Monthly Archives: February 2013

Celebrating African-American Contributions to Public Health

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By: Jonca Bull, M.D.

As a medical doctor and director of FDA’s Office of Minority Health, I am highly conscious of health disparities in the United States. Certain racial and ethnic populations respond differently to some medical products. FDA ensures that these differences are considered in its review of marketing applications for medical products.

But as an American, I am also highly aware how much all of us in this country have in common, a simple truth that emerges with particular clarity during this African American History Month, when we commemorate the 150th anniversary of President Lincoln’s Emancipation Proclamation and the 50th anniversary of the March on Washington.

Both events have had a profound effect not only on African Americans, but on our entire nation. The Emancipation Proclamation was critical to President Lincoln’s efforts to end the Civil War and advance freedom. The March on Washington brought us Dr. Martin Luther King’s unforgettable call to America to live up to his “dream.” His vision of equality and racial harmony has been a steadfast guide for all Americans as we strive toward “e pluribus unum”— “Out of Many, One”, the ideal enshrined in the Seal of the United States.

This common bond unites us in many other ways.

As an ophthalmologist and a physician, I have a great admiration for the many distinguished African Americans who advanced medical science.

For example, Dr. Charles Drew discovered a method for the preservation of blood that was used extensively during World War II by the British military to save the lives of wounded soldiers. After the war, Drew was appointed the first director of the American Red Cross Blood Bank.

He was only one of many outstanding African-American scientists.

This honor roll includes Percy Julian, an Alabama native who earned a Ph.D. from the University of Vienna, and in 1935 synthesized physostigmine, a drug for the treatment of glaucoma, and cortisone for the treatment of rheumatoid arthritis.

It includes Daniel Hale Williams, the first surgeon to successfully perform open-heart surgery, Dewey Sanderson, the inventor of the urinalysis machine, Otis Boykin, who designed a control unit for the heart pacemaker, and Michael Croslin, who computerized blood pressure devices.

As a civil servant, I celebrate the history of African Americans by remembering their leadership in public health. The first to earn this distinction was Patricia Harris, a Howard University professor, who in 1979 became the Secretary of Health, Education and Welfare. Ten years later, the department was entrusted to the leadership of Dr. Louis Sullivan, the founder of the Morehouse School of Medicine. And in 1993, Dr. Joycelyn Elders, a pediatrician and public health administrator, became the first African-American to be named Surgeon General.

And as an FDA employee, I am proud to be working with many outstanding administrators and scientists of African-American ancestry who every day contribute to the public health and help advance FDA’s mission.

I have mentioned just a few of the distinguished narratives in our history that have been authored by remarkable African Americans—intellectuals, professionals, soldiers and artists of outstanding achievement.

I celebrate these women and men as fellow Americans whose extraordinary spirit, talent and efforts advance better health for all. I celebrate them as an inseparable part of my own proud heritage as an African American citizen.

Jonca Bull, M.D., is Director of FDA’s Office of Minority Health

No Longer In the Dark

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By: William Maisel, M.D., M.P.H.

We take so much for granted.

That was my thought when I first heard about the Argus II Retinal Prosthesis System, a new device approved by the FDA today.

This small electronic device, implanted in the eye, may improve the visual function of patients with advanced retinitis pigmentosa, or RP.

What is RP? It’s a rare genetic eye disease that affects approximately 100, 000 people in the U.S. RP gradually damages the light-sensitive cells that line the retina (a membrane within the eye). In time, a person’s limited ability to tell light from dark can erode. Far too often, the outcome is total blindness.

After carefully reviewing data from a clinical study of 30 patients with RP, FDA is giving the go-ahead to Second Sight Medical Products Inc. to sell the Argus II, the first device of its kind. It has three components: a small electronic device surgically implanted in the eye, a tiny video camera mounted on a pair of glasses and a control unit that is carried by the patient.  

The camera captures images. The images become an electric signal. The brain perceives this signal as light.

I could say a lot about how this device works and what it can do for those with RP, but instead, I’ll let patients from the study speak for themselves. Here are excerpts from their comments at the public forum of FDA’s advisory meeting:

“The biggest thing to me was being able to see the crosswalk lines on the street so I can safely cross streets in Manhattan.”

“The most exciting day to me was October 27th, in 2009. It was the first time I was able to see letters on the monitor screen (during a test of visual perception). I had not seen letters since 1994, so that was huge.”

“I have a son who is 17 years old and … I don’t mind telling you how much — I mean, how happy that made me, not only to see the silhouette of my son, but to hear that voice coming and saying, ‘Yeah, it’s me, Dad. I’m here and I love you.’ ”

For many of the approximately 1,300 individuals who will develop the disease this year, this technology may change their lives. And FDA, on the cutting edge of regulatory science, played an important role in that.

It’s the difference between night and day.

William H. Maisel, M.D., M.P.H., is Deputy Director for Science and Chief Scientist at FDA’s Center for Devices and Radiological Health

 

FDA is asking the public to send in ideas for combating drug shortages

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By: Valerie Jensen

FDA has made progress over the last year or so in preventing and resolving shortages of important drugs — including chemotherapies, anesthetics and antibiotics. Nevertheless, the agency believes that even more can be done and is therefore turning to the American public for advice, as explained in a Federal Register notice published this week. What the public tells FDA will help inform the agency’s development of a strategic plan that will ultimately enhance FDA’s response to preventing and mitigating drug shortages.

FDA has long been tackling the problem of drug shortages, and since October 2011, has stepped up its efforts to encourage drug and biological product manufacturers to report if they know of any circumstances that could lead to a drug shortage, including temporary interruptions in manufacturing. Such early notification is the agency’s most powerful tool to address drug shortages—we can’t work to prevent, mitigate, or resolve a shortage if we don’t know about it. Along these lines, FDA supported efforts to expand the FD&C Act’s early notification requirements as part of the Food and Drug Administration Safety and Innovation Act (FDASIA), enacted on July 9, 2012.  Happily, these efforts have been paying off.  For example:

  • The number of shortages is now less than half of what it used to be. There were 117 in 2012, down from 251 in 2011.
  • Many more shortages are now being averted. We prevented 195 in 2011. Last year, we prevented 282.

We expect the requirements in FDASIA to further enhance FDA’s efforts to work with manufacturers and other stakeholders to prevent or alleviate shortages. When notified of a potential or actual shortage, FDA can take a number of actions, as appropriate, including: expediting inspections and reviews of regulatory submissions, working with the manufacturer to solve the underlying problem contributing to the shortage, identifying alternative manufacturing sources, exercising enforcement discretion for the shipment of a critically needed drug with special instructions to healthcare providers, and using enforcement discretion for the temporary importation of non-U.S. product.

One shortage of a drug that improves or saves the life of even one patient is one shortage too many. More can be done to prevent shortages.

As required by FDASIA, FDA has also formed an internal Drug Shortages Task Force to develop a strategic plan to enhance the agency’s efforts to address and prevent drug shortages. Among other things, the strategic plan will include blueprints for enhanced coordination, communication, and decision making within FDA and with other federal agencies; and plans for effective communication in the event of a shortage, including who should be alerted about potential or actual drug shortages and what information should be shared.

FDA wants to hear from all interested stakeholders on the strategic plan. The agency published a Federal Register notice, posted Feb. 11, which provides additional information and seeks input on six targeted questions related to the Strategic Plan and to preventing and mitigating drug and biological product shortages. Comments will be accepted through Thursday, March 14, 2013.

Valerie Jensen, a pharmacist and expert on drug shortages, is associate director at FDA’s Center for Drug Evaluation and Research  

Trying to Meet a Desperate Need: Treatments for Alzheimer’s Disease

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By: Russell Katz, M.D.

If no treatments are developed to prevent, cure or slow the progression of Alzheimer’s disease (AD), the number of Americans suffering from this disease will grow from 5.4 million to as many as 16 million by 2050, according to estimates by the Alzheimer’s Association.

The aging of baby boomers is fueling what could turn a public health problem into a public health crisis. 

So it is with a sense of urgency that FDA has issued a new draft guidance to assist, encourage, and facilitate those working to develop drugs to treat the early stages of AD. The ultimate goal: preventing or arresting AD before the brain suffers too much damage.

AD is a degenerative disease that kills brain cells over time, eventually impairing patients’ thinking and function. There is no simple laboratory test for AD, so diagnoses are generally based on patients’ symptoms. The lack of a laboratory test creates an obvious challenge: identifying people at risk of developing AD, as well as those who are in the earliest stages of the disease – when symptoms are difficult to perceive.  Yet these are precisely the people one would want to enter into studies of treatments to prevent AD, or to delay progression of AD in its early stages.

FDA’s guidance addresses the challenge of choosing such patients for clinical trials to study drugs with the potential to help treat AD.  The guidance also considers the evidence drug makers can use to show that a new medicine can effectively treat symptoms of AD or, better yet, slow progress of the disease.

