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

Rating

3 Star

MT Pharma America Announces FDA Approval of RADICAVA™ (Edaravone), the First New Treatment Option for ALS in More Than 20 Years

Our Review Summary

If this release from MT Pharma America on its new amyotrophic laterals sclerosis (ALS) drug had provided a clear enumeration of the benefits of the drug, cost information, and some explanation of any conflicts of interest in the physician being quoted, it would be a solid release. In many ways, this release provides good information for reporters or even the general public. It follows the same basic tenets of most television pharmaceutical ads in that it hews to the evidence used to clear FDA hurdles and provides an ample list of side effects.

 

Why This Matters

ALS is a progressive neurological disorder with limited treatment options, none of which cure the disease. It belongs within a wider group of conditions known as motor neuron diseases that are caused by gradual deterioration and death of motor neurons, or nerve cells. The Centers for Disease Control and Prevention (CDC) estimated that between 14,000 and 15,000 Americans had ALS in 2016.  “Currently, there is no cure for ALS and no effective treatment to halt, or reverse, the progression of the disease, ” according to the National Institute of Neurological Disorders and Stroke (NINDS). The existing drug, Rilutek, has shown in clinical trials that it can extend life by a few months, and the newly approved drug described here, Radicava, was shown to slow decline in daily functioning. With so few treatment options available, the new drug is being welcomed as another option among very limited offerings.

Criteria

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 adequately explain/quantify the harms of the intervention?

Satisfactory

The release does provide a rather long list of potential harms. And then it goes on to say, “These are not all the possible side effects of Radicava. Call your healthcare provider for medical advice about side effects. You may report side effects to MT Pharma America, Inc. at 1-888-292-0058 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.”

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

Satisfactory

We’re giving it a pass here because it explains quite a bit about the study. It would have been better had it also explained whether the findings had been published in a peer-reviewed journal or undergone any other outside vetting.

Does the news release commit disease-mongering?

Satisfactory

There is no disease mongering in the release. It also provides some context about the prevalence of ALS.

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.”

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

Satisfactory

The release makes it clear that the drug is only now approved for prescription in the U.S. after having been on the market in Japan and South Korea. It says, “Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and will be commercialized in the U.S. by MT Pharma America. MTPC group companies began researching ALS in 2001 through a comprehensive clinical platform over a 13-year period. In 2015, edaravone was approved for use as a treatment for ALS in Japan and South Korea.”

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

Satisfactory

The release establishes the novelty of the findings in that this is the first new drug for treating ALS in more than 20 years.

Total Score: 6 of 10 Satisfactory

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