Balancing the Prevention of Opioid Abuse and Appropriate Pain Management

By: Margaret A. Hamburg, M.D.

Earlier today I had the opportunity to address the Rx Abuse Summit in Atlanta, Georgia, an important three day conference that brings together local, state and federal agencies, business, academia, clinicians, treatment providers, counselors, educators, state and national leaders, and advocates to share ideas and strategies for reducing the growing number of tragedies resulting from prescription drug abuse.

Margaret Hamburg, M.D.For FDA, stemming the rise in prescription opioid misuse, abuse, addiction, and overdose is an issue of the highest priority. The issue is complicated by the need to balance it with another major public health priority: managing the pain that affects an estimated 100 million Americans.

This balance goes to the heart of FDA’s mission to protect and promote the public health — and to our role as drug regulators. FDA has a responsibility to demand the best science and data to uphold its high standards to ensure a proper benefit/risk profile for these drugs, and to foster discussion and research that leads to a better understanding of pain and pain drugs.

FDA shares the concerns about safe and appropriate use of all prescription opioid analgesics and the public health consequences associated with misuse and abuse of these drugs. I encourage you to read my remarks from today, in which I detail the many actions FDA has taken to address prescription opioid abuse, targeting key drivers of the problem.

Nothing can erase the tragedy that so many people have had to face as a result of the misuse, abuse and addiction of opioids. But progress to reduce and prevent our nation’s prescription drug crisis is attainable through a comprehensive and collaborative approach. For our part, FDA is committed to reducing abuse of opioids and ensuring appropriate access to pain medicines for patients in need.

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

Collaboration and Medical Countermeasures: Furthering Regulatory Science

By: Dr. Stephen Ostroff

“I hope no one ever needs these products,” isn’t something you hear too often, particularly from those developing drugs, biologics, or devices. That point of view—and a universal desire to protect the American public—is what sets biodefense professionals apart.

Stephen OstroffWithin FDA, our Medical Countermeasures initiative (MCMi) does things a little differently as well. Managed by the Office of Counterterrorism and Emerging Threats, in the Office of the Chief Scientist, MCMi thrives on collaborations. We work proactively with industry, academic and federal research centers, FDA product centers and offices, and other government entities in our mission to help protect the U.S. from chemical, biological, radiological, nuclear, and emerging infectious disease threats.

Collaborating is particularly important to advancing regulatory science: developing the tools, standards, and approaches needed to evaluate FDA-regulated products—in this case, medical countermeasures—for safety, efficacy, quality, and performance. When it comes to developing the necessary data for regulatory decisions, medical countermeasures often present unique and complex challenges since the diseases they target rarely occur naturally.

To support regulatory decision-making for medical countermeasures, FDA manages a robust research portfolio under the MCMi Regulatory Science Program. Working with other federal agencies to ensure alignment with U.S. priorities, we fund both intramural and extramural research programs to drive innovation in regulatory science.

Ongoing research projects include:

  • Developing models of radiation damage in lung, gut, and bone marrow organs-on-chips, and using these models to test candidate medical countermeasures to treat such damage;
  • Mapping immune responses to certain biothreat agents and medical countermeasures in humans and animal models to create species-specific immune function maps;
  • Assessing the feasibility of using electronic health record systems to conduct near real-time monitoring of health outcomes, including serious or unexpected adverse events associated with medical countermeasures used during public health emergencies.

For those interested in these and other medical countermeasure-related regulatory science developments, FDA will be hosting our 2014 MCMi Regulatory Science Symposium at FDA headquarters in Silver Spring, Maryland, June 2-3, 2014. The meeting is free and open to the public (pre-registration required).

We welcome poster abstract submissions until April 27, 2014, in topic areas including: animal models, biomarkers and correlates of protection, devices, diagnostics, emergency communication, new technologies, product quality, and surveillance.

I also encourage you to read more about MCMi in action in our Fiscal Year 2013 Program Update.

Stephen Ostroff, M.D., is FDA’s Acting Chief Scientist

FDA Works with China to Ensure Medical-Product Safety

By: Christopher Hickey, Ph.D.

