SCIENTIFIC STORYTELLING: Patient Centricity Powers Progress in Rare Disease

Deerfield Agency discusses patient centricity in rare disease and their perspectives with Tim Pigot, Chief Commercial Officer of Aerovate, and Colleen Brunetti, PAH patient and advocate.

Too often, companies delay engaging the patient community until they have early evidence of a drug’s potential benefit. Companies can and should engage patient advocates as active partners in advancing
new treatments, providing guidance on everything from trial design to marketing and engagement activities. Nowhere is this patient centricity more critical than while working in rare disease.

Deerfield Group LLC works with emerging and pioneering health companies, such as Aerovate Therapeutics, many of which are innovators in the rare-disease space. Aerovate is a pioneer in
one particular rare disease, pulmonary arterial hypertension (PAH), a condition associated with a thickening and narrowing of pulmonary arteries. PAH is treatable but ultimately progressive, incurable, and often fatal, and scientists, industry, and patients have rallied to accelerate therapeutic progress.

We sat down with Tim Pigot, Chief Commercial Officer of Aerovate, and Colleen Brunetti, PAH patient and patient advocate, to hear their perspectives on how the evolving PAH-treatment landscape has benefitted from patient involvement as clinical-stage treatments advance.

How are you bringing a patient-centric approach to the development of your investigational drug for PAH, AV-101?

Tim: Aerovate has been very intentional about listening to patients and applying key learnings and insights throughout the development of AV-101 in real time versus after the fact. We developed a patient advisory panel early on to solicit guidance, which has been invaluable as we thought about clinical trial design, patient education, data communications, and product packaging. We traveled all around the country to visit with different patients to gain insights and obtain feedback on activities, including in product design. We also leveraged a “patientfriendly” clinical trial design—minimizing clinic visits while maximizing data outputs.

Rare-disease patients are often well educated about their disease—how have you leveraged this level of awareness?

Colleen: We are a very proactive community and accustomed to being the educator in the room with anyone but our PAH specialist. We stay well informed as a form of self-preservation, which gives us a heads up when good things are coming down the pike.

I am part of an advocacy organization, the Pulmonary Hypertension Association (PHA), and they are excellent at providing in-depth educational activities and support for patients, as well as funding research and getting the word out about new treatments as they become public. I rely a lot on other patients on social media for that first-person perspective. Patients are highly knowledgeable in a way that their doctors are not, particularly about managing side effects and day-to-day life. Talking to patients about clinical trials is also exciting.

What have you learned along the way about drug design and rollout through the lens of the patient experience?

Tim: Over the course of my career, I’ve learned that hindsight is 20/20. I can remember at a prior organization, receiving feedback following a drug approval about decisions made without sufficient provider and patient input. Product packaging and design may not often be thought about at such an early stage, but it is a good example. Working closely with patients on this aspect opened up more insights about the patient journey that informed many of our clinical and communications activities.

Colleen: Allowing patients to handle the materials prior to commercialization is great. I have had the opportunity to play with the devices for a company and pointed out that we have a patient population with scleroderma, which can impact the hands and make access to the medication a challenge. It may not feel intuitive for companies to think about the effects of secondary diseases associated with pulmonary hypertension. By allowing patients to engage with devices or communications prior to availability, it only helps uptake when it finally does arrive.

How can companies do a better job of including the patient perspective?

Colleen: Consistent decision-maker exposure to patients through patient/caregiver advisory boards is critical. When I speak to a company’s employees, I am often the first patient they have had personal contact with in their career, let alone working for that PAH-related organization. I also think an openness to not only hearing but also applying patient feedback is important, and would encourage companies to trust our experience versus deferring to the overarching results of a clinical trial when making decisions that will ultimately impact patients. We know our voices have been heard when a company follows up to show us how our thoughts were implemented, which is highly validating.

What impact does engaging patients have on engaging employees?

Tim: We see tremendous value in bringing patients into our internal company discussions. Aerovate employees hear us say how difficult the disease is and how poor the prognosis is, so they all understand that they are working for a noble cause, but they can relate even more when they directly engage with a patient because everyone understands the challenge of managing an illness. That is a universal story.

Patients are happy to engage with us. They are enthusiastic to see a clinical-stage company reaching out to them and wanting to learn. They appreciate the opportunity to guide drug development ahead of commercialization. Knowing that their input will make a difference in the final product is a huge home run for them.

What is the benefit for patient advocates in sharing their stories with companies?

Colleen: It can boost morale and spur a need to urgently act. The story I tell is that I did not think I would live to see my son reach kindergarten—he was 18 months old when I was diagnosed. He is graduating from high school this year. I am here because of science, because other patients in clinical trials and companies took a risk. On the other hand, I continue to lose friends to this disease, and I am still pretty sick, which means we still need more. It is extremely powerful to talk to a company and help them really feel the emotion attached—the good and the bad. I like to believe it can do a lot for staff productivity and morale and, ultimately, the broader patient community at large.

  • Amanda Johnson
    Amanda Johnson

    Senior Vice President, Science Deerfield Agency

    Amanda Johnson, PhD is a seasoned communicator with more than 15 years’ experience distilling highly complex scientific topics to a range of audiences in biotech, pharma, government, academic, and nonprofit settings. She leads the science and medical team at Deerfield Group LLC to elevate biotech and pharma clients’ science stories. amanda.johnson@ vergescientific.com

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