A new dawn for neglected diseases?

Breaking with the past

Faiz in India

Despite numerous improvements, drug development remains an expensive and lengthy multi-stage process with there being no guarantees of success. Therefore drug development organizations are often preoccupied with recouping R&D investment upon subsequently marketing a product. Unfortunately, this has resulted in many companies avoiding certain therapeutic areas as they are not perceived as offering sufficient financial rewards for products. Diseases of the developing world fall into this category and are often referred to as ‘neglected diseases’.

In recent years there have been major efforts to rectify the lack of investment in research for neglected diseases. Agencies such as the World Health Organization (WHO) have been urging a better focus on this area, particularly since one sixth of the world’s population suffer from one or more neglected disease (1). This is an issue that has attracted considerable media coverage and represents a challenge for all those involved in healthcare to overcome. To encourage further work in this field, WHO have highlighted the notable successes achieved when coordinated action has been taken. For example, 14.5 million patients have been cured of leprosy through multi-drug therapy since 1980 (1).

Persisting problems

Historically, governments and international agencies have hoped that the general commercial forces that drive R&D innovation would naturally extend to encompass new drug development for neglected diseases. However, this has proved to be an ineffective strategy and few drugs have emerged as a result.
One frequently quoted estimate is that between 1975 and 1999 only 13 new drugs for neglected diseases were developed (2). Since pharmaceutical companies are commercial organizations, the expectation that goodwill alone will vastly improve productivity in this field suggests an oversimplification of the healthcare challenges being faced.

Despite the political and legal battles that have characterized this area, it would be unfair to suggest that pharmaceutical companies have not boosted their own R&D efforts for neglected diseases, but this alone will not solve the problem. Producing a drug for a neglected disease only represents part of the solution in tackling it. Some companies are still reticent to become involved as they believe they will be under pressure to run all healthcare activities beyond the development of a drug (3).

Long-term improvements in tackling neglected diseases require the effective cooperation of all parties in the field of healthcare and must be aimed at setting up reliable infrastructure as well as distribution of the drug for medical services. Furthermore, there will need to be adequate training of the healthcare personnel involved. A system that allows people access to affordable medicines is obviously desirable, particularly in medical emergencies, but it must be accompanied by improvement in other areas of the healthcare systems of these countries in order for long-term progress to be achieved. Thus a dedicated health strategy is required whereby pharmaceutical companies work together with governments, representatives of the national health care systems, non-governmental organizations (NGOs) and involve the public. Yet it would appear that the lack of clarity in defining the practical responsibilities within these initiatives has done much to undermine coordinated action and without trust these projects cannot succeed.

Going forward

Some progress has been made through the establishment of Public-Private Partnerships (PPP), whereby companies, governments and NGOs collaborate on a formal basis. These not-for-profit initiatives have proved popular and have encouraged better relationships to be built between the various stakeholders. One study identified 12 PPPs in existence between 1980 and 1990, but for the period 1990 to 2000 they found 71 such initiatives in operation (4). As more pharmaceutical companies become involved in these initiatives others will be encouraged to enter this field. Much of the attention is focused on the large pharmaceutical companies, but small and medium-sized companies have much to offer and their participation should also be sought. This may necessitate specific incentives to attract them to the field (3).

While further industry involvement should be encouraged, all aspects of the PPP must be examined so that the set-up created has the maximum chance of success. The experience of pharmaceutical companies gained through commercial drug development is crucial, but they must not be the only parties who are critically assessed for performance. As PPPs still represent a new approach to developing drugs for neglected diseases, and since they involve the use of public funds, care must be taken that the various projects being run do not lead to duplication and waste of resources (3).

A 2005 report by a team from the London School of Economics and Political Science suggests the future of neglected disease R&D looks promising (3). They found that 60 relevant projects were in progress with 18 of the drugs being at the stage of clinical trials (3). Interestingly, half of those in clinical trials were in Phase III suggesting that the arrival of new products on the market should be imminent. By their calculations, these projects alone were expected to result in eight or nine new drugs by 2009 (3).


In 2000, 189 world leaders made a commitment to achieve eight Millennium Development Goals by 2015 (5). These ambitious goals focused on dramatically reducing poverty and ensuring marked improvements in the health of the poor. This timeframe should be sufficient for some of the promising drug candidates for neglected diseases to make it through clinical trials. However, it will be important that the continuing R&D in this field accounts for the full range of diseases affecting the developing regions of the world and does not solely target areas that generate greatest media attention. Equally important will be evaluating the R&D set-ups that prove most effective so that the experiences of the various partners can be used to improve the success of other initiatives.


1. Control of Neglected Tropical Diseases. World Health Organization.
2. Pecoul B et al. (1999). Access to essential drugs in poor countries: a lost battle? JAMA. 281 (4): 361-67.
3. The new landscape of neglected disease drug development. Wellcome Trust.
4. Roscigno G (2003). Partnerships for Health: evolution and challenges. Foundation for Innovative New Diagnostics. European and Developing Countries Clinical Trials Partnership. EDCTP Rome 2004.
5. Millennium Development Goals (MDGs). World Health Organization

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