Precision medicine: beyond genomics to targeted therapies

2016 ◽  
Vol 13 (2) ◽  
pp. 97-100
Author(s):  
Ralph Snyderman ◽  
David Spellmeyer
Keyword(s):  
Precision Medicine ◽  
Targeted Therapies

Impact of rapid multigene assays with short turnaround time (TAT) on the development of precision medicine for non-small cell lung cancer (NSCLC).

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 9094-9094
Author(s):  
Shingo Matsumoto ◽  
Takaya Ikeda ◽  
Kiyotaka Yoh ◽  
Akira Sugimoto ◽  
Terufumi Kato ◽  
...  
Keyword(s):  
Lung Cancer ◽  
Clinical Trials ◽  
Precision Medicine ◽  
Targeted Therapies ◽  
Advanced Nsclc ◽  
Genetic Alterations ◽  
Braf V600e ◽  
Gene Testing ◽  
Genomic Screening ◽  
Nsclc Patients

9094 Background: A variety of oncogene drivers have been identified in NSCLC and molecularly-stratified precision medicine has led to improved survival in advanced NSCLC. Next-generation sequencing (NGS)-based testing is utilized to detect actionable gene alterations; however, the TAT of NGS is often too long to translate into clinical decision making. Thus, rapid multi-gene testing alternatives are needed. Methods: A lung cancer genomic screening project (LC-SCRUM-Asia) capturing clinical outcome was established in 2013 to identify patients with oncogene drivers and to support the development of novel targeted therapies. Since February 2013 to May 2019 (LC-SCRUM-Asia 1st-phase), single gene testing and/or a targeted NGS assay, Oncomine Comprehensive Assay (OCA), were used for the genomic screening. Since June 2019 to December 2020 (2nd-phase), a multi-gene PCR assay (Amoy 9-in-1 test) and a rapid NGS assay (Genexus/Oncomine Precision Assay [OPA]) were also implemented as rapid multi-gene testing. Results: A total of 10667 Japanese NSCLC patients, including 6826 in the 1st-phase and 3841 in the 2nd-phase, were enrolled in the LC-SCRUM-Asia. Success rate for OCA: 93%, for 9-in-1 test: 98%, for Genexus/OPA: 96%. Median TAT for OCA: 21 days, for 9-in-1 test: 3 days, for Genexus/OPA: 4 days. The frequencies of genetic alterations detected in the 1st-/2nd-phase were EGFR: 17/24%, KRAS: 15/16%, HER2 ex20ins: 4/3%, ALK fusions: 3/3%, RET fusions: 3/2%, ROS1 fusions: 3/2%, MET ex14skip: 2/2%, BRAF V600E: 1/1%, NRG1 fusions: 0/0.2% and NTRK3 fusions: 0.05/0.04%. Overall percent agreement of 9-in-1 test compared with OCA for EGFR/KRAS/HER2/BRAF/MET/ALK/ROS1/RET/NTRK3 alterations was 98%, and that of OPA compared with OCA was 95%. The rate of patients who received targeted therapies as 1st-line treatment was significantly elevated in the 2nd-phase compared with the 1st-phase (510/3841 [13%] vs. 567/6826 [8%], p < 0.001). Through the genomic screening, 1410 (37%) and 1269 (18%) candidate patients for clinical trials of KRAS, HER2, BRAF, MET, ALK, ROS1, RET or TRK-targeted drugs were identified in the 2nd-phase and in the 1st-phase, respectively. The rate of patients who were actually enrolled into the genotype-matched clinical trials were also significantly higher in the 2nd-phase than in the 1st-phase (222 [6%] vs. 186 [3%], p < 0.001). In 1st-line treatments for advanced NSCLC patients, the median progression-free survival was 8.5 months (95% CI, 7.7−9.4) in the 2nd-phase (n = 1839) versus 6.1 months (95% CI, 5.9−6.3) in the 1st-phase (n = 4262) (p < 0.001). Conclusions: Both the 9-in-1 test and Genexus/OPA had short TATs (3−4 days), high success rates (96−98%) and good concordance (95−98%) compared with another NGS assay (OCA). These rapid multi-gene assays highly contributed to enabling precision medicine and the development of targeted therapies for advanced NSCLC.

Individualized dosing of oral targeted therapies in oncology is crucial in the era of precision medicine

2019 ◽  
Vol 75 (9) ◽  
pp. 1309-1318 ◽  
Author(s):  
Stefanie L. Groenland ◽  
Ron H. J. Mathijssen ◽  
Jos H. Beijnen ◽  
Alwin D. R. Huitema ◽  
Neeltje Steeghs
Keyword(s):  
Precision Medicine ◽  
Targeted Therapies ◽  
Individualized Dosing

scholarly journals The FDA Oncology Center of Excellence and precision medicine

2017 ◽  
Vol 243 (3) ◽  
pp. 308-312 ◽  
Author(s):  
Kirsten B Goldberg ◽  
Gideon M Blumenthal ◽  
Amy E McKee ◽  
Richard Pazdur
Keyword(s):  
Drug Development ◽  
Precision Medicine ◽  
Targeted Therapies ◽  
Treatment Options ◽  
Regulatory Science ◽  
Cancer Drug ◽  
Center Of Excellence ◽  
Life Threatening ◽  
Oncology Center ◽  
The U.S

