References
- Dancey JE, Bedard PL, Onetto N, et al. The genetic basis for cancer treatment decisions. Cell. 2012;148:409–420.
- Hanahan D, Weinberg RA. Hallmarks of cancer: the next generation. Cell. 2011;144:646–674.
- Biomarkers and surrogate endpoints: preferred definitions and conceptual framework. Clin Pharmacol Ther. 2001;69:89–95.
- DiMasi JA, Grabowski HG. Economics of new oncology drug development. J Clin Oncol Off J Am Soc Clin Oncol. 2007;25:209–216.
- Kola I, Landis J. Can the pharmaceutical industry reduce attrition rates? Nat Rev Drug Discov. 2004;3:711.
- Braiteh F, Kurzrock R. Uncommon tumors and exceptional therapies: paradox or paradigm? Mol Cancer Ther. 2007;6:1175–1179.
- Schwaederle M, Kurzrock R. Actionability and precisiononcology. Oncoscience. 2(10):779.
- U.S. Department of Health and Human Services. Developing and labeling in vitro companion diagnostic devices for a specific group or class of oncology therapeutic products- guidance for industry. 2018;11. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/developing-and-labeling-vitro-companion-diagnostic-devices-specific-group-or-class-oncology
- Oldenhuis CNAM, Oosting SF, Gietema JA, et al. Prognostic versus predictive value of biomarkers in oncology. Eur J Cancer Oxf Engl 1990. 2008;44:946–953.
- Schwaederle M, Daniels GA, Piccioni DE, et al. On the road to precision cancer medicine: analysis of genomic biomarker actionability in 439 patients. Mol Cancer Ther. 2015;14:1488–1494.
- Mullard A. FDA drug approvals. Nat Rev Drug Discov. 2019;18:85.
- Drilon A, Laetsch TW, Kummar S, et al. Efficacy of larotrectinib in TRK fusion–positive cancers in adults and children. N Engl J Med. 2018;378:731–739.
- Hay M, Thomas DW, Craighead JL, et al. Clinical development success rates for investigational drugs. Nat Biotechnol. 2014;32:40–51.
- Jardim DL, Groves ES, Breitfeld PP, et al. Factors associated with failure of oncology drugs in late-stage clinical development: a systematic review. Cancer Treat Rev. 2017;52:12–21.
- Nixon NA, Khan OF, Imam H, et al. Drug development for breast, colorectal, and non-small cell lung cancers from 1979 to 2014: cancer drug development from 1979–2014. Cancer. 2017;123:4672–4679.
- Schwaederle M, Zhao M, Lee JJ, et al. Association of biomarker-based treatment strategies with response rates and progression-free survival in refractory malignant neoplasms: a meta-analysis. JAMA Oncol. 2016;2:1452.
- Schwaederle M, Zhao M, Lee JJ, et al. Impact of precision medicine in diverse cancers: a meta-analysis of phase II clinical trials. J Clin Oncol. 2015;33:3817–3825.
- Fontes Jardim DL, Schwaederle M, Wei C, et al. Impact of a biomarker-based strategy on oncology drug development: a meta-analysis of clinical trials leading to FDA approval. J Natl Cancer Inst. 2015;107:djv253.
- Le Tourneau C, Delord J-P, Gonçalves A, et al. Molecularly targeted therapy based on tumour molecular profiling versus conventional therapy for advanced cancer (SHIVA): a multicentre, open-label, proof-of-concept, randomised, controlled phase 2 trial. Lancet Oncol. 2015;16:1324–1334.
- IQVIA Institute for Human Data Science. Global oncology trends 2018 - innovation, expansion and disruption. 2018 May.
- Chawla A, Janku F, Wheler JJ, et al. Estimated cost of anticancer therapy directed by comprehensive genomic profiling in a single-center study. JCO Precis Oncol. 2018;1–11.DOI: 10.1200/po.18.00074.
- Mak K-K, Pichika MR. Artificial intelligence in drug development: present status and future prospects. Drug Discov Today. 2019;24:773–780.
- Shimazawa R, Ikeda M. Drug–diagnostic co-development: challenges and issues. Expert Rev Mol Diagn. 2016;16:187–204.
- U.S. Food and Drug Administration, editor. Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review [Internet]. [cited 2019 Sep 15]. Available from: https://www.fda.gov/patients/learn-about-drug-and-device-approvals/fast-track-breakthrough-therapy-accelerated-approval-priority-review
- Stewart DJ, Whitney SN, Kurzrock R. Equipoise lost: ethics, costs, and the regulation of cancer clinical research. J Clin Oncol. 2010;28:2925–2935.
- Department of Health and Human Services, U.S. Food and Drug Administration. Projects receiving critical path support fiscal year 2008 [Internet]. The critical path initiative Transforming the way FDA-regulated products are developed, evaluated, manufactured, and used; 2009 [cited 2019 Sept 15]. Available from: http://www.fda.gov/oc/initiatives/criticalpath/
- Jardim DL, Schwaederle M, Hong DS, et al. An appraisal of drug development timelines in the Era of precision oncology. Oncotarget [Internet]. 2016;7. [cited 2019 Jul 25]. Available from: http://www.oncotarget.com/fulltext/10588
- Harrington JA, Hernandez-Guerrero TC, Basu B. Early phase clinical trial designs – state of play and adapting for the future. Clin Oncol. 2017;29:770–777.
