‘Radiating Shoe Sales’

By: Vanessa Burrows

Since the discovery of X-rays in the late 19th century, the technology has enhanced health care in a variety of ways. Like many cutting-edge scientific developments, however, it also has inspired uses of uncertain therapeutic value. That was the case with the shoe-fitting fluoroscope, the subject of the latest episode of FDA’s history video series.

Vanessa Burrons

Vanessa Burrows, FDA Historian, next to a shoe-fitting fluoroscope

Marketed as a scientific method for optimizing shoe fit, the fluoroscope appeared in shoe stores nationwide from the 1920s to the 1960s. But the machines not only didn’t do what they promised, they also exposed children, their parents, and store clerks to unhealthy doses of radiation.

In the late 1940s, scientists and regulators began to raise serious concerns about the dangerous levels of radiation. Over the next two decades, individual states gradually took action to either ban or restrict the use of the device.

By the 1970s, concerns grew about radiation emitted from common appliances, such as televisions and microwave ovens. And in 1971, FDA was given authority to regulate radiation-emitting devices. The agency continues this oversight, working to protect consumers from harmful novelty devices like the shoe-fitting fluoroscope. FDA also sets standards for medical imaging, surgical and therapeutic equipment, security systems, and consumer products – all for the protection of the health of American consumers.

We hope you enjoy your visit to FDA’s HistoryVault.

Vanessa Burrows is an FDA Historian

Working to Raise Awareness and Reduce Health Disparities

By: Jonca Bull, M.D.

Minority Health Month imageApril is National Minority Health Month and this year’s theme is “Bridging Health Equity Across Communities.”

FDA’s Office of Minority Health (OMH) is committed to the HHS mission of advancing health equity, and our office works year-round to advance FDA’s message of ensuring the safety and efficacy of our nation’s food supply and medical products to all communities, but with a focus on minority groups. The first HHS Office of Minority Health was established nearly three decades ago and FDA’s own office came into being in 2010.

Jonca Bull, M.D., is Director of FDA’s Office of Minority HealthIn the intervening years, we have made significant progress. But we are reminded daily that there is still more to be accomplished in the fight to reduce and eliminate health disparities.

Today, minority communities and those at the lower socioeconomic rungs still remain disproportionately burdened by chronic disease and are much more likely to succumb to certain illnesses.

Within the past year, we have worked diligently to connect our communities to resources to help educate and raise awareness about these issues. Highlights from our two programmatic teams include:

Outreach and communications –

Released English and Spanish language videos to encourage minority participation in clinical trials, with the most recent video being released this month;

  • Worked closely with the health fraud team in the Office of Regulatory Affairs to raise awareness about tainted and fraudulent products that target some ethnic groups;
  • Exhibited and presented at nearly a dozen conferences throughout the year; and,
  • Spearheaded the agency’s Language Access plan to help ensure that FDA materials are available to all consumers in their native language.

Research and Collaboration –

  • Worked with FDA centers and external partners to support research studies around minority health and health disparities. Projects included: Racial Disparities in Multiple Myeloma, Underutilization of Generic Medications in Underserved Patients, and Advertising and Promotional Labeling in Adult Immunization Disparities.
  • Funded multiple Centers for Excellence in Regulatory Research Science and Innovation (CERSI) projects with Georgetown University (Targeting triple negative breast cancer in African American women), University of California San Francisco/Stanford (Safer Labeling of Pediatric Medications: Reducing Literacy-related Health Disparities among Chronically Ill Adolescents), Johns Hopkins University (Workshop Clinical Trials: Assessing Safety and Efficacy for a Diverse Population);
  • Supported a genomics and health disparities fellow at Harvard University School of Medicine; and,
  • Hosted more than half a dozen fellows and pharmacy students from Howard University, the University of Washington, and Florida Agricultural and Mechanical University, among others.

