America Living With AIDS

approved drug granted orphan status
for a new indication (as was the case
for pentamidine), the loss of orphan
status once high profitability was
established would mean the loss of
exclusivity for that drug and for that
indication. Market forces would then
be expected to lower costs.
This proposed approach has an
advantage over a population limit for
orphan drugs in that it is based on a
demonstrable end point (profits or
sales), whereas population targets are
likely to be based on figures (such as
the number of HIV-infected individuals) subject to considerable debate. A
sales cap of $150 million dollars has
been suggested by the National Organization of Rare Disorders. This
option attempts to return the Orphan
Drug Law to its original intent, and
would not affect drugs with low sales
or profits.
This proposal could result in considerable savings for all payers of
drugs for treatment of HIV disease.
Savings on pentamidine alone could
amount to $900 per patient per year;
administrative costs would be minimal. Such a policy would not be
AIDS-specific and would be expected
to benefit any patient group requiring
an expensive orphan drug whose price
was affected by the amendment.
The Commission firmly believes
that total systemic reform will be necessary to genuinely achieve appropri
ate access to health care for all people
living in the United States. In the
interim, the Commission recommends a series of immediate shortterm steps to address the urgent problem of inadequate coverage for people
with HIV disease.