Americans benefit greatly from medical products produced by other countries. Approximately 40 percent of finished drugs in the United States come from overseas, as well as more than 50 percent of all medical devices. About 80 percent of the manufacturers of active pharmaceutical ingredients are located outside the United States.

Christopher Hickey

Christopher Hickey, Ph.D., testifies April 3, 2014.

However, this rapid globalization of commerce presents challenges to regulators who oversee the safety and quality of medical products. Many of these challenges manifest themselves in China. As FDA’s country director for the People’s Republic of China, I testified on April 3, 2014 before the U.S.-China Economic and Security Review Commission, an advisory panel created by Congress, on our work to ensure the safety and quality of medical products produced in China and imported into the United States.

China is the source of a large and growing volume of imported foods, medical products and ingredients. In the years spanning fiscal years 2007 and 2013, the total number of shipments of FDA-regulated products from China to the United States almost quadrupled.

The challenges we see in China mirror those we see in other countries with developing regulatory systems. These issues include problems with data integrity, inadequate implementation of quality systems in manufacturing, and inconsistent regulatory oversight, among others.

As China’s role on the global stage expands, FDA has significantly increased drug and medical device inspections there, but we need to continue to strengthen our efforts. FDA is currently working to use Congressionally-appropriated funding to increase from eight to 27 the number of U.S. staff it posts in China. Visa issues that arose with the Chinese government over new FDA staff assigned there were addressed during Vice President Joe Biden’s visit to Beijing in December, and FDA continues its work to post new staff in Beijing in the coming months.

FDA recognizes that strategic engagement in China starts first and foremost with Chinese regulators. China’s Food and Drug Administration, or CFDA, is responsible for the regulation of food, drugs, and devices for domestic distribution in China, and for regulation of certain exported drugs and medical devices.

Senate Committee Hearing, April 3, 2014

Christopher Hickey, Ph.D., testifies before the U.S.-China Economic and Security Review Commission.

FDA has established a strong working relationship with CFDA. Our office has trained hundreds of Chinese inspectors in areas that include inspecting for good manufacturing practices and assessing the quality of data from sites that conduct clinical trials. Experts from FDA’s Center for Devices and Radiological Health now meet regularly with their counterparts from CFDA under the auspices of the International Medical Devices Regulatory Forum. These investments will pay long-term dividends for the American people: a stronger Chinese regulatory system can only strengthen FDA’s efforts to promote and protect U.S. public health.

Finally, in the area of inspections and enforcement, CFDA inspectors now regularly observe FDA inspections in China. And since 2012, FDA’s Office of Criminal Investigations has worked closely with CFDA to fight against Internet-based, illegal distribution into the U.S. of falsified, counterfeit and adulterated drugs.

FDA’s priorities in China match its global priorities: we work to ensure the safety and efficacy of FDA-regulated products. Manufacturers are best situated to make certain that appropriate processes are in place to ensure safety and quality in production. Regulatory bodies should hold companies accountable for lapses in the production process. Inspections and testing are important tools in that process, but they must be used as part of a larger system that emphasizes a preventive, approach to the production of safe, effective, high-quality medical products.

And in our globalized world, it’s increasingly important that regulatory partners work together to ensure the safety of products as they move through increasingly complex supply chains. Patients and consumers – whether in Beijing or Boston – deserve no less.

Christopher Hickey, Ph.D., is FDA’s Country Director for the People’s Republic of China.

For more information, please visit this Web link:

China’s Healthcare Sector, Drug Safety, and the U.S.-China Trade in Medical Products

Marsha Henderson Leads Programs to Improve Health Outcomes for Women

By: Kimberly A Thomas, MPH

Kimberly ThomasI have worked with many clinicians and researchers whose dedicated efforts have helped to improve the health care women receive. FDA’s Marsha Henderson stands out among this distinguished group. For over 30 years, Marsha has led research and educational programs that help improve medical treatments for women and provide women with the resources they need to make informed health decisions.

For these efforts, Marsha was recently awarded the first Dr. Estelle Ramey Award for Women’s Health Leadership from the Society for Women’s Health Research. This award was created to recognize leaders like Dr. Ramey who demonstrate exemplary leadership in women’s health and a commitment to the study of the impact of sex differences on health.

