Definitive proof may be lacking that the newly approved Duchenne muscular dystrophy actually works, but most neurologists expect to recommend the medicine for their patients, according to a new survey.

Specifically, 70 percent of the physicians appear fairly convinced that the drug is sufficiently effective to justify its use, and 82 percent expect their patients to start experiencing a benefit within six months, according to the survey of 101 neurologists who treat Duchenne.

The results “confirm the expectation that, given the lack of available options and the devastating nature of the disease, this agent will be widely used and quickly adopted,” wrote RBC analyst Simos Simeonidis in an investor note Tuesday in which he summarized the survey findings conducted by his firm.

advertisement

Indeed, Duchenne is a rare disease that causes steady muscle deterioration, eventually confining boys to wheelchairs and condemning them to an early death. There were no treatments available until last week when the US Food and Drug Administration approved a drug developed by Sarepta Therapeutics.

However, the approval was marred by controversy over the extent to which the drug is truly effective. The dispute enmeshed top FDA officials, but the survey underscores that some doctors feel they have little choice but to prescribe the Sarepta drug, which is called Exondys 51.

Newsletters

Sign up for our Pharmalot newsletter

Please enter a valid email address.

For instance, 21 percent of the neurologists are not convinced the drug offers a clinical benefit, but only 5 percent would not recommend it to patients. And 40 percent said they will recommend it to all or most eligible patients, while another 54 percent expect to first treat a small group of patients and then decide about others.

Granted, this is a small sample, but it provides a glimpse into physician thinking. The RBC analyst estimated that the neurologists surveyed treat about 750 Duchenne patients, which represents roughly 5 percent of the total 15,000 Duchenne patients in the United States. However, only 13 percent of Duchenne patients have the gene mutation for which the Sarepta drug was designed to mitigate.

Beyond treatment decisions, most doctors feel the cost may be an issue.

Sarepta says the “net annual cost” will be about $300,000 per patient per year, although this could vary depending upon the weight of the patient. Nearly 60 percent of the neurologists expect insurance reimbursement could limit access to the drug, despite the lack of available options. Only 2 percent of those surveyed did not expect any issues with reimbursement.

The drug labeling does not have any restrictions based on the patients’ ages or functioning levels, but there is speculation that unless Sarepta strikes risk-sharing or pay-for-performance deals with insurers, coverage may be restricted to the same patient details as in the Sarepta clinical trials.

“I do not think we will be able to get any pricing concessions from Sarepta for this drug, given the lack of competing treatments for this condition.” Michael Sherman, the chief medical officer at Harvard Pilgrim Health Care, which is the second-largest health insurer in New England, told BioCentury.

“I anticipate that we will limit the drug to the 13 percent of those (boys) with DMD with the appropriate mutation, as well as to the demographics consistent with the evidence that has been presented. Any other request would be subject to individual consideration,” said Sherman.

Despite such sentiments, Simeonidis wrote that, since “there are only about 2,000 US boys with DMD (for whom the drug was designed), we do not think this will be the disease US insurance companies will choose to fight the next pricing battle on.”

Leave a Comment

Please enter your name.
Please enter a comment.

  • my son is DMD and now he is 11 years and he is unable to walk since at the age of 9 years, now he is in wheel chair, his test reports 10 and 11 exons (duplication). Please give suggestion/treatment to my son and save his life.
    thanking you

  • really appalling that this drug costs this much for so little, if no efficacy. Id be really surprised to see insurers covering this for anything more than the trial inclusion criteria

  • While – “Simeonidis wrote that, since “there are only about 2,000 US boys with DMD (for whom the drug was designed), we do not think this will be the disease US insurance companies will choose to fight the next pricing battle on.” – I think he has missed the point – there will be ‘off-label use’ and that will join the battle. In his own words, Simeonidis said earlier in the post – “given the lack of available options and the devastating nature of the disease, this agent will be widely used and quickly adopted,”. Note those key words – followede by ‘who can put a price on hope’ – I know I am repeating myself in this forum but it bears repeating.

    • Neurologists had best learn the compassionate use procedures. When the required post marketing study fails to prove a survival benefit, as most think will be the outcome, the drug will be withdrawn from market, neurologists will have to get a compassionate use exemption, and the insurance companies will back out. That is the LESSER of the two evils. The other option is not to prescribe the drug with the certainty that you will be sued for malpractice. In that case you will probably lose your career because neurologists as a group carry less than average malpractice insurance because they don’t get sued very often. Appetizing set of choices.

Sign up for our Daily Recap newsletter

A roundup of STAT’s top stories of the day in science and medicine

Privacy Policy