N. Aaronson, A. Choucair, T. Elliott, J. Greenhalgh, M. Halyard et al., User's Guide to Implementing Patient-Reported Outcomes Assessment in Clinical Practice, International Society for Quality of Life Research, 2011.

M. Baldwin, A. Spong, L. Doward, and A. Gnanasakthy, Patient-reported outcomes, patientreported information: From randomized controlled trials to the social web and beyond, Patient, vol.4, issue.1, pp.11-17, 2011.

A. K. Banerjee, S. Okun, I. R. Edwards, P. Wicks, M. Y. Smith et al., Patient-Reported Outcome Measures in Safety Event Reporting: PROSPER Consortium Guidance, Drug Safety, vol.36, pp.1129-1149, 2013.

E. Basch, A. P. Abernethy, and B. B. Reeve, Assuring the patient-centeredness of patientreported outcomes: Content validity in medical product development and comparative effectiveness research, Value in Health, vol.14, issue.8, pp.965-966, 2011.

E. Basch and A. V. Bennett, Patient-Reported Outcomes in Clinical Trials of Rare Diseases, Journal of General Internal Medicine, vol.29, pp.801-803, 2014.

E. Basch, A. M. Deal, M. G. Kris, H. I. Scher, C. A. Hudis et al., Symptom Monitoring With Patient-Reported Outcomes During Routine Cancer Treatment: A Randomized Controlled Trial, Journal of Clinical Oncology, vol.34, issue.6, pp.557-565, 2015.

D. Benamouzig, Mesures de qualité de vie en santé. Les Cahiers du Centre Georges Canguilhem, , vol.4, issue.1, pp.135-176, 2010.

K. Benjamin, M. K. Vernon, D. L. Patrick, E. Perfetto, S. Nestler-parr et al., Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report, Value in Health, vol.20, issue.7, pp.838-855, 2017.

M. Bin-han-ong, Do patients know best? Industry, NCI committing dollars to studying patient-reported outcomes -The Cancer Letter Publications. The Cancer Letter, 2018.

A. Coulter, L. Locock, S. Ziebland, and J. Calabrese, Collecting data on patient experience is not enough: they must be used to improve care, BMJ: British Medical Journal, vol.348, 2014.

S. Dean, J. M. Mathers, M. Calvert, D. G. Kyte, D. Conroy et al., The patient is speaking": Discovering the patient voice in ophthalmology, British Journal of Ophthalmology, vol.101, issue.6, pp.700-708, 2017.

A. Desomer, K. Van-den-heede, M. Tiemstra, J. Paget, D. De-boer et al., Use of patient-reported outcome and experience measures in patient care and policy, Health Services Research, p.52, 2018.

N. J. Devlin and J. Appleby, Getting the most out of PROMs: Putting health outcomes at the heart of NHS decision-making. King's Fund, 2010.

N. J. Devlin and R. Brooks, EQ-5D and the EuroQol Group: Past, Present and Future. Applied Health Economics and Health Policy, vol.15, pp.127-137, 2017.

L. Dudley, C. Gamble, J. Preston, D. Buck, T. E. Group et al., What Difference Does Patient and Public Involvement Make and What Are Its Pathways to Impact? Qualitative Study of Patients and Researchers from a Cohort of Randomised Clinical Trials, PLOS ONE, vol.10, issue.6, 2015.

. Eular--european, Patient Involvement in Research. A way to success, League Against Rheumatism, vol.24, 2013.

. Eupati, Évaluation des résultats signalés par les patients (PRO), 2016.

E. Group, EuroQol--a new facility for the measurement of health-related quality of life, Health Policy, vol.16, issue.3, pp.199-208, 1990.

, Reflection paper on the use of patient reported outcome (PRO) measures in oncology studies, European Medicines Agency, p.9, 2014.

. Eurordis, Patient Relevant Outcome Measures & Patient Reported Outcomes, 2015.

. Fda, Guidance for Industry. Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. U.S. Department of Health and Human Services Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER), Center for Devices and Radiological Health (CDRH), 2009.

M. Fleischmann and B. Vaughan, The challenges and opportunities of using patient reported outcome measures (PROMs) in clinical practice, International Journal of Osteopathic Medicine, vol.28, pp.56-61, 2018.

J. Greenhalgh, The applications of PROs in clinical practice: What are they, do they work, and why? Quality of Life Research, vol.18, pp.115-123, 2008.

K. D. Gregory, L. M. Korst, S. Saeb, and M. Fridman, The role of patient-reported outcomes in women's health, OBG Management, vol.30, issue.3, pp.18-23, 2018.

C. Grossman, Patient-Reported Outcomes: Design with the End in Mind, 2018.

K. L. Haywood, R. Wilson, S. Staniszewska, and S. Salek, Using PROMs in Healthcare: Who Should Be in the Driving Seat-Policy Makers, Health Professionals, Methodologists or Patients? The Patient -Patient-Centered Outcomes Research, vol.9, pp.495-498, 2016.

. Ichom--lnternational, What is valuebased health care?, Consulté 11 février 2019, à l'adresse ICHOM website, 2018.

S. Jaroslawski, P. Auquier, B. Borissov, C. Dussart, and M. Toumi, Patient-reported outcome claims in European and United States orphan drug approvals, Journal of Market Access & Health Policy, vol.6, issue.1, p.1542920, 2018.

J. R. Johnson and R. Temple, Food and Drug Administration requirements for approval of new anticancer drugs, Cancer Treat Rep, vol.69, issue.10, pp.1155-1162, 1985.

D. Kyte, J. Ives, H. Draper, and M. Calvert, Management of Patient-Reported Outcome (PRO) Alerts in Clinical Trials: A Cross Sectional Survey, PLoS ONE, issue.1, p.11, 2016.