Marsha was honored for her history of serving as a champion for women’s health, and for her leadership of FDA’s Office of Women’s Health. As the assistant commissioner for women’s health, Marsha directs research that helps FDA better understand how sex differences affect the safety and effectiveness of medical treatments.  At FDA, Marsha has also developed outreach programs that make sure that women from diverse communities have access to easy-to-read FDA health and safety information.

Marsha Henderson with award

Marsha B. Henderson, FDA Assistant Commissioner for Women’s Health, with FDA Commissioner Margaret A. Hamburg, MD (left) and Phyllis Greenberger, President & CEO of the Society for Women’s Health Research (right).

When I first came to the FDA in 2004, Marsha immediately showed me how the work we do at FDA can have a positive impact on women’s lives. Her leadership motivated me to find new ways to promote scientific discussion and expand educational outreach.

When Marsha accepted her award she not only acknowledged the tremendous personal honor, but she also recognized all of the scientists, health educators, and general staff at FDA whose daily work helps to improve the outcome of medical treatments for women. Her comments demonstrated why she is so deserving of this leadership award. I congratulate her on this honor and thank her for distinguished career serving as a voice for all women.

Kimberly A Thomas, MPH, serves as a senior advisor for communication and outreach in FDA’s Office of Women’s Health.

FDA Seeks Comment on Proposed Health IT Strategy That Aims to Promote Innovation

By: Bakul Patel

Health information technology (IT) offers many benefits to the American people and health care providers. Health IT products, technologies and services can prevent medical errors, improve efficiency and health care quality, reduce costs and increase consumer engagement. They also can help with identification of, and quick response to, public health threats, and further health research. But while health IT benefits are far-reaching, technology can bring risks to patients if not designed, developed, implemented, or maintained properly.

Bakul PatelI’m glad to share that FDA, along with the Office of the National Coordinator for Health Information Technology (ONC), and the Federal Communications Commission (FCC), has recently released a report outlining our proposed framework for health IT. This report fulfills the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA) requirement that we develop a proposed strategy and recommendations on an appropriate, risk-based regulatory framework pertaining to health IT that promotes innovation, protects patient safety and avoids regulatory duplication.

We’ve developed a proposed framework that fulfills this requirement and would allow Americans to reap the benefits of health IT. This is a growing field with many benefits. For example, electronic health records allow providers to access accurate patient data. And computer-aided detection software can analyze an electrocardiogram (EKG) signal and help determine if a person is having a heart attack, helping providers give timely treatment.

In health IT, the best approach is a risk-based approach. We believe risk assessment should primarily focus on the function of the health IT product, not its platform. So we’ve identified three categories of health IT. The first is administrative and relates to functions such as billing and scheduling. This area has minimal risks for patient safety and does not require additional oversight.

The second relates to health management functions, including provider order entry, electronic communication and patient identification. In this category, we’ve identified four priority areas that can be scaled and applied throughout the health IT product lifecycle:

•              Promote the use of quality management principles;

•              Identify, develop, and adopt standards and best practices;

•              Leverage conformity assessment tools; and

•              Create an environment of learning and continual improvement.

Since safety risks for products in the health management category are sufficiently low, even if a technology in this category meets the definition of a medical device, FDA does not intend to focus oversight on it. Rather, FDA intends to focus our attention on the third category, which relates to medical device functions, such as computer-aided detection software and radiation treatment software. Such products are already FDA’s focus because they generally pose greater risk to patients than the products in the other two categories.

We’re not recommending that new areas of FDA oversight are needed.

Along with ONC and FCC, we will continue to develop this framework through public engagement, especially in fostering the further development of a quality-focused culture for health IT. We do not believe that regulation should be, or needs to be, the first approach used to reach this outcome. The agencies are holding a three day public workshop on May 13, 14, and 15 at the National Institute of Standards and Technology to discuss the framework and its components. We encourage consumers, providers, and health care organizations to register for the workshop and to submit comments. Health IT has brought, and continues to bring, many benefits and we are looking forward to engaging with you on this important topic.

Bakul Patel is senior policy advisor in FDA’s Center for Devices and Radiological Health.

