‘Serious Concerns’ Raised by Watchdog Group on Cost-effectiveness of DMD Therapies

‘Serious Concerns’ Raised by Watchdog Group on Cost-effectiveness of DMD Therapies

The Institute for Clinical and Economic Review (ICER) issued a draft report raising serious concerns about the cost-effectiveness of Sarepta‘s exon-skipping therapies Exondys 51 (eteplirsen), and the investigational golodirsen, as well as the corticosteroid Emflaza (deflazacort; by PTC Therapeutics), for treating Duchenne muscular dystrophy (DMD).

ICER’s worries about these treatments come mostly from the limited evidence backing their efficacy or safety, and their current prices, which are considered too high for the potential health benefits they offer.

Public comments to this draft report are open until June 18. All comments will be taken into account to eventually revise the evaluation, and will be incorporated in the final report, scheduled for July 11, the institute announced in a press release. That “evidence report” will be subject to deliberation during a public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), one of ICER’s three appraisal committees, on July 25.

ICER is a U.S. independent nonprofit organization, best known as the nation’s independent watchdog on drug pricing. The agency provides comprehensive clinical and cost-effectiveness analyses of health treatments, tests, and procedures, and is funded by non-profit foundations, drug makers, insurers, and government grants.

Before issuing the draft report, ICER incorporated the input it received from patients, clinicians, and other stakeholders.

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ICER evaluated Emflaza’s effectiveness based on the results of available clinical trials, involving a small number of patients, that compared Emflaza to prednisone, another commonly used corticosteroid.

“These data as well as additional observational [non-interventional] studies suggest that there may be some greater benefits on motor function with deflazacort, although not all data are consistent and the size of the benefits may be small,” ICER reported.

Most data suggest less patient weight gain with Emflaza, but also reduced growth. Additionally, Emflaza has not been shown to improve pulmonary function outcomes compared with prednisone.

Another concern for ICER was the lack of sufficient evidence to say with confidence that Emflaza is safer than prednisone, especially in terms of behavioral and psychiatric side effects. This is an important aspect, as a main reason for interest in Emflaza was the potentially fewer risks of the treatment.

“Overall, given the evidence on motor function and weight, we have moderate certainty that deflazacort [Emflaza] has comparable or better net health benefits compared to prednisone,” ICER said.

Concerning the two Sarepta exon-skipping therapies — Exondys 51 (eteplirsen) and golodirsen — ICER classified the available evidence on their clinical effectiveness as “insufficient.”

Clinical data for both therapies is very limited, and studies of dystrophin levels show increases that are of “uncertain clinical or biologic importance,” ICER said. Although Exondys 51 has not demonstrated any safety concerns, treated patients have thus far received follow-up only for short periods, which makes it difficult to evaluate long-term risks. For golodirsen, still an investigational treatment, functional outcomes and safety data have not yet been reported.

“While there are important limitations to consider, … the magnitudes of the treatment costs relative to the potential health effects projected for DMD suggest serious concerns regarding the cost-effectiveness of these treatments at current prices,” ICER said.

Emflaza’s incremental cost-effectiveness ratio was projected to stay above $500,000 per quality-adjusted life-year.

For Exondys 51, ICER says it “would not be cost-effective,” given its willingness-to-pay threshold of $150,000 per quality-adjusted life-year, even when assuming the treatment will have extremely favorable efficacy.

Quality-adjusted life-year (QALY) is a measure of disease burden used in health economics, which corresponds to one year in perfect health.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.
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Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.
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7 comments

  1. Ralph Yaniz says:

    I believe there may have been a response from Sarepta about this. It might be useful to cover that and what the response might mean.

  2. Arctic Parrot says:

    It is disappointing that a drug such as “emflaza” (deflazacort) is being marketed for 86,000 per year. My two sons receive this same drug in Canada and the cost is $300 each per year – which we have to pay full cost for as it is not insurable. The drug is made by Sanofi in Switzerland – so this is not some counterfeit knockoff. What ICER should be criticizing are policies that allow drugs to be priced in this way and not the efficacy of the drugs based on outrageous pricing.
    As an aside, one of my sons had to briefly switch to the equivalent dose of prednisone from deflazacort. The change to his mental health was immediate, he said he would never use prednisone again as it made him feel so “weird”.

  3. Jessica Santiago says:

    I have notice weight gain and my son not growing in height, I constantly tell this to his doctor and all I get is ” this is how it is” nothing can be done.

  4. M v A says:

    Agreed Americans are being taken advantage of by drug companies. Open up a free market. Emflaza has been around for a long time at a cheaper price under generic drug makers. It is shameful that Americans cannot reward reliable generic drug makers. $300 CND is much more reasonable than $86000 US. Whose pockets are being lined? Make this an election issue.

  5. Madhuri Chatterjee says:

    In India the price of Deflazacort is about 17000 Indian rupees per year. But the patients have to pay from their own pocket . This is often difficult for parents from economically deprived population. Yet this has benefits as we do not have any medicine to stop the progression of disease. But each country should try to reduce the prices of drugs that are used in rarer diseases

  6. Harold Potts says:

    I think someone has to address whether these DMD treatments are just placebos or not. Eteplirsen was approved by the FDA in 2016 and ataluren was approved by the CHMP in 2014, and no one is yet sure whether either of these incredibly expensive drugs are effective. Confirmatory trials were ordered, but there doesn’t seem to have been any information since approval. Surely, by now, there must be enough evidence to prove/disprove efficacy?

  7. Fred says:

    The American method of doing medicine makes me physically ill, so much so that when I hear this kind of stuff I want to go over to their houses and puke in their shoes. There is a mountain of evidence here in Canada generated by (among others) Dr Douglas Biggar. He is the MD that looked after my son, who at 25 and on Deflazacort for 19 years, is still able to feed himself and raise his hand above his head (with the use of long hair that is). Deflazacort costs us about $450 per year and that is covered 100% by my wife’s drug plan from work. If you live in the US and have a child with DMD, move, anywhere else will do as there are plenty of good English speaking countries who take care of their injured instead of turning a profit on them. In the long run, it will be much cheaper.

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