For an AIDS-Free Generation: Access to Drugs and Diagnostics Is Essential

By: FDA Commissioner Margaret A. Hamburg, M.D. and HHS Assistant Secretary Jimmy Kolker

Margaret Hamburg, M.D.On World AIDS Day this year, tens of millions of people with HIV are now living healthy, productive lives because of access to safe and lower priced medicines. We rejoice in this achievement, because all people, no matter how rich or poor, deserve to have the medicines they need to live their lives in the best health possible.

We can truly see in our future an AIDS-Free generation because of the wide availability of prevention and treatment tools. But the availability of these drugs and diagnostic tools, especially in Africa, was never a given. Ten years ago, in 2004, the U.S. Food and Drug Administration (FDA) committed to support the President’s Emergency Plan for AIDS Relief (PEPFAR) by introducing an expedited review process to make generic and low-cost treatment more readily available for the most affected countries. PEPFAR requires antiretroviral drugs to be safe, effective, and of high quality and supports their distribution to people needing treatment around the globe. But meeting these requirements can be costly and time-consuming. Those suffering from AIDS cannot wait. The FDA, an agency that is part of the Department of Health and Human Services (HHS), applied the tentative approval process in order to increase dramatically the number of products approved for purchase and distribution by PEPFAR.

Thanks to the commitment of FDA scientists, as of today FDA has issued expedited approval decisions for 179 products, including 39 formulations specifically designed for children that allow flexible dosing across multiple weight bands and many innovative formulations, such as fixed-dose combinations and co-packaged products that improve adherence to treatment and reduce the risk of developing resistance. The 179 tentative approvals allowed PEPFAR to purchase products at a lower cost, leading to cost savings of hundreds of millions of dollars. These savings contributed to additional patients being able to receive treatment.

Jimmy KolkerAccording to UNAIDS, by June 2014, 13.6 million people around the world had access to antiretroviral therapy. This is an important success, but many more people still need access.

Unfortunately, too many countries lack the regulatory capacity to conduct product registrations in a timely manner. This makes it difficult for these countries to provide high-quality rapid HIV tests and treatment.

The FDA and the HHS have been working with the Department of State Office of the Global AIDS Coordinator (S/GAC); the World Health Organization; the Global Fund to Fight AIDS, Tuberculosis, and Malaria; and other organizations to help countries build both their health care systems and regulatory capacities.

Importantly, FDA has partnered with host country health ministries to help strengthen regulatory capacities in support of their public health programs. PEPFAR recently contributed $1.5 million in support of this FDA partnership to further regulatory system strengthening in the East African community.

With these improvements, countries battling HIV and AIDS can build the systems necessary to ensure that patients get the high-quality treatment they need, which one day will lead to the realization of an AIDS-free generation.

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

Jimmy Kolker is Assistant Secretary for Global Affairs in the U.S. Department of Health and Human Services

Achieving an AIDS Free Generation – Highlights from the PEPFAR Annual Meeting in Durban, South Africa

By: Katherine Bond, Sc. D. and Jude Nwokike, MSc, MPH

The U.S. Global AIDS Coordinator, Ambassador Deborah Birx, recently described the President’s Emergency Plan for AIDS Relief (PEPFAR) as “one of the greatest expressions of American compassion, ingenuity, and shared humanity in our nation’s rich history.”

Kate Bond and Jude Nwokike

Katherine C. Bond, Director of FDA’s Office of Strategy, Partnerships and Analytics, Office of International Programs and Jude Nwokike, FDA’s PEPFAR Liaison, Office of Strategy and Partnerships, Office of International Programs.

We recently attended the PEPFAR 2014 Annual Meeting in Durban, South Africa. Since its inception in 2003, PEPFAR, the U.S. Government’s initiative to help save the lives of those living with HIV/AIDS around the world, is supporting 6.7 million people on anti-retroviral treatment (ART) and has resulted in one million babies born HIV-free. In FY 2013 alone, PEPFAR supported 12.8 million pregnant women for HIV testing and counseling and as of September 30, 2013 will have supported voluntary medical male circumcisions for 4.2 million men in east and southern Africa.

