Simulating the contribution of a biospecimen and clinical data repository in a phase II clinical trial: A value of information analysis

Abstract

The potential contributions of a centralized data warehouse or repository in clinical research include the expedited accrual of subjects for phase II trials. Understanding the contribution of data warehouses that integrate clinical, biospecimen, and molecular data for the conduct of clinical trials is essential to inform private and public decisions on resource allocation and investment. We conducted a value of information analysis using data from recent trials at the Moffitt Cancer Center and simulated the potential reductions in trial size due to possible alternative scenarios of expedited accrual. In this study, we compared alternative data sets using a single model to assess value of information. Our findings suggest that the reductions in trial size range from 0% to 43%, depending on the amount of censoring in overall survival. The ability to expedite the accrual of patients for clinical trial studies using large data repositories that store data on inclusion/exclusion criteria and response to standard of care therapies demonstrated significant improvement in reducing the number of subjects needed to achieve similar end-results, as evaluated using value of information analysis with a limited number of parameters and a parsimonious model of overall survival.

Keywords

value of information clinical trials phase II neoplasms biorepository

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References

Elseman

Haga

. A handbook of human tissue sources: a national resource of human tissue samples, Santa Monica, CA: RAND Corporation, 2000.

Fullerton SM, Anderson NR, Guzauskas G, et al. Meeting the governance challenges of next-generation biorepository research. Sci Transl Med [serial on the Internet] 2010; 2(15).Available from: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&dopt=Citation&list_uids=20371468 (accessed 8 August 2010).

Ambrosone CB, Nesline MK, Davis W. Establishing a cancer center data bank and biorepository for multidisciplinary research. Cancer Epidemiol Biomarkers Prev[serial on the Internet] 2006; 15(9).Available from: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&dopt=Citation&list_uids=16985014 (accessed 8 August 2010).

Compton CC. The surgical specimen is the personalized part of personalized cancer medicine. Ann Surg Oncol [serial on the Internet] 2009; 2711907; 16(8). Available from: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&dopt=Citation&list_uids=19472010 (accessed 2 July 2010).

Park A. 10 Ideas changing the world right now: biobanks. TIME in Partnership with CNN. 2009.

Silberman S. Libraries of flesh: the sorry state of human tissue storage. WIRED Magazine. June 2010.

Compton C. Getting to personalized cancer medicine: taking out the garbage. Cancer [serial on the Internet] 2007; 110(8). Available from: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&dopt=Citation&list_uids=17763370 (accessed 6 August 2010).

Office of Biorepositories and Biospecimen Research (OBBR). Biospecimen research network. In: Institute NC (ed) Fact sheet. Bethesda, MD: NIH, 2009, pp. 1–2.

Schaffer E. Of biobanks and biospecimens. The NIH Catalyst, 2009; Section 1,pp. 16–18.

10.

National Institutes of Health. ARRA investments in cancer biospecimens and biorepositories. Bethesda, MD: NIH, 2010 [updated 14 Jan 2010]. Available from: http://report.nih.gov/recovery/investmentreports/ViewARRAInvRpt.aspx?csid=274.

11.

Ginsburg GS, Burke TW and Febbo P. Centralized biorepositories for genetic and genomic research. JAMA [serial on the Internet] 2008; 299(11). Available from: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&dopt=Citation&list_uids=18349099 (accessed 6 August 2010).

12.

Goodman MT, Hernandez BY, Hewitt S, et al. Tissues from population-based cancer registries: a novel approach to increasing research potential. Hum Pathol [serial on the Internet] 2005; 36(7). Available from: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&dopt=Citation&list_uids=16084952 (accessed 6 August 2010).

13.

Greely HT. The uneasy ethical and legal underpinnings of large-scale genomic biobanks. Annu Rev Genomics Hum Genet [serial on the Internet] 2007; 8. Available from: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&dopt=Citation&list_uids=17550341 (accessed 6 August 2010).

14.

Haga SB and Beskow LM. Ethical, legal, and social implications of biobanks for genetics research. Adv Genet [serial on the Internet] 2008; 60. Available from: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&dopt=Citation&list_uids=18358331 (accessed 6 August 2010).

15.

Moore HM, Compton CC, Lim MD, et al. 2009 Biospecimen research network symposium: advancing cancer research through biospecimen science. Cancer Res [serial on the Internet] 2009 2782665; 69(17). Available from: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&dopt=Citation&list_uids=19706749 (accessed 6 August 2010).

16.

