Staff from OHE recently attended the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 20th Annual European Congress. We contributed to a variety of sessions, and can now share our presentations from the meeting.
Koonal Shah presented two posters. One examined whether society is willing to pay more for cancer prevention and treatment than for other types of health care. It reported selected highlights from Koonal’s article in the ‘Value and Cancer’ special issue of the Journal of Cancer Policy.
In another poster, Koonal examined how framing effects, such as the use of indifference options and visual aids, affected the extent of public support for an end of life premium observed in an online stated preference survey. The study was a collaboration between OHE and Aki Tsuchiya and Allan Wailoo of the University of Sheffield.
Martina Garau led a workshop on ‘Sustainable funding and fair pricing for orphan drugs. What are the solutions?’ Since the inception of the European regulation on orphan drugs, there has been a debate as to whether conventional economic evaluation methods should be applied to assess these treatments and whether their funding is sustainable in healthcare systems facing substantial budgetary pressures. The evidence in support of paying a premium for orphan drugs is mixed but in practice, many countries in Europe provide access to them. The purpose of this workshop was to discuss options to make the funding of valuable orphan drugs sustainable for healthcare systems and to provide a ‘fair’ reward to manufacturers investing in areas of high unmet need.
Martina introduced the session, providing evidence on the rate of HTA approval and reimbursement of orphan drugs across Europe. Saskia Knies (National Health Care Institute, The Netherlands) offered insights on and learnings from the Dutch approach to appraising orphan drugs. Mike Drummond (University of York) explained a new method to adjust the cost-effectiveness threshold to reflect the difference between the population size of orphan and non-orphan treatments. To engage with the audience, Martina asked, and facilitated discussion around, a series of questions via an online poll relating to the appraisal of orphan drugs.
Martina was also part of the faculty of the short course on “Using Multi-Criteria Decision Analysis (MCDA) in Health Care Decision Making: Approaches & Applications”. Her part explained the rationale for using MCDA in HTA, reviewed the current landscape of MCDA applications, and discussed critical issues which still need to be addressed in the use of MCDA in HTA. Participants engaged in practical exercises for building an MCDA model, selecting criteria and eliciting preferences across them. Participants also took part in a series of polls around opportunities and challenges of applying MCDA in HTA.
Adrian Towse discussed the challenges of conducting HTA of gene therapies as part of an ISPOR issue panel. He explained that gene therapy is making progress – slowly – and that more consideration needs to be given to handling uncertainty; innovative payment mechanisms including outcomes-based payments; relevant thresholds and an appropriate discount rate. Other panellists included Professor Ron Akehurst of the University of Sheffield and Bresmed and Clark Paramore from BlueBird Bio.
In the light of NICE’s recent position statement regarding use of the EQ-5D-5L, a workshop was convened to consider the issues for NICE HTA of using the EQ-5D-5L and its English value set. Rosie Lovett from NICE outlined the key considerations. Allan Wailoo presented analysis undertaken by the NICE Decision Support Unit to understand the impact of the new value set on ICERs. OHE’s Nancy Devlin, representing the EuroQol Group, provided an overview of the evidence comparing the 3L and 5L descriptive systems, concluding that evidence points clearly to the 5L being a better descriptive system than the 3L: it more accurately describes patients’ health. The value sets for the 5L also have some advantages over the 3L value sets: 5L value sets are based on more up to date preference data and draw on improvements in methods developed over the last 20 years. There are important differences between the 5L value set for England and the UK 3L value set, but, to some extent, these are attributable to the ‘unusual’ characteristics of the UK 3L value set. Indeed, there is evidence to suggest that the UK 3L value set may have led to overestimation of QALYs. Nancy concluded by noting that the transitional challenges for NICE, posed by a shift away from the 3L to any new preference-based measure, are inevitable. All speakers’ slides from this workshop (W10) may be downloaded from the ISPOR website.
In an issue panel on the subject of patient-centred care in Europe, three speakers including Nancy Devlin departed from the convention of each giving a presentation, and instead opted to have a ‘live’ conversation/debate around the central topics outlined in the slide deck below. The panel covered wide-ranging issues relating to how value for money is assessed, and what being ‘patient-centred’ means in this context, drawing contrasts between the UK and German health care systems.
An invited issue panel took as its starting point the recent ISPOR report on US value frameworks. Panellists considered how MCDA approaches could be used in health technology appraisal, with a particular focus on how cost and cost-effectiveness could be addressed when multiple criteria of value are used. OHE’s Nancy Devlin began by asking what was more important to stakeholders: consistency of approach (e.g. what criteria are used; what weight is placed on them) or flexibility? She went on to argue that whatever approach is taken to the assessment of value for new medicines should also be reflected in the way opportunity cost is assessed so that benefits and benefits foregone are considered in commensurate terms. In concluding, Nancy noted that MCDA approaches can provide a useful way of structuring deliberation when multiple criteria of value are important. MCDA does not aim to replace the judgement of HTA committees – but can help committees exercise judgements in a systematic way. What specific approach to MCDA is best will depend on the socio-political characteristics of the health care system – there is unlikely to be a simple ‘one size fits all’ solution.
In comparison with adult health states, relatively little research has been undertaken on the value of health-related quality of life in children. There are a number of challenges and issues in how to go about doing this research. This issue panel highlighted a number of methodological and normative issues surrounding the valuing of children’s health states for the purposes of health technology appraisal. OHE’s Nancy Devlin was the moderator, and began the panel by providing an overview of the issues. Panellists included Deborah Morrison from NICE Scientific Advice; Donna Rowen from ScHARR, University of Sheffield, and Oliver Rivero-Arias from University of Oxford.
Adrian Towse presented work from the Office of Health Economics adapting HTA approaches to assess the value of antibiotics, building on a recent Discussion Forum in London with key stakeholders. Adrian gave an overview of the OHE briefing which proposed a value framework for antibiotics, highlighting the elements that are included in traditional HTA and those generally not considered in the HTA process but of particular relevance to antibiotics. Panellists included Douglas Lundin of the Swedish TLV and Professor Alec Morton of the University of Strathclyde and DRIVE-AB.
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