What Patients Told the FDA About Living with Recurrent Respiratory Papillomatosis

On October 27, 2022, the Recurrent Respiratory Papillomatosis Foundation (RRPF) hosted a landmark patient-led listening session with the U.S. Food and Drug Administration (FDA). The purpose was simple but profoundly important: to ensure that regulators heard directly from the people living with recurrent respiratory papillomatosis (RRP) and the caregivers supporting them every day. Click here for the full summary.

For decades, treatment decisions in RRP have largely been shaped by clinical outcomes such as disease recurrence and surgical frequency. While those measures are important, they do not fully capture the lived experience of the disease.

The stories shared during this session revealed something that cannot be measured solely through clinical data: the emotional, psychological, financial, and social burden of a disease that often requires repeated surgeries throughout a patient's lifetime.

The listening session represented an important step toward ensuring that patient voices help guide future regulatory decisions, clinical trial design, and therapeutic development. As RRPF President Kim McClellan explained:

"It is my hope that today's listening session will assist the FDA in making those decisions that impact our community. The patient voice has been missing, and to see our voice now being part of what drives progress is more than we could have dreamed of just a few short years ago."

Why the Session Was Held

The primary objective of the session was to provide FDA staff with firsthand perspectives on what it means to live with recurrent respiratory papillomatosis and what outcomes matter most to patients and caregivers. At a time when the RRP field is beginning to shift from repeated surgical intervention toward systemic and medical therapies, RRPF recognized the importance of ensuring that patient priorities are part of the conversation.

The session brought together patients, caregivers, clinicians, and representatives from multiple FDA divisions, including the Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER), Center for Devices and Radiological Health (CDRH), and the Oncology Center of Excellence.

A Consistent Message: Surgery Is Not Enough

One theme emerged repeatedly throughout the session: patients and families are desperate for treatment options beyond surgery.

For many individuals living with RRP, surgery is not an occasional event. It becomes a recurring cycle that can dominate childhood, adulthood, careers, family life, and mental health.

Current standard treatment relies on repeated surgical removal of papillomas from the airway. While these procedures can restore breathing and voice function temporarily, recurrence is common, and patients may require dozens, hundreds, or even more procedures over a lifetime.

The session summary notes that surgery itself often contributes to long-term morbidity because repeated interventions can permanently alter the delicate structures of the larynx.

As one caregiver explained, surgery often feels like "trimming the grass." The disease returns, and the cycle begins again.

The Real Burden of RRP Extends Beyond the Airway

A major takeaway from the listening session was that RRP affects far more than breathing and voice.

Patients described:

• Repeated disruptions to education

• Lost career opportunities

• Financial hardship

• Social isolation

• Anxiety and depression

• Medical trauma

• Family stress

• Fear of disease progression and cancer

The summary highlighted findings from the International RRP Patient Registry showing that:

• 74% of patients reported feeling limited in their career options because of RRP.

• Nearly 80% reported experiencing social anxiety related to their symptoms.

• Some patients reported spending more than 15% of their annual income on RRP-related healthcare expenses.

These findings underscore that RRP is not simply a disease of the airway. It is a disease that can impact nearly every aspect of a person's life.

"One Less Surgery Matters"

Perhaps the most powerful message shared during the listening session was remarkably simple.

One less surgery matters.

When FDA representatives asked what would constitute a meaningful reduction in surgical burden, patients and caregivers were nearly unanimous.

Any reduction matters.

Kim McClellan, who has lived with juvenile-onset RRP for more than five decades and undergone more than 250 procedures, explained that even one fewer surgery can have profound effects on quality of life.

The discussion highlighted that each surgery represents:

• Time away from school or work

• Recovery from anesthesia

• Voice loss

• Financial costs

• Family disruption

• Emotional stress

• Risk of additional scarring and permanent vocal damage

As surgeon Dr. Simon Best noted during the discussion:

"The correct answer to this question is one." Any avoided surgery reduces the risk of permanent vocal cord damage.

Caregiver Stories Highlight the Need for Better Treatments

Several caregivers shared deeply personal experiences caring for children with severe RRP.

One family relocated internationally to pursue participation in a therapeutic vaccine study after exhausting available treatment options. Another family traveled repeatedly across the country seeking expert care and access to emerging therapies.

Parents described:

• Frequent surgeries every few weeks

• Airway emergencies

• Long-distance travel for treatment

• Insurance battles

• Educational disruptions

• Emotional trauma affecting the entire family unit

These stories reinforced a critical point: access to promising therapies remains inconsistent, and many families face substantial barriers when seeking specialized care.

Mental Health Cannot Be Ignored

The session devoted significant attention to the psychological impact of RRP.

Patients described years of anxiety, depression, social withdrawal, and isolation resulting from repeated procedures and chronic uncertainty. Caregivers spoke openly about the emotional toll of watching their children undergo surgery after surgery.

One parent described decades of counseling and mental health support required for a child living with severe RRP. Another participant shared experiences of depression and suicidal thoughts stemming from the relentless cycle of disease recurrence and treatment.

These testimonies emphasized that successful treatment should not be measured solely by airway outcomes. Mental health and quality of life are equally important outcomes for the RRP community.

Pulmonary RRP Remains a Major Fear

The session also highlighted concerns surrounding pulmonary dissemination of RRP.

Although pulmonary involvement occurs in only a minority of patients, it remains one of the most feared complications of the disease. The summary notes that pulmonary progression occurs in approximately 9% of patients and is associated with a dramatically increased risk of malignancy.

Caregivers shared experiences navigating pulmonary disease, lung surgeries, cancer diagnoses, and ongoing uncertainty. Several participants argued that greater attention should be given to pulmonary screening and monitoring for high-risk patients.

What Patients Want Regulators to Understand

The listening session made clear that the RRP community has a different perspective on risk than individuals who do not live with the disease.

Many participants expressed a willingness to accept significant treatment risks if those risks offered the possibility of reducing surgical burden. Patients and caregivers repeatedly emphasized that the trauma of repeated surgeries often outweighs concerns about potential side effects from new therapies.

The session also highlighted concerns about traditional placebo-controlled clinical trial designs in rare diseases such as RRP, where withholding potentially beneficial treatment may mean continued surgeries, progressive scarring, and worsening disease.

Why This Listening Session Matters

The RRPF-organized FDA Listening Session represented far more than a meeting.

It created an opportunity for regulators to hear directly from those living with the realities of recurrent respiratory papillomatosis.

The stories shared throughout the day illustrated the challenges that statistics alone cannot capture:

• The child who misses school because of surgery.

• The adult who loses a career because of voice limitations.

• The parent who spends years searching for treatment options.

• The family living with fear of pulmonary spread and malignancy.

• The patient who has undergone hundreds of procedures and still hopes for a better future.

Most importantly, the session delivered a unified message from patients and caregivers alike:

The future of RRP treatment must go beyond surgery.

Key Takeaways

• RRPF organized a patient-led FDA Listening Session on October 27, 2022, to provide regulators with patient and caregiver perspectives on RRP.

• Patients emphasized that any reduction in surgeries would be meaningful.

• Repeated surgeries contribute to physical, emotional, financial, and social burdens.

• Participants expressed strong support for development of non-surgical and systemic treatment options.

• Mental health impacts, including anxiety, depression, and trauma, were recurring themes throughout the session.

• Pulmonary RRP remains one of the greatest fears among patients and caregivers.

• The session reinforced the importance of incorporating patient voices into future drug development and regulatory decision-making.

Download the Full FDA Listening Session Summary

To learn more about the experiences shared during this historic event, download and read the complete report: