, Définitions lexicographiques et étymologiques de « stratégie » du Trésor de la langue française informatisé, 2012.

. Sharma, Orphan drug: Development trends and strategies, J Pharm Bioallied Sci, vol.2, issue.4, pp.290-299, 2010.

. Gbi-research, Niche is the new normal: the growth of the orphan drugs market, Pharmaceutical Technology, 2017.

. Fda, Developing products for rare disease & conditions, 2018.

E. , Development of medicines for rare diseases, 2018.

, Definition of Immunotherapy, NCI Dictionary of Cancer Terms, 2018.

, Immunothérapie : mode d'action, 2017.

, Personalized Medicine Coalition, 2015.

E. , Summary of Product Characteristics Cryvista, 2018.

E. , Summary of Product Characteristics Bavencio, 2018.

. Ortho-prep, Les maladies monogéniques et multifactorielles, overblog, 2011.

C. Ferec, La mucoviscidose : du gène CFTR au conseil génétique, 2011.

A. Gennery, Déficit immunitaire combiné sévère par déficit en adénosine désaminase, orphanet, 2012.

E. Warner, Goodbye Glybera! The world's first gene therapy will be withdrawn, 2017.

, LEEM, les procedures d'enregistrement d'un medicament en Europe, Chapitre 4 la règlementation du médicament

E. , Prime : priority medicines, 2018.

. Eurordis, Orphan medicines regulation, 2009.

E. , Orphan Designation : overview, 2018.

, Horizon 2020 -Work program 2018-2020, 8. Health, demographic, European Comission, 2018.

K. A. Hall, The current status of orphan drug development in Euope and the US Intractable Rare Dis Res, vol.3, pp.1-7, 2014.

V. Giannuzzi, Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen, Orphanet journal of rare diseases, vol.12, p.64, 2017.

, Recommendation for removal of orphan designation at the time of marketing authorisation -Emplicti (elotuzumab) for the treatment of multiple myeloma, EMA Committee for Orphan Medicinal Products, 2016.

J. Sheppard, Adaptive clinical trials in orphan drug development, 2012.

, Rare diseases: common issues in drug development, guidance for industry, 2015.

H. Phan, Rare diseases require unique approaches in clinical trial design, clinical leader, 2017.

M. Berdud, Establishing a Reasonable Price for an Orphan Drug, 2018.

. Logviss, Characteristic of clinical trials in rare vs common diseases: A register-based Latvian study, PLoS ONE, vol.13, issue.4, p.194494, 2018.

. Kesselheim, Characteristics of Clinical Trials to Support Approval of Orphan vs Nonorphan Drugs for Cancer, JAMA, vol.305, issue.22, pp.2320-2326, 2011.

M. Dooms, From promising molecules to orphan drugs: Early clinical drug development, Intractable Rare Dis Res, vol.6, pp.29-34, 2017.

A. Stevens, The future of orphan drugs in Europe, GlobalData, p.1445, 2017.

, Fast track, breaktrhrough therapy, accelerated approval, priority review, FDA, 2018.

C. H. Wong, Estimation of clinical trial success rates and related parameters, Biostatistics, vol.00, pp.1-14, 2018.

P. Mincarone, Reimbursed price of orphan drugs: current strategies and potential improvements, Public Health Genomics, vol.20, pp.1-8, 2017.

J. Mycka, Market access trends across the EU5: 2009 to 2016-An update, Value in health, vol.20, p.677, 2017.

K. E. Young, A comparative study of orphan drug prices in Europe, Journal of Market Access & Health Policy, vol.5, issue.1, 2017.

L. , De l'AMM à la fixation du prix : les spécificités des maladies rares à mieux prendre en compte, vol.19, 2016.

J. Sussex, A pilot study of multicriteria decision analysis for valuing orphan medicines, Value in Health, vol.16, issue.8, pp.1163-1169, 2013.

, European network for Health Technology Assessment: HTA Core Model®, vol.30, 2018.

K. Cockerill, Orphan drug pricing in Europe, White paper, Health Advances and Parexel, 2017.

, York; York Health Economics Consortium, 2016.

E. Licking, A roadmap to strategic drug pricing, In Vivo the business medicine report, vol.34, 2016.

, Orphan drug report, 2018.

D. Healthcare, Orphan drug trends, pp.143-144, 2011.

A. Cote, What is wrong with orphan drug policies? Value Health, vol.15, pp.1185-91, 2012.

C. Helfand, The tope 10 drug launches of 2017, 2017.

, Comité économique des produits de santé, 2018.

, Lists of medicinal products for rare diseases in Europe, Orphan drugs collection, Orphanet Report Series, 2018.

. Evaluatepharma, Orphan drug report 2013, 2013.

L. Dezzani, Orphan drugs, IgeaHub pharmaceutical club, 2018.

, Pharmaceuticals -Community register, Register of designated orphan medicinal products, European Comission, issue.2, 2018.

