ALS drug announcement answers many questions but raises others about cost, benefits and a potential conflict of interest

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

Not Satisfactory

It’s almost as if a sentence is missing from the release. It does not say that the drug is expensive or inexpensive, but one must presume that it is expensive because it goes into some detail about a program set up to help people afford the drug. It says, “MTPharma America is helping to make RADICAVA accessible to all patients who need it and has created Searchlight Support, a patient access program for people with ALS who are prescribed RADICAVA. As soon as a person receives a prescription, he or she can access Searchlight Support, including personal case management, reimbursement support and 24/7 clinical support. Some people with ALS who have received prescriptions for RADICAVA may be eligible to receive additional assistance from MTPharma America.”

According to the ALS Association, “The drug’s list price is $1K per infusion, $11K per dosage cycle, which amounts to $146K per year. This does not take into account coverage from insurance or Medicare/Medicaid.”

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

Not Satisfactory

The story does put some numbers to the benefits, but they are in relative terms and not easy to understand.

“The pivotal Phase 3 study (MCI186-19), which evaluated 137 people with ALS, formed the basis for the FDA approval of RADICAVA. Data demonstrated patients who received RADICAVA for six months experienced significantly less decline in physical function —by 33 percent or 2.49 ALSFRS-R points (p=0.0013).”

Although the release rightly states that the intervention improved patient outcomes by 2.49 ALSFRS-R points, there is no discussion about what this means in terms of actual patient function. A change of two and a half points might just equate to a patient not developing worse handwriting and excess saliva. These changes may be important but are minimal with respect to the degree of disability the disease causes. A statement that no change in mortality or independent living has been seen would also have been warranted.

We would also liked to have seen a clear numerical comparison. How many of the 137 received Radicava for six months? How many of those patients saw a significant preservation of physical function? How many saw no benefit?

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

Not Satisfactory

The release is a corporate release. And it’s clear that the drug company that makes the drug in question is hoping to sell the drug. There are two things that are unclear, though. First, the relationship of the only physician quoted in the article to the drug or the company. Second, the relationship of Barbara Newhouse, president and CEO, ALS Association, to the company. Both basically give the findings a lot of fanfare. And Newhouse in particular provides a very positive take by talking about the potential impact on patients, saying.“We applaud the work MT Pharma America and the FDA are doing as they have taken unprecedented steps to get this treatment into the hands of patients as quickly as possible.” Drug companies often sponsor patient support organizations such as the ALS Association. For example, in 2016, the ALS Association announced MT Pharma as a “corporate sponsor” of its walk to raise money for disease research.

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

Not Satisfactory

There is no comparison to alternatives in the release. As Jeff Akst at The Scientist pointed out there, is one other drug on the market, “The drug, manufactured by MT Pharma America, the US subsidiary of the Japanese company Mitsubishi Tanabe Pharma Corporation, is only the second approved ALS treatment in the U.S. The first, riluzole (Rilutek), was approved in December 1995.”