On March 5, at the New Jersey Rare Disease Day event, hosted by the New Jersey Rare Disease Alliance, John Maslowski, CEO of Fibrocell Science, sat down with Rare Disease Report
to discuss clinical development.
While the subject matter might seem like something that doesn’t pertain to anyone other than medical professionals, Maslowski stresses the need for patient voice in the development process for drugs intended to treat the rare disease community.
“All companies need to start communicating earlier,” he says, “and these conversations need to happen before a Phase 1; not as your about to enter a Phase 3 or when you’re going into clinical trials. If you wait, you can learn the hard way that none of your endpoints matter, because none of what you’re evaluating matters to the patient.”
Rare Disease Report:
What exactly is the CEO of a biotechnology company doing at a Rare Disease Day event? These events are typically for patients and advocates.
[Fibrocell] has a focus on rare skin diseases, primarily in children. We’re looking to use gene therapy to locally effect these diseases, and we hope to have a great outcome from a medical standpoint, but an important part of our business is to support rare disease advocacy. That’s really a source where you can learn so much about the disease. Not only are you learning how the disease effects patients, but you’re learning how you can access patients and how they manage their daily care.
What challenges are you facing in trying to develop therapies for rare disease patients?
There have been a lot of advancements as it pertains to approaching clinical development in general, and there’s especially that challenge in rare diseases. I think all advocates, parents and patients know that there are 7,000 rare diseases and 95% of them don’t have FDA-approved therapies. Only 5% of rare diseases are represented and, really, what we want to get across is that industry has to begin to work earlier with advocacy groups and learn more about disease states before the actual scientific work starts. Biotechs and pharma companies have to learn more about what patients really need so that they can develop the proper preclinical models and clinical endpoints. That consideration and respect needs to be established early, and it will help the patients understand that these companies have their patients’ best interests in mind.
What benefits does your company see after attending events like this one?
The one benefit that our company really gets from working with and supporting advocacy groups in general is access to families and patients. When we talk to them, we get that direct conduit of what they really need. Where a company like ours could really go wrong is if they were to develop a product all the way down into a pivotal Phase 3 trial and then realize that the outcomes don’t even benefit the patients. We really want to get there early and make sure that we’ve done our due diligence on the safety of the drug, and that as we progress into later trials, that patient voice continues to be heard. We really need that relationship, and I think that most pharmaceutical companies and biotechnology companies are learning that it needs to start earlier and earlier in development.
In what ways do you think that big companies can help patients directly?
Pharma companies take clinical trials and talk about endpoints and dose escalation and all of these things that can be hard to understand, but then I’ve seen some translate that information into more reasonable text on their websites that explains why these sorts of studies need to be done. It needs to be understood why we need to go through Phase 1, Phase 2 and Phase 3. More importantly, people need to know the process of how and why certain drugs even enter the clinic. People need to understand why samples might be necessary to do some early animal work. I think that translation for the advocacy groups is where the trust comes from. As somebody from one of these companies, we want people from all walks, but especially advocacy groups, to understand what we’re doing.
Are there other ways, besides elaborate events and on your company website, that you can effectively communicate with people who want this information?
One of the most effective tools that we use to communicate with patients and advocates is town hall meetings. There, we can explain our progress to patients and parents. There, we can let people know exactly where we are and where we stand with the FDA, and what has happened in the process up until that exact moment. Being able to communicate to patients and to parents through a group is becoming a bigger part of drug development. People are thinking earlier now about research, and targets and endpoints.
For more from World Rare Disease Day 2018, follow Rare Disease Report