For more information please visit these Web links:

FDASIA Health IT Report

Public Workshop – Proposed Risk-Based Regulatory Framework and Strategy for Health Information Technology, May 13-15, 2014

Proposed Risk-Based Regulatory Framework and Strategy for Health Information Technology Report; Notice to Public of Availability of the Report and Web Site Location; Request for Comments

Creating a New System to Improve the Security of the Drug Supply

By: Ilisa Bernstein, Pharm.D., J.D.

FDA is committed to protecting consumers from potentially dangerous drugs, including those that are counterfeit, stolen or tainted. We are taking steps to create a system that will help identify and trace certain prescription drugs as they are distributed within the United States.

Ilisa BernsteinThe Drug Supply Chain Security Act (DSCSA), signed into law last November, outlines the path to building this electronic tracking system. We are working now to develop standards for this new system and we can’t do it alone. Stakeholders – including drug manufacturers, wholesaler distributors, repackagers, and many dispensers (mainly retail and hospital pharmacies) – will be working with us.

The ultimate goal is to identify each individual prescription drug package in a way that will enable rapid and accurate verification of the legitimacy of the product, which will be an important tool in the fight against counterfeit drugs. If a counterfeit or other harmful drug is discovered in the supply chain, we can immediately notify patients, health care professionals, hospitals and others about the public health risk.

This system, which will include a history of the transactions involving each drug product, will also help to enable more efficient recalls to remove drugs from the market that are considered potentially dangerous.

It will be an “interoperable” system, meaning that different stakeholders in the drug supply chain will be able to communicate with each other and share information about the drug and its location.

We have posted our implementation plan at fda.gov and have opened a docket in the Federal Register with questions that we’d like these stakeholders to answer about any practices or systems they may be using to exchange information related to prescription drug transactions, in paper or electronic format. The docket is open until April 21, 2014.

We will be reviewing all information that comes into the docket as we work on establishing standards for the interoperable exchange of tracing information. On May 8 and 9, 2014, FDA is hosting a public workshop for interested parties to discuss what standards may work, what makes sense and what can be done quickly. We encourage you to participate.

Time is of the essence because the law requires FDA to issue a draft guidance document with initial standards by Nov. 27, 2014, and for many stakeholders to establish systems and processes that will enable them to comply by Jan. 1, 2015.

Experts from all over FDA are working to implement this important law. Provisions of this law will enhance FDA’s ability to help protect consumers from exposure to drugs that may be counterfeit, stolen, contaminated, or otherwise harmful through improved detection and removal of potentially dangerous drugs from the drug supply chain. U.S. consumers deserve safe, effective and high-quality medications.

Ilisa Bernstein, Pharm.D., J.D., is Deputy Director of the Office of Compliance in FDA’s Center for Drug Evaluation and Research

For more information please visit the following Web links:

Drug Supply Chain Security Act (DSCSA) Implementation Plan

Standards for the Interoperable Exchange of Information for Tracing of Human, Finished, Prescription Drugs, in Paper or Electronic Format; Establishment of a Public Docket

Public Workshop: Standards for the Interoperable Exchange of Information for Tracing of Human, Finished, Prescription Drugs, in Paper or Electronic Format

Crossing the Country to Connect with the Cancer Research Community

By: Mitch Zeller, J.D.

One of my goals as the director of FDA’s Center for Tobacco Products (CTP) is to get feedback from everyone interested in our groundbreaking work of regulating tobacco products. We’ve heard that some people outside the Beltway – meaning those beyond the nation’s capital – would appreciate the chance to interact with us. When we can, we make those opportunities happen. We especially want to hear more comments from the public on the scientific issues associated with our regulatory process.

Mitch Zeller

Mitch Zeller speaking to reporters at a media availability during the American Association of Cancer Research annual meeting in San Diego, Calif.

One of those opportunities occurred this week when about 10 FDA staff members and I participated in the annual meeting of the American Association for Cancer Research (AACR) in San Diego. The meeting provides a unique opportunity for members of the worldwide cancer research community to learn about cutting-edge advances, obtain feedback on their own research, and make connections that will foster future collaborations.