The focus of this year’s conference was on delivering a sustainable AIDS Free Generation. We were privileged to represent FDA at the meeting, along with other Health and Human Services operating divisions –including the Centers for Disease Control, the National Institutes of Health, the Health Resources and Services Administration, and the Substance Abuse and Mental Health Services Administration.

FDA has played a critical role in the PEPFAR program. As of March 2014, the Agency had approved or tentatively approved 170 antiretroviral drugs for use by PEPFAR, including 80 fixed dose combinations (FDCs), 24 of which are triple FDCs. Triple FDCs are significant because they have simplified ART from up to 20 pills a day to one pill daily — improving adherence to treatment, reducing the risk of developing resistance, and simplifying the supply chain.

We saw the direct impact of the program during a visit to the KwaMashu Community Health Centre, north of Durban in South Africa’s KwaZulu-Natal Province. Formerly a sugar plantation, the area saw a mass resettlement of poor people in the early 1960’s. It was often the site of political violence during the Apartheid era, and is now characterized by inadequate housing, poor infrastructure, high unemployment and crime, and among the highest rates of HIV in the world.

In 2012, the prevalence of HIV in antenatal women in KwaZulu-Natal Province was 37.4%. With the support of PEPFAR, in 2014 over 12,000 adults and nearly 800 children are receiving anti-retroviral therapy at KwaMashu, extending life expectancy, and giving hope for a better future. This hope was especially apparent in two girls, ages 12 and 14, each living with HIV/AIDS, who spoke eloquently to us about being cared for by grandmothers and a dedicated cadre of area doctors, nurses, pharmacists and community workers.  One girl dreams of becoming a medical researcher and the other aspires to be a lawyer.

At the conference we learned that thirteen low- and middle-income countries (LMICs) are at the tipping point of overcoming the HIV/AIDS epidemic, with the number of those starting therapy exceeding the number of newly infected. This makes the goal of an AIDS Free Generation plausible. PEPFAR is supporting HIV/AIDS response in more than 100 LMICs. Also, promising comprehensive prevention strategies present great opportunities to stem the epidemic’s tide. But, even with PEPFAR’s numerous achievements, challenges still exist. In 2012 alone, there were 1.6 million deaths, 2.3 million new infections, and 260,000 babies born infected with HIV.

Scaling up treatment and effective preventive interventions, and sustaining support and access to care are critical to achieving an AIDS Free Generation.  Essential to sustainability is ensuring product availability, quality, and safety of medical products used in the PEPFAR program.  Several PEPFAR country representatives described challenges in supply chains attributable to weak regulatory infrastructure (for example, limited sources for Tenofovir-containing FDCs used as first line regimen); lack of capacity of PEPFAR country regulators to assure quality of rapid diagnostic kits; seizure of products at border posts because products are not registered or approved in a country; few national standards for diagnostics and medical devices; and limited capacity of local regulators for regulating medical devices. Representatives of several countries called for strong pharmacovigilance and post marketing surveillance.

Despite these challenges, there are promising developments that are likely to bring benefits to regulators in PEPFAR countries, and ultimately, the PEPFAR program’s beneficiaries. In May 2014, African nations voiced unified support for a World Health Assembly resolution on strengthening regulatory systems; reductions in time to register medicines has been reported by the African Medicines Registration Harmonization Initiative; and the WHO global surveillance and monitoring system for substandard, falsified and counterfeit medical products is receiving reports from, and issuing drug alerts based on vigilant reporting by, African regulators.

We held a special session on strengthening regulatory systems with our colleagues from a number of PEPFAR countries and identified several possible areas for future collaboration. Strengthening regulatory systems will be a key component in defining a sustainable path forward.

Katherine C. Bond is Director of FDA’s Office of Strategy, Partnerships and Analytics, Office of International Programs

Jude Nwokike is FDA’s PEPFAR Liaison, Office of Strategy and Partnerships, Office of International Programs

For more information please visit:

PEPFAR BLUEPRINT: Creating an AIDS-free Generation

Approved and Tentatively Approved Antiretrovirals in Association with the President’s Emergency Plan

Stronger Drug and Vaccine Safety Systems Overseas, Safer Products at Home: Highlights from the World Health Assembly

By: Margaret A. Hamburg, M.D. 

