Granulocyte‐macrophage colony‐stimulating factor as adjunct therapy in relapsed lymphoid malignancy

Implications for economic analyses of phase III clinical trials

C. L. Bennett, S. L. George, James Olen Armitage, Julie Marie Vose, J. L. Armitage, J. O. Armitage, N. C. Gorin, S. C. Gulati

Research output: Contribution to journalArticle

25 Citations (Scopus)

Abstract

With the increasing concern over the high cost of health care, policy makers have incorporated economic analyses into phase III clinical trials as the randomized clinical trials can provide important information on the efficacy and potential cost‐effectiveness of new pharmaceutical agents. Economic analyses of single‐hospital experience during phase III trials of granulocyte‐macrophage colony‐stimulating factor (GM‐CSF) as adjunct therapy for high dose chemotherapy with autologous stem cell support found significant shortening of neutropenia with GM‐CSF at each hospital, but shortened hospitalization (and lower costs) at only two of three hospitals. In this study, we added data from three additional hospitals and found that the 103 patients who received GM‐CSF had, on average, 5.7 days shorter durations of severe neutropenia than the 95 patients who received placebo (p < 0.0001) and 3.4 days shorter in hospitalization (p = 0.06). However, the duration of hospitalization, the primary determinant of health care costs, was shorter for GM‐CSF patients in only four of the six centers and the duration of hospitalization of placebo patients was shorter at the other two centers. Careful analyses must be carried out when phase III clinical trial results are used to derive estimates of cost‐effectiveness of new pharmaceutical agents. The interpretation of economic analyses of phase III clinical trials raises issues related to the perspective of the investigators, study design, collection of data on resource utilization, learning curve effects and generalizability of the results to other settings.

Original languageEnglish (US)
Pages (from-to)414-420
Number of pages7
JournalSTEM CELLS
Volume13
Issue number4
DOIs
StatePublished - Jan 1 1995

Fingerprint

Phase III Clinical Trials
Hospitalization
Economics
Neutropenia
Neoplasms
Placebos
Costs and Cost Analysis
Learning Curve
Therapeutics
Health Policy
Administrative Personnel
Pharmaceutical Preparations
Health Care Costs
Primary Health Care
Stem Cells
Randomized Controlled Trials
Research Personnel
Drug Therapy

Keywords

  • Autologous stem cell transplantation
  • Cost‐effective
  • Economics
  • Granulocyte‐macrophage colony‐stimulating factor

ASJC Scopus subject areas

  • Molecular Medicine
  • Developmental Biology
  • Cell Biology

Cite this

Granulocyte‐macrophage colony‐stimulating factor as adjunct therapy in relapsed lymphoid malignancy : Implications for economic analyses of phase III clinical trials. / Bennett, C. L.; George, S. L.; Armitage, James Olen; Vose, Julie Marie; Armitage, J. L.; Armitage, J. O.; Gorin, N. C.; Gulati, S. C.

In: STEM CELLS, Vol. 13, No. 4, 01.01.1995, p. 414-420.

Research output: Contribution to journalArticle

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