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According to the WHO, Neglected Tropical Diseases (NTDs) affect over 1 billion people worldwide, and are devastating to patients in the developing world. What is being done to get treatments to these patients and to speed development of new treatments?

Contributors Respond

Brenda Colatrella

Brenda Colatrella

Executive Director, Corp. Responsibility, Merck

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Neglected Tropical Diseases (NTDs) impact more than a billion people in some of the poorest, most remote parts of the world, blinding, disabling, disfiguring and sickening those infected. They have a negative impact on life expectancy, productivity and childhood education -- all of which create a cycle of poverty and stigma for affected communities.

Today, because of renewed and new commitments, millions impacted by NTDs are being treated, several NTDs are being controlled effectively, and some even eliminated. 

More than 25 years ago, Merck made the unprecedented decision to donate Mectizan® for as long as needed, wherever needed, to eliminate onchocerciasis (river blindness) -- a debilitating, disfiguring disease and once a leading cause of blindness.   In 1998, Merck extended its commitment to the prevention of lymphatic filariasis(LF) in African countries where it co-exists with river blindness.  Through a broad, multi-sectoral partnership, the Mectizan Donation Program now reaches more than 200 million people annually.  Onchocerciasis has been eliminated from Colombia and disease transmission has been interrupted – meaning no new cases have been identified – in three other Latin American countries and nine regions in five African countries.   Steady progress is being made to achieve elimination of both LF and river blindness by 2020 and 2025, respectively.   [More information at www.merckresponsibility.com]

However, the Mectizan Donation Program is no longer unique.  Today, many pharmaceutical companies – including Merck, GlaxoSmithKline, Pfizer, J&J, Sanofi-Aventis, among others – are engaged in the fight against NTDs and are working together, and with other stakeholders, to address these diseases.   

  • As early as 1999, through the Partnership for Disease Control Initiatives, companies and their NGO partners began to share best practices and coordinate their activities where feasible.
  • Through the Road Map for Neglected Tropical Diseases, WHO better defined and advanced the strategy for NTDs, setting targets for the control or elimination of 17 NTDs by 2020. 
  • In January 2012, the London Declaration on Neglected Tropical Diseases was launched, bringing together diverse stakeholders working on NTDs and creating renewed momentum to control or eliminate 10 NTDs by 2020.  WHO, the Gates Foundation, 13 pharmaceutical companies and the World Bank were among the original endorsers of the London Declaration.
    • And in 2013 the World Health Assembly Resolution on NTDs was passed and the African Regional Committee also passed a resolution on NTDs, demonstrating the commitment of the countries. 

Progress is also being monitored through a public scorecard that highlights both successes and areas for improvement.  According to the London Declaration’s first report, pharmaceutical companies are meeting 100% of country requests for medicines and delivered more than one billion treatments in 2012.  Endemic countries are stepping up their commitments with 40 countries developing or having launched multi-year, integrated NTD plans. Donors have committed funds to support integrated NTD programs, scale-up programs, increase resources for mapping, improve program strategies and develop new tools.  And innovative R&D partnerships have been launched to address remaining clinical gaps.

While significant progress has been made, there is still much to be done to sustain and expand existing efforts, and to tackle operational and R&D gaps.   Funding is being mobilized, but more is needed.  The capacity to deliver treatments in some countries needs strengthening.  New diagnostics are needed to better understand when to start and stop treatments.  For some diseases, new and better drugs or treatment strategies are needed to fill gaps where current therapies are limited or lacking.  Endemic country governments need the political will and resources to address NTDs in their countries.   

Additional support, commitment, and innovation will translate into even greater progress against NTDs and improved health for patients and communities.

Dale Kempf

Dale Kempf

Distinguished Research Fellow, AbbVie

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Along with a number of other PhRMA members,AbbVie is committed to helping to bring solutions to the world's recognized health challenges, including Neglected Tropical Diseases (NTDs). In line with our unique position as a biopharmaceutical company, AbbVie approaches NTDs from three perspectives: leveraging the expertise of our people; partnering with leading organizations; and utilizing our financial resources to address this global challenge.

AbbVie’s Executive Council on Neglected Diseases coordinates efforts across the company to contribute innovative technologies, compounds for screening and our scientific expertise. It facilitates coordination of our resources and identifies opportunities for partnerships that address NTDs. In 2012, more than 100 of AbbVie’s most experienced scientists contributed their expertise to research on NTD drug discovery and development in collaboration with partner organizations. We provide laboratory facilities and mentor student interns under a program sponsored by Franciscan University of Steubenville (Franciscan Institute for World Health) that focuses on NTD research.

AbbVie collaborates with leading organizations, providing our large library of diverse chemicals to screen against many NTDs. Partners include Drugs for Neglected Diseases initiative (DNDi), the Gates Foundation TB Drug Accelerator (TBDA), TB Alliance, Medicines for Malaria Venture (MMV), and the Liverpool School of Tropical Medicine (LSTM).  We are working closely with MMV to support three preclinical and early clinical development programs of novel antimalarials. In early discovery efforts, AbbVie scientists engage with DNDi and the TBDA, providing cheminformatics support, characterizing lead compounds from our partners’ programs, and supplying large numbers (up to 100,000) of our compounds for screening against various parasites and tuberculosis. In collaboration with LSTM, we are identifying and advancing novel compounds for filarial (worm) diseases. AbbVie has joined 12 other health care companies, the Gates Foundation, World Bank, NGOs and Governments to collectively sign the London Declaration on NTDs, which aims to eliminate or control 10 NTDs by the year 2020.

The AbbVie Foundation, a nonprofit 501(c)(3) organization, aims to help advance science and medical innovation, expand access to health care and promote science education globally. In line with those objectives, the AbbVie Foundation has provided $3 million to-date to support projects focused on advancing progress against NTDs. We continue to explore partnership opportunities that will increase early detection and intervention to prevent debilitating consequences of NTDs.

Michael Nyenhuis

Michael Nyenhuis

President and CEO, MAP International

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Not one more. Not. One. More.

I said those words after meeting a young boy named Kwazi in Ghana. His right arm and hand had been stricken by a little-known ulcer that left it permanently disfigured and unusable. Other children at his school in rural, central Ghana teased him, he told me. They called him "clutch" because his twisted right arm looked to them like a truck's clutch pedal. All he wanted, he pleaded, was for his right hand to work again.

It did not have to be this way. This could have been stopped. Not one more child should face this. Not. One. More.

Kwazi was stricken by Buruli ulcer, one of the 17 designated "Neglected Tropical Diseases" (NTDs). Buruli, named after the village in Uganda where it was first identified, is a skin and soft tissue infection. When caught early as a small nodule on the skin, it can be easily treated without much impact to the patient. When it opens as an ulcer and spreads, Buruli eats away skin, muscle and tissue, creating permanent disfigurement and disability. As many as 6,000 cases are reported each year, mostly in West Africa.

In combatting other NTDs, mass drug administration is often the best strategy. MDA programs can cost less than 50 cents per person per year to effectively combat trachoma, onchocerciasis, lymphatic filariasis, soil-transmitted helminths and others. A number of pharmaceutical companies have stood up to provide drugs used in MDA programs.

Buruli is different, however. It is not combatted through mass distribution programs. Instead, Buruli requires case-by-case treatment utilizing antibiotics and wound care for weeks and even months. Improved treatment options will come with simplified antibiotic protocols, point-of-care diagnostic tests and better understanding of the mode of transmission.

My organization, MAP International, has implemented Buruli-focused projects in West Africa since 2007, beginning in Côte d'Ivoire, expanding to Ghana and now into Liberia. Alongside WHO and national ministries of health, and with critical support from partners like the American Leprosy Mission and AbbVie, we have strengthened national programs, built treatment wards and ensured the provision of antibiotics and medical supplies. We have brought healing to children like Kwazi who are affected by the ulcer. Still, too many like him are left with lifelong disfigurement and disability.

