Pre-reading material

Kohane, I (2015). Ten things we have to do to achieve precision medicine. Science 349:37.

Breckenridge A and Eichler H-G (2013). Towards a prevention model of health care. Nature Reviews Drug Discovery 12: 563.

Eichler, H.-G. et al. Bridging the efficacy-effectiveness gap: a regulator’s perspective on addressing variability of drug response. Nat. Rev. Drug Discov. 10, 495–506 (2011).

De Wever A, « Comment gérer les rapports coût-efficacité ? » Revue Médicale de Bruxelles ; vol 30, n°4, septembre 2009, 437-440.

Beresniak A, Medina-Lara A, Auray JP, De Wever A, Praet JC, Tarricone R, Torbica A et al., “Validation of underlying assumptions of the QALY outcome : results from the Echoutcome European project” Pharmaco-economics, September 2014, 1170-7690

Cleemput I, Neyt M, Van de Sande S, Thiry N. Belgian guidelines for economic evaluations and budget impact analyses: second edition. Health Technology Assessment (HTA). Brussels: Belgian Health Care Knowledge Centre(KCE). 2012. KCE Report 183. Available at:

Neyt M, Van Brabandt H, Devriese S, Van De Sande S. A cost-utility analysis of transcatheter aortic valve implantation in Belgium: focusing on a well-defined and identifiable population. BMJ Open. 2012 May 4;2(3).

San Miguel L, Hulstaert F. The importance of test accuracy in economic evaluations of companion diagnostics. J Comp Eff Res. 2015 Nov;4(6):569-77. doi:10.2217/cer.15.41. Epub 2015 Nov 3. PubMed PMID: 26529499.

Neyt M, Hulstaert F, Gyselaers W. Introducing the non-invasive prenatal test for trisomy 21 in Belgium: a cost-consequences analysis. BMJ Open. 2014 Nov 7;4(11):e005922. doi: 10.1136/bmjopen-2014-005922. PubMed PMID: 25380810; PubMed Central PMCID: PMC4225226.

Lievens Y, Obyn C, Mertens AS, Van Halewyck D, Hulstaert F. Stereotactic body radiotherapy for lung cancer: how much does it really cost? J Thorac Oncol. 2015 Mar;10(3):454-61. doi: 10.1097/JTO.0000000000000421. PubMed PMID: 25376514.

Hulstaert F, Schwierz C, Nevens F, Thiry N, Gamil M, Colle I, Van de Sande S, Horsmans Y. Should chronic hepatitis B be treated as early as possible? Int J Technol Assess Health Care. 2013 Jan;29(1):35-41. doi: 10.1017/S0266462312000736.Epub 2013 Jan 8. PubMed PMID: 23298548.

Hulstaert F, Neyt M, Vinck I, Stordeur S, Huić M, Sauerland S, Kuijpers MR, Abrishami P, Vondeling H, Flamion B, Garattini S, Pavlovic M, van Brabandt H. Pre-market clinical evaluations of innovative high-risk medical devices in Europe. Int J Technol Assess Health Care. 2012 Jul;28(3):278-84. doi:10.1017/S0266462312000335. PubMed PMID: 22980705.

Hulstaert F, De Laet C. How many CIN2+ lesions can be avoided through HPV 16/18 vaccination? Acta Obstet Gynecol Scand. 2009;88(7):859; author reply 859-60. doi:10.1080/00016340902989005. PubMed PMID: 19449218.

Goldman, C. Compton, BB. Mittleman, Public-private partnerships as driving forces in the quest for innovative medicines. Clin Transl Med. 2 (1):2. (2013).

Papadaki, G. Hirsch, Curing Consortium Fatigue. Sci. Transl. Med. 5, 200fs35 (2013).

Mittleman, G. Neil, J. Cutcher-Gershenfeld, Precompetitive consortia in biomedicine—how are we doing? Nat. Biotechnol. 31, 979-985 (2013).

Rinaldi, A. 2011. Teaming up for biomarker future. EMBO Rep. 12(6): 500–504.

L.G. Baird, G. Hirsch, Adaptive Licensing: Creating a Safe Haven for Discussions, Scrip Regulatory Affairs, 10-11 (2013).

