How putting patients at the center improves biotech clinical operations

Clinical trials allow patients access to investigational therapies that aren’t yet available to the general public. While there’s no guarantee that the treatments will work, this participation allows patients to take a more active role in their treatment with the potential to improve their conditions.

Unfortunately, finding and enrolling in trials may not always be easy.

At BioCryst Pharmaceuticals, the Clinical Operations and Global Patient Advocacy teams are working to put patients at the center of their trials.

“We involve patient advocacy organizations and members of the patient communities during the trial design to ensure the science is really about what patients are wanting, in terms of treatments or a potential cure — we don't want to make assumptions around the most important outcomes or the most important aspects that are measured in a trial,” said Desiree Clemons, vice president of clinical operations at BioCryst. “For example, in designing clinical trials, we try to really listen to what patients are concerned about and what they feel the unmet needs might be for their disease state.”

The team at BioCryst also listens closely to patients in terms of the burden of participation in the trial. For example, some trials may require patients to visit sites a significant distance from their homes or attend frequent visits at their doctor’s office. In order to address this challenge, the company works with partner organizations that essentially act as travel agencies for patients, helping to coordinate travel arrangements and reimburse costs.

By hearing insights from patients during the design phase, potential hurdles like these can be avoided while also ensuring the study is scientifically robust.

“It's important to try and run the trial design through patients and advocacy groups. For example, if a protocol requires a needle biopsy, that's an arduous procedure. How often are patients going to be willing to undergo that? We can talk to doctors, but it's really the patients who are going to be able to help us understand the burden,” said Dakota Fisher-Vance, a senior manager on the Global Patient Advocacy team at BioCryst. “Engaging with patients at the earliest possible phase is really important.”

As a member of the Global Patient Advocacy team, Fisher-Vance and the rest of the team help share feedback and insights from the patient advocacy and rare disease patient communities internally.

“A big part of our job is listening. We go to our advocacy partners, and we hear from them about what the patient community needs — what their priorities are, what they want to address — and we take that back internally to our clinical operations team and work with our colleagues and advocacy partners to try to address those challenges and goals together,” said Fisher-Vance. “It’s not a transactional relationship — we're trying to create enduring and long-term relationships that go both ways. To do that, we need to be open from the earliest possible stages of drug development all the way through a potential launch.”

By removing barriers and maintaining transparency and communication, BioCryst strives to be the clinical trial provider of choice for patient communities and study sites, maintaining a strong reputation within each community in the process.

“Our goal is to be very transparent in terms of our research and earn the trust of trial participants so that we can be viewed as a company that cares about advancing the science for patients today and in the future,” said Clemons. “In doing that, we hope to earn the trust of the research community as a whole, all of which helps us reach the most patients possible who may want to participate in a clinical trial.”

Patient advocacy groups are able to provide tremendous support to patients beyond clinical trials, from offering community-building events to providing general disease education, which is why it is vital to work together. Within the company, employees are encouraged to learn more about the diseases that patients are living with as well as the advocacy groups that help support them.

For example, employees will often reach out to the Global Patient Advocacy team about participating in walks or other events led by patient advocacy groups. BioCryst also regularly invites patient community speakers to share their experiences living with a disease with employees. In encouraging this participation and awareness, the company keeps patients at the heart of everything it does.

“It’s not uncommon for us to have representatives from different patient advocacy groups come and talk with us about what their journey has been like with a rare disease. It helps us in terms of being able to think about an individual, what they may be dealing with and what we're working on that may help them or others who have a similar condition,” said Clemons. “Most of the people at our company have found their home here because they’re really motivated by the idea of lessening the impact of rare diseases on patients so they can live their lives to the fullest.”