E-DRUG: non-commercial diseases / neglected diseases (2)
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Ans,
Being the first author of the Lancet article you are referring to, I would
like to modulate your comments promoted through your abstract, and
contribute to the discussion (see the message of Mary Moran on
non-commercial diseases).
1. Through the orphan drug policies - mainly in the US and the EU, even if
the social aspects of these policies can be debated, significant R&D with
critical outcomes has been done for the past twenty years (cf. Trouiller P
et al. Is orphan drug status beneficial to tropical diseases control?
Comparison of the American and future European orphan drug acts. Trop Med
Int Health, 1999).
2. If you are right when you say - as we wrote it in the 2002 Lancet paper,
that no indication that drug development for neglected diseases will
significantly improve in the near future, you have to put it in context. Our
conclusions were to point out that whether we have indeed nothing
significant to expect from the traditional R&D-based pharmaceutical
industry, we could reverse the current trend with new approaches (see an
article in French on: Access and dissemination of medical innovation in
developing countries, A lost battle? Trouiller P. et al. Revue de Sant�
Publique S�ve, 2004). The very young example of the Drugs for Neglected
Diseases Initiative (DNDi), recently launched by MSF and other partners is
illustrating the fact that there is really some hope.
3. In the Neglected Diseases Group, I have proposed the following definition
for a " neglected disease ":
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Excerpt of a NDG report (MSF, 2001)
1) Evolution of the concept of neglected diseases
Because pathogens causing plague or pestilence in humans have evolved over
millions of years and established a symbiotic relationship with other living
creatures, infectious and parasitic diseases have been labelled with various
epithets, as a reflect of historical, social and/or ideological concerns.
For many centuries while the concepts of "epidemic" and "endemic diseases"
was postulated through the work of Hippocrates (450-370 BCE), common
prevalent plagues and pestilences � probably diphtheria, malaria, smallpox,
tuberculosis and typhus - were seen as local miasmas coming out of the
earth. Much later, in 1546, the concept of contagious diseases was proposed
by Fracastoro, while the world was opening up to new horizons (discovery of
the New World and colonisation of north Africa in 1492) and many infectious
diseases began to be exchanged beyond between national or natural boundaries
(e.g., the export of tuberculosis from Europe to Africa, measles, smallpox
and typhus from Europe to the New World, and vice versa for syphilis), and
others unknown or little known in Europe were discovered, such as
leishmaniasis and sleeping sickness. All were eventually put beneath the
umbrella of what was considered medicine of warm climates. With
colonialization came the classification of tropical diseases, although they
did not constitute a single natural class of pathogen, instead comprising a
medley of infectious, parasitic and non-communicable diseases that are
generally found in the intertropical areas.
All things considered, the health hazards that white men faced abroad while
trading, fighting, preaching or planting in areas such as Africa, the East
Indies or India were portrayed by the emergence of tropical medicine. Once
the consequences of colonial expansion for indigenous people's health had
become increasingly apparent, and few colonies gained their independence,
disease control and public health (tropical health) programmes were expanded
(Manderson, 1999). In addition with the emergence of the "international
community" concept as illustrated by the successive creation of the OIHP and
WHO, tropical diseases shifted from the European countries and US national
top of the agenda to the WHO, but shrunk to a handful of diseases (six
diseases on the WHO/TDR agenda in 1975; ten in 1999). What tropical diseases
lost in number, they seemingly gained in consideration, based on sound
public health contingencies trying to resolve the initial conflict between
tropical medicine and tropical health. <endnote #1> Although there has been
clear progress (e.g., onchocerciasis, guinea worm), WHO/TDR could not meet
by and large its unrealistic missions of "developing new and improved tools
(medicines, vaccines and diagnostics) and help tropical countries to improve
their own research", given its unsuitable organisation to be operational,
recurrent lack of funds, vague agenda, and a more than rational political
decision-taking process (Gelband & Trouiller, 2002).
With the globalisation process highlighting health inequalities and
epidemiological interdependence, the advent of HIV/AIDS pandemic
destabilising fragile health care infrastructures, the re-emergence of
infectious diseases in drug-resistant forms (such as malaria, tuberculosis,
and bacterial pneumonias) and recurrence of others (such as sleeping
sickness), it became clear that:
(i) Many diseases, and not only tropical or "warm climates diseases�, could
no longer be sidelined and treated by mere indigent expedients.
(ii) The main determinant of disease is poverty and other socio-economic
factors rather than a warm climate.
(iii) The importance of many diseases had been neglected. From an initial
ideological approach (i.e. tropical medicine as a "tool of empire"), then a
technical and mechanistic method to fix a specific problem up (i.e. creation
of a "special" programme such as the WHO/TDR to serve tropical health), one
came to the perception that the so-called neglected diseases while being a
biological expression are largely socially determined. Neglected diseases
appear from then on as one of the metaphors of social and health
inequalities, embedded in a complex �bio-social� reality (Farmer, 1999).
(iv) The fragmentation and decline in international health aid, both
portrayed by the exponential and recent development of PPPs, lead to an
increased selectivity in favour of projects judged to have the
characteristics of "good policy" environments and image that guarantee good
returns or reasonable chance of successful outcome (cost-effectiveness
criteria), with the emergence of double level in health interventions
priority-setting through a rationing by exclusion. Wherefore compassionate
epithets such as �neglected� or �indigent� which express the failure to look
after properly one issue because you do not care enough about it.
2) Definition and scope (Depoortere et al., 2001)
Paradoxically there does not exist to date a comprehensive overview of the
possible needs in terms of pharmaceuticals for neglected diseases. Building
a conceptual framework to identify and characterize neglected diseases is
essential for setting a rational and need-driven R&D agenda, and how an
issue is defined strongly affects how it is addressed (Zumla, 2002). An
important criterion is clearly the existence and availability, or not, of
effective, affordable and field-adapted pharmaceuticals (i.e. current
treatment options); other relevant parameters include diseases severity
(e.g., case fatality rate, burden of disease expressed in DALYs) and
magnitude (e.g., disease incidence and/or prevalence), geographic
distribution, recent and ongoing clinical development activities. From a
pharmaceutical R&D perspective neglected diseases could be defined as: