Speech |

Treating Rare and Neglected Pediatric Diseases

Treating Rare and Neglected Pediatric Diseases: Promoting the Development of New Treatments and Cures 

MSF Board Member Suerie Moon made an oral statement in front of the U.S. Senate Committee on Health, Education, Labor, and Pensions on July 21, 2010 on the topic of treating rare and neglected paediatric diseases. Moon highlighted the dual nature of the problem that MSF sees in the field, both the limited access to existing tools to diagnose and treat the neglected diseases and also the need to champion innovation to provide better adapted products for use in the field. In particular, she highlights the need to "de-link" the market for research and development from the market for production manufacturing in order to deliver the necessary tools to diagnose and treat the many neglected diseases encounters with patients around the world.

Innovation and Access for Neglected Diseases: The Experience of Médecins Sans Frontières

To: U.S. Senate Committee on Health, Education, Labor, and Pensions (HELP) Hearing

Thank you, Chairperson Harkin, Ranking Member Enzi, and the Committee.

My name is Suerie Moon and I am on the U.S. Board of Directors of the international medical humanitarian aid organization Doctors Without Borders, known by our French acronym MSF, which stands for Médecins Sans Frontières.

My testimony today is based on our decades of experience as one of the only actors providing care and treatment to people suffering from neglected diseases in the developing world. It is also based on our experience as a founding member of the Drugs for Neglected Diseases initiative – or DNDi, which is a public-private product development partnership (PDP). Our support to DNDi makes us the third largest philanthropic funder of neglected disease research.

In a nutshell, we are facing two types of problems in addressing neglected diseases: on the one hand, there is limited access to the tools that exist to diagnose and treat these diseases. But at the same time, the tools we have are terribly insufficient – we urgently need innovation and new products that are more effective, easier to use, and adapted to field conditions.

Globally, neglected diseases target the bottom billion – those living in the most rural areas, with poor or no access to healthcare, and often living on less than a dollar a day. Therefore, it was welcome news when, in 2008, the U.S. government established the Presidential Initiative on Neglected Tropical Diseases. However, the initiative only focused on five of the 14 most neglected tropical diseases identified by the World Health Organization (WHO). It did not fund diagnosis and treatment of the deadliest neglected diseases, such as Chagas disease, sleeping sickness, and kala azar, These have been identified as the most neglected, and are the focus of a number of MSF programs.

Many of you in this room may never have heard of these diseases. They often occur in remote areas, or in countries undergoing political instability. Because of time constraints, I will not describe each of them to you, but perhaps I can provide some information on Chagas disease, which affects 300,000 people in the United States today, and 15 million people worldwide. It is the largest parasitic killer in the Americas, responsible for about 14,000 deaths per year. This disease is caused by a parasite transmitted by what’s called the “kissing bug” because it bites so gently that victims often don’t even know they may have just been infected. Chagas disease can also be transmitted from mother to child during pregnancy; and through blood transfusions and organ transplantation, and sometimes through oral transmission. If left untreated, it can eventually affect the heart and digestive system – and kills 30 percent of those infected.

Despite its deadly effects, we don’t have many tools to combat Chagas disease. Currently, we only have two medicines: benznidazole and nifurtimox. Both were developed over 45 years ago through research that was not even specifically targeting Chagas disease. Presently, neither of these drugs is adapted for use in children, although we anticipate a paediatric formulation of benznidazole in the coming months. Further, there is no test for cure for Chagas disease – which means that even after a course of treatment with these drugs, the patient cannot be certain that they’ve been cured. We urgently need new diagnostic tests, better medicines, a vaccine, and a test for cure to help prevent, diagnose and treat this disease.

Another area where we desperately need innovation is for diagnostics for tuberculosis (or TB). In 2008, there were 12,900 cases of TB in the US, and 9.4 million worldwide. TB is one of the world’s leading causes of pediatric and adult mortality, causing nearly 5,000 deaths a day. However, the current system for diagnosing TB in most developing countries detects less than half of all TB cases. It performs even worse in children and people living with HIV.

