Rare Disorders Without Borders: An International Strategy

By: Katherine Needleman, Ph.D.

The development of 200 new therapies for rare diseases and diagnostic tests for most rare diseases would alleviate untold suffering. So with great enthusiasm, FDA’s Office of Orphan Products Development (OOPD) has joined a global effort to make those goals a reality by 2020.

These are the goals of the International Rare Disease Research Consortium (IRDiRC),  launched by the European Commission and the U.S. National Institutes of Health in April 2011 to foster collaboration in rare disease research. FDA’s OOPD recently joined IRDiRC’s Executive Committee. When the consortium holds its first major conference in Dublin in April, top experts and researchers from around the world will share information and foster collaborations.

Collaboration is critical. Rare disease resources, including experts, are so limited that we cannot afford to waste a single moment by duplicating work or failing to share promising developments. Products for rare diseases are often called “orphan products” because they generally lack sponsors to develop them.

Some rare diseases affect only a few hundred people, and by FDA’s definition, each one affects no more than 200,000 people in the U.S. Yet taken together, about 7,000 rare diseases afflict about 30 million Americans and a similar number in Europe. In much of the world, they go undiagnosed. Often, they are debilitating or deadly. Many are genetic, so children are often victims.

Today, on Rare Disease Day 2013, the international rare disease community comes together with the theme “Rare Disorders Without Borders,” and FDA continues its commitment to close collaboration with international counterparts, such as the European Medicines Agency.  

FDA is the first regulatory agency to join the consortium’s Executive Committee. In addition, FDA is represented on the consortium’s Therapies Scientific Committee by Marc Walton, M.D., Ph.D., in FDA’s Center for Drug Evaluation and Research. We hope to bring regulatory scientific perspectives that help develop great research ideas into great products that meet regulatory approval standards for safety and effectiveness.

FDA has been working to change the orphan status of rare diseases since 1983, with the passage of the Orphan Drug Act. Since then, FDA has approved over 400 products for the treatment of rare diseases, compared to only 10 developed by industry in the decade before the act.

OOPD’s grant program uses its annual budget of approximately $14 million to fund top scored clinical trials of rare disease treatments. The grants program has been successful, as at least 49 OOPD grants supported clinical trials that contributed to agency approval of these products for rare diseases.

Still, for most rare diseases, there are no treatments, or definitive diagnostic tests.

Recent progress in human genomics and newly emerging sciences increase the prospect for treatments or even cures. A new generation of therapies even shows promise for changing the defective genes that result in some rare diseases. These are among the topics that will be addressed at the upcoming conference.

Our OOPD Director, Gayatri Rao, M.D., J.D., will present FDA’s perspective at the IRDiRC meeting while I will be working on the challenges ahead through the Executive Committee as FDA helps ensure that the energy and expertise brought to the conference is harnessed to bring results to patients.  

The need is great; the time is now.

Katherine Needleman, Ph.D., is director of the Orphan Products Grants Program of FDA’s Office of Orphan Products Development.

Advancing Science and Building a Healthier Society

By: Theresa Castillo

Knowledge and education are critical, but “passion and perseverance” are also needed to eliminate health disparities, Assistant Health and Human Services Secretary Howard Koh, M.D., told a group gathered to pursue the goal of better health for all.

Dr. Koh’s call for even greater commitment was issued to more than 4,500 people who attended the Summit on the Science of Eliminating Health Disparities at the Gaylord National Resort and Convention just outside of the nation’s capital in December 2012. Since then, individuals and groups around the nation have returned to their jobs with new information gained from some of the more than 100 workshops that explored emerging sciences, policies and practices that can help eliminate health disparities that disproportionately affect minority groups.

Here at FDA, we are now in the midst of collaborating with the National Institutes of Health to publish recommendations and best practices that were highlighted at the summit and worthy of adoption on a broader scale. Articles published in peer reviewed journals also will be available at FDA’s Office of Minority Health Web site later this year.

The summit was led by the National Institute on Minority Health and Health Disparities and co-sponsored by FDA’s Office of Minority Health and the U.S. Department of Health and Human Services. Leaders in government, academia, business, medicine, science and public policy joined with advocates and others in the community to learn, share and build new alliances.

The ultimate goal: to share creative and innovative solutions that can be adopted widely to improve health in theU.S.and around the world.

