Orphan Drug Act Gives Hope to Patients with Rare Diseases
Landmark legislation gives hope but changes still needed in IH treatment
February 12, 2008
By Lianna Haywood
(This article appears at www.ohsu.edu)
Imagine having double-vision, headaches so severe nothing can help them, and hearing a whooshing and pounding so loud you can’t fall asleep. And then imagine visiting multiple doctors and being told nothing was wrong with you. This is a normal scenario for patients with intracranial hypertension. And exactly what happened to Megan Hoffart.
Megan visited countless specialists for more than a year before one finally helped her reach a diagnosis. Today, severe headaches with little relief have become her daily norm. Megan still has problems with short-term memory. Vision problems keep her from spending too much time looking at a computer or reading, and a side effect of one of her medications gives her sharp pains in her hands and feet that can keep her from driving.
Intracranial hypertension develops when the pressure of cerebrospinal fluid in the skull is too high. The body naturally produces and then absorbs cerebrospinal fluid daily. When the body can not effectively absorb cerebrospinal fluid, intracranial pressure rises. The increased pressure causes severe headaches, and pressure on the optic nerve can cause vision problems and blindness. Anyone can develop chronic intracranial hypertension, regardless of age, gender, ethnicity, race or body type, though it frequently strikes overweight women of childbearing age.
Intracranial hypertension is known as an “orphan” disease because it affects fewer than 200,000 Americans. This year marks the 25th anniversary of the Orphan Drug Act. This groundbreaking legislation brought real hope for more than 25 million Americans living with one of the 7,000 rare disorders, or orphan diseases, recognized today. The Orphan Drug Act spurred breakthrough drug research and development for little-known diseases like intracranial hypertension while providing a potent catalyst to the growth of the pharmaceutical and biotechnology industries in the United States.
In the past, because of very low prevalence, orphan diseases were overlooked by drug and medical device developers. In the 10 years prior to passage of the Orphan Drug Act, only 10 new drugs for rare diseases were developed by the pharmaceutical industry. In the 25 years since the approval of the act, more than 300 new orphan drugs have been approved in the United States - an average of about 11 new drugs every year. However, no drugs have been specifically developed to treat chronic intracranial hypertension.
The Intracranial Hypertension Research Foundation (IHRF) is the only nonprofit organization in the world devoted to supporting the medical research of chronic intracranial hypertension. The mission of IHRF is to discover exactly why chronic intracranial hypertension occurs and to foster medical research to find better ways to treat, prevent and ultimately cure this disorder. The IHRF maintains a registry of medical data from intracranial hypertension patients that researchers can use for studies. The IHRF also serves as an educational resource for patients, families and physicians.
The IHRF registry is a project of the IHRF and the Oregon Health & Science University Casey Eye Institute.
“That the IHRF exists gives me hope,” said Hoffart, director of alumni and student relations in the Oregon State University/Oregon Health & Science University College of Pharmacy. “It gives me hope that I have a place for education, hope that I have an ally, and hope that someone is fighting for me.”
Oregon Health & Science University is the state’s only health and research university, and Oregon’s only academic health center. OHSU is Portland’s largest employer and the fourth largest in Oregon (excluding government), with 12,400 employees. OHSU’s size contributes to its ability to provide many services and community support activities not found anywhere else in the state. It serves patients from every corner of the state, and is a conduit for learning for more than 3,400 students and trainees. OHSU is the source of more than 200 community outreach programs that bring health and education services to every county in the state.
As a leader in research, OHSU earned $307 million in research funding in fiscal year 2007. OHSU serves as a catalyst for the region’s bioscience industry and is an incubator of discovery, averaging one new breakthrough or innovation every three days, with more than 4,100 research projects currently under way. OHSU disclosed 132 inventions in 2007 alone, and OHSU research resulted in 33 new spinoff companies since 2000, most of which are based in Oregon.