E-drug: Re: Wilson's Disease
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This reaction is fully understandable and deserves more attention.
When one categorizes diseases in function of their priorities on the
pharmaceutical industry agenda, we can consider the following six classes in
descending order of priority :
1. Non communicable diseases with a high level of prevalence (neurological,
cardio-vascular, alimentary/metabolic and musculoskeletal diseases or
disorders... etc.): for those conditions drugs are available because a
creditworthy and profitable market exists ; the only limit being scientific
limits (there is for instance not yet a sufficient medical and biological
knowledge to discover and develop a new candidate drug) or economic limits
(accessibility to available drugs, for instance in the US since there is no
nationwide social insurance scheme).
2. Communicable diseases constituting a threat for the Western countries
(e.g., antibiotics for nosocomial infections, antivirals for HIV): drugs are
available according to the same scheme as drugs of class 1 (e.g., anti-HIV
drug are available but with a limited accessibility due to their high price
when no social insurance scheme exists).
3. Rare diseases (or orphan diseases) but only in few developed countries
which have recognised their importance despite their low prevalence through
specific schemes (e.g. Orphan drug acts existing in the USA, EU and Japan):
governmental incentives push pharmaceutical industry to discover and develop
drugs (orphan drugs) despite the absence of market ; the only limits being
the same as those described for class 1 and 2.
4. Neglected diseases (according to the classification proposed by MSF) such
as malaria and TB, whose prevalence is high. Available drugs are old or
becoming ineffective, pharmaceutical industry has no well-structured R&D
strategy because the market is too "volatile" ; but public awareness on this
problem has increased and solutions could be reached ; moreover those
conditions could pose a threat for the developed world: they are strategic
diseases and public national and/or international bodies will "invest" on
them.
5. Very neglected diseases such as African sleeping sickness (AHT),
leishmaniasis... etc. (generally parasitic diseases) whose prevalence is
medium but without any creditworthy market : drugs are old or becoming
ineffective and there is no public and political awareness because these
diseases have no strategic interests ; those diseases (contrary to malaria
or TB) do not pose a threat for the developed world.
6. Rare diseases in developing countries such as the Wilson's disease case
mentioned by Mary Graper : sometimes drugs are available (see class 3) but
they will not be globally accessible because mainly of their price or their
pricing mechanism (e.g., a one year treatment for the Gaucher's disease
costs more than US$100 000 per patient).
That means there is still a huge work before rare diseases in developing
countries rise up the current priority scale. But nothing is hopeless: one
year ago who could "bet" that HIV triple therapy would drop from 10-15000
US$ to below US$2-300 and probably still lower in a near future.
Patrice Trouiller, PharmD, MB
- Hospital Practitioner
University Hospital of Grenoble, France
- Medecins sans Frontieres (Doctors without Borders, MSF) consultant
Tel : +334 76 76 54 97
Fax : +334 76 76 51 09
Email: PTrouiller@chu-grenoble.fr
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