. Pre-approval inspections for devices requiring premarket approval applications (PMAs) and “for cause” compliance inspections will not be part of the MDSAP pilot.

Manufacturers that choose to participate in the pilot program will help to shape the policies and procedures of the fully operational MDSAP, which is scheduled to begin in 2017. We expect that the MDSAP pilot will enhance confidence in third party audit programs, increasing the footprint of this global endeavor.

The FDA is pleased to be part of this MDSAP pilot. International cooperation promotes global alignment of regulatory approaches and technical requirements, expanding the safety net that protects patients world-wide.

New information about how countries will participate in the MDSAP pilot is available on the FDA’s MDSAP pilot web page.  Manufacturers can find additional information on the MDSAP web pageThis MDSAP page provides information on the auditing organizations involved in the pilot for interested manufacturers to contact directly.

Kim Trautman is Associate Director of International Affairs at the FDA’s Center for Devices and Radiological Health

CDER Approved Many Innovative Drugs in 2014

By: John Jenkins, M.D.

Each year, FDA’s Center for Drug Evaluation and Research (CDER) will typically approve more than 100 new medications. A portion of those are novel new drugs, medications that have not previously been approved by FDA and are often among the most innovative products serving previously unmet medical needs or otherwise significantly helping to advance patient care and public health.

John JenkinsThis year, the news media has been concentrating on the number of novel new drugs – either new molecular entities or new therapeutic biologics – approved by CDER in 2014. And that’s understandable because we approved 41 novel drugs this year, the most in nearly 20 years. But instead of looking at the approval tally, we prefer to focus on the significant benefits that many of these drugs bring to patients and the steps that CDER took to get these products to market in a timely manner while maintaining FDA’s standards for safety, effectiveness, and quality.

Many of the 41 new drugs have the potential to add significant clinical value to the care of thousands of patients with serious or life-threatening diseases. They include eight new drugs for treating patients with various types of cancer, four new drugs to treat type-2 diabetes, four new antibiotics to treat serious infections, and two new products to treat patients with hepatitis C.

Moreover, consider these facts:

  • Seventeen (41%) of the 41 novel new drugs were approved to treat rare diseases that affect 200,000 or fewer Americans. This is the highest yearly total of such drugs ever — surpassing the previous high of 13 from 2012. These approvals are particularly significant because patients with rare diseases often have few or no drugs available to treat their conditions.
  • Seventeen (41%) of the 41 novel new drugs are identified by CDER as “First-in-Class,” one indicator of a drug’s degree of innovation. The total for First-In-Class approvals in 2014 approaches the highest yearly total of 20 reported in 2012.

To expedite the development and review of these products, CDER used a number of regulatory programs, including Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval.

  • Fast Track and Breakthrough Therapy designations are designed to speed the development of promising new drugs intended to treat serious conditions with unmet medical needs. Almost half – 19 or 46% of the 41 novel new drugs approved in 2014 — were designated as Fast Track, Breakthrough, or both.
  • Twenty-five (61%) of the 41 novel new drugs were designated for Priority Review. These are drugs in which CDER sees potential for providing a significant advance in medical care, and sets their review target to within six instead of the standard 10 months.
  • Six (20%) of the 41 novel new drugs were approved under FDA’s Accelerated Approval program, which allows early approval of a drug for a serious or life-threatening illness that offers a benefit over current treatments. Accelerated Approval is based on a “surrogate endpoint” or an intermediate clinical endpoint that is thought to be “reasonably likely to predict clinical benefit.” Additional clinical trials are required after approval to confirm the predicted clinical benefit. A surrogate endpoint is a marker of drug effect (e.g., an effect on a lab value or tumor size) that does not directly represent an improvement in how a patient feels or functions, but is expected to predict such a benefit.

Here are a few other ways we assess our contributions to last year’s approvals:

  • Under the Prescription Drug User Fee Act (PDUFA), sponsors pay fees when they submit a product application. This money is used to provide FDA with additional resources to meet performance goals, such as a goal date for completing its review of the application. In 2014, CDER acted on or before the PDUFA goal date for 40 (98%) of the 41 novel new drugs approved.
  • CDER approved more than three-quarters — 32 (78%) — of the 41 novel new drugs on the “first cycle” of review, meaning without requests for additional information that would delay approval and lead to another cycle of review.
  • Nearly two-thirds of the novel new drugs – 26 (63%) — were approved in the U.S. before approval in another country.

It’s been another strong year for approval of novel new drugs for patients in need. We are proud of our role in helping to safely and efficiently bring important new medications to the American public.

Our Novel New Drug Summary for 2014 provides more details. A current list of CDER’s 2014 novel new drug approvals is available on our Web site.

John Jenkins, M.D., is Director of the Office of New Drugs in FDA’s Center for Drug Evaluation and Research

Another important step in FDA’s journey towards enhanced safety through full-scale “active surveillance”

By: Janet Woodcock, M.D.

They say the longest journey begins with a single step. In 2008, FDA launched the Sentinel Initiative and thus began a long journey toward the challenging goal of developing a full-scale medical product safety monitoring program using an important scientific technique called “active surveillance,” which complements our FDA Adverse Event Reporting System (FAERS). FAERS is already well developed and uses the equally important technique of “passive surveillance.” Today, I’d like to recognize our progress along the way.

Janet WoodcockAfter a successful five-year pilot program, which began in 2009, FDA’s Mini-Sentinel program is now transitioning, as planned, to the full-scale Sentinel System. I’d like to share with you the success of our Mini-Sentinel pilot program and some of FDA’s visions for our new leg of the journey toward full-scale “active surveillance” under the new Sentinel System.

First, a quick discussion of the importance of “active surveillance”: Over many years, FDA’s program that we now call FAERS has been our main tool for assessing the safety of medical products. This system relies on patients, medical professionals, and product manufacturers to report to us potential safety issues of the products FDA regulates.

FAERS is an invaluable asset, and we’re not seeking to replace it. However, the Sentinel System offers us the exciting possibility of not waiting for safety information to come to us in the form of reports, but rather it enables us to go out and get that information, adding greatly to our safety monitoring capability. This is active surveillance.

Over the past five years, the Mini-Sentinel pilot program has established secure access to the electronic healthcare data of more than 178 million patients across the country, enabling researchers to evaluate a great deal of valuable safety information. While protecting the identity of individual patients we can get valuable information from Mini-Sentinel that helps us better understand potential safety issues, and share with you information on how to use medicines safely. We have used Mini-Sentinel to explore many safety issues, helping FDA enhance our safety surveillance capabilities, and giving us valuable input in decision-making on drugs and vaccines.

We’re now well on our way to developing a nationwide rapid-response electronic active surveillance system, Sentinel System, for monitoring the safety of FDA-regulated drugs and other medical products.

So where does our journey take us from here?

  • FDA will build on the successes of the Mini-Sentinel Pilot. We have a variety of safety assessments ongoing under Mini-Sentinel that will continue and we will seek to expand our reach and capabilities with the Sentinel System;
  • Mini-Sentinel gave us an important start, but it is essential to continue to develop and refine existing scientific methods to evaluate the data we access through the Sentinel System;
  • We see Sentinel as a potentially valuable national resource for other safety researchers, besides those at FDA. Looking even further ahead, our hope is that, working with other scientific groups, we will be able to create a National Data Infrastructure that would enable other users (e.g., other governmental agencies, researchers from academia or industry) to access the Sentinel infrastructure for multiple purposes (e.g., medical product research, quality improvement);
  • Not only will such access directly serve the public health, it will also help sustain these programs because stakeholders will have an incentive to provide support (financial and otherwise) for its maintenance and growth.

From the outset, the goals of the Sentinel Initiative have been large and of ground-breaking scale. We knew it would be years in the making, but Mini-Sentinel’s successful completion marks important progress. We look forward to continuing and expanding our active surveillance capabilities as we now transition to the full-scale Sentinel program.

Janet Woodcock, M.D., is the Director of FDA’s Center for Drug Evaluation and Research

Together: A Food Safe America

By: Michael R. Taylor

I recently had the pleasure of speaking at the Consumer Food Safety Education Conference convened by the Partnership for Food Safety Education (PFSE). The conference brought together food safety educators from across the country – people in state and local health departments, universities, extension services, and food businesses who are working every day on the front line, with consumers, to reduce food safety risks by improving consumer food handling practices.

Michael R. Taylor is FDA’s Deputy Commissioner for Foods and Veterinary Medicine.The theme of the conference was “Together: A Food Safe America” – a theme that captures so well the sense of community, high purpose and energy that were present so abundantly at the conference. I shared the podium with two good friends and colleagues representing key FDA partners on food safety – USDA’s Acting Under Secretary for Food Safety Brian Ronholm, and Joe Corby, the Executive Director of the Association of Food and Drug Officials, which represents state and local food safety officials.

