Rare diseases (RDs) are
significant public-health issue and they pose a challenge to the medical
community. These rare diseases are called ‘health orphans’, because research
and developments have neglected them for years due to many RED challenges. Amendments were made to the existing law in
the 1960s, mandating that every drug must be proven to be safe and effective
through ‘adequate and well-controlled studies’ before getting market approval.
Then raising drug development cost ended in drugs for small disease populations
being ‘orphaned’ by many top drug companies.
Congressional Findings for the Orphan Drug Act
(1) There are many diseases and
conditions, such as Myoclonus, Huntington's disease, ALS (Lou Gehrig's
disease), muscular dystrophy and Tourette syndrome, which affect a few individuals
residing in the United States that the diseases and conditions are regarded as rare
in the United States
(2) Satisfactory number of drugs
for treatment of many of such diseases and conditions has not been developed
(3) Drugs for such diseases and
conditions are generally referred to as "orphan drugs"
(4) Because very few individuals
are affected by such rare diseases or conditions, a pharmaceutical company which develops
an orphan drug may expect the drug to generate moderately small sales when
compared with the cost of developing the drug and consequently to incur a
financial loss
(5) There is reason to believe
that some promising orphan drugs will not be developed unless changes are made
in the applicable Federal laws to reduce the costs of developing such drugs and
to provide financial incentives to develop such drugs; and it is in the public
interest to provide such changes and incentives for the development of orphan
drugs.
The Orphan Drug Act began many years ago and
now, many believe that this is not the best use of resources. Attend the
webinar “Orphan Drugs: The Conundrum” by Marybeth Regan, to learn more about
the topic.