In addition, last fall, OMH issued a draft guidance document titled, Collection of Race and Ethnicity Data in Clinical Trials-Guidance for Industry and FDA Staff with the goal to ensure that subpopulation data is collected consistently by industry. This document outlines FDA’s expectations for, and recommendations on, the use of a standardized approach for collecting and reporting race and ethnicity data in submissions for clinical trials for FDA regulated medical products conducted in the United States and abroad. We also hosted a webinar to walk through the document.

Working collaboratively within FDA and with external stakeholders, we will continue promoting and protecting the health of diverse populations through research and communicating of science information that addresses health disparities.

Jonca Bull, M.D., is FDA’s Assistant Commissioner for Minority Health, Office of Minority Health

For more information read: The FDA Supports Research to Reduce Health Disparities

A Shocking “Exercize”

By: Suzanne Junod, Ph.D., and John Swann, Ph.D.

Suzanne Junod and John Swann

Suzanne Junod, Ph.D., and John Swann, Ph.D., holding FDA artifacts.

In this second installment of FDA’s new history video series, the FDA’s historians venture into the “vault” to explore the Relaxacizor, originally sold in beauty salons as a device that stimulated and relaxed muscles, but subsequently marketed as a passive exercise machine.

Ads for the device assured users that they would lose weight while building muscle mass, a promise that was, of course, too good to be true. FDA took legal action against the company and by the early 1960s succeeded in having the devices removed from the market.

Keep an eye out for the reference to the TV show, “Mad Men,” which included a faux advertising campaign for Relaxacizor in the show’s first season.

Enjoy your visit to the FDA History Vault.

If you missed it, watch the first video – an introduction to the series here.

Suzanne Junod, Ph.D., and John Swann, Ph.D., are FDA Historians.

Opening the FDA’s History Vault

By: Suzanne Junod, Ph.D., and John Swann, Ph.D.

Welcome to the FDA’s History Vault, which contains more than 10,000 artifacts that provide a journey through American history and document the critical role played by one of the nation’s oldest public health agencies in support of its mission to promote and protect American health.

Suzanne Junod and John Swann

Suzanne Junod, Ph.D., and John Swann, Ph.D., holding FDA artifacts.

The items featured in this new series of short videos reflect the constant changes in science and society. It is the responsibility of the FDA’s history office to document and share these changes through the collection, management, and display of these rare, and in many cases, irreplaceable items.

Besides collecting and maintaining these articles, our office embraces the broader role of history: to inform, explain, and educate, so that future decisions are made with the best available knowledge and science.

The collection includes deceptive and dangerous foods, medicines, and so-called medical products that the FDA helped remove from commerce and that led to important changes in laws and regulations.

For example, it includes:

  • a sample of Elixir Sulfanilamide, a 1937 wonder drug that was formulated with a poisonous solvent that killed more than 100 people, including many children. The 1937 disaster spurred passage of the Federal Food, Drug, and Cosmetic Act of 1938, the basic law under which the FDA still operates.
  • a can of Bon Vivant vichyssoise soup (contents removed) that sparked an outbreak of botulism in the early 1970s and significant new food protections for consumers
  • the Dalkon Shield intrauterine device, an ill-designed product that left thousands of women sterile during the 1970s, and encouraged Congress to craft legislation that specifically addressed the safety and efficacy of medical devices, and,
  • the Relaxicisor, a passive electric muscle stimulation “exercise” device first made famous during the 1950s, and again, more recently, thanks to the television show “Mad Men.”

Artifacts like these tell the story of the origins of many of our laws and regulations, the ways in which the FDA works to carry out its responsibilities to uphold them, and the interactions between the agency, its stakeholders, consumers, patients, and Congress in the interests of public health and product safety.

Other artifacts in our vault  illustrate how FDA’s essential tools, which once seemed pioneering in their time, are eventually superseded as FDA adopts new approaches in response to continuing advances in science and technology.

The first video released today harkens back to the time of the Bureau of Chemistry, the organization that preceded the FDA, when data on foods and drugs was analyzed using a novel early calculating device.

