7 Key Takeaways of the Novo Nordisk Rare Disease Survey in Sickle Cell Disease from Patients as Partners 2025

At the 2025 event, Dr Ify Osunkwo, Chief Patient Officer of Novo Nordisk Rare Disease shared learnings from the Learnings and Insights into the Sickle Cell Trial Experience (LISTEN) survey conducted amongst the global sickle cell disease (SCD) patient community, and the powerful insights generated on collaborating with and embedding the patient voice sustainably, and the message that real listening is about understanding, acting, and implementing what is heard from the patient community. 

Below are key takeaways from Dr Osunkwo’s keynote. 

1. Redefining "Listening" in Patient Engagement

“[It] is not just hearing, but understanding and incorporating what you hear into making meaningful change,” said Dr Osunkwo. The mission of the LISTEN survey was to understand the lived experiences of SCD patients, in order to translate those insights into tangible action, protocol revisions and long-term change. 

SCD patients voiced frustration about previous engagement efforts where they were consulted late in the process or felt like an afterthought. “[Patients] want equity where they are placed in a level similar to or equal to the HCPs and everybody else we listen to. Don't listen to everybody else and then tell them, ‘This is what we heard,’”said Dr Osunkwo. The takeaway was that patient engagement must begin at the design phase, not after protocols are finalized, and building trust required transparency and shared ownership.

"We want to make sure that we're reaching everyone and involving them in the research that we're doing."

2. Start with Patient Organizations

Dr Osunkwo spoke about the need to flip the traditional model and begin advisory boards with patient organizations, then patients, and then healthcare providers. By beginning with patient groups, you could develop a nuanced understanding of patient needs. Validating those findings would then enable trials to be designed around those needs. “We want to make sure that we're reaching everyone and involving them in the research that we're doing, that they help us iterate the process over time, and that we understand the disease burden.” 

3. Empower Patients as Equal Stakeholders

One of the items that emerged was the desire from patients to be treated as true partners and for their roles as advocates and recruitment partners to be formalized. “It can’t just be that we find one person, put them on an ad board, but whenever they talk, nobody listens,” said Dr Osunkwo. Dr Osunkwo talked about how the process of upskilling patients to actively participate in ad boards was discussed, including who would be doing the training, and how upskilling is an investment that would pay back in trust and impact. 

Osunkwo stressed that when interacting with patient advocacy groups around the globe, some may have different maturities or capabilities. Dr Osunwko noted that, in cases where advocacy groups weren’t as mature and didn’t have some of the logistics set up, it required more time and investment. However, once that had been done, they were willing to mobilize and bring in additional folks to the survey. 

4. Internal Buy-In and Championship

During the Q&A portion, Dr Osunkwo answered a question about how to get patient insights incorporated through the company in the face of internal stakeholder pushback. She acknowledged that at Novo Nordisk, they were lucky to have a CEO with a patient-first mindset, but she also spoke about the internal influencing, networking and negotiating that has to take place as well. 

“I volunteer my time and services to anybody and everybody,” she said. “I drop insights like, ‘Have you thought about this?’ or ‘Look at what somebody else has done that has worked,’” to make it easy for people to opt in. 

"You can't solve the barriers unless you teach people about what clinical trials are."

5. Language and Cultural Competency

One insight from LISTEN was that “language” barriers didn’t mean foreign languages. The language barrier was comprehension and cultural context. The difference between urban and rural language, cultural identity, and tone all impacted trust and understanding. Patients called for language that reflected their reality, not medical jargon, and highlighted that medical literature often failed to reflect the severity and nuances of their daily experience, particularly with pain. 

“Plain language needs to be plain,” she said, not just slightly less sophisticated versions of the main manuscript. Patients want something that is readable, in order to understand and make informed decisions. And part of that requires understanding the full breadth of education in the patient population, and accommodating that. 

