Rare diseases go untreated

Thousands of people
with rare diseases cannot get the help they need

The problem of rendering medical help and drug coverage to
people suffering from rare (“orphan”) diseases came to receive a high level of
media attention in February, on the eve of International Rare Disease Day (29
February).  Non-profit organisations
requested that the Russian Ministry of Health allocate subsidies to the regions
from the federal budget, to finance the supply of medication for people
suffering from rare diseases.  It is
proposed that their treatment should be provided for out of regional
budgets.  To help such patients
effectively, a federal register of rare disease sufferers is being
formulated.  At present, there are 230
names on this list.  The Ministry has
also planned standards of treatment for 24 rare diseases which are recognised
as life-threatening or disabling (the programme “Seven nosologies” and 17
standards of treatment for other rare diseases).

At a press conference on “Equal access to medical and
pharmaceutical help for sufferers of rare diseases in Russia”, doctors and
patients’ representatives explained how the situation has changed in this
area.  The event took place today at RIA

The adoption of the federal law “On the
Fundamentals of Public Health Protection in the Russian Federation” should have
solved many problems faced by patients with rare (orphan) diseases.  Experts consider, however, that standards of
treatment of rare diseases have still not been established, and orphan drugs
registered in the country are not included on the list of vital
medication.  This
means that thousands of people with mucopolysaccharidosis, Fabry’s disease,
pulmonary arterial hypertension, and other rare diseases cannot get the
necessary assistance.

“There were hopes that patients would at least receive care
according to the list of 24 diseases, but the problem of treating rare diseases
is not mentioned in the state programme of healthcare development up to 2020”,
said Snezhana Mitina, president of
the union of patients and patients’ organisations for rare diseases.  She assumed that the issue of providing help
to patients suffering from rare diseases would be included in the
mother-and-child programme, but, in that case, adult patients’ right to
treatment would not be observed.

Professor Elena
, director of the clinical department of orphan diseases at the
Health Ministry’s State Scientific Centre, pointed out that there is a pressing
question of financing drug provision for patients with rare diseases.  At present, their medication is paid for out
of their own pockets and by charitable funds. 
The former management of the Health Ministry, however, raised the issue
of funding drug coverage through the state. 
Lukina expressed the hope that the new leadership will take further
steps in this direction.  Unfortunately,
Health Ministry officials did not attend the press conference; departmental
opinion was not reflected.


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