Ness RB, Samet JM, Camargo CA, et al. American College of Epidemiology weighs in on new NCI Biorepository Guidelines. Ann Epidemiol[serial on the Internet] 2007; 17(1). Available from: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&dopt=Citation&list_uids=17140809 (accessed 6 August 2010).

17.

Berger

Ghosh

Mukhopadhyay

. Approximations and consistency of Bayes factors as model dimension grows. J Stat Plan Infer 2003; 112(1–2): 241–258.

18.

Antonia

Mirza

Fricke

. Combination of p53 cancer vaccine with chemotherapy in patients with extensive stage small cell lung cancer. Clin Cancer Res 2006; 12(3): 878–887.

19.

Bepler

Sommers

Cantor

. Clinical efficacy and predictive molecular markers of neoadjuvant gemcitabine and pemetrexed in resectable non-small cell lung cancer. J Thorac Oncol 2008; 3(10): 1112–1118.

20.

Chiappori

Simon

Williams

. Phase II study of first-line sequential chemotherapy with gemcitabine-carboplatin followed by docetaxel in patients with advanced non-small cell lung cancer. Oncology 2005; 68(4–6): 382–390.

21.

Chiappori A, Williams C, Northfelt D, et al. Obatoclax (GX17-070), a small molecule pan-bcl-2 inhibitor, in combination with docetaxel in a phase I/II trial enrolling patients with relapsed non-small cell lung cancer (NSCLC). EJC Suppl [Meeting Abstract] 2008; 6: 185.

22.

Daud A, Weber J, Urbas P, et al. Phase II trial of sagopilone (ZK-EPO), a novel synthetic epothilone, with significant activity in metastatic melanoma. J Clin Oncol[Meeting Abstract]. 2009;27(15): 9031.

23.

Fishman

Hunter

Soliman

. Phase II trial of B7-1 (CD-86) transduced, cultured autologous tumor cell vaccine plus subcutaneous interleukin-2 for treatment of stage IV renal cell carcinoma. J Immunother 2008; 31(1): 72–80.

24.

Moulder

Valkov

Neuger

. Phase 2 study of gemcitabine and irinotecan in metastatic breast cancer with correlatives to determine topoisomerase I localization as a predictor of response. Cancer 2008; 113(10): 2646–2654.

25.

Ramnath

Sommers

Robinson

. Phase II study of neoadjuvant chemotherapy with gemcitabine and vinorelbine in resectable non-small cell lung cancer. Chest 2005; 128(5): 3467–3474.

26.

Romero

Stanton

DeFelice

. Phase II trial of thalidomide and daily oral dexamethasone for treatment of hormone refractory prostate cancer progressing after chemotherapy. Urol Oncol-Semin Orig Investig 2007; 25(4): 284–290.

27.

Simon

Sharma

. Feasibility and efficacy of molecular analysis-directed individualized therapy in advanced non-small-cell lung cancer. J Clin Oncol 2007; 25(19): 2741–2746.

28.

Simon

Extermann

Chiappori

. Phase 2 trial of docetaxel and gefitinib in the first-line treatment of patients with advanced nonsmall-cell lung cancer (NSCLC) who are 70 years of age or older. Cancer 2008; 112(9): 2021–2029.

29.

Daud

Mirza

Lenox

. Phenotypic and functional analysis of dendritic cells and clinical outcome in patients with high-risk melanoma treated with adjuvant granulocyte macrophage colony-stimulating factor. J Clin Oncol 2008; 26(19): 3235–3241.

30.

Druker

. Translation of the Philadelphia chromosome into therapy for CML. Blood 2008; 112(13): 4808–4817.

31.

Ross

Slodkowska

Symmans

. The HER-2 receptor and breast cancer: ten years of targeted anti-HER-2 therapy and personalized medicine. Oncologist 2009; 14(4): 320–368.

32.

Bates

. Progress towards personalized medicine. Drug Discov Today 2010; 15(3–4): 115–120.

33.

Gonzalez-Angulo

Hennessy

Mills

. Future of personalized medicine in oncology: a systems biology approach. J Clin Oncol 2010; 28(16): 2777–2783.

34.

Garber

Tunis

. Does comparative-effectiveness research threaten personalized medicine? New Engl J Med 2009; 360(19): 1925–1927.

35.

Luce

Kramer

Goodman

. Rethinking randomized clinical trials for comparative effectiveness research: the need for transformational change. Ann Intern Med 2009; 151(3): 206–209.