CTP kicked off its participation with an educational session to introduce cancer researchers to what tobacco regulatory science is – and what it is not. Science grounds all of our efforts to reduce the disease and death toll of tobacco use. This includes both funding and conducting research that helps determine the regulatory steps we must take to protect public health.

Next, CTP hosted a listening session so that our senior scientific staff could hear presentations on topics relevant to science-based regulation of tobacco products. This public session was held in conjunction with the AACR meeting for the convenience of the many researchers who attend and for members of the public living on the West Coast, but participants did not have to be registered for the AACR meeting to attend or present. We were particularly interested in hearing from the public in connection with FDA’s goals for regulating tobacco:

  • Prevent youth tobacco initiation.
  • Encourage adults who use tobacco to quit.
  • Reduce product harms and addictiveness.
  • Develop a science base and continue meaningful product regulation to reduce the toll of tobacco-related disease, disability and death.

Finally, I participated in a symposium panel and press conference marking AACR’s release of a special commemorative publication of the 2014 Surgeon General’s Report: The Health Consequences of Smoking: 50 Years of Progress. As I said in an interview for the special issue, it is amazing that, with everything we’ve learned about the dangers of tobacco use, our progress has slowed in terms of reducing smoking rates nationally. Nearly one in five adults still smokes, and too many kids become regular smokers. At the press conference, our panel of leaders in the field of tobacco research took questions from cancer and research media about current tobacco control efforts. We discussed smoking patterns and the evolution of tobacco control over the last 50 years, as well as highlighted this newest data from the Surgeon General’s Report on tobacco’s negative health effects. I emphasized the critical role that the regulation of tobacco products can play in these efforts.

Tobacco-related cancer researchers are key partners of FDA as we work toward our goal of ending the epidemic of tobacco use. Hearing from this audience – as well as others – was incredibly valuable to us because we use these insights and feedback as part of the regulatory process. We welcome the opportunity to further engage with the research community as we work to reduce the destructive health consequences of tobacco use.

Mitchell Zeller, J.D., is the Director of FDA’s Center for Tobacco Products  

For more information please visit these Web links:

Informing Tobacco Regulation through Research

2014 Surgeon General’s Report: The Health Consequences of Smoking: 50 Years of Progress

Q&A: Mitchell Zeller on the FDA and Tobacco, AACR Journals

Recognizing Those Who Strive to Vanquish Alzheimer’s Disease

By: Margaret A. Hamburg, M.D.

I had the opportunity yesterday to take part in the Alzheimer’s Association’s (AA) Advocacy Forum and engage in a public discussion with the organization’s President and CEO Harry Johns about the FDA’s role in helping advance Alzheimer’s disease research and the development of new treatment options and approaches.

Margaret Hamburg and Harry Johns

Harry Johns, President and CEO of the Alzheimer’s Association, in a public conversation with FDA Commissioner Margaret Hamburg at the group’s recent Advocacy Forum in Washington, D.C.

This event recognizes the tireless efforts of a community determined to make strides in the fight against what is, in no uncertain terms, a devastating disease and world-wide crisis. Indeed, it is difficult to overstate the impact of Alzheimer’s disease on our society.

Only last month new research was published that suggests that deaths due to Alzheimer’s disease in the United States actually have been severely under-recognized. By these new estimates, Alzheimer’s disease may rank as the third leading cause of death, trailing only behind heart disease and cancer. What’s even more alarming is that it is the only cause among the top 10 without a way to meaningfully prevent, treat, or slow its progression.

Fortunately, in stark contrast to these disturbing figures, the commitment of the countless individuals entrenched in the fight against this epidemic remains resolute. The FDA shares in this determination and holds the need to facilitate the discovery of effective treatments for Alzheimer’s disease among its highest priorities.

In January 2011, President Obama signed into law the National Alzheimer’s Project Act (NAPA), which has the goal of creating a coordinated national plan to overcome the Alzheimer’s crisis. The FDA has been closely involved in the evolution of this plan and has taken several important steps toward realizing some of its stated goals.

In February 2013, the FDA published a draft guidance document responding to the shifting focus of the research community towards the earlier stages of Alzheimer’s disease. The hope is that intervening earlier in the disease process (before the onset of dementia) may provide a greater opportunity to alter the course of the condition. Given that these are uncharted waters, it is essential that we provide as much clarity as possible. Of particular note, the guidance discusses the possible use of the accelerated approval pathway as a means of approving drugs for patients in the earliest stages of the disease.

