The R&D hurdles for neglected diseases

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Pharmaceutical Technology Europe

Pharmaceutical Technology Europe, Pharmaceutical Technology Europe-06-01-2009, Volume 0, Issue 0

Modern medicines have dramatically improved the health of millions of people worldwide, but the healthcare situation in industrialized countries is a stark contrast to that in the least developed countries where neglected diseases are still prevalent.

Thanks to medical interventions, conditions that would have been considered life-threatening 100 years ago can now be prevented, cured or controlled. Despite this progress, however, a number of diseases persist in exerting a heavy healthcare burden on marginalized communities in poor countries, even though they have largely been eradicated elsewhere across the globe.

Faiz Kermani

The perception that these diseases are low on the list of global healthcare priorities, as well as the fragmented approach to researching treatments, has resulted in them being grouped under the umbrella term 'neglected diseases'. Although a universally accepted list does not exist, the World Health Organization (WHO) considers neglected diseases to be primarily infectious diseases that thrive in the heat and humidity of tropical climates. According to the WHO, considering these diseases as a group is important for control programmes, because many people are afflicted simultaneously by more than one of them.1

Since 1975, pharmaceutical companies have been responsible for the launch of over 1400 new chemical entities as human therapeutics,2 but relatively few of these have targeted neglected diseases. Subsequently, there has been considerable media and public pressure on the pharmaceutical industry to focus more attention on this area of drug development, as well as on governments to provide incentives for investment in neglected diseases.

The challenges faced by pharma

Efforts to develop specific R&D programmes for neglected diseases have steadily increased, but this has required new approaches to traditional R&D challenges, as well as recognizing other factors that lead to the persistence of neglected diseases in affected regions. For example, although treatments do exist for some of the conditions, the healthcare infrastructure in the affected areas remains poor, and without local government backing, it is difficult for private companies and non-profit organizations to effectively intervene. Furthermore, a proper distribution network needs to be organized to transport medicines to the clinics and training is also required for healthcare personnel to administer the medicines. Aside from treatment options, there is the additional need for the development and implementation of prevention strategies, which necessitates government support.


One of the primary challenges of developing new treatments for neglected diseases is the fact that the drug development process is lengthy, expensive and risk intensive. A recent analysis of the pharmaceutical industry by CMR International found that it took approximately 11.5 years from the identification of a suitable drug target to the introduction of a new medicine,2 while the Tufts Center for the Study of Drug Development has estimated the cost of successfully getting a drug to market at more than $1.3 billion (€979 million).3 Additionally, according to the Pharmaceutical Research and Manufacturers of America, only two in ten medicines ever produce revenues that match or exceed average R&D costs.4

Bearing this in mind, pharmaceutical companies embarking on R&D projects need to be confident that they can recoup their investment. Often, companies tend to focus commercial efforts on North America, Europe and Japan, because these markets represent more than 80% of global pharmaceutical sales and increase the likelihood of making a profit from discoveries. Because pharmaceutical companies have to answer to the demands of investors and the financial markets, it is difficult for their management to justify the expense of developing a medicine exclusively for use in developing countries, as a return on investment is unlikely. This dictates a new approach to tackling neglected diseases, whereby the experience of pharmaceutical companies in innovative R&D is linked to alternative financing mechanisms.

Partnering for progress

The International Federation of Pharmaceutical Manufacturers and Associations (IFPMA) points to collaborative R&D programmes with different public and private partners as a viable solution.5 The IFPMA comprises 26 leading international companies and 44 national and regional industry associations covering industrialized and industrializing countries. These public–private partnerships (PPP) draw on the strengths of participating organizations, and share the cost and risk of developing drugs, thereby minimizing the impact of R&D failures on each of the partners. Partnerships can include governments, nongovernmental organizations (NGOs), pharmaceutical companies and academic groups.

The notforprofit Medicines for Malaria Venture (MMV), established as a foundation in Switzerland in 1999 following discussions between the WHO and the IFPMA,6 is one such PPP. The organization's mission is to reduce the burden of malaria in diseaseendemic countries by discovering and developing affordable antimalarial drugs. Its strategy is based on virtual drug discovery and drug development, which involves outsourcing key activities, but managing their progress through a central unit.