Steer AC, Law I, Matatolu L, Beall BW, Carapetis JR. Global emm type distribution of group A streptococci: systematic review and implication for vaccine development. Lancet Infect Dis. 2009 Oct;9(10):611-6

Sanderson-Smith M., De Oliveira D.M., Guglielmini J., McMillan D.J., Vu T., Holien J.K., Henningham A., Steer A.C., Bessen D.E., Dale J.B., Curtis N., Beall B.W., Walker M.J., Parker M.W., Carapetis J.R., Van Melderen L., Sriprakash K.S. and Smeesters P.R.; the M Protein Study Group. A systematic and functional classification of Streptococcus pyogenes that serves as a new tool for molecular typing and vaccine development. The Journal of Infectious Diseases (2014);210(8):1325-38.

Moreland N.J., Waddington C., Williamson D., Sriskandan S., Smeesters P.R. Proft T., Steer A.C., Walker M.J., Baker E.N., Baker M.J., Lennon D., Dunbar R., Carapetis J. and Fraser J. Working towards a Group A Streptococcal Vaccine: Report of a Collaborative Trans‑Tasman Workshop. Vaccine (2014);32(30):3713-20.

Wimana, Z. Z., Gebhart, G., Guiot, T., Vanderlinden, B., Morandini, R., Doumont, G., Sherer, F., Van Simaeys, G., Goldman, S., Ghanem, G. E., & Flamen, P. (2015, mars). Mucolytic Agents Can Enhance HER2 Receptor Accessibility for

[(89)Zr]Trastuzumab, Improving HER2 Imaging in a Mucin-Overexpressing Breast Cancer Xenograft Mouse Model. Molecular imaging and biology. doi:10.1007/s11307-015-0840-x

Bondue, B., De Vuyst, P., Parmentier, M., Vosters, O., Sherer, F., Van Simaeys, G., Doumont, G., Egrise, D., Rorive, S., Sauvage, S., Lacroix, S., Goldman, S., Yakoub, Y. Y., & Huaux, F. (2015, janvier). PET/CT with 18F-FDG- and 18F-FBEM-labeled leukocytes for metabolic activity and leukocyte recruitment monitoring in a mouse model of pulmonary fibrosis. The Journal of nuclear medicine, 56(1), 127-132. doi:10.2967/jnumed.114.147421

Wens, V., Bourguignon, M., Goldman, S., Marty, B., Op De Beeck, M., Clumeck, C., Mary, A., Peigneux, P., Van Bogaert, P., Brookes, M. J., & De Tiege, X. (2014, avril). Inter- and Intra-Subject Variability of Neuromagnetic Resting State Networks. Brain topography. doi:10.1007/s10548-014-0364-8

Goldman, S. (2014). PET imaging of garbage protein in Alzheimer’s disease: Does it require reappraisal of brain PET analysis? European journal of nuclear medicine and molecular imaging, 41(5), 813-815. doi:10.1007/s00259-014-2740-8

Challenges with advanced therapy medicinal products and how to meet them. Committee for Advanced Therapies (CAT);  Nat Rev Drug Discov. 2010 Mar;9(3):195-201.

Marketing Regulatory Oversight of Advanced Therapy Medicinal Products (ATMPs) in Europe: The EMA/CAT Perspective. Salmikangas P1,2, Schuessler-Lenz M3,4, Ruiz S5,6, Celis P7, Reischl I8,9, Menezes-Ferreira M10,11, Flory E12,13, Renner M14, Ferry N15,16. Adv Exp Med Biol. 2015;871:103-30.

Regulatory evaluation of Glybera in Europe – two committees, one mission. Melchiorri D, Pani L, Gasparini P, Cossu G, Ancans J, Borg JJ, Drai C, Fiedor P, Flory E, Hudson I, Leufkens HG, Müller-Berghaus J, Narayanan G, Neugebauer B, Pokrotnieks J, Robert JL, Salmonson T, Schneider CK.   Nat Rev Drug Discov. 2013 Sep;12(9):719.

Bohlin N., Kurosawa M., Luyten D., Brennan J. and R.A. Wadman (2015). Embracing the consumer health opportunity. How new market entrants are changing the sector. Arthur D. Little Prism, 1.

Bohlin N., Brennan J., Kaltenbach T. and F. Thomas (2014). Innovation in the healthcare space. How new technologies are driving fundamental changes in the pharmaceutical business model. Arthur D. Little Prism, 1.

Steinberg D., Horwitz G. and D. Zohar (2015). Building a business model in digital medicine. Nature Biotechnology, 33(9).