K. L. Miller, Do investors value the orphan drug designation?, Orphanet Journal, 2017.

A. Bondain, Stratégie : Embouteillage dans l'oncologie et conséquences, 2018.

O. Wellman-labadie, The US Orphan Drug Act: rare disease research stimulator or commercial opportunity?, Health Policy, vol.95, pp.216-228, 2010.

F. Fréry, Stratégique, 11ème édition, Éditions Pearson, pp.415-449, 2017.

A. Teste-de-sagey, Les fusions acquisitions dans l'industrie pharmaceutique, outil de croissance et de création de valeur ? Le cas Norgine, Thèses, 2014.

K. Arnold, Value drivers in licensing deals, Nature Biotechnology, vol.20, issue.11, pp.1085-1094, 2002.

S. Guetta, Gamida cell ppt_englih_5-5-2010, slideshare linkedin, 2014.

S. Korea, C. Japan, and A. , RegeneRx completes license expansion for RGN-137 in Europe, 2017.

. Prnewswire, RegeneRx licensee GtreeBNT enter into joint venture to globally develop RGN-137 for treatment of epidermolysis bullosa, 2018.

A. Pena, Joint venture plans to advance potential EB therapy RGN-137 into phase 3 trial, Epidermolysis Bullosa News, 2018.

É. Littré and ;. Le-littré, Définition de synergie, Dictionnaire de la Langue française, pp.1872-1877

L. Ramsey, We spoke to the CEO of Roche Pharmaceuticals about how the giant became a deal machine, 2018.

B. Booth, Getting struck twice: acquisitions and spinouts in biotech, 2017.

R. Rooswinkel, Acquiring orphans, Biotentrepreneur, 2014.

, Jazz Pharmaceutical to acquire orphan medical; combines Orphan Medical's growing central nervous system product and commercial team with jazz pharmaceutical's development pipeline, Jazz-Pharmaceuticals-Acquire-Orphan-Medical-Combines-Orphan, 2005.

C. Hefland, Xyrem -Jazz Pharmaceuticals, Fierce Pharma, 2016.

M. Nissen, Alexion's deal discipline sure beats a previous disaster, Bloomberg Opinion, 2018.

, United Securities and Exchange Comission, SEC Fillings Forms D

A. Kay, What is in-liensing, Pharmaceutical Investing News, 2018.

A. Smith, Big Pharma thinks small, 2006.

J. Dillon, Advanced business development course prepared for Bio's, Dillon Capital Strategies, 2015.

P. Giglio, The Art of the deal -licensing trends in orphan drugs, 2014.

R. Mason, The economics of licensing contracts, Nature, 2008.

J. Caroll, Pfizer snags orphan drug in $70M licensing deal with Repligen, FierceBiotech, 2013.

. Globalnewswire, Santhera obtains worldwide exclusive license from polyphor to develop and commercialize clinical stage candidate for cystic fibrosis and other pulmonary diseases, Santhera-Obtains-Worldwide-Exclusive-Licensefrom-Polyphor-to-Develop-and-Commercialize-Clinical-Stage-Candidate-for-Cystic-Fibrosis-and-Other-Pulmonary-Diseases.html, 2018.

. Sobi, Sobi strengthens inflammation franchise by acquiring global rights for emapalumab from Novimmune, Press realease, 2018.

. Sobi, Sobi to acquire Synagis US rights from AstraZenecacreates a platform for global growth, Press release, 2018.

P. Recordati and . Realease, Recordati acquires Orphan Europe, a European group specialized in rare diseases, 2007.

. Reuters, Australia's sigma buys Orphan for A$130 mln, 2007.

A. Gilmour, Control premium study 2017, insight into market dynamics, financial dynamics and other factors, 2017.

R. Skowronski, M&A Market, structure, volatility and acquisition premiums, Bank Zachodni WBK, p.34, 2011.

C. Göbel, Start-up valuation of biotech companies with real options, 2016.

P. Frei, Pharma-Biotech company valuation -an introduction, The Valuation Experts, Fit for Health 2.0, Venture Valuation group, 2015.

W. Bratic, Navigating through a biotechnology valuation, vol.8, pp.207-216, 1997.

S. Weingust, Using the Monte Carlo method to value early stage, technology-based intellectual property assets, 2013.

K. D. Keegan, Biotechnology valuation: an introductory guide, 2008.

P. Kodukula, Project valuation using real options: a practitioner's guide. Fort Lauderdale, 2006.

E. Baduns, Realistic Investment Valuation: A Comprehensive Real Options Model, Journal of Business Management, issue.7, pp.58-71, 2013.

F. Schmachtenberg, Deal structures in the life sciences industry and their financial statement implications, dissertation of the university of St Gallen, Difrodruck GmbH Bamber, p.34, 2014.

K. Smith, The Art of M&A Strategy: A Guide to Building Your Company's Future through Mergers, Acquisitions, and Divestitures, 2012.

M. E. Camacho-molina, L'analyse en options réelles comme méthode de valorisation de projets R&D en entreprise, Louvain school of management, 2016.