In the decade before the
Orphan Drug Act was passed by Congress and signed by President Ronald
Reagan, only 10 treatments had been developed for rare diseases by the
pharmaceutical industry. In the 25 years since then, more than 1,100
treatments for rare diseases have entered the research pipeline and more
than 300 have been approved by the U.S. Food and Drug Administration for
marketing. In addition, the Act has proven to be a potent catalyst to
the growth of the pharmaceutical and biotechnology industries in the
United States.
A rare or “orphan” disease is defined by the U.S. Food and Drug
Administration (FDA) as a disease or condition that affects fewer than
200,000 Americans. In the past, these diseases of low prevalence were
overlooked by drug and medical device developers. The Orphan Drug Act
provides financial incentives that help companies recover the cost of
developing a drug for small patient populations.
During 2008, NORD and its members, along with the National Institutes of
Health (NIH), the U.S. Food and Drug Administration (FDA), and the
pharmaceutical and biotechnology industry organizations, celebrated the
25th anniversary of both the Orphan Drug Act and of the founding of NORD.
To raise awareness of rare
diseases and the need for safe, effective treatments, people around the
world will join together to observe the 2nd Annual Rare Disease Day on
the last day of February (Feb. 28, 2009). NORD is coordinating this
project in the U.S., and is inviting all patient organizations,
caregivers, researchers, and companies developing orphan products to
join in this observance. The purpose is to focus attention on rare
diseases, the challenges encountered by those affected, and the
importance of research to develop diagnostics and treatments.
The 1st Rare Disease Day was organized in Europe last year by EURORDIS,
the European Rare Disease Organization. It was very successful, with
many political leaders and members of royal families helping to draw
attention to the activities. This year is the first time for Rare
Disease Day to be observed in the U.S. The hope is that this will become
an annual global event on the last day of February.
Rare Disease Day Partners
All organizations, societies, and companies working with rare diseases
and/or orphan products are invited to become Rare Disease Day Partners.
(For information on this, write to rarediseaseday@rarediseases.org).
NORD is compiling a list of Partners that will be published soon on the
NORD website. Partners are also being asked to:
* Write to their state governors requesting that February 28, 2009, be
designated Rare Disease Day in their state.
(CLICK HERE FOR A SAMPLE LETTER)
* Post the Rare Disease Day logo on their websites and in their
publications.
* Make their members aware of Rare Disease Day.
* Encourage media coverage of Rare Disease Day by suggesting stories to
their media contacts for that day or the week leading up to it. (NORD
will provide a press kit soon to all partners that can be personalized
and used as needed.)
* Share information about any activities their group may sponsor related
to Rare Disease Day so that other organizations can benefit from reading
about what they are doing.
* Share human interest stories about individuals and families affected
by rare diseases to rarediseaseday@rarediseases.org, for possible
posting on this website or for possible use by reporters covering Rare
Disease Day.
* Nominate researchers to a Rare Disease Hall of Fame to be published on
the website. This can be done by sending a brief (one page or less)
summary of the what the researcher is doing, why it is important, and
any results to date that have improved the lives of people with rare
diseases. If possible, nominations should include a photo of the
researcher.
Watch for More Information Soon
NORD will be posting new information regularly on their page and in
emails to Partners. If you have questions or would like to share
information related to Rare Disease Day, write to rarediseaseday@rarediseases.org.