This incredibly short news brief reported on the FDA’s expanded approval of a drug for relapsing multiple sclerosis called Gilenya (fingolimid) to treat children and adolescents 10 and older. The drug was previously approved for use in adults.
This news flash was targeted at investors and addressed the availability and novelty of this drug. Still, we think it should have gone beyond what was contained in an FDA news release to mention the high cost and potential harms of this drug along with the limited evidence about its long-term safety and effectiveness in young people.
We’ve written about how MS patients can be harmed when news media outlets take news releases about treatments at face value, without exploring the limitations of the evidence. This is a case in point.
There’s no cure for MS, an inflammatory, autoimmune disease of the central nervous system that disrupts communication between the brain and other parts of the body. It’s hoped that new drugs may be able to prevent episodes of worsening function and appearance of new symptoms, called relapses, which may lead to progressive declines in function and greater disability.
News reports should offer cautious reporting. That’s particularly true with drugs such as this one, which was approved via the FDA’s Breakthrough Therapy designation, an accelerated review process that allows approvals of drugs for serious conditions based on preliminary evidence that they might offer a substantial improvement over existing therapies.
There was no cost data. Gilenya costs $8,210 for a 30-day supply, according to the web site drugs.com.
The story stated:
In a clinical trial 86 percent of patients receiving Gilenya remained relapse-free after 24 months of treatment, compared to 46 percent of those who were administered another MS drug.
However, it didn’t explain how many relapses patients in either groups experienced, so readers can’t tell how much of an impact these drugs actually had.
No potential harms were mentioned.
The FDA said side effects of Gilenya in pediatric trial participants were “similar to those seen in adults,” with the most common being headache, liver enzyme elevation, diarrhea, cough, flu, sinusitis, back pain, abdominal pain and pain in extremities.
The FDA added there are other serious risks with the drug including slowing heart rate, infections including a rare brain infection, vision problems, swelling and narrowing of the blood vessels in the brain, respiratory problems, liver injury, increased blood pressure and skin cancer, as well as potential harm to a developing fetus.
The story didn’t give a sense of the strength of evidence behind this drug. For example, it didn’t tell readers what the comparison drug was in the trial, nor how many children were in the trial.
According to the FDA, the clinical trial evaluating the effectiveness of Gilenya in treating pediatric patients with MS included 214 patients aged 10 to 17 and compared it to the drug interferon beta-1a. Novartis said it was the first clinical trial of the drug specifically designed for children and adolescents with relapsing MS.
The story also could have mentioned that it’s unclear whether the frequency of relapses will accurately predict a patient’s degree of disability years later.
The story stated that MS is “among the most common causes of neurological disability in young adults,” but numbers would have helped.
According to the FDA, 2-5% of people with MS get symptoms before age 18 and its estimated that 8,000 to 10,000 children and adolescents in the U.S. have MS.
There were no independent sources, and the fact that Novartis funded the study to support the FDA approval isn’t mentioned.
The story mentioned that Gilenya was compared with another drug, but it doesn’t go into specifics. It also doesn’t mention that corticosteroid medications can be used to reduce inflammation.
The story mentioned that Gilenya has already been approved to treat adults with MS. However, it would have been stronger it had mentioned that the expanded indication is for ages 10 and older.
The story reported that Gilenya is the first FDA-approved drug to treat pediatric patients suffering from relapsing MS. This appears to be accurate.
The story appears to have relied solely on the news release. But it was less informative and had fewer details than the news release.