NCHR Testimony at the FDA Panel on Ataluren

Thank you for the opportunity to speak today. I’m Jack Mitchell, director of health policy at the National Center for Health Research. We analyze medical data and provide objective health information to patients, providers, and policymakers. NCHR does not accept funding from drug companies, so I have no conflicts of interest to report.

I’d like to acknowledge the many patients, children and their families who have traveled long distances to share their view with the panel. Patients with rare diseases urgently need safe and effective treatments. That means we need persuasive data based on soundly reviewed science.

We agree with the FDA that substantial evidence of effectiveness must be provided to support approval of a new drug. When the FDA has been flexible in approval criteria for treatments for devastating rare diseases, that has sometimes backfired. In some cases, it has resulted in insurance companies refusing to pay for FDA-approved treatments that the insurance companies deem experimental rather than proven.

This disconnect adversely affects patients who would otherwise have free access to the drugs in clinical trials because the trials are stopped or limited. Patients and their families cannot afford to pay for treatments that insurance companies maintain are not proven to work.

We agree with FDA scientists that the data today do not indicate significant benefit in randomized, double-blind, placebo-controlled trials, such as Study 7. Only after making many post-hoc changes did ataluren show it was effective for ADP patients, but this was not replicated in Study 20. As you know, those post-hoc manipulations do not provide clear evidence of efficacy.

For both studies, 90% and 79% of patients were White. But the CDC reports that Hispanic males are disproportionately likely to have these conditions. It is essential that an adequate number of Hispanic males be analyzed to determine if they can benefit from a treatment such as ataluren.

Finally, we have concerns regarding safety. Elevated blood lipids and blood pressure are not benign side effects, especially in children. Those risks are substantially increased in children taking chronic corticosteroids. In addition, the effects of ataluren on kidney function blood tests are also a matter of concern, especially in children taking many other drugs that could be harmful to the kidneys.

We agree with the FDA scientists that no study conducted as planned had sufficiently positive results. A possible signal of treatment effect for patients deserves further study, but current data are not sufficient to warrant FDA approval.

Patients and their loved ones deserve the benefits of the most rigorous research. We urge this Advisory Committee to advocate for scientific rigor and vote that the data suggest that ataluren has not been proven sufficiently effective.

I respectfully recognize that the families here today so not shatter that viewpoint. Their stories are both moving and meaningful. Among other things, we’re a patient advocacy organization, so
this is not an easy position to take. But we believe further study of the drug is needed.

Thank you for the opportunity to share our perspective.

Ten out of eleven members of the Peripheral and Central Nervous System Drugs Advisory Committee voted that the data for ataluren were ineffective and that more work will be needed to establish whether the drug is effective. One member voted that the data were sufficient to conclude that ataluren was effective.