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FDA release offers inadequate presentation of auto-inflammatory drug approval

FDA approves expanded indications for Ilaris for three rare diseases

Our Review Summary

fdaThis news release issued by the U.S. FDA extends indications for a drug called Ilaris (canakinumab). The new indications are Tumor Necrosis Factor Receptor Associated Periodic Syndrome (TRAPS), Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD) and Familial Mediterranean Fever (FMF).

TRAPS and HIDS/MKD are both rare but serious autoimmune diseases for which there are currently no approved therapies. Unfortunately, lacking any information about the quality of the evidence, cost or benefits, this release is notably lacking in key details that any person suffering from these conditions would need to make an informed decision about the treatment.


Why This Matters

If there are additional useful indications for Ilaris (canakinumab), in this case to treat Tumor Necrosis Factor Receptor Associated Periodic Syndrome (TRAPS) and Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD), then this could be a good thing for patients suffering from these conditions. Unfortunately, we don’t learn from this news release what is currently used to treat patients suffering these conditions, or anything else about these rare-sounding conditions. It is impossible to assess the impact of the treatment without more details of the prevalence of the diseases in question.


Does the news release adequately discuss the costs of the intervention?

Not Applicable

FDA releases don’t typically assess cost, but seeing as this treatment is already on the market, it would not have been much of an effort to include that information in the news release. One might assume that the costs of treating these rare diseases are substantial but the release gives no indication on cost, or whether the drug might be covered by insurance.

According to one trusted source on average retail drug prices, llaris prescriptions cannot be filled at a typical pharmacy. Instead, the medicine is administered and distributed at a hospital or clinic. This suggests the drug may be very expensive.

Does the news release adequately quantify the benefits of the treatment/test/product/procedure?

Not Satisfactory

All we learn about the benefits for patients is that the treatment “may help improve their quality of life.”  It would be hard to give less information about the benefits of the treatment. The statement that “approvals for the new indications were based on clinical studies, including safety, efficacy and pharmacokinetic data” is inadequate.

Does the news release adequately explain/quantify the harms of the intervention?


The release includes a discussion of harms warranting a borderline satisfactory rating. But the side effects information is similar to that found on product labeling — it follows the regulatory requirements concerning product labels — but provides no data on harms from the trials behind the drug’s approval. It could have been more informative with a few more details.  In addition, the information on harms appeared to be contradictory. One paragraph says common responses to the drug were minor reactions and susceptibility to colds; the next paragraph lists serious side effects.

Does the news release seem to grasp the quality of the evidence?

Not Satisfactory

We would have welcomed the faintest of details of the clinical studies, and the “safety, efficacy and pharmacokinetic data” used to approve Ilaris for these new indications.  This was a lost opportunity to inform.

Does the news release commit disease-mongering?


The release doesn’t engage in fear-mongering, but neither does it tell us how prevalent and disabling these diseases are. It would have been nice to get more information about the new indications, the numbers who suffer from them and the way they may be currently treated even if there are no approved drugs.

Does the news release identify funding sources & disclose conflicts of interest?

Not Applicable

There’s no mention of funding or conflict of interest, but these don’t (or shouldn’t) apply to regulatory approvals. It’s presumed that the studies will have been conducted by the company seeking approval.

Does the news release compare the new approach with existing alternatives?

Not Satisfactory

Even if there are no approved drugs for these indications, surely there is some sort of therapy that patients suffering from them are prescribed, even if it’s limited to palliative care. To leave out details on how patients with these rare disorders are currently treated does a disservice to the readers.

Does the news release establish the availability of the treatment/test/product/procedure?


Since the drug has already been approved for two other conditions, it’s safe to assume that the drug is already on the market and generally available.

Does the news release establish the true novelty of the approach?

Not Satisfactory

The release claims novelty with the following statement: “For the first time, patients with TRAPS and HIDS/MKD, two painful and life altering diseases, have access to a treatment that may help improve their quality of life,” according to an FDA official.

But is novelty demonstrated? All we learn is there are “new” indications for the drug, two of which have no previously approved treatments. This is presented as a “take our word for it” kind of way which seems unacceptable.

Does the news release include unjustifiable, sensational language, including in the quotes of researchers?


The release doesn’t engage in unjustifiable or sensational language.

Total Score: 4 of 8 Satisfactory


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