Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries

T Zelei, MJ Molnár, M Szegedi, Z Kaló - Orphanet journal of rare diseases, 2016 - Springer
Background In case of orphan drugs applicability of the standard health technology
assessment (HTA) process is limited due to scarcity of good clinical and health economic …

A review of international coverage and pricing strategies for personalized medicine and orphan drugs

I Degtiar - Health Policy, 2017 - Elsevier
Background Personalized medicine and orphan drugs share many characteristics—both
target small patient populations, have uncertainties regarding efficacy and safety at payer …

Commonalities and differences in HTA outcomes: a comparative analysis of five countries and implications for coverage decisions

E Nicod, P Kanavos - Health Policy, 2012 - Elsevier
OBJECTIVE: To identify diverging HTA recommendations across five countries, understand
the rationale for decision-making in specific therapeutic categories, and suggest ways …

[HTML][HTML] Health benefit assessment of pharmaceuticals: An international comparison of decisions from Germany, England, Scotland and Australia

KE Fischer, T Heisser, T Stargardt - Health Policy, 2016 - Elsevier
Background Little is known on the performance of the newly introduced health benefit
assessment process, AMNOG, in Germany compared to other health technology …

Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders

M Schlander, S Garattini, S Holm… - Journal of …, 2014 - becarispublishing.com
Drugs for ultra-rare disorders (URDs) rank prominently among the most expensive
medicines on a cost-per-patient basis. Many of them do not meet conventional standards for …

Shining a light in the black box of orphan drug pricing

E Picavet, T Morel, D Cassiman, S Simoens - Orphanet journal of rare …, 2014 - Springer
Background The pricing mechanism of orphan drugs appears arbitrary and has been
referred to as a “black box”. Therefore, the aim of this study is to investigate how drug-and …

Acceptance of health technology assessment submissions with incremental cost-effectiveness ratios above the cost-effectiveness threshold

EA Griffiths, JK Hendrich, SDR Stoddart… - ClinicoEconomics and …, 2015 - Taylor & Francis
Objectives In health technology assessment (HTA) agencies where cost-effectiveness plays
a role in decision-making, an incremental cost-effectiveness ratio (ICER) threshold is often …

[HTML][HTML] Which factors enhance positive drug reimbursement recommendation in Scotland? A retrospective analysis 2006–2013

M Charokopou, IM Majer, J De Raad, S Broekhuizen… - Value in Health, 2015 - Elsevier
Objectives To identify the factors that influence the Scottish Medicines Consortium (SMC) in
deciding whether to accept pharmaceutical technologies for use within the Scottish health …

Stated and revealed preferences for funding new high-cost cancer drugs: a critical review of the evidence from patients, the public and payers

TE MacLeod, AH Harris, A Mahal - The Patient-Patient-Centered …, 2016 - Springer
Background The growing focus on patient-centred care has encouraged the inclusion of
patient and public input into payer drug reimbursement decisions. Yet, little is known about …

Access to medicines-a systematic review of the literature

N Abbas, SS Hasan, L Curley - Research in Social and Administrative …, 2020 - Elsevier
Background Budgetary constraints and the rising cost of new innovative medicines are the
key challenges for access to medicines. Multiple research studies explored diverse …