Early detection is key to stopping the effects of the disease. If Buruli can be stopped before it becomes an ulcer - when it is still a nodule on the skin - no more children will suffer Kwazi's fate. That is why we have also invested heavily in education and awareness campaigns targeting mothers, health workers and others.

In one of our Buruli treatment wards I met two young girls. One had a small nodule on her cheek. Her mother had been to a Buruli sensitization event in her village. Concerned, she brought the daughter in to be checked. After a simple treatment, she was on her way home. A second girl had her shoulder and upper arm wrapped in bandaging. Her mother did not know about Buruli and the ulcer spread. After weeks and weeks of treatment, the girl would soon go home to contend with a lifelong disability.

Yes, we need improved antibiotic treatment and diagnostics. Equally, we need the resources to spread the message of early detection so that not one more child suffers the permanent scars of Buruli. 

Eduardo Pisani

Eduardo Pisani

Director General, IFPMA

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Since 2005, IFPMA has published a directory of global health partnerships, many of which focus on neglected tropical diseases (NTDs).  The nature of these partnerships – ranging from R&D to healthcare system strengthening to donations – reflect the comprehensive approach needed to reduce human suffering caused by NTDs. Prominent among these partnerships, as well as individual company activities, are focused R&D activities to develop new therapies for patients around the world. So what is currently being done to speed development of therapies? Lots!

Earlier this year, IFPMA released a status report on pharmaceutical R&D to address NTDs specifically. Among our companies, we identified the 132 R&D projects focus on the following diseases prioritized by the World Health Organization's (WHO) Special Program for Research and Training in Tropical Diseases (TDR): tuberculosis, malaria, human African trypanosomiasis (sleeping sickness), leishmaniasis, dengue, onchocerciasis (River blindness), American trypanosomiasis (Chagas disease), schistosomiasis, leprosy and lymphatic filariasis. These 132 programs represent a 40 percent increase in the number of projects versus the previous year. Because many global health successes in recent years have occurred through partnerships that combine expertise of different players, it is important to point out that most (85 percent) of these 132 R&D initiatives are collaborations between industry, research units such as product development partnerships and universities, and independent researchers around the world.

As more research compounds progress into late-stage development, increasing strain will be placed on the clinical trial and regulatory infrastructures in developing countries, where many trials take place and new therapies are eventually approved by regulators While capacity in both areas is slowly improving, more needs to be done. Because the time required for regulatory approval in some developing countries can be significantly longer than in US or Europe, focusing some attention and resources on strengthening regulatory capacity can help speed patient access to new therapies.

While researchers are leading the way to tomorrow’s therapies, people are in need today.  That is why the research-based pharmaceutical industry is donating 14 billion treatments this decade to eliminate or control of nine NTDs that represent more than 90 percent of the global NTD burden.

Finally, speeding development of and access to new treatments requires coordination.  That is why we all depend on the WHO’s leadership to ensure that R&D, capacity-strengthening, and access efforts have the best chance of relieving suffering among the nearly one billion afflicted by these diseases. 

Andy Wright

Andy Wright

VP, Global Health Programs, GlaxoSmithKline

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Clara Marr and Matt Azzara may seem unlikely partners in the fight against one of South Asia’s most deadly health challenges, visceral leishmaniasis or "black fever," but they represent an expanding model of pharmaceutical cooperation to address stubborn health challenges like neglected tropical diseases (NTDs).

Marr is a clinical research and infectious disease specialist working in England for GlaxoSmithKline. Azarra is a market researcher and oncology strategist for Pfizer in the United States. Together they served on a six-month volunteer assignment to assist CARE India in researching ways to eliminate black fever, which infects an estimated 500,000 people each year. It is especially prevalent in South Asia, and is transmitted by small sand flies.