We regulators have a responsibility, through our oversight of the food industry, to do everything we reasonably can to make sure that the foods consumers bring into their homes are as safe as they can be. We are doing this by building into our food safety standards and compliance programs modern concepts and techniques for preventing the contamination that can make people sick.

Under the FDA Food Safety Modernization Act (FSMA), we at FDA have a new mandate to build a farm-to-table system of prevention, encompassing work that must be done to make food safe at four major stages of the commercial food system. These pillars of prevention include:

  • Production of produce on the farm,
  • Practices in food processing and storage facilities,
  • Transportation of food, and
  • Practices in grocery stores and restaurants.

But there’s a fifth pillar of prevention, and that’s the consumer. We all know that, even with the best of efforts by commercial food producers and handlers, consumers still must play a crucial role in preventing the introduction and spread of contamination – by keeping their hands and food surfaces clean, by keeping raw meat and produce separate, and by being sure to cook food to proper temperatures and chill food through prompt refrigeration.

It seems like common sense – and the basic ideas are – but food safety educators know that it’s far from simple to provide consumers the information, tools, and motivation they need to turn common sense into sustained behavior change. But they are out there, every day, doing the hard work.

We in government and the food industry need to better support our food safety educators.  FDA, USDA, and the Centers for Disease Control and Prevention (CDC) do some good work on food safety education. For example, at FDA, our current programs include targeting groups and individuals who are especially vulnerable to foodborne illness and partnering with the National Science Teachers Association to incorporate food safety into the science curriculum at the middle and high school levels. But there is more we can do to support food safety educators at the front line, in their daily work with consumers in clinics, in schools and in communities – where most of the food safety education, and all of the behavior change, takes place.

At FDA, we will be building food safety education into our risk-based priority setting paradigm, which means documenting better the contribution that education makes to reducing risk, evaluating what works to sustainably improve consumer practices, and targeting resources where they will make a real difference. Federal food safety agencies – and their finite resources – are overwhelmingly focused on the congressional mandate to prevent hazards arising from the commercial supply chain, which makes sense: that’s what we regulate. But, backed up by the right analysis, we can effectively target and increase our investment in consumer education in ways that will make a real difference for public health.

But the federal government can only do so much. And that’s where PFSE comes in. The Partnership brings together government, industry and consumer leaders to pool their expertise, share their perspectives, and collaborate on the hard work of food safety education. I applaud and thank the consumer groups, food companies, and trade and professional associations that are contributing their time, creative energy and resources to the work of the Partnership. And I salute the PFSE’s Executive Director Shelley Feist for her leadership and her sustained commitment to food safety and consumer education.

Working hard, and working together, we can have a Food Safe America.

Michael R. Taylor is FDA’s Deputy Commissioner for Foods and Veterinary Medicine

FDA’s Janet Woodcock, M.D., recognized by the Institute for Safe Medication Practices: Receives Lifetime Achievement Award for her career in public service

By: Margaret A. Hamburg, M.D.

FDA’s mission is to protect and promote the health of the American public. The FDA employees who dedicate their careers to this worthy goal do so not for personal reward or public recognition but because of an extraordinary commitment to improving public healthcare. Which is why it is even more special when these employees receive public acclaim.

Margaret Hamburg, M.D.

Margaret A. Hamburg, M.D., Commissioner of the Food and Drug Administration

One such individual is Dr. Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research (CDER). Janet recently was awarded the Institute for Safe Medication Practices (ISMP) Lifetime Achievement Award, recognizing “an individual who has had a significant career history of making ongoing contributions to patient safety and has had a major impact on safe medication practices.” This award is well deserved.

During her nearly 30 year career with FDA she has served in several different capacities in addition to her current position, including Director of the Office of Therapeutics Research and Review in the Center for Biologics Evaluation and Research (CBER) and as FDA’s deputy commissioner and chief medical officer. Throughout her career, Dr. Woodcock has helped the Agency elevate and transform its approach to medical product safety, personally leading the way on many key safety initiatives from their beginning to implementation.

