Eur J Cancer. 2017 Oct;84:325-334. doi: 10.1016/j.ejca.2017.07.028. Epub 2017 Sep 4.
Societal challenges of precision medicine: Bringing order to chaos.
Salgado R1,
Moore H2,
Martens JWM3,
Lively T4,
Malik S5,
McDermott U6,
Michiels S7,
Moscow JA8,
Tejpar S9,
McKee T10,
Lacombe D11;
IBCD-Faculty.
Becker R12, Beer P13, Bergh J14, Bogaerts J15, Dovedi S16, Fojo AT17, Gerstung M18, Golfinopoulos V19, Hewitt S20, Hochhauser D21, Juhl H22, Kinders R23, Lillie T24, Herbert KL25, Maheswaran S26, Mesri M27, Nagai S28, Norstedt I29, O'Connor D30, Oliver K31, Oyen WJG32, Pignatti F33, Polley E34, Rosenfeld N35, Schellens J36, Schilsky R37, Schneider E38, Senderowicz A39, Tenhunen O40, van Dongen A41, Vietz C42, Wilking N43.
- 1
- EORTC Pathobiology Group, Belgium; Department of Pathology GZA, Antwerp, Belgium; Breast Cancer Translational Research Laboratory, Jules Bordet Institute, Belgium.
- 2
- National Cancer Institute Biorepositories and Biospecimen Research Branch (BBRB), Rockville, MD, USA.
- 3
- Department of Medical Oncology, Erasmus MC Cancer Institute and Cancer Genomics Netherlands, Erasmus University Medical Center, Rotterdam, The Netherlands.
- 4
- Cancer Diagnosis Program, Division of Cancer Treatment and Diagnosis, US National Cancer Institute, Rockville, MD, USA.
- 5
- Investigational Drug Branch, Cancer Therapy Evaluation Program, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
- 6
- Wellcome Trust Sanger Institute, Cambridge, UK.
- 7
- Service de Biostatistique et D'Epidémiologie, Gustave Roussy, CESP, INSERM, Univ. Paris-Sud, Univ. Paris Saclay, Villejuif, France.
- 8
- National Cancer Institute, Cancer Therapy Evaluation Program, Rockville, MD, USA.
- 9
- Molecular Digestive Oncology Unit, University Hospital Gasthuisberg, Leuven, Belgium.
- 10
- Cancer Diagnosis Program, Division of Cancer Treatment and Diagnosis, Diagnostic Biomarkers and Technology Branch, National Cancer Institute, Bethesda, USA.
- 11
- EORTC Headquarters, Brussels, Belgium. Electronic address: denis.lacombe@eortc.be.
- 12
- US Food and Drug Administration (FDA), Silver Spring, MD, USA.
- 13
- 14M Genomics, Medical Genomics, Hinxton, Cambridge, UK.
- 14
- Karolinska Institutet and University Hospital, Stockholm, Sweden.
- 15
- EORTC Headquarters, Statistics Department, Brussels, Belgium.
- 16
- MedImmune, Oncology, Cambridge, UK.
- 17
- Columbia University Medical Center, New York, USA.
- 18
- European Bioinformatics Institute EMBL-EBI, Cancer Genome Project, Hinxton, UK.
- 19
- EORTC Headquarters, Medical Vice Director, Brussels, Belgium.
- 20
- National Cancer Institute (NCI), Bethesda, MD, USA.
- 21
- UCL Cancer Institute, Dept. of Oncology, London, UK.
- 22
- Indivumed GmbH, CEO, Hamburg, Germany.
- 23
- Frederick National Laboratory for Cancer Research, Laboratory of Human Toxicology and Pharmacology, Frederick, MD, USA.
- 24
- Merck Sharp & Dohme Limited, Global Clinical Development, London, UK.
- 25
- Duke University Medical Center, Surgery Department, Durham, NC, USA.
- 26
- Massachusetts General Hospital, Cancer Center, Charlestown, MA, USA.
- 27
- National Cancer Institute (NCI), National Institutes of Health (NIH) OCCPR, National Cancer Institute (NCI), Bethesda, MD, USA.
- 28
- The University of Tokyo and Pharmaceuticals and Medical Devices Agency, Companion Diagnostics Working Group, Tokyo, Japan.
- 29
- European Commission, Innovative and Personalised Medicine, Brussels, Belgium.
- 30
- Medicines & Healthcare Products Regulatory Agency (MHRA), Licensing Division, London, UK.
- 31
- International Brain Tumour Alliance (IBTA), Chair and Co-Director, Tadworth, Surrey, UK.
- 32
- The Institute of Cancer Research, Sutton, UK.
- 33
- European Medicines Agency (EMA), London, UK.
- 34
- Mayo Clinic, Rochester, USA.
- 35
- University of Cambridge, Cancer Research UK Cambridge Institute, Cambridge, UK.
- 36
- The Netherlands Cancer Institute - Antoni Van Leeuwenhoek-ziekenhuis, Amsterdam, The Netherlands.
- 37
- American Society of Clinical Oncology (ASCO), Office of the CEO, Alexandria, VA, USA.
- 38
- New York State Department of Health, Wadsworth Center, Albany, NY, USA.
- 39
- Cerulean Pharma Inc., Clinical Department, Waltham, MA, USA.
- 40
- Finnish Medicines Agency, Oulu, Finland.
- 41
- VU University Medical Center Amsterdam, Dept. of Radiology & Nuclear Medicine, Amsterdam, The Netherlands.
- 42
- Foundation Medicine, Inc., Regulatory Affairs, Cambridge, MA, USA.
- 43
- Skane University Hospital, Lund, Malmo, Sweden.
Abstract
The increasing number of drugs targeting specific proteins implicated in tumourigenesis and the commercial promotion of relatively affordable genome-wide analyses has led to an increasing expectation among patients with cancer that they can now receive effective personalised treatment based on the often complex genomic signature of their tumour. For such approaches to work in routine practice, the development of correspondingly complex biomarker assays through an appropriate and rigorous regulatory framework will be required. It is becoming increasingly evident that a re-engineering of clinical research is necessary so that regulatory considerations and procedures facilitate the efficient translation of these required biomarker assays from the discovery setting through to clinical application. This article discusses the practical requirements and challenges of developing such new precision medicine strategies, based on leveraging complex genomic profiles, as discussed at the Innovation and Biomarkers in Cancer Drug Development meeting (8th-9th September 2016, Brussels, Belgium).
KEYWORDS:
Assay validation; Biomarkers; Drug development; Preanalytical and analytical validation; Precision oncology; Regulatory sciences
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