,

P. Marino, M. Bannier, J. Moulin, and G. Gravis, Rôle et utilisation des Patient reported outcomes (PROs) dans la prise en charge des patients en oncologie, Bulletin du Cancer, vol.105, issue.6, pp.603-609, 2018.

S. P. Mckenna, Measuring patient-reported outcomes: Moving beyond misplaced common sense to hard science, BMC Medicine, vol.9, 2011.

S. G. Mills, A. Jonker, L. Lau, and S. Aymé, Patient-Centered Outcome Measures Initiatives in the Field of Rare Diseases (p. 30 pages), Available on the IRDiRC-International Rare Diseases Research Consortium's website, 2016.

T. Morel and S. J. Cano, Measuring what matters to rare disease patients -reflections on the work by the IRDiRC taskforce on patient-centered outcome measures, Orphanet Journal of Rare Diseases, vol.12, issue.1, p.171, 2017.

H. Nabarette, J. Peterka, J. A. Urtizberea, and T. N. Brignol, Médicaments orphelins : un nouveau concept à l'épreuve du temps. Les Cahiers de Myologie, pp.5-10, 2018.

J. Nicklin, F. Cramp, J. Kirwan, M. Urban, and S. Hewlett, Collaboration with patients in the design of patient-reported outcome measures: Capturing the experience of fatigue in rheumatoid arthritis, Arthritis Care & Research, vol.62, issue.11, pp.1552-1558, 2010.

E. Nilsson, L. Orwelius, and M. Kristenson, Patient-reported outcomes in the Swedish National Quality Registers, Journal of Internal Medicine, vol.279, issue.2, pp.141-153, 2016.

C. Pascoal, S. Brasil, R. Francisco, D. Marques-da-silva, A. Rafalko et al., Patient and observer reported outcome measures to evaluate health-related quality of life in inherited metabolic diseases: a scoping review, Orphanet Journal of Rare Diseases, vol.13, issue.1, p.215, 2018.

V. Rabeharisoa and L. Doganova, Making rareness count: testing and pricing orphan drugs, I3Working Papers Series, p.17, 2016.
URL : https://hal.archives-ouvertes.fr/hal-01379153

V. Rabeharisoa, T. Moreira, and M. Akrich, Evidence-based activism: Patients', users' and activists' groups in knowledge society, BioSocieties, vol.9, issue.2, pp.111-128, 2014.
URL : https://hal.archives-ouvertes.fr/hal-00976588

M. Shapiro, D. Johnston, J. Wald, and D. Mon, Research Triangle Park, NC: RTI International. Prepared for Office of Policy and Planning, Office of the National Coordinator for Health Information Technology, 2012.

A. Slade, F. Isa, D. Kyte, T. Pankhurst, L. Kerecuk et al., Patient reported outcome measures in rare diseases: a narrative review, Orphanet Journal of Rare Diseases, vol.13, issue.1, p.61, 2018.

C. F. Snyder, N. K. Aaronson, A. K. Choucair, T. E. Elliott, J. Greenhalgh et al., Implementing patient-reported outcomes assessment in clinical practice: A review of the options and considerations, Quality of Life Research, vol.21, issue.8, pp.1305-1314, 2012.

M. Stefanou and I. Amygdalos, Patient-led research in amyotrophic lateral sclerosis: Quo vadis? Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, vol.16, pp.418-422, 2015.

S. Stefanovic, M. Wallwiener, U. Karic, C. Domschke, L. Katic et al., Patient-reported outcomes (PRO) focused on adverse events (PRO-AEs) in adjuvant and metastatic breast cancer: clinical and translational implications, Supportive Care in Cancer: Official Journal of the Multinational Association of Supportive Care in Cancer, vol.25, issue.2, pp.549-558, 2017.

J. C. Streuli and E. Vayena, The Promising Revolution of Participant-Led Research in Rare Neurological Diseases. Potential Benefits and Pitfalls, vol.32, pp.177-182, 2015.

R. E. Tractenberg, A. Garver, I. H. Ljungberg, M. M. Schladen, and S. L. Groah, Maintaining primacy of the patient perspective in the development of patient-centered patient reported outcomes, PloS One, vol.12, issue.3, 2017.

C. Treadwell, Clinical Outcome Assessments: Nothing About Me Without Me, Cancer Policy Monitor, 2018.

E. Vayena, R. Brownsword, S. J. Edwards, B. Greshake, J. P. Kahn et al., Research led by participants: A new social contract for a new kind of research, Journal of Medical Ethics, vol.42, issue.4, pp.216-219, 2016.

T. Weldring and S. M. Smith, Patient-Reported Outcomes (PROs) and Patient-Reported Outcome Measures (PROMs), Health Services Insights, vol.6, pp.61-68, 2013.

P. Wicks, Patient, study thyself, BMC Medicine, vol.16, 2018.

B. Wiering, D. Boer, and D. Delnoij, Patient involvement in the development of patientreported outcome measures: A scoping review, Health Expectations, vol.20, issue.1, pp.11-23, 2017.

K. Williams, J. Sansoni, D. Morris, P. Grootemaat, and C. Thompson, Available on the Australian Commission on Safety and Quality in Health Care's website, p.91, 2016.

R. J. Willke, L. B. Burke, and P. Erickson, Measuring treatment impact: a review of patientreported outcomes and other efficacy endpoints in approved product labels, Controlled Clinical Trials, vol.25, issue.6, pp.535-552, 2004.

W. A. Wood, A. V. Bennett, and E. Basch, Emerging uses of patient generated health data in clinical research, Molecular Oncology, vol.9, issue.5, pp.1018-1024, 2015.