Good afternoon. My name is Diane
Edquist Dorman, Vice President for Public Policy for the National Organization
for Rare Disorders (NORD). I have no personal financial relationship with
Questcor. For the past few years, however, NORD has administered an allotment of
free product for the uninsured through our Acthar Gel Patient Assistance Program
Questcor also provides a donation to our Infantile Spasms Co-payment Assistance
Fund for the under-insured.
In 1996, the FDA received word from
concerned pediatric neurologists that Acthar Gel® was no longer going to be
manufactured. Shortly thereafter, NORD received notification from the FDA that
NORD would serve as the official "gatekeeper" for the remaining supply until
additional drug became available.
As a result of the shortage, an
emergency process was put in place to funnel all requests for Acthar Gel®
through NORD. Doctors were referred to the Call Center and had to
document that he/she was prescribing for infantile spasms or for severe exacerbations association with MS. All requests for the use of Acthar Gel®
for other conditions had to be turned away due to limited supply. The remaining
supply was housed at one specialty pharmacy in order to ensure that prescribing
physicians received Acthar Gel® within a 24 hour period. As a result
of the emergency plan that was in place for a year, patients received the
therapy free of charge. Hundreds of infants receiving Acthar Gel®
were spared profound brain injury or death.
I would like to share with the
Committee an email we received recently:
"I am the mother of a child that has
been diagnosed with West Syndrome. The treatments that we have tried have not
worked. We needed to get ACTH and weren't sure how to get the money for it. This
program helped answer our prayers by paying for the med and getting it to us
faster than the staff believed possible. My daughter's have almost completely
stopped and her EEG is reading close to normal. It's been only two days. Thank
you so much. It's the most wonderful thing to see my five month old able to lay
still and alert. I don't know where she would be right now without your help."
As you continue to deliberate today, I
ask only that you keep two things in mind: 1) patients affected by rare diseases
are willing to take on a far great degree of risk than those affected by more
widely understood diseases affecting larger populations; and 2) because there
are only 357 orphan products that treat few rare diseases, patients and their
doctors have very limited treatment options available to them. Acthar Gel®
will give physicians, patients, their families a life-saving and critically
important alternative.
Thank you.
Contact Information: Diane
Edquist Dorman
Vice President, Public Policy
National Organization for Rare Disorders
1779 Massachusetts Avenue, NW, Suite 500
Check here to read about events on Capitol Hill, funding for rare-disease research, and other topics of interest from NORD's office in Washington, DC.