GSK’s and Pfizer’s commitment to work together with CARE India reflects the willingness among corporations to collaborate to solve big problems.

There is no doubt that NTDs are a big problem. One in six people in the world are affected by an NTD, a group of parasitic and bacterial infections. Financial incentives to develop treatments do not exist because NTDs mostly affect the poorest people who lack finances to afford treatment.

The World Health Organization (WHO) has set some very specific goals around combating NTDs by 2020. A coalition of global pharmaceutical companies and leading organizations signed the landmark London Declaration in January 2012 to eliminate or control 10 NTDs.

Progress against these commitments over the last year are outlined in a report, From Promises to Progress. The amazing news is that in 2012 pharmaceutical companies donated more than one billion treatments to meet 100 per cent of drug requests by endemic countries for NTD programs.

We are proud to be an active partner in one of the world’s biggest and boldest public health initiatives, led by the WHO, to rid the world of Lymphatic Filariasis (LF). LF is transmitted by mosquitoes. It is more commonly known as elephantiasis, a name which describes the hardening and thickening of the skin that frequently accompanies massive swelling in the arms, legs, breasts and genitals. In 1998 we announced our commitment to donate to WHO all the albendazole needed to treat the 1.3 billion people in over 72 countries who are at risk, until LF is eliminated. So far we have donated four billion treatments of albendazole, alongside grants and other support for the global initiative.

The recommended regimen for treating LF is a single dose of two medicines given together - albendazole plus either DEC (diethylcarbamazine) or ivermectin. Merck has agreed to donate an unlimited supply of Mectizan (ivermectin) for African countries and Yemen. In January 2012 Eisai committed to donate DEC to WHO over a period of seven years to benefit some 250 million people.

In support of the WHO goals, we expanded our donation to an additional two billion albendazole tablets by 2020 to treat school-age children for intestinal worms which adversely affect children’s nutrition and educational attainment.

Research still is needed to find treatments and prevention for NTDs. We have a dedicated drug discovery unit at Tres Cantos in Spain which focuses primarily on malaria and tuberculosis, but also NTDs such as Leishmaniasis, Chagas Disease and Sleeping Sickness. Research decisions at Tres Cantos are prioritised on their socio-economic and public health benefits.

The potential success of innovative teamwork, whether individuals like Matt and Clara or multinational companies, reinforce the need for collaborative approaches to address global health challenges, whether it is one disease at a time or one patient at a time.

Bennett Shapiro

Bennett Shapiro

Board Chair, Drugs for Neglected Diseases Initiative (DNDi) North America

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When we speak of neglected tropical diseases (NTDs), we are actually talking about neglected patients, "the bottom billion" – poor, marginalized, voiceless populations living primarily in the developing world, and for whom access to health care and research and development (R&D) for new medicines are sorely lacking. These "bottom billion" are each afflicted with one or more of the 17 NTDs as defined by the World Health Organization (WHO) – diseases that are sometimes fatal and always debilitating.

For some of these NTDs, adequate drugs and health tools do exist, so disease control is primarily a matter of getting these drugs into the field and into the mouths of patients – this is especially true for the handful of NTDs that the US government focuses on through its US Agency for International Development (USAID) NTD Program, which include intestinal-worm diseases such as schistosomiasis and soil-transmitted helminthiasis, and blindness-causing parasitic diseases including onchocerciasis and trachoma.

However, a dozen other important NTDs are not included in the USAID NTD Program – including the three with the highest death rates: African sleeping sickness, Chagas disease, and leishmaniasis. In advanced stages of disease, untreated patients with these NTDs will die. And current drugs are wanting. They cause harsh side effects, require long treatment durations, and are complicated and difficult to administer in the field, often remote or conflict-ridden, resource-poor areas. In short, proper control of these most-lethal NTDs is extremely challenging without new, adapted treatments.