The FDA is also partnering closely with many public-private initiatives, advocacy groups and consortia in the Alzheimer’s community. As one example, the agency is a member of the recently announced NIH-led Accelerating Medicines Partnership (AMP), which is attempting to uncover biomarkers that may help predict clinical benefit in drug development. We are also working with the Coalition Against Major Disease (CAMD) to develop novel biomarkers to signal the onset of the disease and clinical-trial goals through the agency’s Drug Development Tool Qualification process, and have recently endorsed a clinical trial simulation tool they have developed. These represent just a few of our many collaborations within the Alzheimer’s disease field.

The Alzheimer’s Association used the occasion of their Advocacy Forum to honor the contributions of a number of individuals who have dedicated themselves to the vision of a world without the scourge of Alzheimer’s disease. We at FDA join in recognizing those who have made these important contributions and commitments. We also recognize and applaud the contributions of each and every member of the Alzheimer’s Association who have come to Washington this week to learn more, but also to educate policymakers through their experience, advocacy, and personal stories. Like all of them, we at FDA are committed to doing our part to meet the immense scientific and social challenges of this disease.

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

For more information please visit these Web links:

The National Alzheimer’s Project Act (NAPA)

FDA Guidance for Industry, Draft Guidance, Alzheimer’s Disease

FDA is Working Closely with Manufacturers of Meningitis B Vaccines

By: Karen Midthun, M.D.

Meningitis has been in the news recently because of outbreaks of a specific strain (called “serogroup B” or “MenB”) on college campuses. Infections caused by MenB are uncommon in the U.S, but can be very serious. According to the Centers for Disease Control and Prevention (CDC), 160 of the 500 cases of meningococcal disease in the U.S. in 2012 were caused by MenB. There are vaccines licensed (approved) in the U.S. to prevent meningitis, but none include this strain.

Dr. Karen MidthunTo address this critical public health need, FDA worked closely with CDC, in order for CDC to make an unapproved MenB vaccine available as quickly as possible to the universities where CDC determined outbreaks had occurred. This was accomplished under FDA’s expanded access program for investigational (or unapproved) products. The program allows the use of unapproved drugs or vaccines to treat or prevent serious or immediately life-threatening conditions when other options are not available.

FDA has been working closely with manufacturers pursuing the development and approval of MenB vaccines for the U.S. The approval of any vaccine in the U.S. is an extensive process that requires submission of a Biologics License Application (or BLA) by a manufacturer. FDA medical and scientific staff then perform a detailed review of data supporting the safety and effectiveness of the vaccine, and FDA staff inspect the quality of the manufacturing process. Because the potential usefulness of a preventative vaccine must be weighed against any unintended side effects, the evaluation of each submission includes a careful assessment of the benefits and risks to public health.

The agency has a variety of regulatory tools – breakthrough therapy designation, accelerated approval, the fast-track program, and priority review – that have enabled FDA to help make innovative and effective new treatment options available to patients more rapidly for serious conditions such as MenB. More information about these programs is available on FDA’s web site.

Although the law generally prohibits FDA from disclosing the existence of pending applications, Novartis has given the agency permission to disclose that the firm plans to submit a BLA for Bexsero (serogroup B meningococcal vaccine) for review in the second quarter of 2014. Pfizer, which also is developing a serogroup meningococcal B vaccine, issued a statement on March 20, 2014, in which it acknowledged receiving breakthrough designation from the FDA for its vaccine, and that it intends to submit an application for review by mid-2014.

FDA is committed to working with manufacturers to bring important medical products to patients as quickly as possible. The health and well-being of patients is our top priority.

Karen Midthun, M.D., is the director of FDA’s Center for Biologics Evaluation and Research

Opioid Auto-Injector Can Help Prevent Overdose Deaths

By: Dr. Douglas C. Throckmorton

The Food and Drug Administration has today made an important advance in helping to save lives when overdoses from drugs known as opioids occur: the approval of a drug that can actually reverse that overdose.