It’s worth noting that like other federal agencies, FDA also hired women to be human “computers,” an important role that was brought to greater attention in the recent movie “Hidden Figures,” which depicted three such women who worked at NASA. By the 1940’s and 1950’s, women at the FDA regularly used statistical methods to distinguish products with therapeutic merit from those “which merely had good copywriters.” Their work played an important role in the analysis of the earliest “cooperative” clinical drug trials. Of course, just like NASA, data analysis at FDA is now made possible by computers and supercomputers.

The series of videos we begin today are designed to be entertaining, informative, fun, as well as highlight some of the items in our collection securely stored in our White Oak facility. We plan to release them once a month, always on Thursday, as part of the popular social media tradition of “Throwback Thursday.” The goal is to educate and increase the understanding of the ways that the FDA has, for more than 100 years, embraced scientific advances to ensure the well-being of the American public.

We hope you enjoy your visit to “the FDA’s History Vault.”

Suzanne Junod, Ph.D., and John Swann, Ph.D., are FDA Historians.

FDA Advisory Committees: Independent, Informed, Essential, and Evolving

By: Robert M. Califf, M.D.

One of the most common concerns raised when I meet with medical leaders is the need to improve the function of FDA’s Advisory Committees (ACs). ACs play a key role in FDA’s decision-making process by providing independent expert advice on extraordinarily complex issues. Just as importantly, they offer a forum for open and transparent discussion about these processes. As their name suggests, ACs are only advisory, but they can yield unique insights into understanding the balance of benefits and risks of products.

Not every product is brought to an advisory committee — when the answers are clear, the FDA makes decisions without consulting an AC. But when products present challenging issues or involve developing areas of science, the views of experts in relevant fields can provide essential perspective needed to make good decisions.

They also provide a barometer for the public on Agency thinking in a given field and offer insight into Agency decision-making and requirements for successful product development in a particular setting. The views expressed and votes taken can have financial impacts on companies and can lead to changes in how investments are made in therapeutic areas. So it is not surprising that the deliberations and views of ACs often receive significant media attention.

ACs have been the subject of ongoing discussions concerning their impartiality, their transparency, and how they affect decisions made about FDA-regulated products. In response to these concerns, the FDA is taking a closer look at the AC meeting process to determine what changes may be needed to ensure that ACs remain able to provide crucial expert advice relevant to the uncertainties that prompt such meetings.

Robert Califf

The process of engaging the expertise needed for ACs requires careful consideration, and the goal of ensuring that such a critical function leads to the best advice with optimal public trust by eliminating or managing conflicts is embedded in both law and culture at FDA. Experts who comprise ACs generally are classified as “special government employees” (SGEs) of the FDA. As such, they must declare any potential conflicts of interest and undergo a rigorous financial screening to ensure that they do not have a conflict or apparent conflict that could preclude their participation. SGEs are also expected to be free of intellectual bias that may foreclose their ability to consider the data and questions with an open mind.

Sometimes, a compelling interest can justify allowing a SGE with a potential conflict to participate. In such a case, the prospective AC member must be granted a waiver or appearance authorization, which provide a mechanism for clearly delineating the reasons for allowing that person to participate and requires disclosing the conflict. This aspect of the AC process has evolved over time, becoming increasingly complex and burdensome.

In 2007, the Food and Drug Administration Amendments Act (FDAAA) restricted the FDA’s ability to use waivers for SGEs as part of an effort to reduce bias among AC members by allowing minimal or no financial conflicts. This led to concerns from multiple stakeholders about whether the FDAAA provision was in fact discouraging the most qualified experts from serving on ACs and thus depriving FDA of the best possible guidance on important scientific issues.