As part of the solution to address this gap, the not-for-profit R&D organization Drugs for Neglected Diseases initiative (DNDi) is working to develop safe, effective, affordable, field-adapted new treatments for these three deadly NTDs, which make up the core of DNDi's disease focus. These are the NTDs being seen in the field by Doctors Without Borders, which conceived of and launched DNDi 10 years ago this year, along with six global public health partners in Kenya, Brazil, India, Malaysia, and France, and at the WHO. DNDi was created to address the market and policy failures in developing new medicines to treat patients infected with these NTDs, most of whom are poor and do not represent a profitable market for traditional pharmaceutical R&D. DNDi's business model is to de-link the cost of R&D from final product price, considering medicines as public goods, and to bring together and coordinate public and private partners – academia, public health institutes, governments, pharmaceutical and biotechnology companies, non-governmental organizations, and funders – to speed up neglected-disease R&D. DNDi works closely with the pharmaceutical industry to harness their expertise in translating the fruits of the biomedical research revolution into new treatments for historically neglected patients, and pharmaceutical partners of DNDi have been active players in this effort to develop safe and effective therapies.

To bring new drugs to neglected patients, support is needed from the pharmaceutical industry and the other many partners mentioned; however, the full solution for accelerating new drug development for NTDs and other pressing global health needs requires more than just one or even several organizations or companies – more than a piecemeal strategy. The real solution requires a global R&D framework to prioritize, coordinate, and sustainably and innovatively finance new health-tool development for neglected patient needs, including pharmaceutical products. A WHO-led global health "R&D treaty" of this sort is currently being explored, with initial steps including set-up of a R&D "observatory", and review of 22 R&D "demonstration projects" submitted by different countries. However, the jury is still out as to whether these steps will lead to the establishment of a global health R&D framework for essential health.

A recent analysis showed that only 1% of ongoing clinical trials for new drugs and vaccines are directed towards neglected diseases. Filling this nearly empty pipeline must be sped up to save as many lives as possible among the "bottom billion" afflicted by some of the world's most neglected diseases. 

Dr. Neeraj Mistry

Dr. Neeraj Mistry

Managing Director, Global Network for Neglected Tropical Diseases

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We are now closer than any other point in history to making neglected tropical diseases (NTDs) the next great success story in public health, thanks in large part to an innovative public-private partnership model that draws on unique competencies of various sectors and organizations.

The pharmaceutical industry has been an indispensable partner in the fight against NTDs through its mass-scale donation of existent drugs to programs around the world. Through this sustained drug supply, many mass drug administration (MDAs) programs have reached a critical threshold of treatment coverage which, when conducted over consecutive years, have placed many countries at the precipice of eliminating some NTDs in different parts of the world. To build on this momentum, most recently, in the 2012 London Declaration, 13 pharmaceutical companies expanded their commitment by pledging 14 billion doses of pills for free by the end of this decade. We now have all of the treatments required to control and eliminate the seven most common NTDs – which account for 95 percent of the global burden – by 2020.

The prevalence of NTDs in the poorest communities across a wide range of countries, including Myanmar, Honduras and Kenya, among others, has required the development of critical public health infrastructure, including innovative supply chains, forecasting and monitoring tools, which have been proven highly effective at scale. India, for example, which accounts for 35 percent of the total global burden of NTDs, recently ran two of the world’s largest deworming initiatives: in Bihar, 17 million school-age children in over 67,000 schools received treatment; and in Rajasthan, 16 million children from 90,000 government schools and 70,000 community health centers were targeted.

Still, many other countries must demonstrate the political and funding commitments to build the capacity and logistical processes required to carry out such large-scale and sustainable treatment programs.

Over the past year, we have seen some encouraging signs of progress from regional and national bodies in NTD-endemic regions.  Thirty-six African nations have developed multi-year national plans to control and eliminate NTDs by 2020. Also, at WHO’s 63rd Regional Committee for Africa, countries adopted a regional strategic plan recommending increased access to treatments, resource mobilization, advocacy, and monitoring and evaluation, surveillance and research.  In the past several months, the World Health Assembly, Organization of American States and African Union have all adopted resolutions against NTDs and strengthen efforts to integrate NTD programs into other health sectors. 

