Rare Daily Staff

The Institute for Clinical and Economic Review released a report on the comparative clinical effectiveness of a set of DMD therapies in which they found that there was low value for the money despite public criticism from patient advocates at a July public meeting over the inadequacy of the criteria the organization used.

The independent non-profit research institute that analyzes the value of drugs had issued a draft report at the end of May that examined Sarepta’s eteplirsen and its experimental drug golodirsen, which is under review at the U.S. Food and Drug Administration, as well as PTC Therapeutics’ steroid deflazacort. ICER called the underlying evidence for evaluating the drugs “sparse.” Nevertheless, it said deflazacort is projected to have high costs relative to its benefits and that eteplirsen (and golodirsen if it’s priced similarly) had “no plausible treatment effects” that would make it reach cost-effectiveness below $150,000 per quality-adjusted life year.

David Rind, ICER’s chief medical officer, said that during the public hearing, parent testimony provided a window into the hope and frustration that families are feeling as new treatments become available. He said he heard about how deflazacort, an inexpensive drug that was available overseas for years and could be easily brought into the U.S. became “unaffordably expensive” with FDA approval when the U.S. manufacturer massively increased its price. With eteplirsen, he said he heard about the hopes of parents that the treatment is beneficial but called the evidence base from the manufacturer “wholly inadequate.”


“We are left where no one can know whether a useful therapy is being administered to a small number of children while others around the world are denied therapy by payer barriers and regulatory approval, or whether the patients receiving eteplirsen are being given a useless, high-priced treatment,” said Rind. “Three years after approval, the manufacturer has provided no high-quality evidence of benefit, many patients and families are surely losing out, and only the manufacturer truly benefits from this deplorable situation. Families and patients with DMD deserve better.”

The New England Comparative Effectiveness Public Advisory Council, an ICER program that convenes three times a year to review evidence reports, voted that the evidence suggests that deflazacort achieves a net health benefit over prednisone, a more affordable corticosteroid. However, the panel did not find sufficient evidence to show a net health benefit of either eteplirsen or golodirsen over supportive care alone.

During their deliberation, panel members also weighed the therapies’ other benefits and contextual considerations, highlighting that all three therapies target a condition of high severity and a high lifetime burden of illness, and that the use of deflazacort may reduce caregiver burden. The organization noted that for ultra-rare conditions like DMD, insurers and other decision-makers often give added weight to contextual considerations that lead to acceptance of prices higher than those that would meet traditional cost-effectiveness ranges.


Nevertheless, ICER said considering all of the evidence as well as the patient input on the non-clinical factors associated with these treatments, an overwhelming majority of the CEPAC voted that both deflazacort and eteplirsen represent a low long-term value for money.

The CEPAC did not vote on golodirsen’s long-term value for money because the treatment’s price is not yet known.

To reach commonly cited cost-effectiveness thresholds, ICER said deflazocort’s annual net price would need to be between $19,900-$31,700, representing a 73 percent to 83 percent discount off the current wholesale acquisition cost.

It said no price can be suggested as a fair value-based price for eteplirsen or golodirsen because no persuasive evidence yet exists to demonstrate the clinical effectiveness of either drug. At its current cost of more than $1 million per year, eteplirsen would still far exceed commonly cited cost-effectiveness thresholds even if the most favorable assumptions were granted around the potential efficacy of the treatment.

In a post on the Parent Patient Muscular Dystrophy website, the advocacy organization said thatwhile it sees some of its views impacted the underlying modeling and some of the recommendations included within the final ICER report, it remains concerned that the report fails to reflect many of the key items that it believes are foundational in Duchenne.


“We are concerned that ICER’s current framework and construct does not yet consider patient experience and input meaningfully into the underlying equation,” the organization wrote. “Patient experience and innovative qualitative and quantitative patient experience data is valid – and is more than a contextual consideration or controversy. Indeed, it is the lens through which value-based decisions are made and it is our belief that these inputs must be a more-central element of ICER’s Value Assessment Framework for Ultra-Rare Diseases.”

In an open letter to payers, the organization said slowing or halting disease progression and the impact of doing so both on patients and caregivers should be reflected. It argued that when only one approved on-label product exists within a therapeutic class, individuals with Duchenne should not have to go through the process of prior authorization or N-of-1 trials to compare toxicities of approved products with off-label therapies.

The organization also said care decisions should be made by patients and providers, and that gaps or delays in care undermine the effectiveness, and therefore the value, of treatments.

It also called step therapy policies “unethical” in conditions such as Duchenne where such requirements will cause irreversible loss of function. And, it said that therapies that provide even incremental benefit should be prioritized and considered foundational.

Following the voting session, a policy roundtable of experts – including clinicians, patient advocates, and payer representatives – convened to discuss the implications of the evidence for policy and practice. Sarepta Therapeutics and PTC Therapeutics both declined to participate.

Sarepta previously called ICER’s approach for evaluating treatments “fatally flawed.” “Through its conclusions, ICER sends a clear message to innovators that developing rare disease therapeutics is not worth the effort, and to patients – often children who are dying with no other treatment options – that their lives are not worth the investment.”

Photo: David Rind, chief medical officer of ICER

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