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Accelerating the Drug Delivery Pipeline for Acute and Chronic Pancreatitis

Summary of the Working Group on Drug Development and Trials in Recurrent Acute Pancreatitis at the National Institute of Diabetes and Digestive and Kidney Diseases Workshop

Lowe, Mark E., MD, PhD*; Goodman, Marc T., PhD, MPH; Coté, Gregory A., MD, MS; Glesby, Marshall J., MD, PhD§; Haupt, Mark, MD; Schork, Nicholas J., PhD; Singh, Vikesh K., MD, MS#; Andersen, Dana K., MD**; Pandol, Stephen J., MD††; Uc, Aliye, MD‡‡; Whitcomb, David C., MD, PhD§§

doi: 10.1097/MPA.0000000000001164
Conference Reports

Recurrent acute pancreatitis (RAP) is a complex clinical syndrome with significant morbidity, unpredictable outcomes, and limited treatment options. The National Institute of Diabetes and Digestive and Kidney Disease sponsored a workshop on July 25, 2018, in Pittsburgh, Pennsylvania, to address research gaps impeding development of effective therapies for pancreatitis. The RAP working group identified challenges to clinical progress using existing definitions, risk assessment, diagnostic and severity criteria, disease trajectories, outcomes, and research methods. Recurrent acute pancreatitis includes all the risk of acute pancreatitis and often progresses to chronic pancreatitis with variable complications of chronic pain, exocrine insufficiency, diabetes, and pancreatic cancer. However, the great variability among individuals with RAP requires better precision in defining the risks, individual episodes, as well as their frequency, pathogenic pathways, and specific outcome measures for each of the systems affected by pancreatic inflammation. Because of disease complexity, few patients are similar enough for traditional studies and methods to conduct clinical trials with small sample sizes are required. The need for genetic testing, biomarker development, and better imaging methods was highlighted. Adaptive and N-of-one study designs, better endpoints, and outcome measures including patient-reported outcomes should considered early in developing future therapeutic trial design and include all stakeholders.

From the *Department of Pediatrics, Washington University School of Medicine, Saint Louis, MO;

Department of Medicine, Cedars-Sinai Medical Center, Los Angeles, CA;

Department of Medicine, Medical University of South Carolina, Charleston, SC;

§Department of Medicine, Weill Cornell Medical College, New York, NY;

ARIEL Precision Medicine, Pittsburgh, PA;

Department of Quantitative Medicine, The Transcriptional Genomics Research Institute, Phoenix, AZ;

#Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD;

**Division of Digestive Diseases and Nutrition, National Institutes of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health, Bethesda, MD;

††Division of Digestive and Liver Diseases, Department of Medicine, Cedars-Sinai Medical Center, Los Angeles, CA;

‡‡Division of Gastroenterology, Hepatology, Pancreatology and Nutrition, Stead Family Department of Pediatrics, University of Iowa Carver College of Medicine, Iowa City, IA; and

§§Department of Medicine, University of Pittsburgh School of Medicine and UPMC, Pittsburgh, PA.

Received for publication August 6, 2018; accepted August 23, 2018.

Address correspondence to: Mark E. Lowe, MD, PhD, Washington University School of Medicine, 660 South Euclid Ave, MPRB, 4th Flr, Campus Box 8208, Saint Louis, MO 63110 (e-mail:

This study was supported by the National Institutes of Health (NIH) Consortium for the Study of Pancreatitis, Diabetes and Pancreatic Cancer (CPDPC) U01 DK108334 (to A.U., D.C.W., M.E.L., S.J.P., M.T.G.) and 1R13DK118902 (to D.C.W.).

M.E.L. receives royalties from Millipore Inc and UpToDate and is a consultant to ARIEL Precision Medicine. M.H. is an employee of Ariel Precision Medicine and may own stock and options. A.U. is a member of American Board of Pediatrics, Subboard of Pediatric Gastroenterology. M.J.G. receives research funding to his institution from Gilead Sciences. V.K.S. is a consultant to AbbVie, Akcea Therapeutics, and Ariel Precision Medicine. D.C.W. receives royalties from UpToDate, serves as a consultant for AbbVie, Regeneron, and Ariel Precision Medicine, and may own stock and options. The other authors declare no conflict of interest.

The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

All authors developed the concept for the article through teleconferences and a face-to-face meeting. M.E.L., M.T.G, G.A.C., M.J.G., M.H., N.J.S., V.K.S., and D.C.W. wrote the first draft. All authors reviewed and edited the manuscript and approved the final version.

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