FDA and Health Canada: Working Together for an Efficient Pathway for Drug Applications

By:  Robert Yetter, PhD 

At FDA, we work closely with national regulatory agencies around the world on issues relating to the safety, efficacy and availability of medical products. An exciting example of such collaborative efforts is the Common Electronic Submissions Gateway (or CESG), an outcome of the US-Canada Regulatory Cooperation Council (RCC). Through a cooperative research and development agreement, FDA worked with our counterparts in Health Canada, to share technology that will make it more efficient for industry to submit applications to both the U.S. and Canada for the approval of pharmaceutical and biological products. A common infrastructure would enable industry to submit to both countries using the same electronic format for technical documents. 

Robert YetterThe RCC Initiative was announced in February 2011 by President Barack Obama and Prime Minister Stephen Harper. Its goals are to promote economic growth, job creation and benefits to consumers and businesses through increased regulatory transparency and coordination. The electronic submissions gateway is one such project designed to meet those goals. 

So just what is this gateway? It’s an electronic “post office” that uses secure Internet connections to receive electronic versions of medical product applications and related documents from industry sponsors seeking regulatory approval. The technology was developed under contract, and implementation at FDA was led by the Center for Biologics Evaluation and Research.  FDA’s Electronic Submissions Gateway (ESG) has been in operation since 2006. It has now been modified to accommodate submissions from both Canada and the U.S. using the same interface and technology, and subsequently sending those submission transmissions to one or both regulatory authorities. 

The collaboration on the Common Electronic Submissions Gateway has the potential to yield long-term positive outcomes for both FDA and Health Canada. The collaboration continues the work between the two regulatory partners to streamline both agencies’ submission requirements while maintaining consistency in regulatory requirements. It could also lead to cost reductions for regulated industry, which would not have to follow separate technical requirements for submission to the two countries. 

We’re very proud of our work with Health Canada to make this technology accessible in a relatively short amount of time, going from concept to delivery in 26 months. This is yet another example of the steps FDA is taking as part of our Global Initiative, which envisions enhanced collaboration with our regulatory partners. 

Robert Yetter, PhD, is the Associate Director for Review Management in FDA’s Center for Biologics Evaluation and Research

Keeping Foods Safe During Transport

By: Michael R. Taylor

We made great strides in 2013 toward fulfilling the mission set forth by the FDA Food Safety Modernization Act, often known simply by the acronym FSMA. This law is making it possible for us to reinvent ourselves, becoming a force for the prevention of foodborne diseases rather than being mainly first-responders to food safety problems in progress.

Michael TaylorToday we are proposing a new food safety rule that would help prevent the contamination of food during transportation by establishing requirements for sanitary transportation practices.  Certain shippers, carriers, and receivers engaged in transporting food by motor or rail vehicles would be required to follow common sense sanitary practices, such as properly refrigerating food, adequately cleaning vehicles between loads, and properly protecting food during transportation.

This is an important part of the food handling process, one that can introduce contamination even after proper safeguards have been taken by the food producers and processors. Truthfully, it’s uncommon for a foodborne illness to be caused by contamination during transportation. But we have received reports of unsanitary practices, and we want to minimize this potential source of illness.

This proposed Sanitary Transportation of Human and Animal Food Rule is the seventh and final major proposed rule in FSMA’s central framework aimed at systematically building preventive measures across the food system. It builds on the six rules proposed in 2013 to keep produce safe, enact preventive controls in human and animal food-producing facilities, modernize oversight of imported foods, and guard against intentional contamination.

As this next-generation safety network takes shape, the food safety law gave us new authorities that empower us to take immediate action in the meantime to protect public health. Administrative detention enables us to keep a suspect food product out of circulation. Mandatory recall enables us to pull products off store shelves if we have reason to believe they may be unsafe to eat.  We can suspend the registration of a facility if the food it produces, packs or stores presents a serious health hazard.  By using these tools now, we’re practicing what we preach as we plan for the future.

As we’ve traveled the world to explain these new food-safety standards, I’ve developed a mantra of sorts: We’re all in this together. I mean that as much for this transportation proposed rule as I have for each of the ones we proposed last year. When these rules become final, they will be the product of an unprecedented collaboration between food regulators, the food industry, scientists, advocates and consumers.

So let’s start talking about what we need to do to transport foods safely.  As with the other proposed rules, we will work with all stakeholders – including consumers, industry and researchers – to ensure that what we’re proposing is practical and feasible while meeting our food-safety standards.  We look forward to your comments.

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

Another Strong Year for Novel New Drug Approvals

By: John K. Jenkins, M.D.

Last year marked another strong year for FDA approvals of novel new drugs, known as new molecular entities (NMEs).  In 2013, FDA’s Center for Drug Evaluation and Research (CDER) approved 27 NMEs last year – about the same as the 26 average NME approvals per year since the beginning of this decade.

John JenkinsMore important than the quantity of novel new drugs approved in 2013 is their quality – and the important new roles many of these drugs can serve in advancing medical care and the health of patients. We now have new safe and effective treatments for a wide range of serious medical conditions, such as late-stage breast cancer, chronic hepatitis C, metastatic melanoma, mantle cell lymphoma, chronic lymphocytic leukemia, homozygous familial hypercholesterolemia, pulmonary arterial hypertension, and many more. Some of these medications offer new hope to patients who previously had few or no treatment options.

Here are a few highlights of these approvals:

  • One-third of the NMEs approved in 2013 were identified by FDA as “first-in-class,” for example, drugs that use a new and unique mechanism of action for treating a medical condition;
  • One-third were also approved to treat rare or “orphan” diseases that affect 200,000 or fewer Americans who often have few or no drug treatment options;
  • Almost half of the 27 NMEs approved last year (13 of 27), were designated in one or more categories of Fast Track, Breakthrough, Priority Review, or Accelerated Approval. Each of these designations helps speed the development and/or approval process and is designed to help bring important medications to the market as quickly as possible;
  • Although FDA’s regulatory processes differ widely from those of foreign regulatory authorities, almost three-quarters (74%) of the NMEs approved by FDA in 2013 were approved first in the United States before any other country.

All of us at FDA are pleased and proud to be part of a team that helped bring these new drugs to market as safely and efficiently as possible. As always, while striving for efficiency in our review and approval of applications for new drugs, compromises were not made in our standards. To be approved, each NME had to demonstrate that it was safe and effective before being approved.

My colleagues and I look forward to another productive year serving the American public!

For more details about 2013’s approvals, please visit The Novel New Drugs Summary at: http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DrugInnovation/UCM381803.pdf

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

Setting the Bar for Blood Glucose Meter Performance

By: Courtney Lias

Courtney Lias is Director of the Division of Chemistry and Toxicology Devices within the Office of In Vitro Diagnostics and Radiological Devices at FDA’s Center for Devices and Radiological HealthMany of the nearly 19 million Americans diagnosed with diabetes must monitor their blood glucose (sugar) frequently throughout the day using an at-home meter to make sure that their blood glucose is within a safe range. The ability to measure blood glucose at home has given people with this serious and chronic condition the ability to better control their blood sugar and thus avoid potential complications.

In the last 10 years there has been much advancement in the development of glucose meters. They are now smaller, require a smaller blood sample for each test and produce faster results.  However, their accuracy has improved little.

At FDA’s public meeting in March 2010 on this topic, the clinical and patient communities challenged the agency to improve performance of glucose meters. Feedback gathered from that meeting directly informed the creation of two draft guidance documents released this week. These documents set forth recommendations, which are justified to help ensure that these important devices are designed to be more accurate and reliable for the patients who need them. To address this need, this week we are proposing new recommendations for labeling, meter performance evaluation, manufacturing controls, and cleaning and disinfection procedures to help improve the accuracy and performance of blood glucose meters.

FDA recognized the need to optimize the safe use of blood glucose meters in two distinct settings: self-monitoring using devices purchased over-the-counter, and use in a clinical setting by health care professionals. FDA believes that by distinguishing where these devices are used, they can be better designed to meet the needs of their intended populations and ensure greater safety and efficacy.

Historically, devices used in these two settings have been studied using the same methods and standards. However, it has become increasingly clear that meters used in these different settings have unique characteristics and different design specifications. For example, critically ill patients in health care settings may have physiological variables, like abnormal oxygen levels, that could interfere with the accuracy of the blood glucose meter. Patients who use over-the-counter glucose meters and test strips at home vary in age, how much they know about how to use blood glucose tests, and other critical factors that might affect the  accurate use of the device.

To distinguish between FDA recommendations for blood glucose meters used in health-care facilities, and those intended for self-monitoring by lay-persons, the agency is issuing separate draft guidances for each one, that is:

  • prescription-use blood glucose meters, for use in point-of-care professional health-care settings, and
  • blood glucose devices purchased over-the-counter, intended for self-monitoring by lay-persons.

We believe that these recommendations will help ensure that glucose meters meet critical standards for accuracy in the hands of people with diabetes, who rely on them to manage their disease. Please help us in this effort by providing specific comments to these draft guidance documents to let us know if you agree with our recommendations or whether you have suggestions to further improve them.

Improving the quality of blood glucose meters will not solve all challenges for those who live with diabetes, but it may help millions of people to avoid complications and better achieve their health goals.

Courtney Lias is Director of the Division of Chemistry and Toxicology Devices within the Office of In Vitro Diagnostics and Radiological Devices at FDA’s Center for Devices and Radiological Health

Gregory Reaman Helps Make the World a Better Place for Children

By: Richard Pazdur, M.D.

I am privileged to work every day with many physicians and other health care professionals dedicated to advancing public health for all Americans. One of them is Dr. Gregory Reaman, who has been awarded the Leukemia & Lymphoma Society’s prestigious Return of the Child Award. Greg has devoted his career to finding better ways to treat and improve the outcomes for children with cancer.

Richard Pazdur, M.D.This award, presented in December in New Orleans, is given each year to a person who has made a major and lasting scientific or humanitarian contribution to the better understanding, management or treatment of pediatric hematological malignancies. (Hematological malignancies are the types of cancer that affect the blood, bone marrow and lymph nodes.)

Greg was honored for his exceptional leadership and accomplishments in the field of pediatric hematology and oncology during his lengthy career as a clinician and academic researcher and, since 2011, as a member of my team here at FDA. His primary work has focused on clinical trials for children with acute lymphoblastic leukemia (ALL) and the early phases of developing experimental drugs for children. Greg’s leadership in these areas has led to significant improvements in the care of children with ALL, as well as the facilitation of new drug development for children with cancer.

Dr. Gregory Reaman accepts award

Dr. Gregory Reaman (left) accepts The Return of the Child Award Presented by LLS CEO John Walter

Greg is a previous recipient of the Leukemia & Lymphoma Society’s Tree of Life Award, which honors those who have played a major role in improving the quality of life of patients and their families. Through these and many other efforts, his extensive research has helped to advance how pediatric blood cancers are treated today.

At FDA, where he is associate director of oncology sciences, Greg is using new mechanisms created by recent laws to facilitate public discussion and promote drug research and development for children with cancer.

Greg is also executive director emeritus and senior attending physician at Children’s National Medical Center in Washington, D.C. Throughout his distinguished career, Greg has held other leadership positions at Children’s and at the National Childhood Cancer Foundation. He is also a tenured professor of pediatrics at the George Washington University School of Medicine and Health Sciences. 

I know of no greater endeavor in life than to dedicate one’s work to making this world a better place for children, and I know of no individual more deserving of an award for such efforts than Greg. I know I speak for my entire staff and all of us at FDA when I congratulate Greg on his achievements.

Richard Pazdur, M.D., is director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research