Vincent Wille, Pieter-Jan Mermans and Edouard Croufer. Making new product launch decisions in an uncertain environment, Arthur D.Little Prism magazine

Edouard Croufer, Françoise Simon, François Meurgey and Alexandre Meire. Putting patients at the center of a new biopharma business model, Arthur D.Little Prism magazine

Bubela T., FitzGerald G.A., Gold E.R., Recalibrating Intellectual Property Rights to Enhance Translational Research Collaborations.Science Translational Medicine (2012). Vol.4(122).
Goldman M., Seigneuret N., Eichler H-G., The Innovative Medicines Initiative: an engine for regulatory science. Nat.Rev.Drug Discov. (2015). Vol.14: 1-2.

Snyder H.M., Kim H., Bain L.J., Egge R., Carrillo M.C., Alzheimer’s disease public-private partnerships: Update 2014. Alzheimer’s & Dementia (2014).Vol.10:873-880.

Said M., Zerhouni E., The role of public-private partnerships in addressing the biomedical innovation challenge. Nat.Rev.Drug Discov.(2014). Vol.13:789-790.

Stevens H., Van Overwalle G., Van Looy B., Huys I., Perspectives and Opportunities for Precompetitive Public-Private Partnerships in the Biomedical Sector. Biotech.Law Report (2013).Vol.32(3):131-139.

van Zimmeren E., Vanneste S., Matthijs G., Vanhaverbeke W., Van Overwalle G., Trends in Biotechnol.(2011).Vol.29(11):569-576.

M.Verlinden, H.Nys, N.Ectors, I.Huys, Access to biobanks: harmonization across biobank initiative, Biopreserv Biobank. 2014 Dec;12(6):415-22. doi: 10.1089/bio.2014.0034.

Michiel Verlinden, Timo Minssen, Isabelle Huys, IPRs in Biobanking: Risks and Opportunities for Translational Research, [2015] I.P.Q., Issue 2, 2015

Stevens et al. The Role of Public-Sector Research in the Discovery of Drugs and Vaccines. N Engl J Med 2011; 364: 535-541.
Schmidt C. Debate Re-Ignites on Contribution of Public Research to Drug Development. Nat Biotechnol 2011; 29 (6): 469-470.

Editorial. Scientific Writing 101. Nat Struct Mol Biol 2010; 17 (2): 139.

Zhang W. Ten Simple Rules for Writing Research Papers. PLoS Comput Biol2014; 10 (1): e1003453. doi:10.1371/journal.pcbi.1003453

Koh et al. The Arc of Health Literacy. JAMA 2015; Published online August 06, 2015. doi:10.1001/jama.2015.9978

Feasibility, safety, and efficacy of totally endoscopic coronary artery bypass grafting: multicenter European experience. de Cannière D, Wimmer-Greinecker G, Cichon R, Gulielmos V, Van Praet F, Seshadri-Kreaden U, Falk V. J Thorac Cardiovasc Surg. 2007 Sep;134(3):710-6.

Integrated coronary revascularization: percutaneous coronary intervention plus robotic totally endoscopic coronary artery bypass. Katz MR, Van Praet F, de Canniere D, Murphy D, Siwek L, Seshadri-Kreaden U, Friedrich G, Bonatti J. Circulation. 2006 Jul 4;114(1 Suppl):I473-6.

Robotic Mitral Valve Repair for Simple and Complex Degenerative Disease: Midterm Clinical and Echocardiographic Quality Outcomes. Suri RM, Taggarse A, Burkhart HM, Daly RC, Mauermann W, Nishimura RA, Li Z, Dearani JA, Michelena HI, Enriquez-Sarano M.Circulation. 2015 Nov 24;132(21):1961-8

How can innovative forms of clinical research contribute to deliver affordable cancer care in an evolving health care environment? Susen Burock, Françoise Meunier, Denis Lacombe – European Journal of Cancer (2013)49, 2777-2783

Academia-Industry Partnerships: Are we ready for new models of partnership? The point of view of the EORTC, an academic clinical cancer research organisation. Denis Lacombe, Susen Burock, Françoise Meunier – European Journal of Cancer 15 October 2012 (on-line publication exclusively), 1-7

The dream and reality of histology agnostic cancer clinical trials. Denis Lacombe, Susen Burock, Jan Bogaerts, Patrick Schoeffski, Vassilis Golfinopoulos, Roger Stupp – Molecular Oncology 8(2014) 1057-1063

European perspective for effective cancer drug development . Denis Lacombe, Sabine Tejpar, Roberto Salgado, Fatima Cardoso, Vassilis Golfinopoulos, Daniela Aust, Gunnar Folprecht, Arnaud Roth and Roger Stupp – Nature reviews, august 2014, volume 11 no.8

Lancet Oncol. 2015 May;16(5):522-30. Adjuvant ipilimumab versus placebo after complete resection of high-risk stage III melanoma (EORTC 18071): a randomised, double-blind, phase 3 trial.

