In the complex and expensive world of research and development for drugs, there are therapies for conditions known as “orphan diseases,” those that affect fewer than 200,000 Americans. According to a recent article in the Wall Street Journal, “there are about 7,000 such maladies, most of them serious, that have few or no drugs to treat them.”
Examples of such orphan diseases range “from adenoid cystic carcinoma, a rare head and neck cancer, to Zollinger-Ellison syndrome, which is associated with a tumor that causes the production of high levels of stomach acid.”
Usually, the difficulty of creating enough financial support to research orphan diseases is extremely difficult however, WSJ reports that “staff members at the Food and Drug Administration are doing something unusual: leaving Washington to help drug makers take a crucial step in developing drugs for orphan diseases.”
Consequently, these “staffers are helping to administer the Orphan Drug Act, which provides incentives to create therapies for so-called orphan diseases.” As a result of these incentives, “doctors may end up prescribing drugs developed for other diseases off-label, but not all insurers will cover this kind of use.”
A drug is designated as an orphan-drug once the FDA approves the medicine for sale in the U.S., which also gives the drug seven years' marketing exclusivity and tax breaks. IN 2009, only 250 requests for orphan-drug designation were filed, and 160 received it. In response to these numbers, Timothy Coté, director of the FDA's Office of Orphan Products Development noted that industry and the FDA are “barely scratching the surface." For example, there are roughly 350 orphan drugs approved, covering about 150 rare diseases.
One reason there are so few orphan drugs are because “most companies that are developing orphan drugs are small,” according to Tim Cunniff, vice president of global regulatory affairs at Lundbeck Inc. This trend is starting to change however, as “big companies are starting to get more interested in rare diseases.”
This positive shift of industry working with FDA is important, but a “key issue is the high cost of developing a drug and the typically long time it takes to move it from a lab into a clinic as a treatment that gets prescribed.” Consequently, going into the development of creating an orphan drug requires a “to feel there is a reasonable chance of making a profit. Another positive sign to get companies to start funding research on orphan drugs was when Dr. Coté's office announced “two workshops with on-the-spot regulatory advice.”
“The first workshop, held last month at the Keck Graduate Institute, drew 29 potential sponsors, from major drug companies to academic centers, small biotechs and even some patient advocates. In a follow-up survey, 74% said they had never before filed an application for orphan drug designation.” Such a turnout is evident of the clear benefits that industry and government relationships can help patients—no matter how rare the disease is.
The workshop gave participants regulatory advice, not an alternative pathway to orphan-drug designation. In fact, teams of participants met “four times over the course of two days with FDA staffers who offered advice on nine critical issues in filling out an application.”One of the crucial pieces of information discussed was “providing evidence—preferably either trial data or published reports of animal studies—that a drug exists and holds promise for treating a rare disease.”
One example of the usefulness of the workshop was highlighted in Barbara Fant’s experience. As president and chief executive of Clinical Research Consultants Inc., she attended the workshop to prepare an application for a drug-company client. One of the FDA staffers who worked on her application noted parts that would have “delayed the designation process” to become an orphan drug however, because she attended the workshop, some of those problems were avoided.
Evidence of the success of the first two day workshop resulted in 14 submitted applications of the 29 who attended. As a result, Dr. Coté ‘s goal of doubling the number of applications this year seems well on its way, and he “considered the workshop a success.”
The second workshop will be held at the University of Minnesota in August, and can accommodate up to 50 more organizations, and “Dr. Coté said he was considering a workshop in Europe.” We will try to update you on information pertaining to this workshop.
Accordingly, his goal for the next workshop is to “weed out applicants who can't file at the end of two days.” While such an idea is admirable, just because “resources are still limited” should not be a reason to scare away interested entities. The alternative and better approach would be to find another form of funding, and since Congress is not likely to foot the bill, maybe a pool of private sources.
Although there are other factors that determine final approval for marketing—such as safety and effectiveness—Dr. Coté hoped “that by increasing the pool of applicants for designation, they will increase the chances of getting more approvals.” By opening up this workshop and opportunities for industry to make smart investments his hopes are sure to bring new research and development into orphan diseases and his efforts should be applauded.