A Case Study Analysis: Challenges in the NICE Evaluation of Multi-Indication Medicines for Rare and Ultra-Rare Diseases
A selection of the posters and presentations delivered by OHE at the ISPOR 22nd Annual International Meeting in Boston, May 2017, are available below.
Adrian Towse participated in an invited issues panel, alongside: Peter Neumann (Tufts Medical Center) (Moderator), Lou Garrison (University of Washington and OHE Senior Visiting Fellow), Eleanor Perfetto (National Health Council, US) and Edmund Pezalla (Enlightenment BioConsult), that looked at: ISPOR’s special task force report on us value assessment frameworks: what does it say and is it helpful? The white paper’s key points and recommendations were presented, and its contributions and limitations discussed from the perspective of three key stakeholder groups: patients, payers, and the non-US audience. Adrian gave the non-US perspective.
ISPOR Special Task Force on US Value Frameworks: A Non-US Perspective from Office of Health Economics
Nancy Devlin, along with co-panellists Shelby Reed, F. Reed Johnson and Sachin Kamal-Bahl discussed: Moving Beyond the QALY in Patient-Centered Value Frameworks: But, in what direction? The panel explored different perspectives on the way that patients’ preferences and views could best be incorporated into value frameworks. Nancy argued that there is a case for systematically considering evidence patient preferences as part of HTA processes. However, ultimately HTA decisions involve weighing up both benefits to patients affected by the technology and benefits foregone to other patients so there remains an important role for societal preferences.
Moving Beyond the QALY in Patient-Centered Value Frameworks: But, in What Direction? from Office of Health Economics
Adrian Towse participated in an issues panel entitled: Developing and paying for gene therapies: can we resolve the conflicts? Co-panelists were Bill Dreitlein (ICER) (moderator), John Watkins (Premera Blue Cross) and Clark Paramore (BlueBird Bio). The panel discussed the tension between manufacturers of gene therapies seeking compensation for their innovation at high prices, payers struggling with affordability, and technology assessment organisations wrestling with how to measure value. Adrian gave a European perspective.
Developing and Paying for Gene Therapies: Can We Resolve the Conflicts? A European Perspective from Office of Health Economics
Adrian Towse also moderated an issues panel on: Perspectives on the relationship between cost effectiveness and affordability. Adrian introduced the topic, highlighting relevant papers and questioning what we really mean by affordability in various contexts. Panellists Mark Sculpher (University of York), Patricia Danzon (University of Pennsylvania) and Steve Pearson (ICER) discussed cost-effectiveness analysis, budget thresholds, and the potential effect of budget impact on the cost-effectiveness threshold.
Perspectives on the Relationship Between Cost Effectiveness and Affordability from Office of Health Economics
Paula Lorgelly presented three posters:
- Comparing HTA outcomes for centrally authorised medicinal products in Great Britain 2011-2015. Authors: Maignen, F., Zamora, B., O’Neill, P., Garau, M., Lorgelly, P.
Most new substances are approved in the European Union via the centralised procedure (coordinated by the European Medicines Agency); many health care systems in Europe then evaluate new technologies according to their effectiveness and cost effectiveness. This poster presents a comparison of outcomes of HTA evaluations for centrally authorised products in Great Britain. - Adjusting for differential item functioning in the EQ-5D-5L using externally-collected vignettes. Authors: Lorgelly, P., Knott, R. (Monash University).
There is concern that responses to questions on subjective scales will be inaccurate if certain groups of people systematically differ in their interpretation and use of the response categories – known as differential item functioning (DIF). It is possible to correct for DIF by using vignette responses collected externally to the main dataset of interest. This poster shows how this approach can be applied to the EQ-5D-5L to obtain QALY measures that are comparable across different population groups. - Comparing cancer-specific preference-based outcome measures: the same but different. Authors: Lorgelly, P., Norman, R. (Curtin University).
Disease-specific outcome measures for use in economic evaluations are growing in popularity. Within cancer there are now two preference-based measures, the EORTC-8D and the QLU-C10D. This poster presents research that compares these two measures.
Comparing HTA Outcomes for Centrally Authorized Medicinal Products in Great Britain 2011-2015 from Office of Health Economics
Adjusting for Differential Item Functioning in the EQ-5D-5L Using Externally-Collected Vignettes from Office of Health Economics
Comparing Cancer-Specific Preference-Based Outcome Measures: The Same but Different from Office of Health Economics
The OHE team were also actively involved in a number of ISPOR events held in Boston alongside the conference, including:
- Nancy Devlin was an instructor on an ISPOR short course on MCDA, along with Maarten Ijzerman and Kevin Marsh
- Adrian Towse was an instructor on an ISPOR short course on Risk-Sharing/Performance-Based Arrangements for Drugs and Other Medical Products, along with Lou Garrison and Josh Carlson
- Nancy Devlin participated in Board of Directors meeting, and chaired a meeting of the Management and Advisory Board of ISPOR journals
- Nancy Devlin participated in a Patient Representatives Roundtable Forum.
For more information on any of the above please contact the relevant author via our meet the team webpage.
