Which Metrics Are Appropriate to Describe the Value of New Cancer Therapies?

Johnson P, Greiner W, Al-Dakkak I, Wagner S (2015)
BioMed Research International 2015: 865101.

Zeitschriftenaufsatz | Veröffentlicht | Englisch
 
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Autor*in
Johnson, Peter; Greiner, WolfgangUniBi; Al-Dakkak, Imad; Wagner, Samuel
Abstract / Bemerkung
Patients with certain cancers are treated with curative intent, but for others the results are less favorable and different therapeutic approaches are needed. Early data suggest that new therapies, which modulate immune responses to cancers, may have potential for long-termsurvival in a proportion of cases. Therefore, it is timely to consider whether metrics generally used to describe the medical value of therapies for patients with common solid tumors remain appropriate for therapies with curative potential. Literature reviews were conducted to define how various stakeholders describe cure in oncology and to identify the endpoints used in clinical trials for selected solid tumors. The results showed that "cure" is described using various terms that can be divided broadly into lack of disease progression, eradication of cancerous cells, and survival. The review of trial endpoints showed frequent use of median overall survival (OS) and progression-and response-related endpoints. Because these endpoints were mainly described in the context of chemotherapies that are not generally curative, they may not adequately capture outcomes of new therapeutic modalities with potential for long-term survival. More appropriate endpoints may include mean OS, cure fraction, and OS rate at landmark time points.
Erscheinungsjahr
2015
Zeitschriftentitel
BioMed Research International
Band
2015
Seite(n)
865101
ISSN
2314-6133
Page URI
https://pub.uni-bielefeld.de/record/2764394

Zitieren

Johnson P, Greiner W, Al-Dakkak I, Wagner S. Which Metrics Are Appropriate to Describe the Value of New Cancer Therapies? BioMed Research International. 2015;2015:865101.
Johnson, P., Greiner, W., Al-Dakkak, I., & Wagner, S. (2015). Which Metrics Are Appropriate to Describe the Value of New Cancer Therapies? BioMed Research International, 2015, 865101. doi:10.1155/2015/865101
Johnson, P., Greiner, W., Al-Dakkak, I., and Wagner, S. (2015). Which Metrics Are Appropriate to Describe the Value of New Cancer Therapies? BioMed Research International 2015, 865101.
Johnson, P., et al., 2015. Which Metrics Are Appropriate to Describe the Value of New Cancer Therapies? BioMed Research International, 2015, p 865101.
P. Johnson, et al., “Which Metrics Are Appropriate to Describe the Value of New Cancer Therapies?”, BioMed Research International, vol. 2015, 2015, pp. 865101.
Johnson, P., Greiner, W., Al-Dakkak, I., Wagner, S.: Which Metrics Are Appropriate to Describe the Value of New Cancer Therapies? BioMed Research International. 2015, 865101 (2015).
Johnson, Peter, Greiner, Wolfgang, Al-Dakkak, Imad, and Wagner, Samuel. “Which Metrics Are Appropriate to Describe the Value of New Cancer Therapies?”. BioMed Research International 2015 (2015): 865101.

43 References

Daten bereitgestellt von Europe PubMed Central.

Review of oncology and hematology drug product approvals at the US Food and Drug Administration between July 2005 and December 2007.
Sridhara R, Johnson JR, Justice R, Keegan P, Chakravarty A, Pazdur R., J. Natl. Cancer Inst. 102(4), 2010
PMID: 20118413
Raising the bar: the curative potential of human cancer immunotherapy.
Rosenberg SA., Sci Transl Med 4(127), 2012
PMID: 22461638
Pooled analysis of long-term survival data from phase II and phase III trials of ipilimumab in metastatic or locally advanced, unresectable melanoma
Schadendorf D., Hodi F., Robert C.., 0
Immunotherapy of cancer in 2012.
Kirkwood JM, Butterfield LH, Tarhini AA, Zarour H, Kalinski P, Ferrone S., CA Cancer J Clin 62(5), 2012
PMID: 22576456
Development of ipilimumab: a novel immunotherapeutic approach for the treatment of advanced melanoma.
Wolchok JD, Hodi FS, Weber JS, Allison JP, Urba WJ, Robert C, O'Day SJ, Hoos A, Humphrey R, Berman DM, Lonberg N, Korman AJ., Ann. N. Y. Acad. Sci. 1291(), 2013
PMID: 23772560
Efficacy and safety of ipilimumab in metastatic melanoma patients surviving more than 2 years following treatment in a phase III trial (MDX010-20).
McDermott D, Haanen J, Chen TT, Lorigan P, O'Day S; MDX010-20 Investigators., Ann. Oncol. 24(10), 2013
PMID: 23942774
Survival and long-term safety in patients with advanced solid tumors receiving nivolumab (anti-PD-1; BMS-936558; ONO-4538)
Hodi F., Topalian S., Brahmer J.., 0
Safety and tumor responses with lambrolizumab (anti-PD-1) in melanoma.
Hamid O, Robert C, Daud A, Hodi FS, Hwu WJ, Kefford R, Wolchok JD, Hersey P, Joseph RW, Weber JS, Dronca R, Gangadhar TC, Patnaik A, Zarour H, Joshua AM, Gergich K, Elassaiss-Schaap J, Algazi A, Mateus C, Boasberg P, Tumeh PC, Chmielowski B, Ebbinghaus SW, Li XN, Kang SP, Ribas A., N. Engl. J. Med. 369(2), 2013
PMID: 23724846
Clinical activity, safety and biomarkers of PD-L1 blockade in non-small cell lung cancer (NSCLC): additional analyses from a clinical study of the engineered antibody MPDL3280A (anti-PDL1)
Soria J., Cruz C., Bahleda R.., 0
How cancer patients value hope and the implications for cost-effectiveness assessments of high-cost cancer therapies.
Lakdawalla DN, Romley JA, Sanchez Y, Maclean JR, Penrod JR, Philipson T., Health Aff (Millwood) 31(4), 2012
PMID: 22492883
Cure models as a useful statistical tool for analyzing survival.
Othus M, Barlogie B, Leblanc ML, Crowley JJ., Clin. Cancer Res. 18(14), 2012
PMID: 22675175
Minimal residual disease and discontinuation of therapy in chronic myeloid leukemia: can we aim at a cure?
Melo J., Ross D.., 2011
Is going for cure in chronic myeloid leukemia possible and justifiable?
Mahon F.-X.., 2012
New challenges in endpoints for drug development in advanced melanoma.
Ribas A, Hersey P, Middleton MR, Gogas H, Flaherty KT, Sondak VK, Kirkwood JM., Clin. Cancer Res. 18(2), 2011
PMID: 22142824
Methodological aspects of lung cancer clinical trials in the era of targeted agents.
Di Maio M, Gallo C, De Maio E, Morabito A, Piccirillo MC, Gridelli C, Perrone F., Lung Cancer 67(2), 2009
PMID: 19875193
Treatment of patients with advanced non-small cell lung cancer
Dooms C., Vansteenkiste J.., 2004
Endpoints, patient selection, and biomarkers in the design of clinical trials for cancer vaccines.