6. Address Knowledge Gaps Before Participation Barriers

The LISTEN survey collected data from over 1,500 patients and 360 healthcare professionals across 17 countries and discovered that nearly half of respondents had never heard of a clinical trial for sickle cell disease. Only 20% had ever participated. “You can't solve the barriers unless you teach people about what clinical trials are,” said Dr Osunkwo, because participation can’t be expected if the foundation isn’t there. But, Dr Osunkwo noted, once people did participate, they had positive experiences. 

The point was that rather than rushing to solutions after the initial survey, when patients revealed they lacked basic understanding of clinical trials, the focus shifted to education first. The team co-created infographics, videos, and workshops to lay the groundwork for future phases.

"If there is a scientific rationale for the visit that cannot be overcome, explain that to patients." 

7. Trial Design That Reflect Patient Lives

Patients flagged numerous logistical issues: study visits that were too frequent, burdensome travel (and travel from literally door to door), long appointments, and lack of support systems. Patients also expressed a desire for navigators/chaperones who were peers and “trial passports” to help track appointments and requirements, similar to ones that are created for patients going through cancer treatments. 

However, the survey also found that if certain aspects of the trial that might be more burdensome were explained, patients were very receptive to participating. Dr Osunkwo said that once they explained, for example, why a patient-reported outcome needed to take one hour to complete, patients said that they would do that. “If there is a scientific rationale for the visit that cannot be overcome, explain that to patients.” 

Dr Osunkwo ended her presentation with the encouragement that embedding the patient voice takes both process and implementation, coupled with empathy and consistent communication. “But at the end of the day, do not ever feel like you’re not making a difference.” 

Ifeyinwa (Ify) Osunkwo, MD

Chief Patient Officer

Novo Nordisk Rare Disease

Ifeyinwa (Ify) Osunkwo, MD

Chief Patient Officer
Novo Nordisk Rare Disease

Ifeyinwa (Ify) Osunkwo, MD, MPH, is the chief patient officer of Novo Nordisk Rare disease. Prior to this she served as the inaugural chief patient officer and senior vice president at Forma Therapeutics. In her role, Dr Osunkwo is responsible for realizing Novo’s vision to transform the lives of patients by improving access and care through partnerships with global patient and community stakeholders while driving an internal culture of patient focused medicine development. Dr Osunkwo is an internationally acclaimed expert in the research, treatment and care delivery of sickle cell disease (SCD), pediatric to adult care transition, and the management of chronic pain. She has a passion for health equity and has focused her research on identifying the comparative effectiveness of interventions in eliminating health disparities in Black and brown populations. She has more than 25 years of experience in clinical management, population health using the chronic disease management model and health literacy. She is an accomplished clinician educator adept at training clinical and non-clinical audiences and is also a visionary leader in pragmatic program development and implementation using quality improvement. Dr Osunkwo is the Founder & Director of the Sickle Cell Disease Enterprise at Levine Cancer Institute and a Professor of Medicine and Pediatrics at Atrium Health - a vertically integrated healthcare system serving 1400+ adults and 400+ children with sickle cell disease in NC/SC/GA. Previously, she was the medical director of the Comprehensive Sickle Cell Disease Program at Children’s Healthcare of Atlanta @ Egleston and Assistant Professor of Pediatrics at Emory University. She directed the Teen Scene Transition Program that provided care coordination and education to facilitate the smooth transition of young adults from pediatrics to adult care. Dr Osunkwo earned a medical degree from the University of Nigeria College of Medical Sciences and Dentistry and an MPH from Johns Hopkins University School of Hygiene and Public Health. She received her pediatric residency training at the University of Medicine and Dentistry of New Jersey and completed her fellowship in pediatric hematology, oncology and bone marrow transplantation at Columbia University College of Physicians and Surgeons. Dr Osunkwo is board certified in pediatric hematology oncology and has led a robust research enterprise garnering over $15 million in research funding and has served as principal investigator on numerous clinical trials over the course of her career.