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

Mark E. Lowe, Marc T. Goodman, Gregory A. Coté, Marshall J. Glesby, Mark Haupt, Nicholas J. Schork, Vikesh K. Singh, Dana Andersen, Stephen J. Pandol, Aliye Uc, David C. Whitcomb

Research output: Contribution to journalArticle

Abstract

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.

LanguageEnglish (US)
Pages1193-1199
Number of pages7
JournalPancreas
Volume47
Issue number10
DOIs
StatePublished - Nov 1 2018

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National Institute of Diabetes and Digestive and Kidney Diseases (U.S.)
Chronic Pancreatitis
Pancreatitis
Education
Pharmaceutical Preparations
Outcome Assessment (Health Care)
Genetic Testing
Pancreatic Neoplasms
Chronic Pain
Sample Size
Biomarkers
Clinical Trials
Inflammation
Morbidity
Therapeutics
Research

Keywords

  • drug trials
  • pancreatitis
  • patient-reported outcomes

ASJC Scopus subject areas

  • Internal Medicine
  • Endocrinology, Diabetes and Metabolism
  • Hepatology
  • Endocrinology

Cite this

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.; Goodman, Marc T.; Coté, Gregory A.; Glesby, Marshall J.; Haupt, Mark; Schork, Nicholas J.; Singh, Vikesh K.; Andersen, Dana; Pandol, Stephen J.; Uc, Aliye; Whitcomb, David C.

In: Pancreas, Vol. 47, No. 10, 01.11.2018, p. 1193-1199.

Research output: Contribution to journalArticle

Lowe, Mark E. ; Goodman, Marc T. ; Coté, Gregory A. ; Glesby, Marshall J. ; Haupt, Mark ; Schork, Nicholas J. ; Singh, Vikesh K. ; Andersen, Dana ; Pandol, Stephen J. ; Uc, Aliye ; Whitcomb, David C. / 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. In: Pancreas. 2018 ; Vol. 47, No. 10. pp. 1193-1199.
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