Three Questions: Bryce Warren, Atlantic Research Group
CWWeekly presents this feature as a spotlight on issues faced by executives in clinical research. This week, writer Karyn Korieth spoke with Bryce Warren, Ph.D., chief science officer of Atlantic Research Group, a Charlottesville-based CRO focused on rare and orphan diseases, and vice-chairman of the Histiocytosis Association, one of the largest patient organizations for a disorder without a cure.
Q: What are some of the differences in conducting clinical trials for non-rare diseases and those for rare and orphan diseases?
A: Orphan diseases can be exceedingly rare. There are so few patients that only one case may present to an institution per year. We need to stay in contact with investigators without pestering them. When you are talking about the rare patient, you cannot let opportunities go by because someone was asleep at the wheel.
We have to think about where the rare patient will first present. Do they have a crisis and go to the emergency room? Are they in a general practice clinic? Are they in some sort of specialty clinic? When we managed an adjunct safety study for a phase III neurogeneic orthostatic hypotension study, for example, we met the patient enrollment deadline in just four months by realizing that undiagnosed patients were actually floating around in various neurology, cardiology and Parkinson’s disease clinics simply because doctors could not explain their aberrant blood pressure.
To address these challenges, we build teams not just inside of the CRO, but also at the site. We educate each other. Building a team makes conducting a clinical trial more elegant, efficient and, quite honestly, possible. The whole clinical team has to be constantly thinking of the trial, going over the protocol and making sure that, before the next patient visit, the right things are captured. Treaters on the front line need to be able to identify patients and have meaningful communication with them to explain the protocol and why certain things need to be done. Making those things as simple as possible is important to keeping patients coming to the clinic, keeping them compliant and being able to statistically power the clinical trial.
Q: How can engaging patients and advocacy groups benefit drug development for rare and orphan diseases?
A: Over the last two years, patient advocacy groups for rare diseases, medical and science groups and even government groups have put together patient registries, built natural history data and worked to take some risk away from product development. This is fairly unique in the rare disease world. Patients who suffer from rare diseases often have to advocate for themselves. They have tied together these groups and are sharing as much information as they can for the chance to share in the clinical development or experience of a particular product. The importance of supporting or even being a part of these groups and helping them lift patients toward treatment and sharing that information globally can’t be overstated.
Understanding the disease state and what it means to the patient can inform what the protocol should or shouldn’t do, what it can and can’t do and how it might capture needed data points. Some of the challenges of rare diseases and disorders are not obvious. Many are very personal.
These populations are often very invested in their care. It’s not hard to keep them on a trial as long as it makes sense and doesn’t hurt them physically. There is a lot of hope wrapped up in some of these clinical tests and trials. Many of these people—especially the older patients—have been living with a disease their entire lives. The rare disease patient is a partner as much as they are a patient.
Q: As the CRO space consolidates and many large sponsors have adopted outsourcing strategies that call for partnering with just a few strategic or integrated CRO partners, what is the role of a niche CRO in today’s clinical research landscape?
A: Any CRO should be able to knit the regulatory pieces together to buttress a clinical trial, but a niche organization can bring the patient experience and specific expertise in rare and orphan conditions to the front. Knowing what the patients feel, see and hear—and how the clinical test influences those things—is the key piece in the rare disease and disorder world. We have subject matter experts who have a true connection to patients through having conducted rare disease clinical trials as an investigator or being part of advocacy groups. Being able to understand the patient care experience and conduct the trial in a meaningful, expeditious way without upending patient lives is difficult to do without a boots-on-the-ground, frontline understanding about the rare disease state.
A niche CRO can bring their clinical experience to the process before the protocol is signed and recommend changes that can help reduce time-consuming, expensive amendments. Maybe mobility is an issue. Maybe sleep is an issue. You have to really think about the patient experience. The sooner that we can start having these conversations, the more beneficial they are to patients. If they are beneficial to patients, chances are those patients are going to be compliant and companies will be able to power the test statistically, which means there is a good shot of moving that molecule to medicine.
This article was reprinted from Volume 21, Issue 13, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »