Table 1 Overview of value assessment frameworks relevant to OMPs
Name of framework | Year of publication | Country or region | Description of framework | Number of domains and/or criteria in frameworka | Reference | |
|---|---|---|---|---|---|---|
OMP-specific frameworks | Transparent Value Framework (TVF) by the Working Group on Mechanism of Coordinated Access to Orphan Medicinal. Products (MoCA-OMP) | 2012 | Europe | Instrument to assist value-based pricing in a transparent way listing important criteria contributing to the value of an OMP. Semi-quantitative framework for determining the degree to which the individual criteria are met | 4 criteria | [25] |
Framework by Hughes-Wilson et al. | 2012 | Europe | Assessment system based on several weighted evaluation criteria and their corresponding potential parameters, which would serve as a tool for Member States, to value an OMP | 9 criteria | [26] | |
Multi-criteria decision analysis (MCDA) Framework by Sussex et al. | 2013 | Europe | Framework for informing value definition of OMPs and providing an explicit understanding of trade-offs for decisions on their eligibility for funding | 2 domains 8 criteria | [21] | |
MCDA for ranking rare diseases in Russia by Fedyaeva et al. | 2014 | Russia | MCDA used to estimate relative importance of 16 criteria to rank and prioritise rare diseases | 2 domains 16 criteria | [27] | |
MCDA framework by Schey et al. | 2014 | Europe | MCDA framework using criteria suggested by Hughes-Wilson et al. A supplementary literature review identified other attributes described in the application of MCDA in rare diseases. | 13 criteria | [28] | |
Decision-making framework by Paulden et al. | 2015 | Worldwide | Decision factors related to the reimbursement of OMPs were identified in a scoping review and synthesised into a decision-making framework | 3 domains 29 criteria | [29] | |
EVIDEM (EVIdence based Decision-Making) framework by Wagner et al. | 2015 | Worldwide | Quantitative MCDA framework to address rare disease issues and policies The framework is regularly updated, the latest version is V3.1 | 6 domains 15 criteria | [30] | |
Non-OMP frameworks | Conceptual MCDA framework by Kanavos et al. | 2013 | N/A | MCDA framework to assess the value of a new drug in a value-based context | 4 domains 12 criteria | [31] |
MCDA framework in Hungary by Endrei et al. | 2014 | Hungary | MCDA framework used in the evaluation of medical technologies in hospitals | 6 criteria | [32] | |
Framework by Williams et al. | 2014 | Case studies from the UK, Germany, Spain to illustrate the MCDA process | Process for using MCDA by a pharmaceutical company to estimate the probability of a positive recommendation for reimbursement for a new drug given drug and environmental attributes | Participants were asked to select up to 10 criteria out of a long list of reimbursement criteria | [33] | |
MCDA framework in Germany by Wahlster et al. | 2015 | Germany | Case study exploring the use of an MCDA approach to appraise a pulmonary heart sensor in Germany using the EVIDEM V2.2 framework | Not mentioned (EVIDEM framework V2.2) | [34] | |
The European Society for Medical Oncology magnitude of Clinical Benefit Scale (ESMO-MCBS) | 2015 | Europe | Tool to assess the magnitude of clinical benefit for cancer medicines to derive a relative ranking between new treatments | 3 to 5 criteria Number of criteria depend on the type of therapy (curative vs palliative) | [35] | |
The American Society of Clinical Oncology (ASCO) Conceptual Framework | 2015 | USA | Framework for assessing the value of treatment options and was designed to eventually assist in facilitating shared decision making with patients about clinical benefits. | 3 criteria | [36] |