Bilusic M, Gulley JL., Cancer Immunol. Immunother. 61(1), 2011
PMID: 22120693
Improved survival with ipilimumab in patients with metastatic melanoma.
Hodi FS, O'Day SJ, McDermott DF, Weber RW, Sosman JA, Haanen JB, Gonzalez R, Robert C, Schadendorf D, Hassel JC, Akerley W, van den Eertwegh AJ, Lutzky J, Lorigan P, Vaubel JM, Linette GP, Hogg D, Ottensmeier CH, Lebbe C, Peschel C, Quirt I, Clark JI, Wolchok JD, Weber JS, Tian J, Yellin MJ, Nichol GM, Hoos A, Urba WJ., N. Engl. J. Med. 363(8), 2010
PMID: 20525992
Safety, activity, and immune correlates of anti-PD-1 antibody in cancer.
Topalian SL, Hodi FS, Brahmer JR, Gettinger SN, Smith DC, McDermott DF, Powderly JD, Carvajal RD, Sosman JA, Atkins MB, Leming PD, Spigel DR, Antonia SJ, Horn L, Drake CG, Pardoll DM, Chen L, Sharfman WH, Anders RA, Taube JM, McMiller TL, Xu H, Korman AJ, Jure-Kunkel M, Agrawal S, McDonald D, Kollia GD, Gupta A, Wigginton JM, Sznol M., N. Engl. J. Med. 366(26), 2012
PMID: 22658127
The ends justify the mean: outcome measures for estimating the value of new cancer therapies
Davies A., Briggs A., Schneider J.., 2012
American Society of Clinical Oncology perspective: Raising the bar for clinical trials by defining clinically meaningful outcomes.
Ellis LM, Bernstein DS, Voest EE, Berlin JD, Sargent D, Cortazar P, Garrett-Mayer E, Herbst RS, Lilenbaum RC, Sima C, Venook AP, Gonen M, Schilsky RL, Meropol NJ, Schnipper LE., J. Clin. Oncol. 32(12), 2014
PMID: 24638016
Prednisone plus cabazitaxel or mitoxantrone for metastatic castration-resistant prostate cancer progressing after docetaxel treatment: a randomised open-label trial.
de Bono JS, Oudard S, Ozguroglu M, Hansen S, Machiels JP, Kocak I, Gravis G, Bodrogi I, Mackenzie MJ, Shen L, Roessner M, Gupta S, Sartor AO; TROPIC Investigators., Lancet 376(9747), 2010
PMID: 20888992
Cabazitaxel or mitoxantrone with prednisone in patients with metastatic castration-resistant prostate cancer (mCRPC) previously treated with docetaxel: estimating mean overall survival (OS) for health economic analyses from a phase III trial (TROPIC)
Oudard S., Joulain A., de A., Sartor O.., 2011
Paclitaxel plus oxaliplatin for recurrent or metastatic cervical cancer: a New York Cancer Consortium Study.
Kuo DY, Blank SV, Christos PJ, Kim M, Caputo TA, Pothuri B, Hershman D, Goldman N, Ivy PS, Runowicz CD, Muggia F, Goldberg GL, Einstein MH., Gynecol. Oncol. 116(3), 2009
PMID: 19931137
Persistent overall survival benefit and no increased risk of second malignancies with bortezomib-melphalan-prednisone versus melphalan-prednisone in patients with previously untreated multiple myeloma.
San Miguel JF, Schlag R, Khuageva NK, Dimopoulos MA, Shpilberg O, Kropff M, Spicka I, Petrucci MT, Palumbo A, Samoilova OS, Dmoszynska A, Abdulkadyrov KM, Delforge M, Jiang B, Mateos MV, Anderson KC, Esseltine DL, Liu K, Deraedt W, Cakana A, van de Velde H, Richardson PG., J. Clin. Oncol. 31(4), 2012
PMID: 23233713
Bevacizumab maintenance in patients with advanced non-small-cell lung cancer, clinical patterns, and outcomes in the Eastern Cooperative Oncology Group 4599 Study: results of an exploratory analysis.
Lopez-Chavez A, Young T, Fages S, Leon L, Schiller JH, Dowlati A, Brahmer JR, Johnson DH, Sandler A., J Thorac Oncol 7(11), 2012
PMID: 23059774
