ICER Faces New Foe As Patient, Disability Alliance Takes Aims At Reports On Mayzent, Spravato
The Pink Sheet
June 22, 2019 Saturday 4:25 PM GMT
A recently-launched initiative supported by organizations for patients and the disabled called Value Our Health is challenging the value assessments conducted by the Institute for Clinical and Economic Review, starting with ICER's reports on Novartis AG's Mayzent for multiple sclerosis and Janssen Pharmaceutical Cos.'s Spravato for treatment-resistant depression.
Value Our Health comprises the Partnership to Improve Patient Care and 37 other advocacy organizations, many of which have relationships with biopharma manufacturers. PIPC members include patient and physician organizations as well as the Pharmaceutical Research and Manufacturers of America and the Biotechnology Innovation Organization.
The initiative responds to ICER's growing influence among payers in the US, VOH organizers said during a 21 June call with reporters.
One example of that influence is CVS Health Corp.'s announcement in August 2018 that it would rely on ICER assessments to threaten non-coverage for any new drug whose price exceeds a certain cost effectiveness threshold, with the aim of lowering list prices.
Value Our Health takes issue with ICER's use of quality-adjusted life years (QALY) as a metric for evaluating cost effectiveness, a practice manufacturers have long criticized as a one-size fits all approach that overlooks the needs of certain patients.
"It is disappointing that ICER continues to reference the QALY as the 'gold standard' despite that it distorts and misrepresents how patientsvalue their own lives, and it can lead to insurers and the government to deny care to people who would benefit from it," PIPC executive director Sara van Geertruyden said.
"Value determinations based on QALYs and similar average metrics often fail to acknowledge side effects and differences in effectiveness for populations that are far from average," added PIPC disability advocate Ari Ne'eman.
"If you are evaluating efficacy of two drugs for same condition, MS or depression, you shouldn't use QALY as ICER does, you should use measures specifically developed and normed to those certain conditions which would have a much better ability to capture nuance in terms of improvements," Ne'eman argued.
The organization did not cite a specific method of evaluating cost effectiveness that could replace QALY and acknowledged that none have gained the support to do so in the US so far.
"There are other ways of conducting value assessment, it's just they just really don't have the investment that ICER has," Geertruyden noted. "The only reason you don't hear about these ... alternatives is they don't receive the same amount of attention and investment."
ICER received nearly $14m for 2017-2020 from the Laura and John Arnold Foundation to fund its work. That followed a $5m grant from 2015-2017. The organization also receives funding from other sources.
ICER Value Framework Update Underway For 2020
ICER responded to criticism about how its focus on QALY benchmarks could discriminate against vulnerable patient groups or those with disabilities by introducing a complementary metric known as "equal value of life years gained" at the end of 2018.
The metric is "not as flexible as the QALY in capturing benefits to quality of life but does measure any gains in length of life exactly the same across all conditions, regardless of age, severity of illness, or level of disability," the group explained.
ICER is also in the process of a planned 2020 update to the methods used in its value framework and has solicited public comments to inform the effort. Draft revisions will be released 16 August for further comment.
"We recognize we won't fully satisfy all stakeholders, particularly those who would prefer a status quo where manufacturers are able to charge any price they'd like for a new drug in this country," an ICER spokesperson said. "But we're pleased that ICER continues to play an important role in convening public discussions in the US on how best to align a drug's price with its benefits for patients."
Mayzent Report Questions Cost Effectiveness
In the 20 June final report on Mayzent (siponimod), ICER concludes the treatment "does not have a unique role in therapy for any phenotype of MS" and "given its similarities to fingolimod [Novartis' Gilenya], siponimod should be considered among a group of highly effective disease modifying therapies for relapsing forms of MS."
The report also noted that "payers may wish to specifically consider granting preferential formulary status to fingolimod when its generic formulation comes to market."
Mayzent launched at a list price of $88,561 per year, which "exceeds commonly accepted thresholds for cost effectiveness of $50,000-$150,000 per QALY gained, when compared to best supportive care in patients with SPMS [secondary progressive multiple sclerosis]," ICER said.
The analysis focused only on patients with secondary progressive MS, even though Mayzent is approved for the broader indication of relapsing MS. ICER chose to focus on patients with SPMS because that was the population studied in the Phase III trial on the drug, it said.
Novartis took issue with ICER's approach in a statement. "ICER established the cost-effectiveness of Mayzent based on a comparison to no treatment (best supportive care) while, in practice, most SPMS patients are today treated with drugs that have not been proven in a trial prospectively powered to demonstrate efficacy in a SPMS population," the company said.
In addition, "Mayzent demonstrated a significant effect in delaying disability progression in a representative SPMS population," Novartis maintained. "The EXPAND trial was not powered to demonstrate significant differences between groups in the non-active SPMS sub-population."
ICER Suggests Up To 52% Discount On Spravato
ICER's final report on Spravato (esketamine), also released on 20 June, concludes the drug's $32,400 annual list price would require a 25%-52% discount to reach a "fair" value-priced benchmark.
Because the drug's potential patient population is so large, Spravato at $32,400 would have a significant impact on US health care spending, ICER added, triggering an "access and affordability alert" among payers.
"Potentially only 16% of eligible Americans with TRD [treatment resistant depression] could be treated with esketamine per year before crossing ICER's potential budget threshold of $819m," the report points out.
Janssen disagreed with the report, maintaining it "underestimates the proven short- and long-term benefits that this treatment, which was granted FDA breakthrough therapy designation, brings to TRD patients in need," according to a statement.
Pink Sheet: ICER Faces New Foe As Patient, Disability Alliance Takes Aims At Reports On Mayzent, Spravato
An article in The Pink Sheet highlighted the Partnership to Improve Patient Care (PIPC)'s involvement with Value Our Health -- a new initiative supported by organizations representations patients and people with disabilities. The article highlights Value Our Health's opposition to the Institute for Clinical and Economic Review's (ICER) use of the quality-adjusted-life-years (QALY) metric, nothing that the patients groups have long criticized the method as a one-size-fits-all approach to addressing the needs of patients. "It is disappointing that ICER continues to reference the QALY as the 'gold standard' despite that it distorts and misrepresents how patients value their own lives, and it can lead to insurers and the government to deny care to people who would benefit from it," said PIPC executive director Sara van Geertruyden. "There are other ways of conducting value assessment, it's just they just really don't have the investment that ICER has."
The article in its entirety can be read below.