Suchergebnisse

Rare diseases are a heterogenic group of disorders with a little in common
except of their rarity affecting by less than 5 : 10.000 people. In the world
is registered about 6000-8000 rare diseases with 6-8% suffering population
only in the European Union. In spite of rarity, they represent an important
medical and social problem due to their incidence. For many rare diseases
have no treatment, but if it exists and if started on time as being available
to patients, there is a good prognosis for them to be able for normal life.
The problems of patients affected by rare diseases are related to the lack of
diagnosis and timely undergoing as well as their treatment or prevention.
Orphan drugs are products intended for treatment, diagnosis or prevention of
rare diseases, but for their development and marketing the industry has not
been interested in yet because of their marketing reasons. Patients suffering
from a rare disease although belonging to the vulnerable group for their
specific health needs, is becoming invisible in the health care system due to
their additional needs un properly recognized. Ethical problems faced by
patients, but also health care professionals are related to the allocation of
medical diagnostics, unequal approach to health care, inappropriately
specialized social services as well as therapy and rare orphan drugs
unavailability. Ethical questions related to clinical trails on orphan drugs,
population screening and epidemiology testing on rare diseases will also be
discussed in this paper.