Estimating and modeling the cure fraction in population-based cancer survival analysis.
Lambert PC, Thompson JR, Weston CL, Dickman PW., Biostatistics 8(3), 2006
PMID: 17021277
The cure fraction of glioblastoma multiforme.
Smoll NR, Schaller K, Gautschi OP., Neuroepidemiology 39(1), 2012
PMID: 22776797
Temporal trends in the proportion cured for cancer of the colon and rectum: a population-based study using data from the Finnish Cancer Registry.
Lambert PC, Dickman PW, Osterlund P, Andersson T, Sankila R, Glimelius B., Int. J. Cancer 121(9), 2007
PMID: 17640061
Population-based survival-cure analysis of ER-negative breast cancer.
Huang L, Johnson KA, Mariotto AB, Dignam JJ, Feuer EJ., Breast Cancer Res. Treat. 123(1), 2010
PMID: 20130982
Guideline on the evaluation of anticancer medicinal products in man
AUTHOR UNKNOWN, 0
Ipilimumab for previously treated advanced (unresectable or metastatic) melanoma
AUTHOR UNKNOWN, 0
Review of meta-analyses evaluating surrogate endpoints for overall survival in oncology.
Sherrill B, Kaye JA, Sandin R, Cappelleri JC, Chen C., Onco Targets Ther 5(), 2012
PMID: 23109809
Guidelines for the evaluation of immune therapy activity in solid tumors: immune-related response criteria.
Wolchok JD, Hoos A, O'Day S, Weber JS, Hamid O, Lebbe C, Maio M, Binder M, Bohnsack O, Nichol G, Humphrey R, Hodi FS., Clin. Cancer Res. 15(23), 2009
PMID: 19934295
Evolution of end points for cancer immunotherapy trials.
Hoos A., Ann. Oncol. 23 Suppl 8(), 2012
PMID: 22918928
Sipuleucel-T immunotherapy for castration-resistant prostate cancer.
Kantoff PW, Higano CS, Shore ND, Berger ER, Small EJ, Penson DF, Redfern CH, Ferrari AC, Dreicer R, Sims RB, Xu Y, Frohlich MW, Schellhammer PF; IMPACT Study Investigators, Ahmed T, Amin A, Arseneau J, Barth N, Bernstein G, Bracken B, Burch P, Caggiano V, Chin J, Chodak G, Chu F, Corman J, Curti B, Dawson N, Deeken JF, Dubernet T, Fishman M, Flanigan R, Gailani F, Garbo L, Gardner T, Gelmann E, George D, Godfrey T, Gomella L, Guerra M, Hall S, Hanson J, Israeli R, Jancis E, Jewett MA, Kassabian V, Katz J, Klotz L, Koeneman K, Koh H, Kratzke R, Lance R, Lech J, Leichman L, Lemon R, Liang J, Libertino J, Lilly M, Malik I, Martin SE, McCaffrey J, McLeod D, McNeel D, Miles B, Murdock M, Nabhan C, Nemunaitis J, Notter D, Pantuck A, Perrotte P, Pessis D, Petrylak D, Polikoff J, Pommerville P, Ramanathan S, Rarick M, Richards J, Rifkin R, Rohatgi N, Rosenbluth R, Santucci R, Sayegh A, Seigne J, Shapira I, Shedhadeh N, Shepherd D, Sridhar S, Stephenson R, Teigland C, Thaker N, Vacirca J, Villa L Jr, Vogelzang N, Wertheim M, Wolff JH, Wurzel R, Yang C, Young J., N. Engl. J. Med. 363(5), 2010
PMID: 20818862
Overall survival analysis of a phase II randomized controlled trial of a Poxviral-based PSA-targeted immunotherapy in metastatic castration-resistant prostate cancer.
Kantoff PW, Schuetz TJ, Blumenstein BA, Glode LM, Bilhartz DL, Wyand M, Manson K, Panicali DL, Laus R, Schlom J, Dahut WL, Arlen PM, Gulley JL, Godfrey WR., J. Clin. Oncol. 28(7), 2010
PMID: 20100959
Meaningful change in oncology quality-of-life instruments: a systematic literature review.
Bedard G, Zeng L, Lam H, Cella D, Zhang L, Lauzon N, Chow E., Expert Rev Pharmacoecon Outcomes Res 12(4), 2012
PMID: 22971034

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