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FDA and Rare Disease Drug Development, from FDA Week

June 29, 2010 // [FDA Week, http://insidehealthpolicy.com/]

FDA is currently in the middle of a flurry of activity aimed at better focusing its regulation of rare disease treatments, but the agency disappointed some outside advocates by delaying the possibility of creating a special rare disease review division within FDA for at least a year. That news came to advocates from FDA staffers an hour before a Senate Appropriations subcommittee looking at federal regulation of these diseases last Wednesday (June 23), a source said. More surprising to the source were comments from National Organization for Rare Disorders leadership during the hearing that the establishment of the new agency division would be a mistake.

FDA has two working groups addressing the issue and a number of rare disease regulatory advocates are proposing improvements to the agency’s regulatory regime. Senate appropriators are also eying whether legislation is needed.

The concept of a rare disease review division has been championed by the Emil Kakkis and his foundation, which has long advocated for Congress to provide FDA $10 million to create the special division. Kakkis and other rare-disease treatment advocates argue that gathering FDA’s experts in the field into one group would help accelerate the regulatory process.

The move would have an “immediate impact on new treatments” and would signal to the biotechnology industry that the agency was “serious” about this effort, Kakkis told the Senate FDA appropriations subcommittee. A special division would encourage more investment and allow for products already in the pipeline to be more quickly acted on, Kakkis said.

FDA should “have a division that has particularly trained people in the diseases that are both chemical, muscular or some of the other genetic rare disorders that take specialized expertise,” Kakkis told the subcommittee. The best regulation would come from consolidating FDA’s experts who are “scattered throughout” the agency, adding staff and building a division where staffers can avoid “having their expertise diffused and not leveraged over time,” Kakkis said.

The problem without a dedicated division also comes with a lack of centralized responsibility for reviewers, Kakkis added. “There are definitely people [in FDA] with expertise who can review these, but often they are called on to do other tasks,” Kakkis said. “This is a way to get better decision-making.”

But a Kakkis foundation staffer told FDA Week that meetings between advocates and FDA staffers prior to the hearing indicated that, while conceptually supportive of the idea of specialization, the timing did not work for the division to be created this year. That impression became formal an hour before the hearing.

The source said his reading of the situation was that FDA was possibly waiting until after drug user fees are reauthorized in 2012 to create the office.

The “biggest shock” from the day, the source said, came from Diane Dorman, the National Organization for Rare Disorders’ vice president for public policy, who said NORD flat-out disagreed with the concept. Dorman said NORD was “somewhat concerned” because of the worry that a separate division would create “narrow silos” within FDA.

By way of example, Dorman described the National Institutes of Health’s office on rare diseases as a nimble group with which the office’s director can leverage opportunities across all NIH offices and institutes without barriers. There’s a possibility to create an ad hoc group, “but creating an additional silo [within FDA] in our opinion may cause problems to access,” Dorman told the subcommittee.

In his response, Kakkis said without the review division FDA “cannot alter the review process for drugs in a way that is fundamental.” Creation of the division would let staffers be “personally responsible to make decisions,” he added.

“I think you’d want your drugs to be reviewed by people who are trained in the area, that specialized in it, and really understand it,” Kakkis said.

FDA Deputy Commissioner for Science and Public Health Jesse Goodman said the agency is aggressively addressing orphan drug issues, pointing to two new expert working groups FDA established in March, the Rare Disease Review Group and the Neglected Disease Review Group.

Those workgroups “will look at all the ideas we’ve heard here” before issuing a report to Congress, Goodman said. The two groups were created to help implement language in the FY 2010 Appropriation Act, which directed FDA to establish the internal review groups to address rare and neglected diseases, and to report to Congress one year after establishing the groups before issuing guidance.

That language came at a request from Sen. Sam Brownback (R-KS), the ranking member on the subcommittee.

Other work recently undertaken by FDA include the agency’s Office of Orphan Products Development starting use of a new tool, the Rare Disease Repurposing Database, which identifies drugs deemed promising for rare illnesses and are already approved by FDA for another disease (see related story).

