University of Miami, Leonard M. Miller School of Medicine Miami, FL, United States
Jodie A. Barkin, MD1, Yasmin G Hernandez-Barco, MD2, Rahul Pannala, MD3, Trudi Delk, PharmD4, Nathalie Erpelding, PhD5, Caryl Kahn, BS5, Abbe Steel, MS6, Dennis L. Decktor, PhD, FACG7, David C Whitcomb, MD, PhD8 1University of Miami, Leonard M. Miller School of Medicine, Miami, FL; 2Massachusetts General Hospital, Boston, MA; 3Mayo Clinic, Scottsdale, AZ; 4Aimmune Therapeutics, a Nestle Health Science Company, Bridgewater, NJ; 5HealthiVibe, a Division of CorEvitas LLC, Arlington, VA; 6HealthiVibe, a Division of CorEvitas, LLC, Arlington, VA; 7Aimmune Therapeutics, a Nestle Health Science Company, Dresher, PA; 8University of Pittsburgh, Pittsburgh, PA
Introduction: Exocrine pancreatic insufficiency (EPI) is caused by deficiency of functional pancreatic enzymes resulting in maldigestion and malnutrition. The patient experience is not well understood, making the diagnosis and treatment challenging. Treatment of EPI relies in part on pancreatic enzyme replacement therapy (PERT); however, inappropriate PERT dosing may increase disease burden and negatively affect quality of life (QOL). The aim of this study was to characterize unmet needs by conducting a qualitative study of patients with EPI due to chronic pancreatitis (CP) and treating physicians through in-depth interviews, as well as conducting two advisory board meetings with key opinion leader (KOL) pancreatologists.
Methods: Virtual one-on-one semi-structured interviews with patients with EPI due to CP and treating physicians along with two KOL meetings were conducted to collect feedback on unmet needs, diagnosis, and treatment. Interviews were recorded, transcribed, and thematically analyzed.
Results: Eight patients with EPI due to CP (2 males; 36–63 years old), and six physicians (5 males; in practice for an average of 13 years seeing about 22 EPI patients/month) provided consent to participate in this research. Five KOLs participated in two advisory board meetings. Patients reported that severe abdominal pain and gastrointestinal symptoms were important determinants impacting their QOL. Patients identified a lengthy path to correct diagnosis, poor relationships with their physicians, suboptimal EPI symptom management, high pill burden, and lack of guidance as important unmet needs. Physicians and KOLs identified improved patient adherence to PERT, simplified and consistent dosing instructions across PERT brands, increased PERT titration options, and need for easy to use non-fecal diagnostic tests for EPI as important unmet needs from their perspective. Patients and physicians agreed that there is a need for long-term studies to better understand EPI and improve disease management.
Discussion: EPI is associated with high disease and QOL burden due to persistent pain/gastrointestinal symptoms, and challenges with diagnosis, patient-physician interactions, and symptom management. Feedback collected through in-depth interviews and interactive advisory boards reinforced the need for more real-world evidence. These data will help inform the design and conduct of future patient-centric, multicenter observational studies of CP patients with EPI.
Disclosures: Jodie Barkin: AbbVie – Other Financial or Material Support, Scientific Advisor. Nestle Health Sciences & HealthiVibe – Other Financial or Material Support, Scientific Advisor. Yasmin G Hernandez-Barco: Nestle health science – Advisory Committee/Board Member. Rahul Pannala: HCL Technologies – Consultant. Nestlé Health sciences – Advisory Committee/Board Member. Trudi Delk indicated no relevant financial relationships. Nathalie Erpelding: HealthiVibe, a division of CorEvitas, LLC. – Employee. Caryl Kahn: HealthiVibe, LLC – Employee. Abbe Steel: Aimmune Therapeutics, a Nestlé Health Science company – Consultant. Dennis Decktor: Nestle Health Sciences – Employee. David C Whitcomb: Ariel Precision Medicine – Advisory Committee/Board Member, Consultant, Patent Holder, Stockholder/Ownership Interest (excluding diversified mutual funds). BioNTech – Advisory Committee/Board Member, Consultant. Nestle – Advisory Committee/Board Member, Consultant. Novartis – Advisory Committee/Board Member, Consultant. Regeneron – Advisory Committee/Board Member, Consultant, Grant/Research Support. Samsung – Advisory Committee/Board Member, Consultant.
Jodie A. Barkin, MD1, Yasmin G Hernandez-Barco, MD2, Rahul Pannala, MD3, Trudi Delk, PharmD4, Nathalie Erpelding, PhD5, Caryl Kahn, BS5, Abbe Steel, MS6, Dennis L. Decktor, PhD, FACG7, David C Whitcomb, MD, PhD8. P0037 - Characterizing Unmet Needs in Patients With Exocrine Pancreatic Insufficiency Due to Chronic Pancreatitis: A Patient-Centered Observational Approach, ACG 2021 Annual Scientific Meeting Abstracts. Las Vegas, Nevada: American College of Gastroenterology.