FDA Working to Keep Patients Well Informed

By: Steve L. Morin R.N., B.S.N.

Steve Morin_2823My job in the Food and Drug Administration’s Office of Health and Constituent Affairs (OHCA) is to serve our nation’s patients in two ways: by listening to their concerns regarding FDA’s policy and decision-making and advocating for them in our agency;  and by informing many patients and patient organizations about FDA’s mission and its work to advance the development, evaluation and approval of new therapeutic products.

This dialogue was formalized and greatly expanded in 2012 when, after a series of listening sessions with many patient advocacy organizations, OHCA created the Patient Network.

Specifically designed for patients, caregivers, patient advocates and disease-specific patient advocacy organizations and the communities that advocate on their behalf, this program serves two goals. It facilitates patient engagement with FDA policy and decision makers, and it educates its audience about the process that brings new medications – both prescription and over-the-counter ­– and medical devices from a concept to the marketplace.

Our Patient Network covers a range of FDA-specific topics and conducts numerous activities that are of interest to patients and patient advocates. One of these activities were webinars with information about upcoming public meetings hosted by FDA.

For example on March 31, 2014, OHCA was pleased to host the first-of-its-kind “LiveChat” with the diabetes community. This online discussion gave patients an opportunity to interact with FDA experts and to better understand a recently released draft guidance dealing with the studies and criteria that FDA recommends be used when submitting premarket notifications (510(k)s) for blood glucose meters.

On September 10, 2014, our Third Annual Patient Network Meeting titled “Under the Microscope: Pediatric Product Development” brought together more than 100 patients, patient advocates, representatives of academia and industry, and FDA leaders. The participants discussed pediatric product development and the ways patient advocates can participate in it.

And on September 17, 2014, our Patient Network webpages were upgraded. The “For Patients” section on FDA’s website is presented in a clear manner with easy-to-use formats. Also, a “For Patients” button is located on our homepage.

We have continued to pursue our goals of informing the public and engaging with patients by building upon the patient-centered webpages and enhancing activities that express our desire to be helpful and transparent. This is our philosophy that has helped the Patient Network evolve to what you see today.

As the Patient Network program continues to grow, I hope to expand it to have more interactive webinars like the “LiveChat” that address specific concerns  of the patient communities. Also, we will continue to make it possible for patients to learn from FDA experts who approve medical products.

The FDA realizes that listening to the “patient voice” and conducting our dialogue is important, and it continues to develop its model for patient involvement through the Patient-Focused Drug Development Meetings and other OHCA sponsored meetings and webinars. We hope patients and those who care for them will join us in that effort, and make it still more helpful in protecting and promoting the public health.

Steve L. Morin, R.N., B.S.N., is a Commander of the United States Public Health Service and the Manager of the Patient Network in FDA’s Office of Health and Constituent Affairs

FDA Uses Web Tool to Better the Odds for Food Safety

By: Ted Elkin

When most people hear the words, “Monte Carlo,” they may think about high-stakes gambling.

We, however, think about reducing the risk in food safety through the use of FDA-iRISK, an innovative Web-based food safety modeling tool developed by the Food and Drug Administration and our partners.

Launched in October 2012, FDA-iRISK uses mathematical logic and Monte Carlo simulation (a computer program named for the gambling mecca) to integrate data and generate results that compare and rank risks of the contamination of foods by various hazards.  Unlike a traditional risk assessment of a single food and a single contaminant, FDA-iRISK allows users to compare multiple hazards – microbial or chemical – in multiple foods.

How does FDA-iRISK work?

Through extensive outreach to and collaboration with partners, we developed built-in templates and other features that allow the user to create real-world (or hypothetical) food safety scenarios.

The user provides the data for seven elements: the food(s), the hazard(s), the population of concern (for instance, elderly or immune-compromised), the production or processing system being used for the food, the consumption patterns, the dose response (what level of exposure will have a health impact), and how the health effects are to be calculated.

This allows the user flexibility, for instance, to look at the impact of potential interventions at various stages of the food production system as well as the populations affected.  And it’s easy to use.

Of particular benefit to the user is FDA-iRISK’s ability to generate reports that measure the health impact of an intervention in terms of the widely used public health metric, DALYs (“Disability-Adjusted Life Years,” meaning years of healthy life lost to illness or death).  This measure lets us know the “bang for the buck” of a particular intervention.

FDA-iRISK is quickly gaining acceptance and use in the food safety community.  As of the middle of May, almost 500 users had established accounts with FDA-iRISK and they came from every continent. Because it is web-based, FDA-iRISK is available to anyone in the world who sets up an account, and it is free to use.

Therefore, the knowledge and sharing power of FDA-iRISK is exponential.  As more users use it and generate reports that are then available to the other users, a more consistent, well documented, systematic, structured and quantitative picture of risk in the food supply will emerge, as well as scenarios for reducing risk.

“Information provided by iRisk can aid in developing global scientific exchanges aimed at maintaining and developing agricultural markets around the world,” according to Jamilah (Fagbene) Cassagnol, an international trade specialist at the U.S. Department of Agriculture.

FDA-iRisk is supported by an exceptional project team. FDA staff members Sherri Dennis, Yuhuan Chen, David Oryang, Regis Pouillot, Karin Hoelzer and Susan Cahill developed the tool in collaboration with Risk Sciences International, RTI International and the Institute of Food Technologists.

Ultimately, for food safety, Monte Carlo shouldn’t mean taking a gamble.  Rather, it’s all about using a quantified, standardized and transparent methodology to better understand what interventions and controls will reduce the risk and improve our public health.

Ted Elkin is Director, Office of Analytics and Outreach, at FDA’s Center for Food Safety and Applied Nutrition.

“Breakthrough” Designation … Another Powerful Tool in FDA’s Toolbox for Expediting the Development and Review of Promising New Drugs for Serious Conditions

By: Janet Woodcock, M.D.

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

In fiscal year 2012, FDA approved 35 novel new drugs, also known as “new molecular entities.” Among these new products were drugs to treat patients with unmet medical needs, such as a groundbreaking treatment for a form of cystic fibrosis, the first FDA-approved human cord blood product for hematopoietic reconstitution, used to help patients with blood forming disorders, and the first drug to treat advanced basal cell carcinoma (a form of the most common skin cancer).

To enable our ongoing efforts to bring innovative drug products to the public as efficiently as possible, FDA relies heavily on several expedited development and review tools such as fast track designation, the accelerated approval pathway and priority review designation. For instance, 56 percent of the novel drugs approved by the Center for Drug Evaluation and Research in calendar year 2012 used some combination of these tools to speed promising therapies to patients with serious conditions. And any given drug may have received multiple expedited program designations. (See a brief summary of how each of these tools helps FDA shorten the development and review of promising new therapies.)

In July 2012, a provision in the new law called the Food and Drug Administration Safety and Innovation Act, or FDASIA for short, gave FDA another powerful expedited development tool, known as the “breakthrough therapy” designation. This new designation is now helping FDA assist drug developers expedite the development of new drugs with preliminary clinical evidence that indicates the drug may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. Although the designation is not yet even a year old, FDA has received 62 requests to grant this new designation to products under development. We have been very active on this subject, meeting with companies and discussing ways to expedite the drug development process for drugs that show striking early results. We have already granted the breakthrough designation to 20 potential innovative new drugs that have shown encouraging early clinical results.

Drug developers should have a clear understanding of all of FDA’s expedited development and review tools. To help industry better understand each tool, including when the tools can be used and the features of each, we have just published an industry draft guidance titled Expedited Programs for Serious Conditions — Drugs and BiologicsAmong other important information, the draft guidance describes FDA’s policies and the threshold criteria for each expedited program, defines and discusses important concepts, including serious condition, unmet medical need, and available therapy, and provides some general considerations for products utilizing an expedited program, such as manufacturing and product quality, nonclinical considerations, and clinical inspection considerations.

The breakthrough therapy designation gives us another tool in our “toolbox” to help expedite the development and review of new drugs to treat patients with serious medical conditions and little or no treatment options. We’ll continue to use the new breakthrough therapy designation and our existing tools to help make our expedited programs even more effective.

We’ve said it before — and I believe it’s worth repeating — our decision-making on whether to approve a drug always involves an evaluation of many factors, such as the seriousness of the disease.  However, ultimately any drug approved must show that its benefits outweigh its risks and regardless of which expedited development or review program or programs are used, FDA does not compromise its safety or efficacy standards in exchange for rapid approval. Like all drugs we approve, those approved after having been designated as breakthrough therapies will meet our usual rigorous standards for safety and effectiveness.

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

Setting the Bar High for FDA

By: Margaret A. Hamburg, M.D.

Rick Pazdur receiving ASCO Public Service Award

Rick Pazdur, accompanied by Margaret Hamburg, receiving ASCO Public Service Award

To say that Rick Pazdur faces enormous challenges in his job is an understatement. To say that he faces each day with energy, insight and resolve still falls short of the mark.

It’s my privilege to tell you that the American Society of Clinical Oncology (ASCO) has awarded Dr. Pazdur with its prestigious Public Service Award for his dedication to improving the lives of people living with cancer.

As director of the Office of Hematology and Oncology Products (OHOP) at FDA, Dr. Pazdur leads a staff of more than 130 oncologists, toxicologists and other specialists.

Their mission is making safe and effective drugs for cancer and hematologic (blood-related) conditions available to the patients who need them. The office is committed to facilitating rapid development, review and action on promising new treatments for these diseases.

Dr. Pazdur sets the bar high. His demand for excellence in his staff as well as in the treatments they review is unparalleled. Ultimately, Dr .Pazdur and his staff must decide whether or not an investigational drug can be tested in a clinical trial and, after testing, be approved for more widespread use. Sometimes, after careful investigation, they conclude that a drug has not been proven effective enough to outweigh the potential risks. These are the types of challenges and the tough decisions that Dr. Pazdur faces on a daily basis. A man of personal integrity with great compassion for those who are ill, he nonetheless is  often the recipient of criticism from patients, advocacy groups, drug companies and others. I have heard him say ruefully, but with characteristic humor, that you can’t win in this job—that if he approves a drug, he’s accused of lowering standards.  And if he doesn’t, he is insensitive to the plight of patients with cancer. Nothing could be farther from the truth.

Since his arrival at FDA in 1999, Dr. Pazdur has worked tirelessly to speed the development and availability of drugs that treat serious diseases, especially when the drugs are the first available treatment or have advantages over existing therapies. He has made a special effort to reach out to patient and advocacy groups, professional associations and foreign regulatory agencies. In 2012, nearly 40 percent of the new molecular entities approved in the Center for Drug Evaluation and Research were to treat cancer, often when few therapeutic options previously existed.

Members of Dr. Pazdur’s staff speak with warmth and enthusiasm of his dedication to cancer patients and his unflagging efforts to streamline the drug approval process. They call him not just a manager, but “a visionary,” and “one of the most unique people I know.” I quite agree.

To one of the most dedicated and accomplished people I know: It’s a pleasure to work at your side, Dr. Pazdur. Congratulations for this well-deserved honor.

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

We’re Working to Offset Ameridose Impact

By Margaret A. Hamburg, M.D.

Drug shortages are two words that no one wants to hear—not patients, not health care professionals, and not me.

Margaret Hamburg, M.D.FDA has been working hard to prevent and mitigate drug shortages. In 2011, the number of medications in short supply hit 251. Addressing drug shortages must be a top priority for us at FDA because these are medications that people need to stay healthy, to treat their illnesses, and even, in some cases, to stay alive.

This year, we’ve taken significant steps to expand our efforts and to engage in new ways with industry. Between Jan. 1 and Sept. 30, 2012, FDA worked with drug manufacturers to help avert the shortage of 145 drugs. Many critical medicines used to treat cancer and conditions such as attention deficit hyperactivity disorder (ADHD) are no longer in short supply.

However, drug shortages are still a serious problem, one that may be temporarily impacted by Ameridose LLC’s voluntary recall of all of its unexpired products. Ameridose, located in Westborough, Mass., is managed by some of the same people as the New England Compounding Center—which produced the drug that is implicated in the deadly, multi-state outbreak of fungal meningitis. An inspection of Ameridose was initiated as part of FDA’s ongoing investigation of the outbreak.

FDA recommended that Ameridose recall its sterile drugs because we could not be assured of the sterility of those products. However, this recall may affect supplies of certain life-saving drugs for some health care systems. FDA has identified a number of Ameridose products—including drugs used during surgery and to treat medical conditions that include congestive heart failure—that were on the current drug shortages list before the recall.

We also know that the supply of other drugs may be affected by the Ameridose recall. That’s why FDA is taking proactive steps to minimize the impact this recall may have on current drug shortages, and to prevent other shortages from occurring.

For recalled medications on the current drug shortages list, FDA is taking the same actions it has used successfully to mitigate other shortages.

  • FDA is working with manufacturers of these drugs, requesting that they ramp up production if they are willing and able to do so.
  • For any manufacturers of these drugs that may be experiencing manufacturing or quality problems, FDA is offering assistance to enable them to produce shortage drug products that are safe and high quality.
  •  As with shortages of any critical products, FDA will expedite the reviews of any pending applications that could help with addressing the shortages.
  • FDA is identifying any additional manufacturers willing to initiate or increase production.
  • If manufacturers of critical drugs are not able to meet U.S. patient needs, FDA will explore overseas companies that are willing and able to import foreign drugs to address the shortage. In these instances, FDA evaluates the imported drug to ensure that it is of adequate quality and that the drug does not pose undue risks for U.S. patients.

Since the beginning of the year, the number of advance notifications to FDA of potential shortages has greatly increased. If we know that a problem is on the horizon, we’re able to proactively work with industry, organizations, patients and stakeholders to address it. We have doubled the number of staff members who work in drug shortage prevention and response.

We at FDA are committed to doing everything we can, using all available tools, to prevent or mitigate drug shortages and help keep critically needed products on the market.

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