The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment

Author:

Whittal AmandaORCID,Meregaglia MichelaORCID,Nicod ElenaORCID

Funder

Horizon 2020 Framework Programme

Publisher

Springer Science and Business Media LLC

Reference60 articles.

1. US FDA. Patient-reported outcomes (PROs) in medical device decision-making, [Internet]. 2019, https://www.fda.gov/about-fda/cdrh-patient-engagement/patient-reported-outcomes-pros-medical-device-decision-making#whatare. Accessed 25 June 2019.

2. Smith A. What are PROMs. York Health Econ Consort. 2014;44:1–2.

3. Bell JA, et al. A pragmatic patient-reported outcome strategy for rare disease clinical trials: application of the EORTC item library to myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia. J Patient-Rep Outcomes. 2019. https://doi.org/10.1186/s41687-019-0123-4.

4. Brazier J, Ara R, Rowen D, Chevrou-Severac H. A review of generic preference-based measures for use in cost-effectiveness models. Pharmacoeconomics. 2017;35(s1):21–31. https://doi.org/10.1007/s40273-017-0545-x.

5. Sabino G, Mills A, Jonker AH, et al. Patient-Centered Outcome Measures in the Field of Rare Diseases: International Rare Diseases Research Consortium (IRDiRC), 2016. http://www.irdirc.org/wp-content/uploads/2016/03/PCOM_Post-Workshop_Report_Final.pdf. Accessed 5 Nov 2019.

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