FDA’s New Breakthrough Designation Process: A Patient Perspective

Teresa Barnes at American Thoracic Society Conference in 2013.

With the FDA holding its first patient-focused drug development meeting in October of last year, regulators are starting to take notice of the impact disease-based organizations can have in improving drug trial design for new therapies-particularly for rare diseases. According to Teresa Barnes, Vice President of Patient Outreach & Program Support for the Coalition for Pulmonary Fibrosis, whether a drug is allocated to the FDA’s fast-track, breakthrough, or accelerated approval pathways, the patient voice needs to be included in the conversation. In the first of a new online series on the “Patient Voice,” Barnes spoke with PharmExec to outline some ways to make sure patients obtain this seat at the table-the earlier, she says, the better.

PE: How can patient groups help structure the conversation between companies and regulators early on in the process of clinical trials?

TB: With clinical trials, depending upon how many procedures are required and what the inclusion/exclusion criteria are, drug companies may or may not be able to recruit the best people. Our recommendation is, talk to us early, and let us provide a perspective from those who actually have the disease and are committed to finding better treatments. Then we can help companies step-by-step with patient-relevant factors in mind when they’re designing their trials, and establishing the protocols. That puts the cards in their favor. We can’t guarantee success, but in this way we can help confirm that patients know about the trial, and know what the requirements are.

PE: What do you recommend to ensure the FDA is involved in making this happen?

TB: Our suggestion to the FDA is that they consider the breakthrough process as a triangle instead of a two-way street. The triangle’s three points would be the FDA, industry and/or researchers, and patients. If FDA were to make a specific requirement that patient groups be involved early on and often in the process, more companies would embrace the idea of working with us. But at least within my area of fibrotic disease, that’s not the way it’s done. In fact, most companies won’t talk to us early; they don’t feel empowered. My other point to FDA is not only that they recommend it, but with everyone’s agreement, actually facilitate bringing us to the table. Obviously, if the pharmaceutical company is not sure what they’re going to do, then that’s not the best time. But at whatever point FDA feels it’s relevant, having us involved directly in the conversation can only help the process to be more predictable-which, in this industry, makes ultimate success more likely. We would also ask that when looking at drugs for deadly diseases with little or no therapies, the FDA truly understand the patients’ willingness to accept and tolerate risk. As William Gahl of the National Institute of Health (NIH) put it, “We want to be protected, but we don’t want to be protected to death.”

PE: What is the FDA doing now in patient consultations that can be leveraged more effectively?

TB: FDA uses patient representatives in the process of drug review-primarily at the very end of the registration process.  With that in mind, we’d suggest lay people who are active in disease areas be recognized as experts in the disease in their work with the FDA.  Lay people are generally not trained medical experts (though there are exceptions) and though they are very knowledgeable on the diseases they advocate for, they may have limited understanding of other diseases.  Therefore, we’d ask that FDA work harder to have people who are truly representative of the patient population affected by the dossier for which they are discussing in review.

PE: What’s the current state of play in the fight against pulmonary fibrosis?

TB: The good news is that there are many clinical trials underway in PF.  The bad news is that there is still no FDA approved treatment for the disease.  I have been in this area since my father died from PF in 1996, while I was in college (he was diagnosed in 1994).  There is virtually no difference in prognosis and treatment for patients now compared with then; there is a 100% mortality rate and a life expectancy of 2.8 years post diagnosis.  There are two drugs in Phase III testing (Boehringer-Ingelheim and InterMune drugs) and a growing number of pharmaceutical companies are entering into the space.  Also, last year, I worked with the American Thoracic Society to convene the first “fibrosis across organs” meeting to bring together experts across disease areas and organ areas.  Since 45% of all deaths in the industrialized world are attributed to some form of fibrosis or scarring (such as lung, liver, kidney, skin and heart), it was and is our belief that a path forward is to look at fibrosis across silos to learn more faster.  If companies can find promising therapies in one area of fibrosis, it is our hope that those therapies could be helpful in other sites for the disease.

PE: Right now, how do you think industry and FDA are doing in incorporating the patient perspective?

TB: The FDA is trying to understand the patient perspective, so I commend it for that. The patient community is not here to slow anything down or cause trouble. We have information about our patients that could be helpful to the regulatory process; ultimately, that benefits our both the process and the patient. That’s why we want to be involved, and that’s why I think FDA understands that we’re really a valuable component that hasn’t been used systematically to inform the decision-making process. I think industry and FDA will actually embrace this-it’s simply a matter of time and adjusting everyone’s comfort zone.