The evidence landscape in precision medicine

Evidence landscapes can help to coordinate research and address challenges in implementing precision medicine. Precision medicine is beginning to make an impact on the treatment of different diseases, but there are still challenges that must be overcome, such as the complexity of interventions, the need for marker validation, and the level of evidence necessary to demonstrate effectiveness. In this Perspective, we describe how evidence landscapes can help to address these challenges.

[1]  Vinay Prasad,et al.  Precision oncology: origins, optimism, and potential. , 2016, The Lancet. Oncology.

[2]  P. Queirolo,et al.  BRAF-mutant melanoma: treatment approaches, resistance mechanisms, and diagnostic strategies , 2015, OncoTargets and therapy.

[3]  M. Buyse,et al.  Precision medicine needs randomized clinical trials , 2017, Nature Reviews Clinical Oncology.

[4]  A. Kesselheim,et al.  Countering imprecision in precision medicine , 2016, Science.

[5]  J. Witte,et al.  The Scientific Foundation for Personal Genomics: Recommendations from a National Institutes of Health–Centers for Disease Control and Prevention Multidisciplinary Workshop , 2009, Genetics in Medicine.

[6]  K. Bloom,et al.  Multisite analytic performance studies of a real-time polymerase chain reaction assay for the detection of BRAF V600E mutations in formalin-fixed, paraffin-embedded tissue specimens of malignant melanoma. , 2012, Archives of pathology & laboratory medicine.

[7]  W. Sauerbrei,et al.  Reporting recommendations for tumor marker prognostic studies (REMARK). , 2005, Journal of the National Cancer Institute.

[8]  R. Porcher,et al.  Evidence for Treatment-by-Biomarker interaction for FDA-approved Oncology Drugs with Required Pharmacogenomic Biomarker Testing , 2017, Scientific Reports.

[9]  Heidi Ledford Cutting-edge cancer drug hobbled by diagnostic test confusion , 2018, Nature.

[10]  A. Kesselheim,et al.  Research ethics for emerging trial designs: does equipoise need to adapt? , 2018, British Medical Journal.

[11]  D. Ransohoff,et al.  Biomarker studies: a call for a comprehensive biomarker study registry , 2011, Nature Reviews Clinical Oncology.

[12]  Soonmyung Paik,et al.  Use of archived specimens in evaluation of prognostic and predictive biomarkers. , 2009, Journal of the National Cancer Institute.

[13]  K. Flaherty,et al.  Inhibition of mutated, activated BRAF in metastatic melanoma. , 2010, The New England journal of medicine.

[14]  C. Weijer,et al.  Accumulating Evidence and Research Organization (AERO) model: a new tool for representing, analyzing, and planning a translational research program , 2013, Trials.

[15]  C. Compton,et al.  Breaking a Vicious Cycle , 2013, Science Translational Medicine.

[16]  A. Hauschild,et al.  Dabrafenib in BRAF-mutated metastatic melanoma: a multicentre, open-label, phase 3 randomised controlled trial , 2012, The Lancet.

[17]  J. Bozell Breaking the vicious cycle. , 2007, Mental health today.

[18]  G. Botti,et al.  BRAF/NRAS mutation frequencies among primary tumors and metastases in patients with melanoma. , 2012, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[19]  T. Eberlein,et al.  Improved Survival with Vemurafenib in Melanoma with BRAF V600E Mutation , 2012 .

[20]  B. Dréno,et al.  Is a single BRAF wild-type test sufficient to exclude melanoma patients from vemurafenib therapy? , 2014, The Journal of investigative dermatology.