Singular Voices, Collective Ideas
In the early '80s, as patients and physicians everywhere were grappling with AIDS and wondering what to do, another group
of patients—or rather, several small groups—had other diseases on the mind. The National Organization for Rare Disorders (NORD)
was founded in 1982, when Abbey Meyers realized that the trouble she was having in getting pharma to manufacture a drug for
her son's rare disease was actually a problem across the board (see Abbey Meyers profile, "The "Mother" of Invention"). Meyers'
efforts and the work of NORD quickly spawned the Orphan Drug Act of 1983—a clear example that a solitary voice, when banded
with others, could be heard.
Today, NORD is continuing to aim for the goals set by Meyers 29 years ago. "We see ourselves as the voice for patients with
rare diseases," says Peter Saltonstall, president and CEO of NORD. The organization serves as an umbrella for many smaller
rare disease advocacy groups across the nation—providing advice, raising funds, shaping legislation, and offering expertise
to those smaller groups in an effort to pool resources and collaborate on behalf of the patients.
A current focus for NORD is assessing the cost of bringing a drug for a rare disease to market versus not bringing that drug to market, especially in terms of burden on the healthcare system. "I ran a large hospital in Boston,
and I can tell you that patients who didn't get diagnosed or didn't have their ongoing medication for whatever reason would
always end up at the emergency room," says Saltonstall.
Going forward, Saltonstall believes that increased collaboration between pharma and NORD (and all advocacy groups) will be
key. Though he says that smaller pharmas have traditionally been the ones developing orphan drugs, Big Pharma is now getting
in the game. "Big Pharma has a different kind of business model," cautions Saltonstall. "They historically have not been plugged
into the patient as much as I think they need to be. So NORD's task is to educate them, to help them master what it is to
bring a rare disease therapy to market."
Thankfully, he says, Big Pharma welcomes the expertise NORD brings to the table. "One of the big players has come to us and
asked us to help structure their rare disease program. We're helping them think through what this whole business unit needs
to look like, what elements it needs to be successful from our perspective. They sought us out—that's very encouraging."
For some patients, however, storming the NIH or writing letters to The New York Times to call attention to their cause just isn't an option. "Let's be honest—the Alzheimer's population is not as vocal in advocacy
as others, because the disease really restricts and sometimes removes that voice," says Eric Hall, president and CEO of the
Alzheimer's Foundation of America (AFA). "All the breast cancer walks and other advocacy efforts of other groups are great.
They are able to find individuals who get up and say, 'I had this illness, and thanks to leading therapies and treatment and
care, I'm now cancer free.' And that's marketing for the cause. But we don't have anyone making those statements—Alzheimer's
doesn't have any survivors."
Like NORD, the AFA is an umbrella organization, uniting the efforts of smaller advocacy groups across the nation. But the
main focus here is not on R&D and finding a cure—it's on supporting the patients and caregivers through their daily struggle
with managing the disease. When the AFA was founded in 2002, says Hall, "One thing I sensed was that there was an enormous
amount of attention being paid to research, and the idea that we would find a cure, and that was the most important objective.
There was so much attention being paid to research for a cure, but in the meantime, there are all these other needs."
Based on the number of individuals currently affected with Alzheimer's and demographic projections, Hall emphasizes the need
to focus on proving support for patients and caregivers, and on what to do about the financial impact Alzheimer's will have
on the country. "This is an almost unprecedented impact on the national economy—in the absence of a cure, what happens? How
does any one country end up caring for this onslaught?"
AFA has helped carve a noticeable chunk out of the challenges associated with Alzheimer's through such efforts as National
Memory Screening Day, a magazine devoted to caregivers, a training program for healthcare professionals called Dementia Care
Professionals of America, and an online support community for teens who are assisting with the at-home care of a grandparent
or other Alzheimer's patient.
As the AFA and other patient advocacy groups continue working toward their goals, Hall, like Saltonstall, says the most important
element going forward will be collaboration. "It's natural for organizations to be focused on their own objectives, and to
have their own goals and missions ... it's not easy to say, 'This won't be about us. We'll do it for the greater good.' But
AFA is an example of what can happen when organizations come together. ... I think advocacy is best served when organizations
unify their voice."