S. Nestler-Parr

Rare Disease Terminology and Definitions-A Systematic Global Review: Report of the ISPOR Rare Disease Special Interest Group.

D. Hughes, Z. Khan, T. Tesoro, 2015, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group.

S. Simoens, C. Pashos, C. Blanchette, 2018, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report.

E. Perfetto, D. Patrick, L. Burke, 2017, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

A survey exploring caregiver burden and health-related quality of life in hereditary transthyretin amyloidosis

S. Acaster, S. Nestler-Parr, S. H. Lo, 2023, Orphanet Journal of Rare Diseases.

Rare Disease Terminology and Definitions-A Systematic Global Review: Report of the ISPOR Rare Disease Special Interest Group.

D. Hughes, Z. Khan, T. Tesoro, 2015, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.