In response to these concerns, Congress included a provision in the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) that encouraged FDA to weigh an AC member’s conflicts against the need for that participant’s scientific expertise. However, despite this added flexibility, there are many who believe FDA has not been aggressive enough in advocating for waivers — a circumstance that they believe has sometimes resulted in difficulty obtaining the optimal expertise needed to address the complex problems typically brought to ACs. And some outside the Agency have wondered whether this means FDA is moving to reduce use of ACs.

The process for AC participation itself has led to other criticisms. Across academia, the AC system is seen as overburdened with unnecessary paperwork. Additionally, FDA has faced criticism that the concept of an “imputed interest” is interpreted so that academic leaders with significant experience and insight are considered to have conflicts relating to grants and contracts held by faculty members at the same institution — even if they themselves have no involvement with the project. The proliferation of roadblocks to serving as an SGE has led some within FDA and key leaders in various scientific fields to question the value of ACs in their current form.

After indepth discussion with the medical product and tobacco Centers, OMPT initiated a process improvement evaluation using Lean concepts, which comprise an industrial engineering toolset used for process improvement. These tools were applied to the AC process to fully understand the administrative requirements for planning meetings and screening potential SGEs. We are confident that administrative processes, both inside FDA and for SGEs, will be streamlined as a result.

The next step will be to evaluate current policies and identify areas where the evaluation of conflicts of interest for SGEs can be modernized. We must consider questions such as the criteria for disqualifying AC members from specific activities, the appropriate scope of “imputed interests,” and the interrelationship between the advisory role of AC members and the decisional role of Agency employees.

Even more importantly, we must engage in wide-ranging discussions inside and outside FDA about the best ways for the Agency to get the advice it needs to make critical decisions that protect and promote the health and safety of all Americans. To obtain the best expertise possible, we must optimally configure and administer our ACs.

There is no question that we must appropriately address potential conflicts for our SGEs.  However, we must also ensure that experts working in their fields are not unnecessarily foreclosed from participation in the AC process. As we continue to improve the mechanics of ACs and to reduce unnecessary administrative burdens, we must also address the appropriate mix of expertise on committees, so that FDA scientists and staff get the advice they need to make the best decisions on behalf of the American public.

Robert M. Califf, M.D., is Commissioner of the U.S. Food and Drug Administration

Clacker Balls and the Early Days of Federal Toy Safety

By: John P. Swann, Ph. D.

As many of us scramble to find the perfect toy for the children in our lives this holiday season, it’s interesting to note that at one time FDA could very well have been known as the Food Drug and Toy Administration.

John SwannThe 1966 Child Protection Act gave FDA authority to ban toys that had chemical, flammability, or radioactivity hazards and the 1969 Child Protection and Toy Safety Act further defined FDA’s responsibility for ensuring the safety of toys, which the agency pursued until the formation of the Consumer Product Safety Commission in 1973.

During FDA’s brief stint as toy regulator, the agency dealt with flammable dolls; infant and toddler playthings that posed serious puncture, laceration, and crushing risks; and the infamous lawn darts. To appreciate FDA’s role at the time, consider what happened with clacker balls. Their origin was unclear, but clacker balls were a veritable craze by January 1971, to the consternation of parents and school districts everywhere. Sold under a variety of other names such as Kerbonkers, Click-Clacks, and Bo-Los, they could vary somewhat in design but typically, they were plastic, wooden, or steel balls of about two-inches in diameter. The balls were attached at each end of a two-foot cord that had a ring or knot in the middle. By holding the cord in the middle and moving your hand up and down, you could cause the balls to strike each other at the top and bottom of an arc with great force and abundant noise. By the spring of 1971, over 100 manufacturers had sold millions of clacker balls, according to FDA’s Bureau of Product Safety, which oversaw such commodities. And it was not a uniquely American phenomenon. The village of Calcinatello in Italy held an international competition of clacker ball enthusiasts in August 1971. In lieu of a trophy the winner received a variety of local delicacies.

picture of clacker balls

Assorted clacker ball sets, including some that ruptured.