A “novel feature and major advantage of this database” is its focus on drugs that have already gone through the FDA approval process, Goodman noted in its written testimony. “Thus, repurposing of these drugs for a new rare disease indication might be attainable quickly, relatively inexpensively, and at great benefit to the patients involved.”

In February, FDA created a position of associate director for rare diseases in the Center for Drug Evaluation and Research, which serves as the center’s focal point to the orphan drug development community “in navigating the complex regulatory requirements for bringing safe and effective treatments to patients,” Goodman wrote.

The agency is also collaborating with other entities to help ramp up its rare-disease treatment efforts.

In October 2010, FDA will co-sponsor its first rare disease investigator training course, in collaboration with NORD and NIH. FDA is also planning a series of scientific workshops to address difficult rare disease research issues. And FDA and NIH are co-sponsoring an Institute of Medicine study to review national policy for rare disease research and related medical product regulation, with recommendations due this September.

Experts suggest that there are roughly 200 FDA-approved treatments for the nearly 7,000 rare diseases that exist globally. So “while there have been many successes, the unmet needs are huge,” Goodman told the subcommittee. Rare diseases are defined as diseases affecting fewer than 200,000 people in the United States.

The FDA estimates that a billion people worldwide are affected by at least one neglected disease like tuberculosis, malaria and leprosy.

These issues are FDA’s concern because “infectious diseases know no boundaries,” Goodman said. “Threats to health anywhere are threats to everyone.”

The agency also is preparing for public hearings on June 29-30 focusing on how it approves and encourages medical products for rare diseases. — Seth Freedland (sfreedland@iwpnews.com)

FDA Hearing on Rare and Tropical (Neglected) Diseases

Just finished up watching the FDA hearing webcast from earlier this week.

Loving Sen. Sam Brownback.

If you have a rare disease, “God bless ya” because we “got no treatment for you.” The billion dollar per drug model doesn’t work for 7,000 rare diseases. We need a new different model – adjust the system so that it costs less and still provides quality drugs. Time to stop walking by the sick people. We can do better.

and Sen. Mark Pryor.

Not so sure how much I love about 50 minutes into the webcast. Ouch. Diane Dorman of NORD referring to Emil Kakkis of Kakkis EveryLife Foundation‘s specialized review division as — an “additional silo.”

// Catherine Calhoun

Advocacy Update from Emil Kakkis of Kakkis Everylife Foundation

Thanks to all our Partners and Friends for your calls to Senators Brownback and Kohl — it is working!

by Emil D. Kakkis

24. April 2010

It was exciting to see grassroots politics in action through your 100+ calls in support of our $10 million request to create a specific review division at the FDA. The respective offices were inundated with calls from very motivated patient community leaders, and the numerous passionate callers were noticed.

While we will have to wait until June for the first draft of the appropriations bills to be released, there is still much work to be done. Members of the House and Senate FDA Appropriations Subcommittees have significant power to move this forward and we are continuing to push the issue with DC staff. We have sent out a letter for patient organizations, academic institutions, biotechnology firms and venture capitalists to endorse the request, and more than 50 organizations have already signed on. We are also working with patient organizations to arrange constituent meetings in district offices with key Members of Congress. Additionally we will launch our CongressWeb™ function next month on our websites enabling you to automatically e-mail a letter of support to appropriate representatives in Congress.

Thanks in part to your calls, Senator Kohl (D-WI) (the Chair of the Appropriations Subcommittee) has agreed to Senator Brownback’s (R-KS) request to hold a hearing on rare disease regulatory issues in June, giving us a special forum. Senator Brownback is very interested in doing something important for rare disease patients and we should help support that goal in his last year in the Senate. Keep checking back for news on this development.

The new review Division is just a start. Combining new specialized staff with existing staff in a focused Division is an important step toward creating the new policies and guidance documents needed to ignite the development of treatments.

As always, thanks for all your support and please send me your thoughts.

Emil