TY - JOUR
T1 - Accelerating the drug delivery pipeline for acute and chronic pancreatitis
T2 - 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
AU - Lowe, Mark E.
AU - Goodman, Marc T.
AU - Coté, Gregory A.
AU - Glesby, Marshall J.
AU - Haupt, Mark
AU - Schork, Nicholas J.
AU - Singh, Vikesh K.
AU - Andersen, Dana K.
AU - Pandol, Stephen J.
AU - Uc, Aliye
AU - Whitcomb, David C.
N1 - Publisher Copyright:
© 2018 Wolters Kluwer Health, Inc. All rights reserved.
PY - 2018/11/1
Y1 - 2018/11/1
N2 - 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.
AB - 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.
KW - drug trials
KW - pancreatitis
KW - patient-reported outcomes
UR - http://www.scopus.com/inward/record.url?scp=85055081016&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85055081016&partnerID=8YFLogxK
U2 - 10.1097/MPA.0000000000001164
DO - 10.1097/MPA.0000000000001164
M3 - Article
C2 - 30325857
AN - SCOPUS:85055081016
SN - 0885-3177
VL - 47
SP - 1193
EP - 1199
JO - Pancreas
JF - Pancreas
IS - 10
ER -