The Office of Health Economics

Scientific advancements offer significant opportunities for better patient outcomes, but also present new challenges for value assessment, affordability and access. Alternative payment models (APMs) can offer solutions to the ensuing payer challenges. However, a comprehensive framework that matches the spectrum of challenges with the right solution, and places them within a framework for implementation, is currently missing. To fill this gap, we propose evidence-based steps for the effective selection and implementation of APMs. First, contracting challenges should be identified and mapped to potential APM solutions. We developed a decision guide that can serve as a starting point to articulate core problems and map these to APM solutions. The main problem categories identified are: budget impact and uncertainty, value uncertainty, and the scope of value assessment and negotiation. Sub-categories include affordability, uncertainty of effectiveness, and patient heterogeneity, which map onto APM solutions such as outcome-based agreements, instalments, and subscription models. Just as important are the subsequent identification and assessment of the feasibility of potential solutions as well as collaboration to reach agreement on the terms of the APM and lay the groundwork for effective implementation. We adduce recent examples of APM implementation as evidence of how commonly cited implementation barriers can be overcome by applying pragmatic design choices and collaboration. This step-by-step framework can aid payers and manufacturers in the process of effectively identifying, agreeing on, and implementing APMs to advance patient access to cost-effective medicines, while at the same time providing appropriate incentives to support future innovation.

In the last decade progress has been made in identifying solutions to the "technical problem" of attributing the value of combinations between component parts, but not in adapting mechanisms to implement solutions. We propose a way forward to address the "mechanism problem", arguing that it is essential HTA bodies and/or pricing and reimbursement authorities get actively involved in setting out attribution rules or methods. HTA and pricing/reimbursement authorities have, in essence, adopted one of three strategies: (i) "Do nothing"; (ii) Take a simplistic and arbitrary approach, such as the German law imposing a "haircut" of 20% on the prices of products used in combination or (iii) "Passing the parcel" to the companies and to competition authorities, hoping they will solve the problem for them. Even if a competition law compatible solution is possible, three challenges remain. First, the cost and effort of using it may be too high in relation to any likely gains. Second, the bargaining power of the backbone owner under current HTA / pricing rules is so high that, likely, no solutions that incentivise add-on therapy development will emerge from a process from which HTA bodies absent themselves. Third, most solutions emerging from such a process which give any returns to the add-on likely need the backbone to have a different price (i.e. lower) in combination use as compared to monotherapy use, requiring payer approval for multi-indication pricing. Resolution of the combination challenge thus requires HTA and reimbursement bodies involvement in value attribution.

People with Parkinson’s disease can experience psychological distress and have difficulties accessing face-to-face psychological support due to symptom burden and limited availability of psychological services. Digital options for psychological support can bridge this gap. We have developed an app based on acceptance and commitment therapy (ACT) to support people with Parkinson’s to improve psychological wellbeing. To assess the acceptability of the app and the feasibility of conducting a randomised controlled trial (RCT) to evaluate the effectiveness of using the app to improve wellbeing for people with Parkinson’s. We will conduct a parallel-group randomised controlled feasibility trial comparing a digital app based on ACT (intervention group) to usual care (waitlist control group). We will recruit 60 people with Parkinson’s, 40 to the intervention group and 20 to the control group. Primary feasibility outcomes include recruitment and retention rate, intervention engagement and satisfaction. Secondary outcomes include measures of clinical effectiveness (anxiety and depression), quality of life and cost-effectiveness. Interviews will be conducted to assess acceptability of the app. Primary feasibility outcome data will be analysed descriptively and compared against pre-defined feasibility criteria. Secondary outcomes will be analysed based on an intention-to-treat principle, and a cost-consequence analysis will be used to estimate cost-effectiveness. Interviews will be analysed using a deductive thematic analysis based on the Theoretical Framework of Acceptability. This trial will provide data on the feasibility of conducting a full-scale RCT of the effectiveness and cost-effectiveness of the app to improve psychological wellbeing for people with Parkinson’s disease.

OBJECTIVE:The three-level and five-level child-friendly versions (EQ-5D-Y) are widely employed for assessing health-related quality of life (HRQoL) in children. However, their effectiveness in asthma remains understudied. We aimed to assess their psychometric properties in asthmatic children. METHODS:Seventy-six dyads of patients and their parents completed EQ-5D-Y-5L and EQ-5D-Y-3L, respectively, at two visits. Test-retest reliability and patient-caregiver agreement were assessed using intraclass correlation coefficient (ICC). Known-groups validity was assessed by comparing scores across varying asthma control levels. Responsiveness was assessed in children showing improved overall health and asthma control. RESULTS:ICCs for test-retest reliability of EQ-5D-Y-3L and EQ-5D-Y-5L summary scores ranged from 0.744 to 0.898 (self-report) and 0.525 to 0.767 (proxy-report), indicating satisfactory reliability. As expected, EQ-5D-Y summary scores for both versions were better in well- or partially-controlled patients, with fewer problems reported for each dimension as well. Effect sizes ranged from 0.19 to 1.00 for the self-completed version, from 0.39 to 0.83 for the proxy version, and from 0.26 to 1.40 for EQ VAS in patients showing improvement. Patient-caregiver agreement ranged from moderate to good. CONCLUSION:The results support the use of the self-complete and proxy versions of both EQ-5D-Y-3L and EQ-5D-Y-5L to assess health outcomes in children with asthma. The self-complete EQ-5D-Y-3L is preferred where possible, but parental carer reports can be used as a reasonable proxy when necessary. The EQ VAS offers a valuable complementary perspective on overall health. Further investigation of the EQ-5D-Y-5L is recommended once a value set becomes available.

The choice of perspective in valuation tasks is likely to affect the scale of EQ-5D-Y-3L value sets, but less is known about how it affects the relative importance of different dimensions. The aim of this study was to examine how preferences for EQ-5D-Y-3L health states differ according to different perspectives utilising two methods: the Online elicitation of Personal Utility Functions (OPUF) tool and a discrete choice experiment (DCE). An online survey was designed containing the OPUF tool and a DCE. Adult respondents from the United Kingdom were randomised to one of five different perspective arms: (1) 4-year-old child, (2) 10-year-old child, (3) a child of unspecified age, (4) another adult, and (5) own health. The resulting OPUF value sets (social utility functions), and relative importance scores for the five dimensions from both methods, were compared across perspectives. Results differed by perspective in both valuation tasks. In both tasks, ‘looking after myself’ was less important and ‘pain or discomfort’ was more important in the child perspectives than in the adult perspectives. Furthermore, the scale of the value sets produced by the OPUF tool differed by perspective, with the value of the worst health state being significantly lower in the adult perspectives than in the child perspectives. Our results suggest that the valuation of the EQ-5D-Y-3L is affected by the perspective that adult respondents are asked to take. Researchers should be aware of the potential impact and ensure that relevant stakeholders understand this when designing valuation studies.