The guidance recognizes, for example, that patients with very early AD may not yet show problems in performing daily life tasks—in other words, their symptoms may be difficult or impossible to detect. In studies of drugs to treat more advanced AD there is an effort to show effects on both thinking ability and function in daily activities. When there are not yet problems with daily function, the guidance says that showing evidence that a drug delays impairment of thinking may provide sufficient evidence to support “accelerated” approval, where there is a requirement to show, after the drug is approved, that the effect on thinking persists over time.

Another issue addressed in the guidance is how to demonstrate that a new medicine is actually modifying or slowing AD. In other words, the drugs now approved for AD may improve thinking and daily function, but despite these improvements, the disease continues to progress, worsening as it would without the drug.

FDA is seeking comments on the guidance, and these comments will be considered before FDA publishes a final guidance. The draft guidance is in the Federal Register.

FDA is devoted to changing the trajectory of this devastating, life-stealing disease, and looks forward to seeing new medicines that can help accomplish this.

Russell Katz, M.D., is director of FDA’s Division of Neurology Products, which regulates and reviews new drugs and marketing applications for treatment of neurological conditions, including Alzheimer’s disease.

Early communication: A key to reduced drug development and approval times

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By: Anne Pariser, M.D.

From “test tube” to market typically takes a new drug more than 10 years.  FDA has been working hard at many points along a drug’s developmental path to reduce this time and bring safe and effective new therapies to Americans as efficiently as possible. 

Much has been said about FDA’s success in using its “expedited approval” tools, specifically Priority Review and Accelerated Approval, to support innovative new drugs. These are important tools that FDA can use once a marketing application is submitted. For instance, last year, FDA’s Center for Drug Evaluation and Research (CDER) approved 39 novel medications, almost half of which benefited from one (or both) of these expedited approval tools. According to a recent FDA report, this is a 63% increase over the average number of annual approvals since 2002. 

But less has been said about FDA’s “expedited development” tools, which help foster new drug innovation during the investigational phases of drug research and development, well before a marketing application for a new drug is even submitted to FDA. Among these tools are more frequent and earlier opportunities for communication between FDA and drug developers. FDA’s Fast Track designation for drugs with the potential to address unmet medical needs is an example. For many years, Fast Track has helped speed new drug development by encouraging more communication early in the development process. In 2012, about 40% of CDER’s novel new drug approvals were drugs that were given this Fast Track designation.     

Just this past year, the Food and Drug Administration Safety and Innovation Act (FDASIA) authorized FDA to use a new Breakthrough designation for investigational new drugs when preliminary clinical data suggest that the drug may provide a substantial improvement over existing therapies for patients with serious or life-threatening diseases. The concept behind Breakthrough is that, with increased communication, FDA will work with new drug developers to help design efficient ways to study the safety and effectiveness of their drug. This early assistance can help ensure that the results of clinical trials provide the evidence that FDA must have to determine whether or not a drug is safe and effective for approval. A growing number of drug developers are already taking advantage of Breakthrough.

But even before Breakthrough had been authorized by FDASIA, FDA was working to encourage communication opportunities for drug developers to meet with FDA to help make sure their clinical trial designs and development plans offered the best chances of efficient, safe, and timely development and approval. These opportunities are available at the start of a drug’s clinical development cycle: right before the earliest phases of human testing known as the “pre-investigational new drug (IND) phase” (fittingly called pre-IND meetings) and continue throughout drug development.

Early communication in action

Recently, FDA has taken a look at the development times of new drugs that were approved with the benefit of pre-IND meetings and compared them to the development times for drugs that were approved without such meetings. The findings underscore the value of early communication. For those new drugs for which a pre-IND meeting between the drug developer and FDA was held, average clinical development times were substantially shorter than when a meeting was not held. For instance, for all new drugs approved between 2010 and 2012, the average clinical development time was more than 3 years faster when a pre-IND meeting was held than it was for drugs approved without a pre-IND meeting.

For orphan drugs used to treat rare diseases, the development time for products with a pre-IND meeting was 6 years shorter on average or about half of what it was for those orphan drugs that did not have such a meeting. Early communication is especially important for orphan drugs because these products require special attention and thus early talks can be especially beneficial.

Many factors can influence the speed and efficiency of a drug development program. Nevertheless, FDA strongly believes in the value of effective communication during the drug development and approval process, especially for novel development programs when established regulatory pathways do not exist. FDA is committed to working with drug developers to ensure efficient and effective drug development programs whenever possible.

Thirty-nine novel new drug approvals last year is encouraging – one third of which are indicated to treat rare diseases – and many of these new drugs are now making valuable contributions to public health inAmerica. FDA will continue to do its part to help bring safe and effective new drugs to market as soon as possible. We will continue efforts to enhance communication as a critical part of the drug research, development, and regulatory process – especially since it is so clear that communications can make a big difference.

Anne Pariser, M.D., is Associate Director for Rare Diseases, Office of New Drugs, Rare Diseases Program at FDA’s Center for Drug Evaluation and Research

Celebrating American Heart Month—Making Heart Healthy Choices in 2013

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By: Janelle Derbis, PharmD

Each year, nearly half of all Americans make New Year’s resolutions. They often include losing weight, starting an exercise program, quitting smoking, and making healthier food choices—all of which contribute to a heart-healthy lifestyle. February is American Heart Month, and the timing couldn’t be better to make these lifestyle changes, especially since heart disease is the leading cause of death in the U.S.

FDA joins in the commemoration of American Heart Month by highlighting agency initiatives to help Americans reduce their risk of heart disease.

Achieve a healthy weight. Obesity contributes to a number of health conditions, including high blood pressure and high cholesterol. To help obese and overweight Americans who have been unsuccessful in getting their weight under control with diet and exercise alone, FDA approved two long-term weight management medications in 2012—Belviq and Qsymia. These are the first medications the agency has approved for the treatment of chronic weight management in 13 years.

Quit smoking. Smoking and tobacco use contribute to many health hazards, including heart disease. Nearly half of adult Americans are at risk for heart disease and stroke, and over 20% are at risk due to cigarette smoking. To address the huge public health problem of tobacco use, FDA is building a national tobacco product regulation program to reduce the impact of tobacco use on the nation’s health. Using powerful new regulatory tools provided by the law, FDA’s work supports the objective of the Department of Health and Human Services to end the epidemic of tobacco-related death and disease in America. In November 2012, HHS announced the availability of a new comprehensive tobacco website, BeTobaccoFree.gov, a providing one-stop access to the best and most up-to-date tobacco-related information from across its agencies. This consolidated resource includes general information on tobacco, federal and state laws and policies, health statistics, and evidence-based methods on how to quit.

Eat right. Consumers can eat for a healthy heart and choose foods that are lower in salt, cholesterol, and trans-fat by reading the Nutrition Facts label on food and beverage packages.  In January 2013, the agency announced it is planning to update the Nutrition Facts label based on the latest science-based nutrition recommendations. The updates are still being formulated, and public input will be sought when they are proposed.

Lower cholesterol levels.  Making lifestyle modifications can help reduce cholesterol levels. However, hereditary issues can make some people more likely to have high cholesterol levels regardless of diet and exercise. The good news is there are treatment options for people who are unable to lower their cholesterol levels. There are several FDA-approved cholesterol lowering medications on the U.S. market.

In December 2012, FDA approved Juxtapid for a rare cholesterol disorder called homozygous familial hypercholesterolemia (HoFH), an inherited condition that makes the body unable to remove the “bad” cholesterol (LDL cholesterol) from the blood, which  can lead to heart attacks and death before age 30. The approval of Juxtapid is an example of how FDA provides the scientific and regulatory advice needed to bring new treatment options to market.

Control high blood pressure.  Adopting a healthy lifestyle can help prevent high blood pressure. If lifestyle modifications are not enough to lower your blood pressure so that it is within the normal limit (less than 120 over 80), medications are often prescribed. There are many FDA-approved medications to treat high blood pressure so talk with your health care provider to determine which is best for you.

In April 2012, FDA approved the first generic versions of Avapro (Irbesartan) and Avalide (Irbesartan and Hydrochlorothiazide) for the treatment of high blood pressure.  Generic drugs such as these provide safe and effective alternatives to brand-name drugs. 

Exercise. And lastly, physical activity is an essential component of a healthy lifestyle and when done in combination with healthy eating can help prevent heart disease. In 2013, make a commitment to exercising on a routine basis and keep your heart strong!

To receive up-to-date information on heart-related drug and device approvals, safety announcements, and notices of upcoming meetings, subscribe to FDA’s CardioBeat or visit FDA’s cardiovascular webpage.

Janelle Derbis, PharmD, co-manages the Cardiovascular and Endocrine Liaison Program (CELP), at FDA’s Office of Special Health Issues.