During her distinguished career Dr. Woodcock:

  • Conceived and oversaw creation of the Adverse Event Reporting System (AERS) system, to manage the increasing number of spontaneous reports of adverse drug reactions submitted to FDA;
  • Co-led the FDA Task Force on Risk Management, one of the Agency’s first efforts to clearly delineate pre- and postmarket safety surveillance and management of medical product risks;
  • Chaired the Council on Pharmaceutical Quality, launched in 2007, presaging many of the Agency’s subsequent safety initiatives;
  • Led the launch of Safety First, a program created to help ensure alignment between premarket drug safety review and postmarket surveillance;
  • Led the creation of the Sentinel Initiative, a data-driven national system that allows active—close to “real time”—safety surveillance using electronic data from healthcare information holders;
Janet Woodcock

Janet Woodcock, M.D., Director of FDA’s Center for Drug Evaluation and Research

Through all of these accomplishments, and many others, Janet Woodcock has helped ensure that FDA can fulfill its mission effectively. She has championed the use of innovative new tools and approaches, and she has forged and enriched many partnerships with industry, academia, healthcare providers, patients and colleagues in government, including across the FDA.

Her work has helped lead FDA into a new century, an extraordinary time of transformation and opportunity in medical science. With these changes, FDA’s responsibilities have also grown enormously. We must continue to ensure that our capabilities for drug product evaluation, oversight, and regulation keep pace with these developments. Thanks to Janet’s vision and hard work, along with many of her colleagues at FDA, I am confident that FDA is up to the task.

I want to thank Dr. Woodcock for her years of dedicated service to the American public, and congratulate her on this most recent recognition of her many contributions.

Margaret A. Hamburg, M.D., is Commissioner of the Food and Drug Administration

2014 Drug Approvals: Speeding Novel Drugs to the Patients Who Need Them

By: Margaret A. Hamburg, M.D.

Preliminary data announced earlier today shows that 2014 is shaping up to be another strong year for novel drug approvals, which is certainly good news for many patients and their families.

Margaret Hamburg, M.D.With a few weeks left in December, our Center for Drug Evaluation and Research (CDER) has so far approved 35 novel new drugs in 2014 compared to 27 in 2013. These numbers include both new molecular entities (NMEs), submitted to CDER in New Drug Applications (NDAs) and new therapeutic biologics submitted to CDER in Biologics License Applications (BLAs).

But the numbers don’t tell the full story. What really matters is that many of these new products offer significant clinical value to the care of thousands of patients with serious and life-threatening diseases. That’s certainly the case for patients with rare diseases that affect 200,000 or fewer Americans. So far this year we’ve reached a milestone with a record 15 approvals for rare diseases. The previous high was 13 drugs in 2012. These results are all the more significant because patients with rare diseases often have few or no drugs available to treat their conditions.

And here’s another point of interest – to date, 15 of the approvals have been first in their class drugs, another indicator of their potentially strong clinical impact.

To ensure that 2014’s novel drugs get to patients as quickly as possible, CDER effectively employed a variety of regulatory tools including FDA’s expedited development and review programs – fast track, priority review, accelerated approval and our new breakthrough therapy designation. Early and repeated communications with sponsors have also been helpful in speeding these products to market.

Consider for example, Blincyto, approved just last week to treat Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukemia. CDER employed all of its expedited review programs to help get this drug to market as early as possible, five months ahead of its review goal date. The sponsor also benefited from incentives for drugs that treat rare diseases.

Another example is Harvoni, the first combination pill approved to treat chronic hepatitis C virus genotype 1 infection and the first approved regimen that does not require administration with interferon or ribavirin. With this and other recent approvals, we are helping to change the treatment paradigm for patients living with hepatitis C. Harvoni received breakthrough therapy designation and was assigned priority review.

One of the more challenging areas of drug development has been the rather barren field of antibacterial drugs. Among our 2014 approvals to date are three new antibacterial drugs – Dalvance, Sivextro and Orbactiv—to treat skin infections, specifically acute bacterial skin and skin structure infections (ABSSSI). These drug approvals represent a welcome but modest increase in activity in this product area. Prior to 2014, only five new systemic antibacterial drugs were approved during the period from 2004 – 2013.

I want to congratulate the management and review staff at CDER for these very impressive preliminary numbers. Thanks in large part to CDER’s hard work and dedication, 34 of the 35 drugs approved so far in 2014 were approved before or on their Prescription Drug User Fee Act (PDUFA) review goal date and 23 of the 35 drugs were available to patients in the United States before they were available to patients in Europe.

In this holiday season of joy and reflection, we have much to be grateful for in the work that CDER does every day on behalf of patients.

Margaret A. Hamburg, M.D., is Commissioner of the Food and Drug Administration

Implementing the Unique Device Identifier System into health care systems is critical for reaching its potential to benefit public health

By: Jeffrey Shuren, M.D., J.D.