The work on health disparities arises from a critical need. For example, African American men are 2.4 times more likely to die from prostate cancer than non-Hispanic white men, 10 times more likely to die of AIDS, and 60% more likely to die of stroke. Native American women are almost twice as likely to die of diabetes than non-Hispanic white women. And while Asian and Pacific Islanders are less than 5 percent of the population, they account for more than 50% of Americans living with chronic Hepatitis B and associated liver cancer.

The numbers reflect a complex web of causes, including unequal access to health care, environmental issues, genetic differences and lifestyle, to name a few. These variables and more were addressed at the summit.

The breadth of the effort is exemplified by listing even a few of the more than 100 workshop and roundtable titles: “Approaches for Identifying and Addressing Environment Health Disparities,” “Public Policies and Strategies to Address Obesity Prevention,” and “Reducing Health Disparities through Innovation.”

The summit brought together some of the nation’s foremost experts. Speakers included former U.S. Health and Human Services Secretary Louis Sullivan, M.D., NIH director Francis S. Collins, M.D., Ph.D.,  Mary Woolley, head of Research!America,  Kira Fortune, Ph.D. of the Pan American Health Organization, and David Fukuzawa of the Kresge Foundation.

The energy, commitment and knowledge gathered in one place left me inspired and convinced that together, we are on a path that will lead to improving health for all.

Theresa Castillo is a public health advisor in FDA’s Office of Minority Health

We’re Taking Steps to Enhance the Safety of Cantaloupe for Consumers

By: Michael M. Landa

The growing and harvest season for cantaloupe in the United States is beginning and FDA is working with its state partners and the produce industry to make sure that this is a good, safe year for lovers of this nutritious fruit.

As FDA works on implementing the produce safety standards mandated by the FDA Food Safety Modernization Act, it is essential that those involved in growing, harvesting, and distributing cantaloupe – and produce in general – follow agricultural practices that FDA and the produce industry have identified as effective in minimizing the risk of contamination. 

FDA is now issuing a letter to firms that handle cantaloupe to help reduce the chance that the fruit may be contaminated by harmful bacteria. Our letter has two key messages.

First, it reminds firms to follow good agricultural and handling practices to protect the safety of their product. Second, it informs these same firms that FDA will be inspecting packinghouses over the course of the upcoming growing and packing season to, in part, assess current practices and identify conditions that may adversely affect the safety of cantaloupe.

Two major outbreaks of foodborne illness in 2011 and 2012 were associated with cantaloupe. Preventing this from happening again has been a team effort.

The cantaloupe industry has worked hard since then to revise and update its own food-safety guidelines. In California, the industry has required implementation of its new best practices via a state marketing agreement.

Our colleagues in state agriculture departments are planning their own efforts with growers to enhance cantaloupe safety.

FDA stands ready to provide technical assistance to industry and to work collaboratively with our state partners, retailers and others in pursuit of our common goal to enhance food safety.

Michael M. Landa is Director of FDA’s Center for Food Safety and Applied Nutrition


Weaving New Threads in FDA’s Global Safety Net

By: Margaret A. Hamburg, M.D.

Drugs manufactured in countries around the world account for 40 percent of the medicines we use in the United States. And an astonishing 80 percent of the active ingredients in the drugs we consume come from abroad.

Margaret Hamburg, M.D.Therefore, it is absolutely essential that we have regulations and procedures in place not just here at home but abroad as well to ensure that the medicines you and I rely on are safe, no matter where they come from. After all, lives and well-being are at stake if drugs are of questionable quality.

One of the greatest threats to safety involves substandard, falsified and counterfeit medical products in the supply chain. For the past several years, FDA has been engaged in global efforts to improve collaboration in preventing, detecting, and responding to this threat.

We have developed strong partnerships with the World Health Organization and the Asia Pacific Economic Community and we also collaborate with such development agencies as the U.S. Agency for International Development and the World Bank.

To further extend our global understanding and impact, in 2011 FDA commissioned a committee at the Institute of Medicine (IOM) to look at how falsified (fake) and poor quality drugs affect the health of people around the world. A report on its findings and recommendations was recently released.

It confirmed FDA’s belief that these fraudulent and inferior drugs are an international problem requiring international cooperation.

Briefly, here are some of the findings of the IOM report.

