A recently published review paper based on 15 real-world evidence studies indicates that riluzole extends survival in ALS patients beyond what was previously shown in the drug’s clinical trials.
Riluzole was approved by the U.S. Food and Drug Administration (FDA) in 1995 for use in patients with ALS. To date, it is the only FDA-approved therapy that has been shown through clinical trials to prolong survival in people with ALS. How riluzole works in treating ALS is still unknown.
Importantly, researchers recently suggested that riluzole may be even more helpful for ALS patients than previously recognized.
Real-world Evidence of Riluzole’s Effectiveness
In a new medical paper, the authors discuss how the gold standard approach for evaluating how well drugs work against certain diseases – termed randomized clinical trials, or RCTs –don’t always paint the entire picture about a drug’s benefit. RCTs are designed to be deliberately stringent so that we can determine with some degree of certainty how well a drug works on a specific subset of patients. While this approach is robust from a scientific perspective, it is limited in terms of its ability to produce results that are generalizable to all patients with the relevant condition.
The RCTs that led to the approval of riluzole in ALS patients showed that the drug was associated with longer survival times and more time until a tube needed to be placed in patients’ windpipes through a procedure known as tracheostomy. While these results are positive and point to some of the value of riluzole, the studies may have shown even more promising results had they included patients who were not as sick. To address this issue, the authors of the new paper, titled Real-world evidence of riluzole effectiveness in treating amyotrophic lateral sclerosis, conducted a comprehensive review of other types of studies – known as population studies – on the effectiveness of riluzole in ALS.
Median Survival and Riluzole: Longer Than Previously Thought?
The authors identified 15 studies. To be included in this analysis, each study had to have two groups of patients. One group was treated with riluzole and the other group did not take riluzole. Most of the studies showed a 6 to 19 month increase in median survival with riluzole treatment. This survival time is longer than the 2 to 3 months observed in the RCTs conducted on patients who had long-standing disease and may have been resistant to treatment. The authors also point to research that suggests that riluzole may work in different ways in people with early stages of ALS versus those whose disease is more progressed. The evidence, they say, points to benefits for both early intervention with this drug as well as prolonged therapy.
Based on what is known so far about riluzole in ALS and the questions that the current data highlight, there is now ongoing research to help clarify what specific benefits riluzole therapy may have for the types of patients who are not represented in the RCT data – namely, those who are in earlier stages of the disease. More data on the effects that the drug has on subpopulations of ALS patients and how the drug may work differently in these different subpopulations will equip clinicians with better information on how to customize treatment.
Taking the Full Dose of Riluzole for the Drug’s Full Benefit
As with any medication, it is imperative that patients are able to take the full, prescribed dose of riluzole in order to get the full benefit of the drug. At some point, many people living with ALS experience swallowing problems, also known as dysphagia (pronounced “dis-FAY-ja”). As swallowing problems develop in ALS patients, doctors typically recommend crushing riluzole tablets and mixing them with foods, such as yogurt or applesauce.
While this has been the longstanding approach to administering the medicine for those with swallowing difficulties, choosing to crush riluzole tablets may prevent ALS patients from getting the full effect of the medicine, since the full portion of the dose might not fully ingested. A suspension form of riluzole is available to meet the needs of people with ALS who have difficulty swallowing or have a feeding tube. When ALS patients take the full dose of riluzole every day, they have the best chance of maximizing the drug’s full benefit.
Click here to get information on how your doctor can prescribe a suspension form of riluzole today.
Andrews, JA et al. (2020). Real-world evidence of riluzole effectiveness in treatment amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. doi.org/10.1080/21678421.2020.1771734
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