In January 2017, the U.S. Food and Drug Administration (FDA) formally established the Oncology Center of Excellence (OCE) to streamline the development of cancer therapies by uniting experts from FDA product centers to conduct expedited review of drugs, biologics, and devices. In May 2017, the FDA approved a cancer treatment based on a biomarker, without regard to the tumor’s site, by granting accelerated approval to pembrolizumab for patients with solid tumors that have the microsatellite instability-high or mismatch repair deficient biomarker. We describe here the OCE’s role in this first site-agnostic approval and OCE programs for further advancement of oncology-related regulatory science and policy. In addition, the FDA’s four expedited review programs that enable transformative therapies to reach patients with life-threatening malignancies earlier in the development process are key to the continued rapid development of safe and effective therapies for patients with few or no other treatment options. These changes at FDA are taking place in the context of recent progress in the understanding of the genetic and immunologic foundations of cancer, resulting in the development of targeted therapies and immunotherapies. The traditional system of phased clinical trials has evolved as early trials of breakthrough therapies use expansion cohorts in a process known as seamless drug development. Increasingly, FDA approvals of targeted therapies are likely to have contemporaneous approvals of companion diagnostics to identify patients whose cancers harbor actionable abnormalities. Impact statement This publication describes the U.S. Food and Drug Administration’s (FDA) first site-agnostic oncology drug approval, a landmark event in the history of cancer drug development. The role of the FDA’s newly established Oncology Center of Excellence (OCE) in this approval is described, as are several OCE programs to advance excellence in regulatory science in the era of precision medicine. Also provided is an overview of FDA’s expedited drug review programs, which are important to the continued acceleration of therapeutics development for patients with life-threatening diseases and few or no other treatment options.

scholarly journals Access to precision medicine in Thailand: a comparative study

2021 ◽  
Vol ahead-of-print (ahead-of-print) ◽  
Author(s):  
Nisita Jirawutkornkul ◽  
Chanthawat Patikorn ◽  
Puree Anantachoti
Keyword(s):  
Health Insurance ◽  
Cancer Risk ◽  
Precision Medicine ◽  
Risk Prediction ◽  
Targeted Therapies ◽  
Dose Adjustment ◽  
Insurance Coverage ◽  
Health Insurance Coverage ◽  
Economic Evaluations ◽  
Content Type

PurposeThis study explored health insurance coverage of genetic testing and potential factors associated with precision medicine (PM) reimbursement in Thailand.Design/methodology/approachThe study employed a targeted review method. Thirteen PMs were selected to represent four PM categories: targeted cancer therapy candidate, prediction of adverse drug reactions (ADRs), dose adjustment and cancer risk prediction. Content analysis was performed to compare access to PMs among three health insurance schemes in Thailand. The primary outcome of the study was evaluating PM test reimbursement status. Secondary outcomes included clinical practice guidelines, PMs statement in FDA-approved leaflet and economic evaluation.FindingsCivil Servant Medical Benefits Scheme (CSMBS) provided more generous access to PM than Universal Coverage Scheme (UCS) and Social Security Scheme (SSS). Evidence of economic evaluations likely impacted the reimbursement decisions of SSS and UCS, while the information provided in FDA-approved leaflets seemed to impact the reimbursement decisions of CSMBS. Three health insurance schemes provided adequate access to PM tests for some cancer-targeted therapies, while gaps existed for access to PM tests for serious ADRs prevention, dose adjustment and cancer risk prediction.Practical implicationsEvidence of economic evaluations likely impacted the reimbursement decisions of SSS and UCS, while the information provided in FDA-approved leaflet seemed to impact the reimbursement decisions of CSMBS. Three health insurance schemes provided adequate access to PM tests for some cancer-targeted therapies, while gap existed for access to PM tests for serious ADRs prevention, dose adjustment and cancer risk prediction.Originality/valueThis was the first study to explore the situation of access to PMs in Thailand. The evidence alerts public health insurance schemes to reconsider access to PMs. Development of health technology assessment guidelines for PM test reimbursement decisions should be prioritized.

New challenges to psycho-oncology research: Precision medicine oncology and targeted therapies

2017 ◽  
Vol 26 (2) ◽  
pp. 144-146 ◽  
Author(s):  
Daniel C. McFarland ◽  
Liz Blackler ◽  
Jimmie Holland
Keyword(s):  
Precision Medicine ◽  
Targeted Therapies ◽  
Oncology Research ◽  
New Challenges

Precision Cancer Medicine: The Future Is Now, Only Better

2014 ◽  
pp. 61-69 ◽  
Author(s):  
Apostolia M. Tsimberidou ◽  
Alexander M. M. Eggermont ◽  
Richard L. Schilsky
Keyword(s):  
Precision Medicine ◽  
Targeted Therapies ◽  
Therapeutic Potential ◽  
Tumor Biology ◽  
Computational Techniques ◽  
Measurement Science ◽  
The Future ◽  
Small Clinical Trials ◽  
Selection Of

The promise of precision medicine for cancer is already being realized with the recent introduction of many targeted therapies, some with companion diagnostic tests that identify patients most likely to benefit from treatment. The utility of molecular profiling of cancer to identify actionable aberrations has been suggested by several small clinical trials conducted in patients with advanced cancer and by many anecdotes but is yet to be proven in well-designed, prospective, randomized trials. Several trials that will definitively test this strategy are now underway or soon to be launched. Melanoma, a disease once largely untreatable when metastatic, may be a paradigm for understanding how the molecular drivers of a disease can lead to highly effective targeted therapies, as well as for realizing the enormous therapeutic potential of unleashing the immune system against cancer to produce long-term disease control. Looking to the future, advanced omics technologies and computational techniques will enable assessment of not only genomic variants, as performed today, but also of pathway and network aberrations that will greatly facilitate selection of drug combinations likely to benefit specific patients. As our deepening understanding of tumor biology converges with rapid advances in measurement science and technology and computational analysis, we have an enormous opportunity to create a future for precision medicine in oncology that provides for highly specific, minimally toxic, and dramatically effective treatment for each patient.

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