title: Pragmatic study designs for older adults with cancer: Report from the U13 conference.
abstract: Cancer is a disease occurring disproportionately in older adults. However, the evidence base regarding how best to care for these patients remains limited due to their underrepresentation in cancer clinical trials. Pragmatic clinical trials represent a promising approach for enhancing the evidence base in geriatric oncology by allowing investigators to enroll older, frailer patients onto cancer clinical trials. These trials are more accessible, less resource intensive, and place minimal additional burden on participating patients. Additionally, these trials can be designed to measure endpoints directly relevant to older adults, such as quality of life, functional independence and treatment tolerability which are often not addressed in standard clinical trials. Therefore, pragmatic clinical trials allow researchers to include patients for whom the treatment will ultimately be applied and to utilize meaningful endpoints. Examples of pragmatic studies include both large, simple trials and cluster randomized trials. These study designs allow investigators to conduct clinical trials within the context of everyday practice. Further, researchers can devise these studies to place minimal burden on the patient, the treating clinicians and the participating institutions. In order to be successful, pragmatic trials must efficiently utilize the electronic medical record for data capture while also maximizing patient recruitment, enrollment and retention. Additionally, by strategically utilizing pragmatic clinical trials to test therapies and interventions that have previously shown efficacy in younger, fitter patients, these trials represent a potential mechanism to improve the evidence base in geriatric oncology and enhance care for older adults with cancer.
authors.affiliation: Massachusetts General Hospital Cancer Center & Harvard Medical School, Department of Medicine, Division of Hematology & Oncology, Boston, MA, USA. Electronic address: RNipp@MGH.Harvard.edu.
authors.forename: Ryan D
authors.initials: RD
authors.lastname: Nipp
authors.affiliation: University of Virginia Cancer Center, Department of Public Health Sciences, Charlottesville, VA, USA.
authors.forename: Nengliang Aaron
authors.initials: NA
authors.lastname: Yao
authors.affiliation: University of Texas MD Anderson Cancer Center, Department of Health Services Research, Division of OVP, Cancer Prevention and Population Sciences, Houston, TX, USA.
authors.forename: Lisa M
authors.initials: LM
authors.lastname: Lowenstein
authors.affiliation: Mayo Clinic, Department of Oncology, Division of Medical Oncology, Rochester, MN, USA.
authors.forename: Jan C
authors.initials: JC
authors.lastname: Buckner
authors.affiliation: Thomas Jefferson University, Philadelphia, PA; La Jolla, CA, USA.
authors.forename: Ira R
authors.initials: IR
authors.lastname: Parker
authors.affiliation: SUNY Upstate University, Department of Medicine, Syracuse, NY, USA; VA Medical Center, Syracuse, NY, USA.
authors.forename: Ajeet
authors.initials: A
authors.lastname: Gajra
authors.affiliation: University of Minnesota, Veterans Affairs Medical Center, Minneapolis, MN, USA.
authors.forename: Vicki A
authors.initials: VA
authors.lastname: Morrison
authors.affiliation: University of Chicago, Section of Geriatrics & Palliative Medicine, Chicago, IL, USA.
authors.forename: William
authors.initials: W
authors.lastname: Dale
authors.affiliation: Weill Cornell Medical College, Division of Biostatistics and Epidemiology, New York, NY 10065, USA.
authors.forename: Karla V
authors.initials: KV
authors.lastname: Ballman
mesh_terms: D000367:Age Factors; D000368:Aged; D019317:Evidence-Based Medicine / Q000592:standards; D006801:Humans; D008495:Medical Oncology / Q000592:standards*; D009369:Neoplasms / Q000628:therapy*; D018579:Patient Selection; D064792:Pragmatic Clinical Trials as Topic / Q000379:methods / Q000592:standards*; D011788:Quality of Life; D012107:Research Design / Q000592:standards*
keywords: Aging; Cancer; Clinical Trial; Frail Older Adults; Geriatrics; Pragmatic Clinical Trials; Quality of Life