Recently I was privileged to be part of a U.S. delegation to the 66th World Health Assembly (WHA) in Geneva, Switzerland, led by the Secretary of Health and Human Services, Kathleen Sebelius. This is an annual meeting of health leaders from the governments of some 194 nations at the headquarters of the World Health Organization (WHO). 

Margaret Hamburg, M.D.While there, I had the honor of co-hosting a special session on drug and vaccine safety with the Honorable Sherry Ayittey, Minister of Health of Ghana. This was the first time the subject (known technically as pharmacovigilance) has been featured in a special session, and my goal was to outline a vision for the future that benefits our collective interests: a global product safety net, where consumers, industry, governments, and international organizations work together to ensure safe and high quality products, no matter where they come from, or where they are consumed. 

The need for such a global product safety net has never been greater. 

Drugs and vaccines are reaching unprecedented numbers of people in low- and middle-income countries. The President’s Emergency Plan for AIDS Relief (PEPFAR) alone makes it possible for roughly five million people to take anti-HIV drugs today. Also, new vaccines for malaria and dengue fever and improved vaccines for cholera and typhoid are being introduced into these countries. With greater access comes the corresponding imperative to ensure that these products are safe and of high quality. 

During the special session on the safety of drugs and vaccines, I outlined what I hope will become a shared vision for a stronger safety system world-wide. 

What the global regulatory community should aspire to is a medical product safety system where product information is immediately and continuously accessible in electronic platforms, allowing optimal detection and analysis of potential problems. Such a system must be able to follow products throughout their lifecycle from development to their ultimate distribution within the global supply chain. 

In addition to adverse events, the system should enable detection of many different kinds of product safety issues, including those that result from programmatic/prescribing errors, substandard manufacturing, and falsified products. 

There are numerous challenges in weaving such a safety net, but fortunately the basic threads are in place. Data on the state of pharmacovigilance systems are being collected. We have global surveillance capacity through programs managed by WHO, and electronic data systems are now available in some countries. In addition, organizations have developed common naming and data reporting standards for regulators to use. Perhaps most importantly, stakeholders, including financial donors such as the Bill and Melinda Gates Foundation, are beginning to make investments in regulatory systems. 

FDA is contributing to many of these advances. In partnership with the U.S. Agency for International Development, we commissioned studies on pharmacovigilance systems in Sub-Saharan Africa and parts of Asia. FDA experts serve on global advisory committees for product safety. FDA has implemented cooperative agreements with WHO to strengthen vaccine safety, and to build a global monitoring system for substandard and falsified products. Further, FDA scientists have recently developed a new counterfeit detection device, which will be field tested in Africa. 

Just as diseases know no borders, in today’s globalized world, product safety and quality know no boundaries. Stronger regulatory systems overseas mean safer products at home. The special session at the World Health Assembly was an important milestone in global efforts to weave a product safety net for the world.

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

Supporting Africa’s Capacity to Review and Approve HIV/AIDS Drugs

By: Beverly Corey, DVM

Sub-Saharan Africa, the portion of Africa that runs from the Sahara Desert to the Cape of Good Hope at Africa’s southern tip, is more heavily affected by HIV and AIDS than is any other region of the world. In South Africa alone, 17.8 percent of the people have HIV. There were an estimated 22.9 million people living with HIV and 1.2 million deaths in Sub-Saharan Africa in 2010.

Against the backdrop of this harsh reality, FDA has played a critical role in helping to ensure the availability of high quality, safe, and effective treatment therapies. The agency has approved or tentatively approved applications for 155 antiretroviral drugs from Dec.3, 2004 to Nov. 8, 2012, thereby making them available for use as part of the President’s Emergency Plan for AIDS Relief, or PEPFAR. This program, begun in 2004, is the U.S.government’s commitment to support HIV/AIDS treatment for millions of people around the world.