Douglas C. Throckmorton, M.D.Opioids include legal prescription drugs, such as OxyContin (oxycodone) and Vicodin (hydrocodone with acetaminophen), used to treat pain, as well as illegal street drugs, such as heroin. In 2010, overdoses of prescription opioids were linked to 16,651 deaths and heroin was linked to 3,036 deaths in the United States.

Whether opioids are used by patients as prescribed by their health care professional or are misused or abused, these drugs have one important thing in common: They can all cause rapid and fatal overdose.

FDA, along with other organizations, has been working diligently on ways to address this major public health issue. That’s why FDA has approved a drug-device combination product that delivers the medication known as naloxone, which is the standard treatment for a drug overdose. This product is potentially easier to use than existing technology and thus could save lives.

Overcoming an Obstacle

Naloxone reverses the effects of opioids. Most significantly, it reverses the severely slowed breathing that can lead to death during an overdose. When naloxone works, the results can be dramatic: Comatose patients can wake up in minutes.

Over the years, emergency health care professionals and others with special training have saved thousands of lives by giving injections of naloxone to people suffering from opioid overdoses. Although effective, this requires expertise – to be able to draw the naloxone up from a vial and then inject it with a needle and syringe. In some cases, the injectable form is adapted so it can be administered through the nose, but there is currently no intranasal form of naloxone approved by FDA.

FDA has now approved  the first naloxone product that is given using an auto-injector, without having to use a separate needle and syringe.  Available via prescription, the product enables anyone – even the general public – to inject naloxone from a pre-filled, single-use device into a person who is overdosing. This is similar to devices used by laypeople to administer epinephrine in cases of severe allergy. In addition, the auto-injector gives recorded instructions to the user describing how to deliver the medication. The instructions also reinforce the need to seek emergency medical attention immediately because while naloxone works rapidly, it only temporarily reverses the effects of opioid overdose. The patient still needs immediate medical care.

The product’s brand name is Evzio, and information on its safe use is available by clicking this hyperlink.

This approval is the culmination of concerted efforts at FDA and throughout the Department of Health and Human Services (HHS) to spur research into alternative forms of naloxone and to expand its availability. On April 12, 2012, FDA co-sponsored a meeting that highlighted the impact of community-based naloxone distribution programs and explained to sponsors the path to FDA approval.

FDA Working on Other Fronts to Combat Opioid Misuse and Abuse

FDA has a responsibility to demand the best science and data to uphold its high standards to ensure a proper benefit/risk profile for drugs that treat pain. We also foster discussion and research that leads to a better understanding of pain and pain drugs. Because we are concerned about the public health issue, the agency has taken a number of actions to help reduce the devastating consequences of opioid misuse, abuse, and fatal overdoses. We are targeting key drivers of the problem with efforts that include:

  • Changing the labeling of certain opioids to help improve their safe use.
  • Requiring that manufacturers conduct studies of the safety of long-term use of certain prescription opioids.
  • Requiring that manufacturers  of certain opioids make training available to practitioners (physicians, dentists and others authorized to prescribe opioids) on responsible prescribing practices and assessing and addressing signs of abuse and/or dependence.
  • Strengthening our surveillance efforts to actively monitor the changing nature of prescription opioid abuse and identify emerging issues.
  • Working with the Drug Enforcement Administration on opioid controls.
  • Encouraging the development of new forms of opioids that are resistant to abuse.
  • Supporting the development of new pain treatments, especially non-addictive treatments.

While FDA plays an important role in mitigating the risks of addiction, abuse and misuse of prescription opioids, we cannot fix the problem alone. A comprehensive approach must be taken by federal and state governments, public health officials, opioid prescribers, addiction experts, researchers, industry, patient organizations, and others. That is why FDA is working closely with our HHS counterparts, including CDC, NIDA, SAMHSA, and throughout the US government.

There’s much work to be done, but today’s approval of the opioid auto-injector could save thousands of lives. FDA will continue to work to prevent the consequences of misuse and abuse of opioids, including preventing fatal overdoses.

Douglas C. Throckmorton, M.D., is Deputy Center Director for Regulatory Programs in FDA’s Center for Drug Evaluation and Research