As the FDA works with manufacturers to launch a new system of identifying medical devices using standard bar codes and numbers, we look forward to the day when the system, called the Unique Device Identifier (UDI) system, will be fully set up— with identifiers on device labels and a corresponding database of identifying information about most of the devices in the U.S. marketplace.

But why does that matter?

Jeffrey ShurenMuch like vehicle identification numbers (VINs) for automobiles, UDIs are intended to streamline the monitoring of devices, improve safety tracking and recall efficiency, and even make it easier to evaluate device performance over time. So while there’s little doubt that UDI can improve patient safety, modernize how we evaluate devices once they are in use, and facilitate future device innovation, these benefits will only become a reality when the UDI system is adopted and integrated into the health care system—when hospitals, doctors’ offices, patient registries, heath care insurance companies, and others incorporate UDI as part of their standard electronic health information systems.

Without the practical implementation on the clinical side, UDI will be codes and a database with limited utility to improve patient care or reach its other critical goals.

The FDA is thinking about this now—not later. While going full steam ahead to fulfill our responsibility for implementing UDI regulations for medical device manufacturers, we are doing everything we can to promote the widespread adoption of UDI in the U.S. health care system.

We commissioned the Brookings Institution to create a “roadmap” for provider systems, patients, payers, supply chain personnel, and many others, to adopt and utilize UDIs. This report, released on Friday, December 5, provides 17 recommendations for adopting UDIs across three major intersections of the health care system—providers (e.g., electronic health records, hospital inventory management, billing records); administrative transactions (e.g., claims data and payment information); and patient-directed tools (e.g., mobile apps and public awareness campaigns).

We’re working hard to create and populate an efficient and useful UDI system for medical devices. But even the perfect system will fail to improve patient care if it’s not properly integrated into electronic health information systems. That process has to start now.

Today, we are co-sponsoring with Pew Charitable Trusts and the Department of Health and Human Services Office of the National Coordinator (ONC) a meeting where some 400 experts are convening to discuss changes that are needed to store and share UDI information throughout the health care system, with the ultimate goal of improving patient care.

The goal is to have the UDI system not only up and running—but actually used as the key to unlock important data that can help patients.

But how does such a system really help patients and the providers who care for them? Consider a possible scenario where the connections made via UDI could make an important difference in patient care.

A patient undergoing knee surgery—we’ll call him John—has the UDI of his knee implant scanned and electronically recorded into his clinical record.

When John is discharged, he can also register the UDI into his personal health record (PHR), available from his provider, through a variety of mobile apps that can enable two-way communication with his provider.

Having the UDI recorded will help John to know if safety alerts apply to his specific implant. It will also help him accurately report any potential adverse event to the provider, the FDA, or the manufacturer, with the confidence that the UDI ensures that all parties know what the type of device may be causing John—and possibly other patients—problems. Importantly, if John hears about knee implants being recalled, he will be able to quickly pinpoint, by using his UDI, if his particular type of implant is involved in that recall. If it’s not, John may avoid needless anxiety; if it is, he can take any necessary action, such as following up with his orthopedic surgeon.

The UDI from John’s surgery is also available to be transmitted to a total joint replacement registry, without any of his personal information. Data from the registry may then be used to support the development of innovative implants and reduce the data requirements for — or replace altogether — postmarket studies conducted by the device manufacturer to demonstrate long-term performance.

The possibilities of UDI are exciting—better and more precise information can lead to better care and better awareness of how medical devices work in the general population. The FDA is working to set up the system, but implementation and integration are critical. The question is—if we build it, will people adopt it?

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

From Wariness to Welcome: Engaging New England on Food Safety

By: Michael R. Taylor

This is the first of two FDA Voice blogs about state listening sessions on updates to four of the rules proposed to implement the FDA Food Safety Modernization Act (FSMA).

What a difference a year makes.

In August last year, my team and I visited New England to talk about the rules proposed in 2013 to implement FSMA. We were met with skepticism and some genuine fear that our produce safety proposals did not take full account of local growing practices and would both disrupt traditional practices and deter innovation. These weren’t easy conversations, but they proved instrumental in FDA’s decision to propose—on Sept. 29, 2014—updates, or supplements, to four of the proposed FSMA rules overseeing human and animal foods, both domestic and imported. These proposals include significant changes in the produce safety proposal and related elements of the preventive controls rules for food facilities.