- Park JJH, Siden E, Zoratti MJ, et al. Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols. Trials. 2019;20:572.
- Le Tourneau C, Borcoman E, Kamal M. Molecular profiling in precision medicine oncology. Nat Med. 2019;25:711–712.
- Sicklick JK, Kato S, Okamura R, et al. Molecular profiling of cancer patients enables personalized combination therapy: the I-PREDICT study. Nat Med. 2019;25:744–750.
- Rothwell DG, Ayub M, Cook N, et al. Utility of ctDNA to support patient selection for early phase clinical trials: the TARGET study. Nat Med. 2019;25:738–743.
- Rodon J, Soria J-C, Berger R, et al. Genomic and transcriptomic profiling expands precision cancer medicine: the WINTHER trial. Nat Med. 2019;25:751–758.
- Mateo J, Chakravarty D, Dienstmann R, et al. A framework to rank genomic alterations as targets for cancer precision medicine: the ESMO scale for clinical actionability of molecular targets (ESCAT). Ann Oncol. 2018;29:1895–1902.
- Meric-Bernstam F, Mills GB. Overcoming implementation challenges of personalized cancer therapy. Nat Rev Clin Oncol. 2012;9:542–548.
- de Gramont A, Watson S, Ellis LM, et al. Pragmatic issues in biomarker evaluation for targeted therapies in cancer. Nat Rev Clin Oncol. 2015;12:197–212.
- Janiaud P, Serghiou S, Ioannidis JPA. New clinical trial designs in the era of precision medicine: an overview of definitions, strengths, weaknesses, and current use in oncology. Cancer Treat Rev. 2019;73:20–30.
- National Cancer Institute. Tumor-agnostic therapy [Internet]. NCI Dict. Cancer Terms. [cited 2019 Aug 28]. Available from: cancer.gov/publications/dictionaries/cancer-terms/def/796871
- Hierro C, Matos I, Martin-Liberal J, et al. Agnostic-histology approval of new drugs in oncology: are we already there? Clin Cancer Res. 2019;25:3210–3219.
- FDA. FDA grants accelerated approval to pembrolizumab for first tissue/site agnostic indication. 2017 May 30 [cited 2019 Sept 19]. Available from: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-pembrolizumab-first-tissuesite-agnostic-indication
- Marcus L, Lemery SJ, Keegan P, et al. FDA approval summary: pembrolizumab for the treatment of microsatellite instability-high solid tumors. Clin Cancer Res. 2019;25:3753–3758.
- FDA. FDA approves entrectinib for NTRK solid tumors and ROS-1. NSCLC. 2019 Aug 16 [cited 2019 Sept 19]. Available from: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-entrectinib-ntrk-solid-tumors-and-ros-1-nsclc
- FDA. FDA approves larotrectinib for solid tumors with NTRK gene fusions. 2018 Dec 14 [cited 2019 Sept 19]. Available from: https://www.fda.gov/drugs/fda-approves-larotrectinib-solid-tumors-ntrk-gene-fusions-0
- Demetri GD, Paz-Ares L, Farago AF, et al. LBA4Efficacy and safety of entrectinib in patients with NTRK fusion-positive tumours: pooled analysis of STARTRK-2, STARTRK-1, and ALKA-372-001. Ann Oncol [Internet]. 2018 [cited 2018 Oct 19]. 29. Available from: https://academic.oup.com/annonc/article/doi/10.1093/annonc/mdy483.003/5200236
- Havel JJ, Chowell D, Chan TA. The evolving landscape of biomarkers for checkpoint inhibitor immunotherapy. Nat Rev Cancer. 2019;19:133–150.
- FDA label. [Internet]. [cited 2019 Nov 18]. Available from: https://nctr-crs.fda.gov/fdalabel/ui/search
- Yamashita K, Iwatsuki M, Harada K, et al. Prognostic impacts of the combined positive score and the tumor proportion score for programmed death ligand-1 expression by double immunohistochemical staining in patients with advanced gastric cancer. Gastric Cancer [Internet]. 2019. DOI:10.1007/s10120-019-00999-9
- Heeke S, Hofman P. Tumor mutational burden assessment as a predictive biomarker for immunotherapy in lung cancer patients: getting ready for prime-time or not? Transl Lung Cancer Res. 2018;7:631–638.
- Hellmann MD, Paz-Ares L, Bernabe Caro R, et al. Nivolumab plus Ipilimumab in advanced non–small-cell lung cancer. N Engl J Med [Internet]. 381:2020–2031. [cited 2019 Nov 18].
- Hellmann MD, Ciuleanu T-E, Pluzanski A, et al. Nivolumab plus Ipilimumab in lung cancer with a high tumor mutational burden. N Engl J Med. 2018;378:2093–2104.
- Smith AD, Roda D, Yap TA. Strategies for modern biomarker and drug development in oncology. J Hematol Oncol. 2014;7:70.
- Greaves M, Maley CC. Clonal evolution in cancer. Nature. 2012;481:306–313.
- Ileana Dumbrava E, Meric-Bernstam F, Yap TA. Challenges with biomarkers in cancer drug discovery and development. Expert Opin Drug Discov. 2018;13:685–690.