Zamora, B., Maignen, F., O’Neill, P., Mestre-Ferrandiz, J. and Garau, M.
The European Commission’s (EC) Orphan Medicinal Products Regulation intended to incentivise the research, development and marketing of new treatments for rare and chronically disabling or life-threatening diseases. Marketing authorisation granted to orphan medicinal products (OMPs) is however only the first step; patients have access to medicines once reimbursement or health technology assessment (HTA) decisions are implemented by national health systems.
Marsden, G., Towse, A., Pearson, S.D., Dreitlein, B. and Henshall, C.
This report presents an analysis of the significant clinical potential of gene therapy and the unique challenges in developing and evaluating evidence on their effectiveness and value. Special attention is given to pricing and payment mechanisms, including new approaches to payment based on long-term amortization of initial costs.
This OHE Seminar Briefing summarises a seminar given by Dr Nicholas Hicks, Co-founder and Chief Executive of COBIC (Capitated Outcomes-Based Incentivised Care). Dr Hicks describes COBIC’s outcomes-centred approach that has been adopted by NHS services across England and that brings together commissioners, providers, other professions, patients and carers.
In 2016 the University of York undertook a review exercise to determine whether NICE’s existing methods and processes are appropriate for assessment of regenerative medicines. The purpose of this OHE report is to explore this review exercise and to assess whether or not the resulting conclusions are appropriate.
The aim of this paper is to consider what normative arguments might exist for advocating the use of any given measure of the average in the context of health state values. We begin by providing examples of the importance and implications of the choice of the measure of central tendency in stated preference studies (including both EQ-5D values and corresponding issues in the willingness to pay literature).
Hernandez-Villafuerte, K., Garau, M., Towse, A., Garrison, L. and Grewal, S.
PhRMA commissioned OHE Consulting to explore options and recommend methods that could help middle-income countries efficiently and effectively identify medicines for formulary inclusion.
This report presents a framework for developing medicines formularies which combines macro-level decision making factors (focusing on the health system organisation and its priority setting), and micro-level factors (looking at intervention-specific effects) to determine the overall value of medicines.
This Research Paper explores decisions by the National Institute for Health and Care Excellence (NICE) that new technologies are to be recommended in line with clinical practice (RiLCP). The authors qualitatively assess documentation associated with RiLCP decisions to create a framework to describe common themes, and assess the level of patient access associated with each RiLCP decision.
Garau, M., Marsden, G., Devlin, N., Amedeo Mazzanti, N. and Profico, A.
Published paper using MCDA to obtain preferences on decision criteria across three stakeholder groups (patients, clinicians and payers) in Italy and to assess the value of obinutuzumab for rituximab-refractory iNHL.
Fischer, A., Hernandez-Villafuerte, K., Latimer, N., and Henshall, C.
This Research Paper reports on a literature review of trials using PFS as a surrogate for OS in oncology between 2012 and 2016; a workshop and interviews involving people from a regulatory agency (EMA) a “payer” organisation (NICE), academics, and from the member organisations comprising POI; and critiques of statistical modelling and economic analysis approaches to surrogacy.