Why don’t we have better tools available to combat neglected diseases? It is precisely because they are poor that the patients who are affected by these diseases are neglected. Let me explain.

The current system that we have to develop new drugs, diagnostics and vaccines, is driven by commercial rewards. A company develops a drug or diagnostic tool, receives a patent that allows the sale of the product at high prices, and the high prices in turn are expected to cover the costs of research and development, or R&D. This system fails miserably to incentivize R&D if patients cannot pay high prices – either because they are too poor or too few.

That is why, between 1975 and 2004, only 1.3% of all new drugs were developed for tropical diseases and tuberculosis, even though these diseases account for 11.4% of the global disease burden.

It is clear, then, that if we want new tools to combat these diseases, we need new incentive mechanisms.

MSF believes that the key principle in evaluating and designing new incentive mechanisms should be “de-linkage.” What do I mean by this term? De-linkage refers to the idea that we can separate the market for R&D from the market for product manufacturing. On one side, we can specify the kind of R&D that we need, generate competition among researchers, and then reward the best innovator. While on the other, once the product has been developed, we can encourage many manufacturers to produce it; then, with robust competition in the production market, prices will fall to the lowest sustainable levels.

The link that we are breaking is that between high medicines prices and R&D: high medicines prices cannot be the only incentive for R&D if there is to be innovation and access to much-needed tools for neglected diseases. De-linkage can stimulate R&D where there is no profitable market – that is, for neglected, rare, orphan or pediatric diseases.

Because the product development process is long and uncertain, we need a range of different funding mechanisms that allow de-linkage, either to “push” R&D via targeted upfront funding (such as through PDPs) or to “pull” R&D to ensure that the right products reach the end of the pipeline.

Prizes are one attractive “pull” mechanism that incorporates the principle of de-linkage. A prize is essentially an award provided for different stages of the R&D process – such as identifying biomarkers, proof of concept, product synthesis, or a finished product. A well-designed prize could offer many benefits, including:

  • The ability to drive R&D based on health needs;
  • Allowing competition to determine the path or team most likely to succeed, (rather than governments or donors);
  • Attracting a broader, more diverse base of potential “solvers” to a problem; and
  • The flexibility to build-in provisions for collaboration, knowledge-sharing, and affordability of end products.

A prize fund could quickly be established, for example, for a TB diagnostic test that could be used at the point-of-care in resource-poor contexts.

In summary, we welcome the increased political attention being paid to the needs of the most neglected patients. However, we urge the US government to include the most neglected tropical diseases (including Chagas disease, sleeping sickness, kala azar; and Buruli ulcer) within the scope of the new Global Health Initiative. We ask the Initiative to provide support for improved access to existing health tools, as well as to support the development of new and improved ones.

In order to develop new and improved drugs and diagnostics, it will be necessary to explore new innovation mechanisms that address the shortcomings of the existing system. The key principle to keep in mind is de-linkage – that is, breaking the link between high medicines prices and the funding of R&D. We believe prize funds are one promising mechanism for generating innovation that meets the health needs of the poorest. We ask this Committee and the US government to support innovative new approaches, such as prizes, which can be established relatively quickly for specific areas such as TB diagnostics; prizes should also be considered for Chagas disease and other neglected areas of innovation as well. At the same time, we urge the US to support more systemic, long-term changes that are needed to provide sustainable financing for health needs-driven R&D that ensures equitable access. Important discussions are now taking place in this regard at the World Health Organization and Pan American Health Organization.

There is increasingly widespread recognition that the existing R&D system is failing – failing patients with neglected diseases, with orphan or rare diseases, and children, among others. Now is the time to begin testing new approaches to generate the innovation that we need to meet global public health needs.

Thank you very much for this opportunity to share our experience with you.