Semin Oncol. 2015 Jun;42(3):429-35. Immune checkpoint inhibitors in melanoma provide the cornerstones for curative therapies

Eur J Cancer. 2013 Feb;49(3):684-95.

The Ebola epidemic: a global health emergency LO Gostin, D Lucey, A Phelan – Jama, 2014 –

From sovereignty to solidarity: a renewed concept of global health for an era of complex interdependence J Frenk, O Gómez-Dantés, S Moon – The Lancet, 2014

Informed consent: where are we in 2015? Foy MA. Bone Joint J. 2015 Sep;97-B(9):1159-61. doi: 10.1302/0301-620X.97B9.36506. Review.

Delayed consent: will there be a shift in approach for US primary percutaneous coronary intervention trials? MacKay CR, Torguson R, Waksman R. Lancet. 2015 Aug 15;386(9994):714-6. doi: 10.1016/S0140-6736(15)60077-0. Epub 2015 May 7.

Intensive care unit research ethics and trials on unconscious patients.

Gillett GR. Anaesth Intensive Care. 2015 May;43(3):309-12

Eizirik DL, Sammeth M, Bouckenooghe T, Bottu G, Sisino G, Igoillo-Esteve M, Ortis F, Santin I, Colli ML, Barthson J, Bouwens L, Hughes L, Gregory L, Lunter G, Marselli L, Marchetti P, McCarthy MI, Cnop M The human pancreatic islet transcriptome: expression of candidate genes for type 1 diabetes and the impact of pro-inflammatory cytokines. PLoS Genetics, 8: e1002552, 2012 (“Faculty of 1000 Recommended”

Santin I, Eizirik DL Candidate genes for type 1 diabetes modulate pancreatic islet inflammation and beta-cell apoptosis.  Diabetes Obes Metab 15 (Suppl. 3): 71-81, 2013

Marroqui L, Lopes M, Santos RSD, Roivainen M, Richardson S, Morgan N, Op de Beeck A, Eizirik DL Differential cell autonomous immune responses determine the outcome of coxsackievirus infections in pancreatic a and b cells. eLife, 4: e06990, 2015

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Tumoral Immune Resistance Mediated by Enzymes That Degrade Tryptophavan Baren N, Van den Eynde BJ. Cancer Immunol Res. 2015 Sep;3(9):978-85
Tumor-Expressed IDO Recruits and Activates MDSCs in a Treg-Dependent Manner. Holmgaard RB, Zamarin D, Li Y, Gasmi B, Munn DH, Allison JP, Merghoub T, Wolchok JD. Cell Rep. 2015 Oct 13;13(2):412-24
Indoleamine 2,3-dioxygenase is a critical resistance mechanism in antitumor T cell immunotherapy targeting CTLA-4. Holmgaard RB, Zamarin D, Munn DH, Wolchok JD, Allison JP J Exp Med. 2013 Jul 1;210(7):1389-402
Discovery of potent competitive inhibitors of indoleamine 2,3-dioxygenase with in vivo pharmacodynamic activity and efficacy in a mouse melanoma model. Yue EW, Douty B, Wayland B, Bower M, Liu X, Leffet L, Wang Q, Bowman KJ, Hansbury MJ, Liu C, Wei M, Li Y, Wynn R, Burn TC, Koblish HK, Fridman JS, Metcalf B, Scherle PA, Combs AP J Med Chem. 2009 Dec 10;52(23):7364-7
Selective inhibition of IDO1 effectively regulates mediators of antitumor immunity. Liu X, Shin N, Koblish HK, Yang G, Wang Q, Wang K, Leffet L, Hansbury MJ, Thomas B, Rupar M, Waeltz P, Bowman KJ, Polam P, Sparks RB, Yue EW, Li Y, Wynn R, Fridman JS, Burn TC, Combs AP, Newton RC, Scherle PA. Blood. 2010 Apr 29;115(17):3520-30
Hydroxyamidine inhibitors of indoleamine-2,3-dioxygenase potently suppress systemic tryptophan catabolism and the growth of IDO-expressing tumors. Koblish HK, Hansbury MJ, Bowman KJ, Yang G, Neilan CL, Haley PJ, Burn TC, Waeltz P, Sparks RB, Yue EW, Combs AP, Scherle PA, Vaddi K, Fridman JS. Mol Cancer Ther. 2010 Feb;9(2):489-98
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