Articles

  • Lanar, S., Acquadro, C., Seaton, J., Savre, I., & Arnould, B. (2020). To what degree are orphan drugs patient-centered? A review of the current state of clinical research in rare diseases. Orphanet J Rare Dis, 15(1), 134. doi:10.1186/s13023-020-01400-0
  • Stauder, R., Lambert, J., Desruol-Allardin, S., Savre, I., Gaugler, L., Stojkov, I., Siebert, U., & Chevrou-Séverac, H. (2020). Patient-reported outcome measures in studies of myelodysplastic syndromes and acute myeloid leukemia: Literature review and landscape analysis. Eur J Haematol, 104(5), 476-487. doi:10.1111/ejh.13389

 

Conference Abstracts and Posters

  • Desvignes-Gleizes C., Vilcot T., Mc Donough G., Pellegrini M., Savre I., Bothorel S., Pereira M.M., Rath A., Arnould B. Facilitating and Accelerating Patient-Reported Outcomes (PRO) Selection to Use for Clinical Research and Rare Diseases (RD) - A European Rare Disease Research Coordination and Support Action (ERICA) Project. Poster presented at ISPOR Europe 2022.

  • Tanguy Vilcot, Gavin Mc Donough, Céline Desvignes-Gleizes, Mar Mañu Pereira, Mariangela Pellegrini, Sonia Bothorel, Ana Rath, Benoit Arnould. Hierarchical clustering of rare diseases based on functional impacts, an ERICA project study. Poster presented at ISOQOL 2022.
  • Céline Desvignes-Gleizes, Benoit Arnould, Mar Mañú Pereira, Mariangela Pellegrini , Gavin Mc Donough, Sonia Bothorel, Ana Rath. Developing a Patient-Reported Outcome Measures (PROMs) repository for use in Rare Diseases. Poster presented at ECRD 2022.
  • Annie Olry, Gavin McDonough, Charlotte Rodwell, Ana Rath, Catherine Acquadro, Laure-Lou Perrier, Celine Desvignes-Gleizes, Benoit Arnould. Meeting challenges in evaluating and measuring functioning in rare diseases: a collaboration between Orphanet and Mapi Research Trust. 10th European Conference on Rare Diseases & Orphan Products 2020
  • Céline Desvignes-Gleizes, Benoit Arnould (Co-authors), Mar Mañú Pereira, Mariangela Pellegrini, Gavin Mc Donough, Sonia Bothorel, Ana Rath. Developing a Patient-Reported Outcome Measures (PROMs) repository for use in Rare Diseases. 1Oth European Conference on Rare Diseases & Orphan Products 2020.
  • Chevrou-Séverac, H., Lambert, J., Savre, I., Koochaki, P., Gaugler, L., Stojkov, I., Sieber, U., & Stauder, R. (2019, June 13-16). Patient-reported outcome measures in clinical studies for myelodysplastic syndromes and acute myeloid leukemia: A literature review and landscape analysis. European Hematology Association (EHA) Congress 2019, Amsterdam, The Netherlands.
  • Arnould B, Desvignes-Gleizes C, Perrier L, Acquadro C, Rodwell C, Olry A, Rath A. Mapping functions, measurement instruments, and rare diseases: challenges and solutions. ISOQOL 2019, San Diego, California, USA
  • B. Arnould, C. Desvignes-Gleizes, L.L. Perrier, C. Acquadro, C. Rodwell, A. Olry, A. Rath. Mapping PROQOLID To Rare Diseases: An On-Going Colaboration Between Mapi Reasearch Trust And Orphanet. Poster asbtract at ISPOR 2019.
  • Arnould B, Desvignes-Gleizes C, Perrier L, Acquadro C, Rodwell C, Olry A, Rath A. Mapping functions, measurement instruments, and rare diseases: challenges and solutions. ISPOR EU 2019, Copenhagen, Denmark