![]() “Autism is a good example of that,” he says. Orphan drug status can be a double-edged sword, too, he says: Clinical trials can contribute to increased awareness and diagnosis of a rare condition, which could nullify a condition’s orphan status. In fact, recruiting participants with a rare condition can be a big challenge because a sufficiently large sample may be spread across the country, Hollander adds. Orphan drugs must go through the same process as any other drug, including multiple rounds of clinical trials. Getting a drug named an orphan doesn’t necessarily make approval easier, though, Hollander says. ![]() And orphan drugs tend to be expensive, as companies take advantage of their period of market exclusivity, causing problems for people whose insurers are reluctant to shell out for the treatments. federal government billions of dollars in tax credits, according to an investigation by Kaiser Health News. In some cases, this repurposing can go so far as carving out new subgroups of patients with the same condition the drug was initially approved for, earning orphan drug status again for the same drug - costing the U.S. The same oral ketamine formulation newly designated for Rett, for instance, has orphan drug designations for four other conditions. Many orphan drugs, including ketamine, are already FDA-approved to treat other conditions - or are at least known entities, in the case of psilocybin. “To make that kind of investment, you want to be able to recoup those costs on the other side.” “There might not be an incentive from a financial standpoint to do it otherwise,” says Eric Hollander, professor of psychiatry and behavioral sciences at Albert Einstein College of Medicine in New York City. The Orphan Drug Act also grants drugmakers seven years of market exclusivity. “I’m sure it isn’t the only reason that companies have been willing to step into that space, but it offers some offset to the risk,” says Jeremy Veenstra-VanderWeele, professor of developmental neuropsychiatry at Columbia University. Once the compound qualifies, its maker benefits from tax credits for clinical trials and exemptions from certain FDA fees - discounts that make its development a bit less of a gamble. To qualify as an orphan drug, a compound must target a condition that affects fewer than 200,000 people. ![]() ![]() This status, created by the Orphan Drug Act, aims to foster drug development for rare conditions that the pharmaceutical industry has “orphaned” because of a lack of financial incentive. Food and Drug Administration (FDA) granted orphan drug designation to oral ketamine for Rett syndrome this month, as well as psilocybin and the experimental drug blarcamesine, both for fragile X, in November. And don’t forget to subscribe to receive Going on Trial in your inbox every month. This newsletter is still pretty new, and I want it to reflect the needs of the research community, so please email me at with your ideas on how to improve it. Thank you to everyone who read our first edition and subscribed to the newsletter, especially those of you who sent me tips and feedback. This month’s issue highlights the federal Orphan Drug Act in the United States, which turned 40 in January, and new orphans for Rett and fragile X syndromes. Welcome to the second edition of Going on Trial, a monthly newsletter that rounds up the latest in clinical trials and drug development for autism and related conditions. ![]()
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