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Note: This story were updated Feb. 14, 2023, to correct that ICER analyses found both Radicava ORS and Relyvrio are not cost-effective for the treatment of ALS.
The recently approved therapies <a class="c-link" tabindex="-1" href="https://alsnewstoday.com/amx0035/" target="_blank" rel="noopener noreferrer" data-stringify-link="https://alsnewstoday.com/amx0035/" data-sk="tooltip_parent" data-remove-tab-index="true">Relyvrio</a> (sodium phenylbutyrate and taurursodiol) and <a class="c-link" tabindex="-1" href="https://alsnewstoday.com/radicava-edaravone-for-als/" target="_blank" rel="noopener noreferrer" data-stringify-link="https://alsnewstoday.com/radicava-edaravone-for-als/" data-sk="tooltip_parent" data-remove-tab-index="true">Radicava ORS</a> (edaravone) are not likely to be cost-effective for treating <a class="c-link" tabindex="-1" href="https://alsnewstoday.com/what-is-als/" target="_blank" rel="noopener noreferrer" data-stringify-link="https://alsnewstoday.com/what-is-als/" data-sk="tooltip_parent" data-remove-tab-index="true">amyotrophic lateral sclerosis</a> (ALS), according to a new analysis from the Institute for Clinical and Economic Review (ICER).
The report, &#8220;<a href="https://www.jmcp.org/doi/10.18553/jmcp.2023.29.2.216">The effectiveness and value of AMX0035 and oral edaravone for amyotrophic lateral sclerosis: A summary from the Institute for Clinical and Economic Review’s Midwest Comparative Effectiveness Public Advisory Council</a>,&#8221; was published in the <a href="https://www.jmcp.org/pb-assets/logos/JMCP_RGB_masthead.png">Journal of Managed Care and Specialty Pharmacy</a>.
Relyvrio and Radicava ORS were approved by the U.S. Food and Drug Administration (FDA) last year. Relyvrio contains two compounds — sodium phenylbutyrate and taurursodiol — thought to protect nerve cells from stress. Radicava ORS is a liquid suspension formulation of Radicava, which has been FDA-approved for ALS since 2017. It can be taken orally or through a feeding tube, whereas the original formulation was administered via infusion into the bloodstream.
&#8220;The oral formulation presents a lower burden for patients and eliminates the risks of intravenous administration, thus most patients are expected to switch to the oral formulation, and more patients overall are expected to begin use of edaravone now that an oral formulation is available,&#8221; the researchers wrote.
ICER is an independent nonprofit that provides evidence-based evaluations of the value of healthcare interventions. It analyzed the value of these two therapies using economic models that accounted for their cost as well as the expected benefit to patients&#8217; quality of life.
Results showed Radicava ORS provided an added 0.04 quality-adjusted life years (QALYs) and 0.05 equal value life years gained (evLYG) compared with standard of care alone. These are two measures that assess the quality and quantity of years gained with a given therapy. However, Radicava ORS was deemed not cost-effective at its list price of about $171,000 a year.
The list price for Relyvrio was not available at the time of the analysis so a placeholder price of $169,000 a year was used. Although patients gained 0.14 QALYs and 0.31 evLYGs when Relyvrio was added to standard of care therapies, results suggested the therapy also was not cost-effective at this price. The medicine’s actual list price is slightly lower, <a class="c-link" tabindex="-1" href="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/" target="_blank" rel="noopener noreferrer" data-stringify-link="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/" data-sk="tooltip_parent" data-remove-tab-index="true">at $158,000 a year</a>.
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<h3>Determining Radicava ORS, Relyvrio&#8217;s cost-effectiveness</h3>
The medicines’ cost-effectiveness analyses were based on data from clinical trials, in which both therapies slowed disease progression. However, the researchers emphasized that available clinical trial data is limited, restraining their analysis.
Relyvrio was approved based on findings from one Phase 2 clinical trial called <a href="https://clinicaltrials.gov/ct2/show/NCT03127514">CENTAUR (NCT03127514)</a> and its extension study, which tested it in 137 ALS patients with rapidly progressing disease. Results showed it <a href="https://alsnewstoday.com/news/amx0035-survival-als-patients-centaur-clinical-trial-analysis-amylyx/">slowed disease progression and appeared to extend survival</a>.
However, the researchers noted concerns about blinding — exit interviews showed most patients given a placebo could correctly guess that they weren&#8217;t getting the active treatment, likely due to the therapy&#8217;s taste and common digestive side effects — and sensitivity analyses by the FDA &#8220;demonstrated lower efficacy and less statistical persuasiveness on the outcomes of slowing disease progression and survival.&#8221;
An ongoing Phase 3 trial called <a href="https://clinicaltrials.gov/ct2/show/NCT05021536">PHOENIX (NCT05021536)</a> is expected to provide more detailed information about Relyvrio&#8217;s clinical profile.
Radicava ORS was approved based on pharmacological data showing it was biologically equivalent to the intravenous version. The FDA&#8217;s approval of the original therapy was supported by data from a small trial, called <a href="https://clinicaltrials.gov/ct2/show/NCT01492686">Study 19 (NCT01492686)</a>, that tested intravenous Radicava in 137 ALS patients with slow disease progression who were in its early stages.
&#8220;Despite its broad FDA label for treatment of all patients with ALS, there are generalizability and replicability concerns regarding the evidence given that the clinical benefit of [Radicava] was demonstrated in a single small trial of a narrow, well-defined population in Japan with ALS in the early stage that only represents 10% or less of the entire population of people living with ALS,&#8221; the researchers wrote.
An ICER advisory committee voted last year to affirm that the available evidence supports a net health benefit to adding Relyvrio to standard of care for ALS. It also voted to affirm that efficacy data for Radicava are sufficient for the specific subset of patients tested in trials, but not for all ALS patients.
The panel also affirmed that Relyvrio and Radicava ORS provided low long-term cost-effectiveness at the current list prices.
It also offered policy recommendations to improve care for the ALS community. Among them, the panel urged insurance companies to develop coverage policies for potential ALS therapies still undergoing review so patients can access them as soon as they&#8217;re approved. It also called on payers to cover home health and assistive devices.
The panel also recommended that therapy developers incorporate uncertainty about effectiveness into pricing decisions when medicines are launched and suggested that the FDA establish a process for conditional approval when evidence of efficacy is unclear.
This study was funded by Blue Cross Blue Shield of Massachusetts, California Healthcare Foundation, The Patrick and Catherine Weldon Donaghue Medical Research Foundation, Arnold Ventures, and Kaiser Foundation Health Plan.

Tag: cost-effectiveness

ICER releases analysis on cost of ALS therapies