Many compared the sudden popularity of clacker balls with the Hula Hoop, but they posed considerably greater risk to the user, as FDA soon discovered. Though advertised to “teach skill and coordination” and to consist of “non-breakable plastic with strong cord,” some of those enthusiasts sustained serious eye and other injuries due to the design and action of the toy, prompting the Society for the Prevention of Blindness to raise an alarm.  The balls could rupture and spew fragments, or become wayward missiles through detachment from or fraying of the cord itself.

The 1969 Toy Safety Act had provided for the banning of toys that represented an electrical, mechanical, or thermal hazard, pending the agency’s publication of the hazardous nature of the item and, if applicable, means of eliminating the hazard. And the agency took that seriously, banning more than 300 individual toys in seven classes through 1971.

After learning  of several injuries to children and adults from clacker balls, FDA issued a public warning in February 1971 as it investigated these reports and studied the toy and its potential to hurt the user—or bystanders.

FDA Toy Review Committee

The recommendations of the Toy Safety Review Committee in the Bureau of Product Safety (pictured here), which included a pediatrician and two engineers, played a key role in FDA’s decisions to ban, further test, mandate product redesign, or create regulations to address the hazards.

With ongoing reports of clacker ball injuries, just two months later, in April, FDA published its intention to ban such toys unless they could meet detailed standards of safety that addressed the integrity of the balls, cords, and connections that held them together.  FDA’s notice stated that the manufacturer would have to carry out the prescribed tests on a proportional number of samples based on the size of the batch produced, maintain records of these results, and make them available to the agency upon request. FDA finalized this plan in November 1971, and all clacker balls marketed thereafter had to either abide by the new safety standards or be subject to the ban.

The rulemaking process rendered clacker balls a safer toy, and those that failed to follow the standards were seized. Over 300,000 sets were put into storage at the Port of Miami while the agency was developing its safety standards for the clacker balls (presumably because the manufacturer knew they wouldn’t pass the safety tests). After a manufacturer tried, unsuccessfully, to find a market for these in South America, a salvage dealer apparently purchased the lot late in 1972, but their subsequent history is unknown.

Ensuring safety was FDA’s goal – and later that of the CSPC. But the toy’s noise was quite another thing. The standards applied by the two agencies never addressed the click-clack-click-clack that tormented parents and others for all these years.

Enjoy the click-clack sound of clacker balls!

John P. Swann, Ph.D., is an FDA Historian

The Race to bring Penicillin to the Troops in WWII

By: John P. Swann, Ph.D.

John SwannThis Veterans Day we remember that nearly 75 years ago dozens of American academic, commercial, nonprofit, and governmental institutions – including FDA – joined together in a race to provide a promising but complex and unstable medicine to troops fighting in World War II — penicillin. Knowing that infection is the major killer in wars, not battle injuries, their goal was to help turn a British discovery into a crucial wartime medical contribution and what would become an indispensable therapeutic agent long after that conflict ended.

Many people are familiar with the story of Alexander Fleming’s 1928 discovery of a Penicillium mold that had contaminated — and surprisingly destroyed — his cultures of pathogenic organisms.

The strain of Penicillium notatum that Fleming discovered at St. Mary’s Hospital in London

The strain of Penicillium notatum that Fleming discovered at St. Mary’s Hospital in London.

Though Fleming and several others in the next decade studied the mold filtrate, known as penicillin, it was Howard Florey and his colleagues at Oxford who uncovered the drug’s chemotherapeutic potential. Their work began with studies in mice in May 1940 and transitioned to a handful of clinical cases nine months later. However, the drug was difficult to purify. Also, it presented an immense challenge to produce in sufficient quantities for study, and with Britain under siege firms there were too involved in other aspects of the war effort to offer much assistance. So Florey and a colleague came to the U. S. in the summer of 1941 for help.

Northern Regional Research Lab

A meeting of NRRL staff in the 1940s (courtesy of the American Institute of the History of Pharmacy).

Among the first sites they visited was the Department of Agriculture’s Northern Regional Research Laboratory (NRRL) in Illinois, which had extensive experience in fermentation work, and from there they contacted several drug and chemical companies to drum up support.  Americans quickly combined forces to tackle the challenge. The federal Office of Scientific Research and Development (OSRD), the federal entity that organized and facilitated investigations to support the war effort, arranged to act as a clearing-house for the latest research on chemical and other studies of penicillin, exchanging data with dozens of organizations in the U.S. and Britain. NRRL developed several production modifications that increased the yield of penicillin by 100 fold.

Penicillin Moisture Testing

An FDA analyst in the 1950s carries out part of the procedure in testing penicillin for moisture content.

FDA’s first experience with the potential wonder drug was around September 1942, when the NRRL Director approached FDA about testing the antibacterial effectiveness of a small quantity of penicillin. A year later, enough of the drug had been produced to confirm in 200 patients what the early results at Oxford had suggested, and penicillin was ready to enter the war. First, however, OSRD asked that FDA certify every lot produced by the half-dozen or so manufacturers, a task the agency also performed for insulin under statutory authority that began in 1941. Six FDA technicians certified samples for potency, absence of fever-producing contaminants, toxicity, sterility, and optimum moisture, which can affect the drug’s stability. So scarce was penicillin that companies always reconditioned the occasional rejected lot rather than destroying it.

By the end of the war, some of the participating firms had increased purity of the drug from the Oxford group’s one percent to about 85 percent. Penicillin was not only more potent, it was also more abundant, its production having increased by a factor of 500 from 1943 to 1945. In fact, by 1945 the output of penicillin, formerly under severe restriction outside of military and scientific use, was now available for most civilian needs as well. In a few years the cost of producing penicillin had decreased so much that the glass used to store ampules of the drug cost more than the drug itself. FDA’s wartime work was codified in the Penicillin Amendment of 1945, which mandated FDA’s certification of penicillin and, through subsequent laws, most other antibiotics — a responsibility that continued for nearly four decades, when the need for government testing no longer existed based on industry’s record of production.

But it all started with an international effort to provide a lifesaving drug to the armed forces, bringing together all sorts of scientific and medical institutions, including FDA. Like so many others participating in this collaboration on a scale unseen up to that point, FDA played a small but critical role to support our troops at this time of global crisis.

John P. Swann, Ph.D., is an FDA Historian

Consumer expenditure on FDA regulated products: 20 cents of every dollar

By: Sheri Walker, Ph.D., and Clark Nardinelli

Sheri Walker

Sheri Walker, Ph.D., is an FDA Senior Economist

One of the much-cited statistics about FDA is this: that FDA-regulated products account for about 20 cents of every dollar of annual spending by U.S. consumers. Add up 20 cents of every dollar and it amounts to more than $2.4 trillion in annual consumption that includes medical products, food and tobacco.

Our staff of 34 economists comes up with this estimate of FDA’s impact every year. We think it helps the public put in perspective the sheer scope of FDA’s responsibilities, especially when you recognize that FDA is only one of dozens of governmental agencies.

We largely rely on personal consumption expenditure data collected by the Bureau of Economic Analysis (BEA) every year to calculate total consumer spending in each of the major FDA product categories. These product categories include food (except alcohol and meat products regulated by USDA), drugs, medical devices, cosmetics, dietary supplements, and (since 2009) tobacco products.

Clark Nardinelli

Clark Nardinelli is FDA’s Chief Economist

Some BEA expenditure categories include more than one FDA product area. For example, biologics and dietary supplements are included in the expenditure for pharmaceutical and medical products (although, legally, dietary supplements are food). Cosmetic products are captured under the BEA expenditure category for personal care products. Pet food and animal drugs are estimated as a percentage of the pet-related products category. The estimate for medical device products is derived using data from the therapeutic equipment products category from the BEA and data from the Annual Survey of Manufacturers collected by the U.S. Census Bureau.

Food products represent the largest share of spending on FDA products, accounting for approximately 11 cents of every dollar of consumer spending. Without the addition of tobacco products, spending on FDA-regulated products would be slightly less than 20 cents per dollar.

20 cents pie chartWe know that some people say FDA oversees 25 cents of every consumer dollar. Maybe it’s an urban legend – or maybe it harkens back to decades ago. The 20 cents (or 20 percent of spending on consumer goods and services) has held steady over the past 5 years. Americans used to spend a much higher proportion of their income on food – with over 25 cents of every dollar going to food during World War II. But since then the share of food and tobacco in total consumer spending has been falling steadily while the share of consumer spending devoted to medical products has been steadily climbing. Whether those trends will continue, and whether FDA’s 20 cents will hold steady for the next 5 or 50 years, is impossible to predict.

Sheri Walker, Ph.D., is an FDA Senior Economist, and Clark Nardinelli is FDA’s Chief Economist

New FDA/EMA rare diseases and patient engagement clusters underway

By: Jonathan Goldsmith, M.D., FACP, and Sandy Kweder, M.D., RADM (Ret.) US Public Health Service

Drug development and approval happens across the globe and we at FDA strive to collaborate with other countries and international regulatory agencies to ensure public health. One of our most valuable collaborators is the European Medicines Agency (EMA) — our counterpart agency for drug regulation in Europe that coordinates a network of 4,500 scientists and evaluates and supervises medicines for more than 500 million people in 31 countries.

Dr. Jonathan Goldsmith

Jonathan C. Goldsmith, M.D., FACP, FDA’s Associate Director Rare Diseases Program, Center for Drug Evaluation and Research, Office of New Drugs

For more than a decade, FDA and EMA scientists have collaborated to help solve some of our biggest challenges. We work with them in groups called “clusters.” The first cluster was initiated in 2004. Since then clusters have been formed to focus on treatments for children; establish effective measures for the development and use of biosimilar medications as cost effective alternatives to brand name biologic drugs; evaluate new treatments for patients with cancer; set standards to help develop medicines personalized to a patient’s genetic makeup, and much more. Both agencies have benefited from this joint work. The EMA summarizes these and our other clusters on its website.

We are excited about the initiation of our most recent cluster activity with our EMA colleagues. Just last month we established a cluster that will work to advance treatments for patients with rare diseases. This cluster’s primary goal is for FDA and EMA scientists to share valuable information about their work and to collaborate on certain review aspects of rare disease drug development programs. FDA’s core members of the cluster include experts from FDA’s Center for Drug Evaluation and Research’s Rare Diseases Program, the Office of Pediatric Therapeutics, the Center for Biologics Evaluation and Research’s director’s office, and the Office of Orphan Products Development, but other experts will be engaged on specific topic areas as the cluster evolves. Among many other important activities, our agencies will collaborate on:

  • Identification and validation of trial end points;
  • Potential trial designs when only small populations of patients are available for testing the safety and effectiveness of prospective new therapies;
  • Ways to apply flexibility in evaluation of drug development programs;
  • Expediting the review and approval of drugs to treat rare diseases to bring new drugs to patients in need as soon as possible.
Sandra Kweder

Sandra Kweder, M.D., Rear Admiral (Ret.) US Public Health Service, FDA’s Deputy Director, Europe Office, and Liaison to European Medicines Agency

Our work also builds on another exciting and recent development — a patient engagement cluster formed in June 2016 to incorporate the patient’s involvement and viewpoint in the drug development process. FDA and EMA are interested in understanding patient’s experiences and gaining input on their tolerance for risk and uncertainty, on current therapy and its benefits or shortcomings and on the benefits that patients seek. This cluster, among other valuable efforts, will:

  • Help each agency learn how the other involves patients in their work, and to develop common goals of expanding future engagement activities with patients;
  • Discuss ways for finding patients that can serve as spokespersons for their community;
  • Explore ideas to help train selected patients and advocates to effectively participate in agency activities, and;
  • Develop strategies for reporting the significant impact of patient involvement.