It is clear that the supply of medications to treat HIV/AIDS in Africa must be increased.  One necessary way to address delayed access to medications is to bolster the expertise of African regulators so that they can conduct timely reviews of drug applications. After all, the FDA approval or tentative approval is just one step. Then the therapies must be registered (or approved) by competent drug regulatory authorities in the countries of use.

Some resource-constrained low and middle income African countries have lacked sufficient expertise to conduct registrations efficiently. Focusing on generic drugs will be particularly important there, because generics are less expensive than the brand name products.

Recently, as part of a longstanding PEPFAR mandate for FDA to provide drug registration training for African regulators, the agency had the opportunity to spearhead and provide such training.

Let me share with you what we accomplished. FDA, in collaboration with the Kilimanjaro School of Pharmacy and Purdue and Howard universities, provided a five-day course on the review of generic drug applications and PEPFAR drug reviews.

Thirty-seven regulators and academicians from 17 African countries participated. This first-of-its-kind training, held in Moshi, Tanzania, was aimed at enabling regulators and pharmaceutical school faculty to familiarize themselves with regulatory and scientific methods applied by FDA.

The most exciting, complementary aspect of the training was to introduce the value of integrating regulatory science training into the curricula of schools of pharmacy and other academic institutions in Africa.

FDA and its partners believe that this training course can eventually be turned into a teaching module for use in academic curricula throughout Africa. Such curricula can support a cadre of regulatory affairs professionals to work in government agencies. This would further the availability and the manufacture of quality, safe and effective drug products for the African population. 

If we can contribute to a global curricula for regulators, what a legacy that would be, for both PEPFAR and FDA!

Beverly Corey, DVM, is the Senior Regional Advisor for  Sub-Saharan Africa, FDA Office of International  Programs, US Embassy, Pretoria, South Africa

FDA is Proud to Have Helped PEPFAR Reach its Latest Milestone: More than 150 Drugs Now Available Worldwide to Help Treat HIV/AIDS

By: Jeffrey S. Murray, M.D.

While Americans today are all too familiar with the acronyms “HIV” and “AIDS,” many of us are not familiar with the strange-sounding acronym, “PEPFAR.”  Despite being less well-known than these other acronyms, PEPFAR, which stands for the President’s Emergency Plan for AIDS Relief, has saved about 1.2 million lives in its global efforts to battle the HIV/AIDS epidemic.  This month marks a historic milestone: As of July, 2012, PEPFAR has now made available more than 150 drugs to help treat people worldwide infected with HIV.

Access to life-saving HIV/AIDS therapy is one of many goals of the PEPFAR program, the U.S. Government initiative to help save the lives of those suffering from HIV/AIDS around the world.  Since PEPFAR’s establishment in 2003, the HIV/AIDS drug program has supported life-saving treatment for more than 3.9 million adults and children worldwide.

FDA is proud to play a role in contributing to PEPFAR’s success.  As part of PEPFAR’s program, FDA conducts an expedited review of antiretroviral drugs produced by manufacturers all over the world and ensures that these products are rapidly reviewed, their quality assessed, and that they are acceptable for purchase with PEPFAR-supported funds.

In 2004, to help increase the availability of safe and effective HIV/AIDS drugs for use under PEPFAR, FDA began an expedited review process for generic HIV/AIDS drugs.  This process respects the patent and exclusivity rights of brand name HIV/AIDS therapies in the U.S., but helps ensure that safe and effective lower-cost generic versions of these therapies can be purchased under PEPFAR for use in other countries.  The PEPFAR program reaches 15 of the hardest hit countries in Africa, Asia, and the Caribbean.  By the end of 2007 more than 90 percent of HIV/AIDS drug products provided in eleven of these countries were generic.

As a result of this expanded use of generic products under PEPFAR, the overall cost of providing treatment has been substantially reduced.  For instance, in one PEPFAR-assisted country it once cost $10,000 per person each year for a typical treatment regimen.  That cost is now less than $200 a year.  Such cost savings have likely helped millions more people receive HIV/AIDS therapy who otherwise would not have access to this treatment.

The goal of PEPFAR is to work with host nations to support treatment of at least 3 million people, prevention of 12 million new infections, and to provide care for more than 12 million HIV-infected and affected people by 2013. In addition, PEPFAR will support training of at least 140,000 health care workers in HIV/AIDS prevention, treatment, and care.

With PEPFAR reaching its historic milestone of making more than 150 drugs available to help fight HIV/AIDS, FDA applauds the program’s efforts, its dedicated workers, and all of PEPFAR’s international partners for their life-saving work.

Jeffrey S. Murray, M.D, is Deputy Director in the Division of Antiviral Products at FDA’s Center for Drug Evaluation and Research


Our Global Imprint in the Fight Against HIV/AIDS

By Edward Cox, M.D., M.P.H.

Since HIV/AIDS first entered the public consciousness in the 1980s, the biology of the disease has both perplexed and astounded us. Although more than 34 million people live with HIV worldwide, in recent years the disease has also served to unite us in a common goal – the search for a cure.

When I joined FDA more than a decade ago, after having spent time as a fellow at the National Institute of Allergy and Infectious Diseases at the National Institutes of Health and in private practice, I could not have imagined the unprecedented level of progress we’d see in the area of drug development for HIV/AIDS. The vast majority of these pivotal breakthroughs have occurred during the last decade and the development programs have often included clinical studies conducted outside of the U.S.

And while our core mission has traditionally focused on protecting the health of Americans while promoting the development of innovative drugs for use here at home, my background as an infectious disease expert allowed me realize early on that FDA would increasingly play an important role in the global public health arena, particularly in areas such as HIV/AIDS, hepatitis C and tuberculosis.

I am particularly proud of our role internationally in supporting the President’s Emergency Plan for AIDS Relief (PEPFAR). Since its inception in 2003, the PEPFAR program continues to serve as a great example of what can be accomplished through collaboration and coordination with our foreign partners.

Since 2004, the agency has worked to ensure that people living with HIV/AIDS worldwide have timely access to safe and effective antiretroviral drugs that have been manufactured by facilities in the U.S. and abroad. As of 2011, FDA has approved more than 137 formulations of drugs and new fixed-dose combination products to treat HIV.

The PEPFAR program itself has directly supported lifesaving antiretroviral treatment for over 3.9 million men, women and children through partnerships with more than 30 countries, including China, Haiti, India, Vietnam and more than a dozen African nations.

And just last week HHS Secretary Kathleen Sebelius and Deborah Autor, FDA’s Deputy Commissioner for Global Regulatory Operations and Policy, met with regulatory officials from India and companies with operations in the region to emphasize the importance of continued dialogue on current and emerging public health issues. Among the many topics of discussion was the ongoing need and value of the PEPFAR program.

Today, FDA continues to actively encourage sponsors worldwide to submit U.S. marketing applications for single entity, fixed dose combination, and co-packaged versions of previously approved antiretroviral therapies. My colleagues in FDA’s Center for Drug Evaluation and Research (CDER) Office of Antimicrobial Products and Office of Generic Drugs stand ready to continue working with companies who are dedicated to providing low-cost therapies to people living with HIV/AIDS around the world.

In December, we commemorated the 23rd anniversary of World AIDS Day and highlighted the 2011 U.S. theme — Leading with Science, Uniting for Action — which Commissioner Hamburg noted was particularly meaningful to FDA given the agency’s role. The success of the PEPFAR program is an excellent example of how science and interaction can quickly translate into meaningful progress for people living with HIV/AIDS.

As a scientist and FDA reviewer, I follow the progress being made when new findings about the disease are released each year. Personally, I can tell you that at FDA we all celebrate the victories that represent progress for patients and express our frustration and disappointment with the setbacks.

While a cure remains elusive for now, we intend to solidify our global imprint by continuing our efforts in support of the PEPFAR program. For our part, FDA will continue encouraging the development of new and effective therapies to treat patients.

More information can be found on “What’s New at FDA in HIV/AIDS.”

Edward Cox is the Director of the Office of Antimicrobial Products in FDA’s Center for Drug Evaluation and Research