Although malaria is a major public health problem in more than 90 countries and causes up to 6 billion new infections worldwide annually, only four antimalarial drugs were developed between 1975 and 1999. However, the MMV has steadily built up a pipeline of 15 projects at different stages of development, which has been cited by the IFPMA as the largest antimalarial drug research portfolio since World War II.5 A number of major pharmaceutical companies are also involved in MMV's projects; in December 2008, the MMV signed an agreement with GlaxoSmithKline (GSK) to develop tafenoquine as a cure for Plasmodium vivax malaria, and in 2009 an agreement with Pfizer was announced that would allow MMV access to the company's library of novel chemical entities to screen for compounds with the potential to be developed into new malaria treatments.

A number of other partnerships involving the pharmaceutical industry have also proved effective. In 2003, the WHO asked for assistance in combating an emerging meningitis epidemic in Africa, where outbreaks of the disease occur frequently during the dry season. The WHO registered 44280 meningitis cases and 5531 deaths in this region during 2002, but collaborating with GSK and the Bill & Melinda Gates Foundation enabled a new meningitis vaccine to be made rapidly available to African countries following the discovery of a new strain of the disease.7 An initial 3 million doses of the vaccine were made available in an area comprising 21 countries. The usual cost of such vaccines is $5–50 (€3.8–38) per dose, making it too expensive for the countries where it was needed most, but the collaborative approach allowed the new vaccine to be made available at $1.50 (€1.1) per dose.

The current status of neglected disease R&D

R&D efforts to combat neglected diseases have increased substantially, but it is often difficult to assess the extent of the improvements and predict which areas have benefited most. In early 2009, the George Institute for International Health released details of a unique study of global public and private investment into R&D for new drugs for neglected diseases and found that more than $2.5 billion (€1.9 billion) was invested in 2007, with nearly 80% of this amount focusing on HIV/AIDS, tuberculosis and malaria.8 While this was considered encouraging, the authors pointed out that a number of diseases still received little attention and that there appeared to be no dedicated global initiatives to tackle them. These included conditions such as Buruli ulcer, caused by infection with Mycobacterium ulcerans, which has been reported in more than 30 countries — mainly with tropical and subtropical climates.

According to the report, most funding for neglected diseases came from the US National Institutes of Health (42% of total funding) and the Bill & Melinda Gates Foundation (18% of total funding). Twelve organizations alone accounted for more than 80% of this global total and approximately one-quarter of funding was devoted to PPPs, such as the International AIDS Vaccine Initiative and the MMV. Unfortunately for most diseases, the funding was not sufficient to create even one new product; for example, sleeping sickness, leishmaniasis and Chagas disease collectively received only $125.1 million (€94.2 million) in funding and diarrhoeal illnesses collectively received only $113.8 million (€85.7 million) in global funding. Buruli ulcer was among a group of neglected diseases that received less than $10 million (€7.5 million), or 0.4% of total global investment.


Since the late 1990s there have been greater efforts by a number of parties, including the pharmaceutical industry, to focus more R&D on launching new drugs for neglected diseases. To date, the most popular approach has been through PPPs, and, although such measures have proved successful in harnessing the different skills of those involved to develop new drugs, such efforts remain targeted to a narrow band of neglected diseases. As such, there still remain other areas that require attention. It will be important for governments, the WHO and pharmaceutical industry associations to encourage new collaborations in these remaining underserved areas. Only in this way can inroads into tackling neglected diseases truly be made.

Faiz Kermani serves as President of the Global Health Education Foundation (GHEF), a non-profit healthcare organization ( .


1. World Health Organization (2009).

2. CMR International, "The CMR International 2007 Pharmaceutical R&D Factbook" (2007).

3. J.A. DiMasi and H.G. Grabowski, Managerial and Decision Economics, 28 (4–5), 469–479 (2007).

4. Pharmaceutical Industry Profile 2009 , (Pharmaceutical Research and Manufacturers of America, WA, USA, April 2009).

5. International Federation of Pharmaceutical Manufacturers and Associations, "Neglected Diseases And The Pharmaceutical Industry" (2003).

6. Medicines for Malaria Venture (2009).

7. World Health Organization, "Partnership moves in record time to provide vaccine against meningitis as epidemic emerges in Africa" (2003)

8. M. Moran et al., PLoS Med, 6 (2) (2009).