Michael R. Taylor is FDA’s Deputy Commissioner for Foods and Veterinary Medicine.We weren’t quite sure what to expect when we flew to Vermont on Sunday, November 16, for a listening session the next day on the proposed supplemental rules. But the tenor of this visit was dramatically different, and very positive, beginning with the detour we took from our FSMA mission on Sunday to visit leading players in Vermont’s local food movement and artisanal cheese-making community.

Accompanied by Vermont Agriculture Secretary Chuck Ross, we first toured the Vermont Food Venture Center (VFVC) in Hardwick, a regional food hub that leases space to small food businesses, providing kitchen equipment, food storage and business consultations. The goal of this modern, well-equipped facility, as executive director Sarah Waring explained, is to strengthen Vermont’s local food network and agricultural economy.

We then toured Jasper Hill Farm in Greensboro, a renowned maker of artisanal cheeses.  We were welcomed by brothers Mateo and Andy Kehler, who have taken an innovative approach to making cheese, using both traditional methods and the latest technology. Their goal is to establish a network of local farms that supply the milk, with Jasper Hill aging and distributing the cheeses in an effort to support small dairy operations.

Our goal was to continue the dialogue we started this year with the cheese-making community to better understand, as food safety regulators, what goes into making artisanal cheeses. We learned a lot, tasted some great cheese, and left impressed by the community-oriented commitment at both VFVC and Jasper Hill Farm, and by their use of top-tier tools to strengthen Vermont’s local food system.

When we arrived back in Montpelier Sunday night, the setting was like something out of a postcard. This picturesque town, the nation’s smallest state capital, was dusted in the season’s first snow, which only accentuated its natural beauty and charm. We were happy to be there.

Monday morning we drove to the Vermont Law School in South Royalton for the FSMA listening session. This school, set in the rolling landscape of rural Vermont, is renowned for its commitment to sustainable environmental practices.

We saw familiar faces. Some had come to the meeting directly from their farm—through the snow. There were people from all over the Northeast—people who had participated in our series of listening sessions throughout New England in 2013. But this time, the response and dialogue were different. We heard acknowledgement and appreciation that we had addressed many of their concerns in our revised proposals by making the proposed rules more feasible, while still meeting our public health goals.

Much of the discussion focused on implementation of the rules, and, interestingly, some of the concerns echoed those we had heard in a November 6 listening session in Sacramento, CA, a place not only on the opposite side of the country but so different in its production systems. Many are finding the complexity of the proposed rules daunting, such as the technical underpinnings of the E.coli benchmark for water quality and the various boundary lines and exemptions that determine who is covered. We’ve always said that we wouldn’t take a “one size fits all” approach, which has contributed to making the rules more complicated. This only underscores our responsibility to explain the rules clearly and to provide education, technical assistance and guidance.

Secretary Chuck Ross said early and often that we need to educate before and as we regulate. And he’s right. I am struck anew by the importance of our partnerships with state leaders. Vermont’s Ross and California Secretary of Food and Agriculture Karen Ross have been invaluable in helping us develop these rules, as they will continue to be as we move towards implementation.

We were grateful for the participation in the listening session by food safety advocates Lauren Bush and Gabrielle Meunier, who each spoke of the devastating effects of foodborne illnesses. Lauren almost died after eating a salad contaminated by E.coli in 2006 and Gabrielle’s young son fought, and recovered from, a Salmonella infection in 2008 after eating tainted peanut butter crackers. Their stories underscore the underlying reason for the effort that so many are making to implement FSMA—to keep people safe.

Some participants expressed the view that even though we decided to defer, pending further study, our decision on an appropriate interval between the application of raw manure and harvest, some kind of interval is needed to protect crops from pathogens. Some suggested that the 90 to 120-day intervals set forth by the U.S. Department of Agriculture’s National Organic Program be adopted as an interim measure.

Others inquired how the FSMA rules would affect them based on very individual scenarios. We asked them, and we’re asking everyone, to comment on the supplemental rules and include those scenarios for us to consider in drafting the final rules. We don’t want to create unintended harmful consequences.

The deadline for commenting on the four supplemental rules for Produce Safety, Preventive Controls for Human Food, Preventive Controls for Animal Food and Foreign Supplier Verification Programs is Dec. 15. Visit our FSMA page on fda.gov for more information.

Our Vermont trip was followed by state listening sessions in Georgia, North Carolina and Florida. I will be filing another FDA Voice blog on what we learned in those Southern states.

Michael R. Taylor is FDA’s Deputy Commissioner for Foods and Veterinary Medicine