While endemic countries accelerate their efforts to implement MDA and fund a larger share of their own NTD programs, we also see an opportunity for donor governments to do more. The US and UK governments have invested significant funds, but a $300 million annual funding gap still exists. Governments including Germany and France can play a leadership role in bridging this gap by integrating NTD investments into their existing development programs.

Improved coordination among a variety of partners may be the key to achieving breakthrough results for global NTD efforts.  The Global Network for Neglected Tropical Diseases, in partnership with the Bill & Melinda Gates Foundation and the World Bank, and with support from the WHO, recently hosted a Development Agency Roundtable attended by representatives from potential donor countries, global development organizations and pharmaceutical companies.

Even as we take the right steps to achieve control and elimination targets by 2020, it is still essential that we plan for the obsolescence of current treatment protocols; the effective tools we have today to combat NTDs will need to evolve to meet drug-resistance challenges that may emerge in the future. In preparation, the pharmaceutical industry once again has stepped-up through innovative PDPs; sharing compound libraries; and are embarking on a bold agenda to replenish the NTD diagnostic, therapeutic and vaccine pipeline.

Why, I am often asked, would pharmaceutical companies do this if there isn’t market potential for these products?

A former company executive who started one of the drug donation programs has a simple answer that speaks to the industry’s moral imperative to combat NTDs: “We had the drug and people needed it, so we just had to get it out there to them!”

And this, I think is the at the heart of the matter – while opting for market-based principles to develop and deliver their products, pharmaceutical companies are essentially working with a purpose to improve the longevity and quality of lives of many people as possible.

Ranjana Smetacek

Ranjana Smetacek

Director General, OPPI

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On 9 September 2013, the Organization of Pharmaceutical Producers of India (OPPI) was proud to partner with the International Federation of Pharmaceutical Manufacturers and Associations and Global Health Progress to convene a high level meeting of stakeholders who are committed to fighting and eliminating neglected tropical diseases (NTDs) in India.

Throughout the world, one billion of perhaps the poorest and most marginalized suffer from neglected tropical diseases. Despite making tremendous progress over the past decade, India remains home to nearly one-third of all of those who suffer from NTDs. 

When stakeholders gathered in Delhi, we talked about understanding the burden of these diseases. We talked about the need to reach the poor and the marginalized. We talked about the importance of directing research and development capability towards these diseases. But throughout these discussions there was one common recurring thread: collaboration – the need for dialogue and the need for sharing, partnering, and integrating the efforts of all stakeholders.

Our meeting highlighted the many examples of what is possible when government, academia, industry, civil society, donors and patients come together and commit resources, both human and financial. NTDs can be controlled and eliminated – even eradicated – but we need to find a mixture of incentives to unite stakeholders. This includes incentives for companies to discover, research, develop and produce new medicines that treat or prevent  theses diseases.

Through a coordinated approach to surveillance, prevention, diagnosis and treatment, India will continue to make progress against NTDs.  The effort to tackle these diseases requires, first and foremost, the leadership of the government. It requires health care providers to integrate approaches to preventing and treating NTDs into primary health care delivery in India. It requires the ongoing engagement of civil society to give voice to the needs of the poor and marginalized. It requires the attention of the media to raise public awareness. It requires an environment that supports and rewards the development and marketing of new medicines and diagnostics.

Recognizing that meaningful progress requires the dedication and support of so many stakeholders even patients themselves to inject a sense of urgency. For 300 million Indians suffering from neglected diseases delay is not an option.

As the report from our meeting highlights: NTDs can be controlled and eliminated. The stakes are too high for failure and the lives and futures of millions of people depend on the decisions and actions we take in the coming months and years.”