NCHR Testimony at FDA Meeting about Duchenne Muscular Dystrophy Clinical Trial

Thank you for the opportunity to speak today. My name is Dr. Megan Polanin. I am a Senior Fellow at the National Center for Health Research. Our research center analyzes scientific and medical data and provides objective health information to patients, providers, and policymakers. We do not accept funding from industry, so I have no conflicts of interest.

We strongly support a drug regulatory process that ensures that patients benefit from safe and effective new treatments as soon as possible. Patients with Duchenne muscular dystrophy urgently need such treatments. In order to improve quality of life and save lives, we need empirical data derived from sound science.

That is why we must conduct high quality clinical trials, comparing new treatments with a control group in a rigorously designed double-blind, placebo-controlled randomized trial. Even for devastating diseases, there is still a placebo effect, which is why a well-controlled clinical trial is so important.

We agree with the FDA’s recommendation of a study that is 18 months to 2 years length, which is essential to establish if there are significant benefits for either of these new drugs. Because this is a longer study, we believe the option of a port-a-cath may be necessary to ensure that as many patients as possible stay in the study long enough to determine the efficacy and safety of the drugs.

We know there are concerns that one third of participants will not receive a potentially beneficial treatment until after the initial 96-week period, and that some of those patients will be exposed to the risks of a central venous access device. There are two reasons why this isn’t as unfair as it seems:

#1: There is clear evidence of a substantial placebo effect for invasive procedures in medicine. For example, the nationally respected cardiologist, Dr. Rita Redberg, concluded that even sham surgeries – surgeries that are conducted as a control group in a clinical trial – have “astonishingly high” benefits for patients with heart disease and other very serious diseases – in some cases equal to the benefits of patients undergoing the “real” surgery. Her article was published in the New England Journal of Medicine. Given that port-a-cath placement would require a surgical procedure, it could have a similar benefit, even when the treatment medication is not involved.

#2: We don’t yet know whether the drugs in this study are beneficial or not. In fact, it is possible that the control group will do as well or even better than the experimental group. This is the purpose of a clinical trial: to determine what works and for whom, in order to benefit a much larger group of patients after the study is completed. Following the first 96 weeks of the study, the control group patients will have free access to SRP-4045 or SRP-4053 for the open-label 96-week extension, if they want it. This free access is a potential benefit that only patients in clinical trials can expect to have.

Given the need for safe and effective treatments for Duchenne muscular dystrophy and the methodological rigor of this study, we believe that there are circumstances in which an indwelling central venous access device should be allowed in this clinical trial. In this case, this would occur when IV utilization is no longer viable, as outlined in the review. Otherwise, we risk losing patients who have invested in the trial, which has potential benefits for themselves and others.

Patients and their families are the ultimate decision-makers regarding participation in this clinical trial. Clear and understandable informed parental consent and patient assent is critical. The implantation and maintenance of a central venous access device must be clearly spelled out so that parents, caregivers, and patients accurately understand risks. In addition, the stress and pain of failed peripheral intravenous access should be discussed with patients and families and compared to the risks of using a central venous access device. Further, the consent and assent forms must be explicit that there is a one third chance that they will not be receiving the drug for the initial 96 weeks of the study and thus experiencing risks without experiencing potential immediate benefit if the experimental treatment works. As I stated earlier, these participants will likely have the benefit of a placebo effect.

Patients with Duchenne muscular dystrophy and their loved ones deserve the benefits of the most rigorous research available. At the same time, investing in a clinical trial requires significant time and effort, and patients ALWAYS face risks without any guarantee of benefits. Patients and families who are willing to participate in a clinical trial that has the potential to benefit many should be fully informed about the potential risks and benefits of their participation in order to make a decision that is best for them.

Thank you for the opportunity to comment today and for consideration of our views.


Food and Drug Administration (2017, May 18). Background Document, Joint Meeting of the Pediatric Advisory Committee and Pediatric Ethics Subcommittee. Retrieved from

Redberg, R. F. (2014, September 4). Sham Controls in Medical Device Trials. New England Journal of Medicine, 371(10), 892-893.