This started as a blog about the second publication on PPMD’s benefit/risk study, which was published in late December in a journal called The Patient (and more on that later). But something remarkable happened this week that helps frame the importance of this work. Here’s that story:

You might recall that there are two major divisions of the FDA. As a simplification, one division is focused on drugs and one is focused on devices. The device group is called the Center for Devices and Radiologic Health (CDRH). The CDRH showed great initiative several years ago in collaborating with researchers to understand the preferences of individuals with obesity about devices to help manage their weight. Similar to what we found in the Duchenne community, individuals with obesity showed a willingness to accept risk and uncertainty for a device that led to improvement.

Fast-forward to this week, when the FDA decided on a submission for an obesity device. In the pivotal clinical trial the device failed to meet the study endpoints; while there was suggestion of benefit, the trial results did not meet the FDA’s most basic standards for demonstrating benefit. And yet the FDA approved the device, based in part on the results of the preferences study that showed that patients would accept uncertainty about benefits. As quoted in the Pink Sheet, an FDA spokeswoman said that the FDA believes patients and caregivers who live with a condition should have a say when approving a product, and may have a different view about striking the right risk-benefit balance. She went on to say that,

“It may be appropriate to approve a device for a specific intended population if adequate evidence indicates that a subset of well-informed patients with that illness would accept the risks as weighed against the benefits of that device. We would require appropriate product labeling that clearly defined the patient sub-population and their benefit-risk preference in the Indications for Use section.” (The Pink Sheet, January 20, 2015)

What does this mean for the Duchenne community and PPMD? It means a new engagement approach with the FDA. It means demonstrating that access to potential treatments for serious and progressive disorders should be prioritized, even when the level of benefit is emerging. It might mean that families take on some risk and uncertainty to have access to the hope and potential of new therapies. We need drugs that work, but we need a different process to get there.

So back to PPMD’s newly-published article. This article is important because it demonstrates how we included an internal validation into our benefit-risk study. The article itself is pretty technical but the message is clear – using two approaches, our participants demonstrate a tolerance for risk with a moderate benefit. We expect that this article will also be of interest to clinical trial sponsors because it shows what percentage of our study participants were willing to accept different simulated treatments (i.e., with different levels of benefits and risks).

This leads to PPMD’s next community engagement effort. The CDRH’s historic decision gives us a clear path for our next benefit-risk study, which is under development. We must assess meaningful benefit and evaluate how much uncertainty in benefit is acceptable to our patient and caregiver community. This data will be meaningful to clinical trial sponsors and will inform our new engagement approaches with the FDA.

We will be coming to you for your input soon!