Given the focus of both of these new clusters, we expect they will address new areas of interest and also draw on expertise from all of the other clusters, such as oncology, pediatrics, and orphan diseases, contributing to more advanced and robust collaborations across both of our organizations.

Focusing on patients with rare diseases and working to advance patient input enhances the value of our cluster activities. With our colleagues at the EMA we look forward to accomplishing more than what we can individually.

Jonathan C. Goldsmith, M.D., FACP, FDA’s Associate Director, Rare Diseases Program, Center for Drug Evaluation and Research, Office of New Drugs

Sandra Kweder, M.D., Rear Admiral (Ret.) US Public Health Service, FDA’s Deputy Director, Europe Office, and Liaison to European Medicines Agency

Where We Are/What We Have Done – Two Years After Releasing Our FDASIA 907 Action Plan

By: Janice Soreth, M.D.

Since it’s been more than two years since FDA unveiled its Action Plan to advance the inclusion of diverse populations in clinical trials, we’d like to update you on how much we have accomplished, and acknowledge that continued commitment is critical in order to build on this foundation.

Janice SorethThe Congressional mandate under Section 907 of the FDA Safety and Innovation Act of 2012 required FDA to develop a report examining the extent to which various demographic groups were included in clinical trials and their outcomes reported in labeling for medical products for which applications were submitted to FDA. The legislation also required FDA to develop an Action Plan based on the report findings and input from stakeholders, issued in August 2014. The Action Plan identified 27 discrete actions for FDA to take within the three priority areas: improving data quality, encouraging greater clinical trial participation, and ensuring more data transparency.

As we discussed at our public meeting on February 29th, we have made progress on nearly every one of our action items and we continue to make strides.

In June 2016, FDA issued the draft guidance, “Evaluation and Reporting of Race and Ethnicity Data in Medical Device Clinical Studies.” We are also updating the 2005 “Guidance for Industry Collection of Race and Ethnicity Data in Clinical Trials.”

Our popular Drug Trials Snapshots, providing information about who participated in clinical trials supporting FDA-approved drugs and biologics, have now been posted on some 75 products. This innovative program developed by our Center for Drugs Evaluation and Research also highlights whether there were any differences in the benefits and side effects among sex, race, and age groups

We have significantly advanced efforts to raise clinical trials awareness. FDA’s Office of Women’s Health instituted a new initiative on “Diverse Women in Clinical Trials” that is disseminating consumer resources in English and Spanish and tools for clinical researchers in partnership with NIH’s Office of Research on Women’s Health. Our Office of Minority Health developed a tool kit and posted several public service announcements on FDA’s YouTube channel aimed at engaging patient participation. And we are currently reviewing the public comments from a range of organizations that we received to the public docket that was opened at the time of the public meeting.

Finally, I want to announce that I recently took over the chairmanship of the steering committee charged with implementing this plan. I am currently the Acting Associate Commissioner for Special Medical Programs, which has oversight of our advisory committee programs, combination products, and pediatric and orphan products programs among other responsibilities. Since joining FDA as a primary medical reviewer 25 years ago, I have served as CDER’s director of the division of Anti-Infectives and Ophthalmology and most recently spent five years in London as Deputy Director of the FDA Europe Office and Liaison to European Medicines Agency.

As we look back at our accomplishments, we believe that transparency in reporting about clinical trial inclusion will make a difference in encouraging broader demographic diversity and want to thank the former chair, Barbara Buch, M.D., of CBER, for her accomplishments. Going forward, I encourage you and all of our key stakeholders – patient and disease advocates, health professionals, and industry to continue partnering with us to advance this important work in ensuring demographic diversity and representation.

Janice Soreth, M.D., is Chair of the FDA Safety and Innovation Act Section 907 Steering Committee and the Acting Associate Commissioner for Special Medical Programs