Diagnostics & Imaging Week Washington Editor

WASHINGTON — The FDA funding merry-go-round has been in the works for better than a decade and a half, but that has not stopped the often outspoken Rep. John Dingell (D-Michigan) and others on Capitol Hill from lambasting the current FDA commissioner, Andrew von Eschenbach, MD, for his failure to play the squeaky wheel.

However, von Eschenbach has reversed field and, in a break with past FDA commissioners, requested additional funding from Congress in a professional judgment letter as Congress pastes together a comprehensive spending bill of roughly $3 trillion.

In response to a letter from Sen. Arlen Specter (R-Pennsylvania), the FDA chief has requested a bolus of $275 million, which FDA would dedicate to programs that ensure the safety of food, drugs and medical devices. Roughly $100 million would go for drug and device safety while $125 million would go toward food safety. The balance would go toward facilities and personnel.

The move took observers by surprise, with some speculating that von Eschenbach decided to unilaterally make the request because he saw the influence of the Bush Administration waning. Others have suggested the possibility that he made the request with the tacit or expressed but not publicized approval of the administration.

Perhaps the most difficult question to answer is whether this will become a precedent or a one-time event, but another question that is difficult to ignore is why Congress took so many years to pay attention to FDA other than to pile on mandates.

Former associate FDA commissioner Bill Hubbard is widely quoted as having said "[i]n 30 years at the agency, I never saw anything like this happen before." Hubbard now serves as a senior adviser to the Coalition for a Stronger FDA (Washington).

In response to von Eschenbach's letter, Sen. Herbert Kohl (D-Wisconsin) has inked in $275 million for FDA in the current fiscal year's budget as part of the emergency supplemental appropriations bill now working its way through Congress. Kohl chairs the Senate Appropriations subcommittee that has jurisdiction over FDA, the clumsily named Subcommittee on Agriculture, Rural Development, Food and Drug Administration and Related Agencies.

Hubbard told Diagnostics & Imaging Week that because von Eschenbach "has been increasingly criticized about not talking about FDA's needs ... I guess he felt he had to step forward" with a position.

He also reiterated that the move is at least "somewhat unusual, and I don't know if he did it with the tacit approval of the Secretary [of Health and Human Services Mike Leavitt] or OMB [the Office of Management and Budget]." Hubbard said von Eschenbach's reluctance to argue for a boost was destroying his credibility. "Group after group has been saying FDA is underfunded," and the FDA chief "was becoming increasingly incredible on that" point, Hubbard said.

"It's [also] possible the administration said 'our budget is out, if you want to get more from Congress, go at it,'" Hubbard said. Von Eschenbach may also have had an eye on the short calendar left to the 110th Congress. The August recess will be followed by the distractions of congressional and presidential campaigns, and "von Eschenbach might have said to himself 'if I'm going to get any money, I need to ask for it now,'" Hubbard said.

NIH to roll out undiagnosed diseases program

The National Institutes of Health (NIH) has traditionally tackled new and poorly understood diseases via traditional research, but the agency said it will henceforth take a more direct approach under the banner of the Undiagnosed Diseases Program (UDP).

NIH director Elias Zerhouni, MD, and several other senior members of NIH laid out the idea in a conference call yesterday. Zerhouni led off with the remark that the goal of the UDP is to "improve disease management for individual patients and to advance medical knowledge in general."

"This great initiative ... could be very transformative," Zerhouni said, adding that the practicing physician may be confronted with any one of 6,000 rare conditions and only about 600 well-known ones.

On the other hand, "even common diseases have many subtypes," he said, including diabetes, a fact that may explain as much as genetics does about why individuals have such different reactions to drugs.

"We're going to see patients earlier and earlier in the natural history of their disease," Zerhouni said, so diagnoses have to happen earlier. He described the program as something that will make NIH "the National Institute of Hope."

William Gahl, PhD, clinical director of the National Human Genome Research Institute, laid out a few more details. He said, "I have long been aware of the need for an undiagnosed diseases program."

However, patients cannot simply e-mail NIH and expect to get into the program. "We've established a fairly stringent referral process," he said, adding that "participants have to be referred" by a provider, which can be an MD or a nurse practitioner. The program will accept one or two patients a week up to 100 per year.

"The care is free for the patient and NIH will pay for travel and lodging," Gahl said. "Dozens of NIH's senior physicians will collaborate" on these cases, bringing a wide range of specialties to bear on these patients. The resulting data will fill out "a phenotype atlas for the country." Gahl, who will direct the program, also said the work should lead to groundbreaking articles in peer review literature.

Stephen Groft, PharmD, director of the NIH Office of Rare Diseases (ORD), said, "this project ... may lead to other clinical trials and studies" based on discoveries made during the course of treatment. He also said NIH had recently conducted a briefing with patient groups, in part prompted by the fact that NIH is "seeing an evolution of the role of patient advocacy groups as funders of projects."

Groft pointed out that this announcement coincides with the 25th anniversary of 1983's Orphan Drug Act and the establishment of the National Organization for Rare Disorders. Only about half the patients whose illnesses put them on this latter organization's list get a correct diagnosis within a year while another 35% wait five years. Given that, "I think the significance of the program is quite understandable," he said.

Amanda Young, 26, of Conyers, Georgia, was a patient in attendance on the call. "For years, my parents searched for the answers" to infections that routinely hit her, she said. Evaluations of her immune system seemed normal "except for one small thing; a continuously low white blood cell count."

Young said that by the age of three, she'd had "an abdominal abscess the size of a cantaloupe" removed along with multiple cases of meningitis. "My first visit (to NIH) was in 1990," she said, and Gallin, who was involved in her care, "said he would never give up on me, and he never has."

Echoing the frustration of anyone who has watched their health spiral down with no apparent cause, Young said, "all we (she and her parents) ever wanted was for my disease to have a name," which turns out to be the oddly named IRAK-4 (interleukin-1 receptor-associated kinase) deficiency.

"I haven't had a major infection in four years" thanks to NIH's work, she said, adding that she nonetheless has to keep an eye on her health. And "although my disease has a name, we don't have a treatment or a cure." However, Young asserted that the announcement of the new program "is like giving someone their life back."