  • Neglecting good manufacturing practices is the root cause of poor quality drugs. However, quality practices require resources. Many of the countries in which people sorely need medical help just don’t have the financial wherewithal and other resources to mandate and monitor these good practices.
  • Bad medicines, whether sold in street markets or on unregulated websites, are a grave public health problem. They often fail to help—and often harm—the very people who most need them. In addition, they may promote antibiotic resistance which harms us all.
  • Crime and corruption drive the business of falsified medicines, and they are rampant overseas. A month’s supply of the lowest price generic ulcer medicine costs more than three days’ wages for the average government worker in much of Africa, Eastern Europe and the Middle East. The poorest patients have little choice but to buy their medicines from vendors who sell products of questionable quality.
  • In modern supply chains, medicines can change hands many times, in many countries, before they reach patients. Each exchange provides the opportunity for unscrupulous behavior on the part of manufacturers and distributors.

The IOM report recommends that FDA continue to reach beyond our U.S. borders to collaborate with our foreign counterparts in building regulatory strategies for the implementation and control of good manufacturing practices. FDA now has 12 posts around the world that are currently working to do just that with our global partners, as well as with industry and scientific and academic communities in these countries.

The IOM committee believes that we must identify where vulnerabilities exist in the legitimate drug supply chain and identify realistic national and international solutions. The panel also urged Congress to establish a track-and-trace system to keep a record of drugs as they travel from the manufacturer to the patient.

Between social media and up-to-the-second news coverage, the world seems like a much smaller place these days. But as this report makes clear, it’s actually larger than ever, and our challenges to provide a global “safety net” are many and formidable. 

The IOM report is an important resource in our quest to strengthen that safety net. For me, this report reinforces the knowledge that when it comes to ensuring the safety of medicines worldwide, we are heading in the right direction.  

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


FDA Cracks Down on Flu Product Scammers

By: Gary Coody, R.Ph.

The severity of this year’s flu season has brought out the scammers promoting fraudulent flu products.

Gary Coody, R.Ph., is FDA’s national health fraud coordinator, FDA Office of Regulatory Affairs, Office of EnforcementThrough our careful monitoring of the Internet, FDA has identified numerous untested and unapproved products being illegally marketed with deceptive claims that they prevent, treat, or cure the flu.

When FDA staff find these fraudulent products, we send a warning letter to the sellers describing how the product violates federal law and instructing them to respond in writing with a description of how they intend to address the violations. If a seller does not respond within 15 days and continues to sell the product without correcting the violations, the products being sold by U.S. companies may be seized, FDA may notify law enforcement officials in the country abroad where a seller maintains its operation, or the federal government may take other legal actions.

In the past week, the agency has sent nine warning letters to firms marketing fraudulent flu-fighting products, including an online seller marketing a product that claims to be an alternative to the flu vaccine, three firms marketing dietary supplements online (letters co-signed by the Federal Trade Commission), and a firm selling an oral spray online and in major retail stores.

The remaining four warning letters were issued to online firms selling what they claim to be generic and other unapproved versions of oseltamivir phosphate, the active ingredient in Tamiflu. Tamiflu is an FDA-approved brand-name drug, but no generic Tamiflu is approved in the U.S.

FDA advises consumers to beware of online “pharmacies” selling generic versions of Tamiflu. If you buy one of these products, you don’t know what you’re getting—it could be counterfeit, contaminated, or not stored properly to maintain quality. It could also have the wrong active ingredient or no active ingredient at all.

Some of the other fraudulent claims addressed in the warning letters include:

  • “the most effective alternative to the flu shot”
  • “natural health and strength can still be yours without flu shots”
  • “fight cold and flu – naturally!”
  • “safeguard you from deadly flu viruses.”

Our concern is that a consumer will buy or use a fraudulent product advertised as an “alternative to the flu vaccine” instead of getting the approved vaccine. The vaccine is the best way to prevent getting the flu.

Any time there is a large outbreak of disease, fraudulent products appear on the market. Bogus remedies were rampant during the 1918 flu pandemic, commonly known as the Spanish flu, which killed an estimated 675,000 Americans. Fast forward to 2009, when FDA sent out more than 100 warning letters to sellers fraudulently promoting their products to prevent the H1N1 (swine) flu.

Today, when a health threat emerges, fraudulent products appear almost overnight because the Internet and social media such as Facebook and Twitter have accelerated how quickly hucksters can reach the unsuspecting public.

FDA can’t track down all of these fraudulent products, so consumers need to beware of unapproved products that make false claims. When in doubt about a product, talk to your doctor, pharmacist, or other health care professional.

For more information about health fraud scams, visit www.fda.gov/healthfraud.

Gary Coody, R.Ph., is FDA’s national health fraud coordinator, FDA Office of Regulatory Affairs, Office of Enforcement

Celebrating African-American Contributions to Public Health

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.

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

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

We take so much for granted.

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

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

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

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