Patient Turned Advocate Shares Insights, Experiences with Clinical Trials

Whether she’s speaking at the United Nations or advising biotechs on new products, advocate Ella Balasa ensures that the patient voice is heard.

By Darcy Grabenstein

At the recent United Nations General Assembly High-Level Meeting on Antimicrobial Resistance (AMR), Ella Balasa shared her personal experience battling antibiotic-resistant infections due to her cystic fibrosis (CF) diagnosis. In her address to over 100 delegates, she urged for consensus among researchers, medicine manufacturers, policymakers, investors, and the public.

As a result of testimony by Balasa and other advocates, global leaders approved a political declaration committing to a clear set of targets and actions, including reducing the estimated 4.95 million deaths associated with bacterial AMR annually 10% by 2030.

Ella Balasa

Balasa, owner of Balasa Consulting, specializes in patient advocacy and engagement. She comes by it naturally, having advocated on her own behalf. That included learning as much as possible about her disease and treatment options. She took it a step further, earning a degree in biology.

She explains that cystic fibrosis causes mucus to build up in the lungs, which traps bacteria from the air that is inhaled. This bacterium colonizes the lungs, causing repeated infections that must be treated with antibiotics, which leads to antibiotic resistance. AMR is a growing global issue; in the UK, antibiotic-resistant infections are at the highest level since COVID-19, according to a recent UK Health Security report.

After graduating college, Balasa worked in a microbiology lab, researching antibiotic resistance from an environmental standpoint. She began to realize the value of her perspective as a patient who understands the science and research.

Balasa says she “bridges the gaps” between patient communities and the research and healthcare industries. “I wanted to empower and educate other patients to gain more awareness about their condition and about ongoing research.”

Firsthand trial experience

Although Balasa had already begun her journey as a patient advocate, she found out about an alternative to antibiotics in a roundabout way. A media company had asked her to write an article about living with cystic fibrosis, to accompany a documentary about a CF patient being treated with phage therapy in Texas. Phage therapy uses a very specialized virus that attacks bacteria. “I had heard about phage before,” she says, “but I did not know that there were patients with CF who were actually getting this treatment. And I was just blown away.”

Putting on her advocate hat, Balasa found out who the researchers were and contacted them directly. Because Balasa’s body was not responding to traditional antibiotics, the researchers were receptive to her case. That’s the catch-22 for many patients seeking to participate in clinical trials; they are either too sick or not sick enough to qualify. “I have been excluded from investigative drug trials for CF because I’ve had very low lung function,” says Balasa, who has written about the fact that inclusion/exclusion criteria (I/E) often lean toward the E in I/E. “It’s very frustrating. I feel there needs to be more focus on adaptive trial design.”

While the researchers were eager to treat Balasa, her doctor was very cautious due to the lack of data. But Balasa was sold. However, in order to receive the treatment, Balasa had to travel from Virginia to Yale University in Connecticut, the closest location offering that treatment.

“I had to travel by car that distance because I was very, very sick,” she recalls. “I was on supplemental oxygen 24/7. And I was in no condition to drive myself. My mom, fortunately, drove the whole way nonstop.” They spent three days in Connecticut, including a full day of treatment. She finished the weeklong treatment back in Virginia. Balasa says the phage therapy did help treat the acute infection she was facing.

She later connected with a research team at Baylor College of Medicine in Houston. She was told they could treat her case and that she could receive the therapy at a local CF clinic. However, her doctor was not familiar or comfortable with requesting emergent use/expanded access from the Food and Drug Administration (FDA) for the experimental treatment for AMR.

Instead, Baylor set up the study protocol so that Balasa could pick up her medication at Duke University Hospital in North Carolina, where she is being seen as part of the lung transplant program. She was not allowed to have the medication shipped to her. Neither could she pick up more than one week’s worth of treatment at a time, even though Baylor sent the full month’s supply to Duke all at once. This likely was due to the fact the blood was drawn at each visit to monitor her progress. That meant she had to travel 2 1/2 hours each way once a week for a month, which took up a full day each time. “It was a lot.”

Amplifying the patient voice

Balasa has volunteered with the Cystic Fibrosis Foundation, providing the patient perspective on advisory boards on protocol development and design for clinical trials. “That's what really started my patient advocacy journey,” she says. “You can say and then from there it just incrementally grew.”

She served as co-chair of the foundation’s inaugural research conference for the patient community, working with researchers and physicians. Aware of the power of the patient perspective, she says, “That helped me to find my voice as a patient and … really gain that confidence to communicate with stakeholders.”

For Rare Disease Day 2020, Balasa spoke on individualized therapies in a panel discussion at the FDA: “Supporting the Future of Rare Disease Product Development.” She founded her consulting business in 2021 and has served on various advisory boards, including the Clinical Trials Transformation Initiative (CTTI) steering committee, the advisory panel on rare disease for the Patient-Centered Outcomes Research Institute (PCORI), and project-related boards for the Center for Information and Study on Clinical Research (CISCRP).

In her consulting practice, Balasa helps start-ups and small biotechs develop a strategy to involve patients in development of their products and solutions.

Diversity and rare disease

While cystic fibrosis may be one of the more commonly known rare diseases, it still qualifies as a rare disease. According to the National Organization for Rare Disorders (NORD), a rare disease is a condition that affects fewer than 200,000 Americans.

Cystic fibrosis is primarily a Caucasian disease. Because of that, Balasa says, “There hasn't been until very recently been really any acknowledgement or focus on the effect that it has on minority populations,” which have different mutations.

Sources: MedlinePlus, American Journal of Respiratory and Critical Care Medicine

Balasa says the Cystic Fibrosis Foundation is leading the charge on efforts to increase diversity, working to understand the diagnostic process, social determinants of health, and how underrepresented communities access care.

She says she is encouraged that many companies are taking the FDA guidance on diversity action plans seriously and beginning to implement it. She does, however, think there could be more grassroots efforts to engage and recruit individuals from underrepresented communities.

The path forward

To other patients, Balasa advises them to self-educate and advocate. “You know your condition and your situation better than anybody else, and that includes even physicians,” she says. She recommends patients educate their doctors, as many do not have the time to research every patient’s specific case.

Balasa underscores the importance of patient community support groups, both from an emotional and social standpoint. “It’s important to seek peer-to-peer connections,” she says, adding that it helps to have “someone who understands you without having to explain yourself.”

She urges patients: “Don’t be afraid to voice your opinion” on the best plan of action. “You have to be the biggest advocate for yourself and your own care.”