Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches

Autor: Elena Nicod, Amanda Whittal, Michael Drummond, Karen Facey

Publikováno v: Orphanet Journal of Rare Diseases, Vol 15, Iss 1, Pp 1-14 (2020)

Abstract Background There is increasing recognition that conventional appraisal approaches may be unsuitable for assessing the value rare disease treatments (RDTs). This research examines what supplemental appraisal/reimbursement processes for RDTs a

Externí odkaz: https://doaj.org/article/1137457c7100465aa7b793aca2bc5141

Consideration of quality of life in the health technology assessments of rare disease treatments

Autor: Karen Facey, Sheela Upadhyaya, Amanda Whittal, Michael Drummond, Elena Nicod, Michela Meregaglia

Publikováno v: Nicod, E, Meregaglia, M, Whittal, A, Upadhyaya, S, Facey, K & Drummond, M 2021, ' Consideration of quality of life in the health technology assessments of rare disease treatments ', European Journal of Health Economics . https://doi.org/10.1007/s10198-021-01387-w

OBJECTIVES: Challenges with patient-reported outcome (PRO) evidence and health state utility values (HSUVs) in rare diseases exist due to small, heterogeneous populations, lack of disease knowledge and early onset. To better incorporate quality of li

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::06eda0046610a31a40e337ef6d742c90
https://doi.org/10.1007/s10198-021-01387-w

Implementing Outcomes-Based Managed Entry Agreements for Rare Disease Treatments: Nusinersen and Tisagenlecleucel

Autor: Angèl Link, Elena Nicod, A. Vagoras, Emma Kent, Jaime Espín, Entela Xoxi, Anna Zaremba, Inneke van de Vijver, Tatyana Benisheva, Sheela Upadhyaya, Karen Facey, Aisling O'Leary

Publikováno v: Pharmacoeconomics
PharmacoEconomics
Facey, K M, Espin, J, Kent, E, Link, A, Nicod, E, O'Leary, A, Xoxi, E, van de Vijver, I, Zaremba, A, Benisheva, T, Vagoras, A & Upadhyaya, S 2021, ' Implementing Outcomes-Based Managed Entry Agreements for Rare Disease Treatments : Nusinersen and Tisagenlecleucel ', PharmacoEconomics . https://doi.org/10.1007/s40273-021-01050-5
PharmacoEconomics, Auckland : ADIS INT LTD, 2021, vol. 39, no. 9, p. 1021-1044

Background and Objective Enthusiasm for the use of outcomes-based managed entry agreements (OBMEAs) to manage uncertainties apparent at the time of appraisal/pricing and reimbursement of new medicines has waned over the past decade, as challenges in

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8213f0ba86166a7749991059bdf8de58
http://europepmc.org/articles/PMC8260322

HTA programme response to the challenges of dealing with orphan medicinal products: Process evaluation in selected European countries

Autor: Karen Facey, Lieven Annemans, Sheela Upadhyaya, Elena Nicod, Anne Lee, Anna Bucsics

Publikováno v: Nicod, E, Annemans, L, Bucsics, A, Lee, A, Upadhyaya, S & Facey, K 2019, ' HTA programme response to the challenges of dealing with orphan medicinal products : Process evaluation in selected European countries ', Health policy (Amsterdam, Netherlands), pp. 140-151 . https://doi.org/10.1016/j.healthpol.2017.03.009

BACKGROUND: Challenges commonly encountered in HTA of orphan medicinal products (OMPs) were identified in Advance-HTA. Since then, new initiatives have been developed to specifically address issues related to HTA of OMPs.OBJECTIVE AND METHODS: This s

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2051855867935b52a1c7c7e13003ba41
https://doi.org/10.1016/j.healthpol.2017.03.009

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

Autor: Amanda Whittal, Elena Nicod, Michela Meregaglia

Publikováno v: The Patient-Patient-Centered Outcomes Research
The Patient

Background Patient-reported outcome measures (PROMs) are used in health technology assessment (HTA) to measure patient experiences with disease and treatment, allowing a deeper understanding of treatment impact beyond clinical endpoints. Developing a

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::672dbe0a273d34101f4d4c683f14ff0c