Guest editor: Martina Garau | Director
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The 2022 OHE Masterclass series: supporting HTA and healthcare decision-making to evolve with science.
In my February Bulletin, I highlighted the existing lag between initial proposals for reform of Health Technology Assessment (HTA) and the implementation of such reforms in the real-world, which can take over a decade to materialise. In an effort
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to shorten this time gap and improve understanding of pressing problems and optimal solutions, OHE, among other initiatives, have been running a Masterclass series since 2019. OHE’s Masterclasses provide a curated learning journey led by the most relevant experts in their subject field. Every Masterclass explores the complexities of a topic from multiple perspectives (including theory and empirical evidence, policy and real-world experience), and provides the opportunity to discuss practical ways forward.
The 2021 Series, for example, started with a Masterclass explaining a broad range of novel and established value elements. In the first 2022 Masterclass we took this to the next level for a specific aspect of a broader value assessment: incorporating disease severity considerations in HTA. Speakers included OHE’s Chris Skedgel and David Mott, Vivian Reckers-Droog from Erasmus University, and Ian Watson from NICE. The motivation to explore severity was that, although public support to prioritise the sickest patients and robust methods to reflect this in HTA decision-making exist, HTA systems have been slow to evolve their value assessment paradigms. Currently, some HTA agencies measure severity using quality-adjusted life year (QALY) shortfalls, which prioritise different age groups. Some HTAs also link shortfall categories to adjustments of their willingness-to-pay for health gains. The main reason to follow this approach is simplicity but categories violate principles of vertical equity. Evidence on the impact of introducing severity adjustments on treatment access is lacking and should be developed based on clear metrics. Ways forward to improve current systems that were discussed by the experts included: introducing continuous rather than categorical adjustments, and aligning severity definitions and premium to public preferences.
In the second Masterclass, the spotlight was on Adaptive Pathways (APs) to provide early access to innovative treatments and generating the necessary evidence. APs combine regulatory tools, HTA methods, and payment models based on an iterative approach to evidence generation and patient access, as explained by OHE’s Isobel Firth. When used appropriately, APs can accelerate access to promising therapies targeting unmet needs while addressing uncertainty around their effects and rewarding innovation (the triple win). Lotte Steuten from OHE then discussed elements driving uncertainty at an early stage of a treatment introduction, including surrogate endpoints, and how different types of uncertainty can be addressed. Finally, OHE’s Adrian Towse showed when risk-sharing agreements are optimal and allow earlier treatment access under specific conditions while generating more evidence. The panel discussion highlighted that to safeguard timely and evidence-based patient access after accelerated approval, manufacturers and HTA bodies should engage early to define optimal evidence generation plans, including appropriate use of surrogate endpoints and risk-sharing agreements.
Finally, our first funded* Masterclass offered a deep-dive into payment models for multi-indication therapies. OHE has been at the forefront of proposing and developing pragmatic solutions for implementing Indication-based Pricing (IBP) – our first publication on the topic is dated 2015, with our most recent contribution published at the end of last year. An exceptional panel of stakeholders and experts explained how access may be delayed or even denied due to challenges in linking payment with a medicine’s value across different indications. Payment models that account for differential value across indications represent an important part of the solution for multi-indication therapies. The panel demonstrated how value recognition at the indication-level could be achieved in multiple ways while acknowledging divergent country health systems and experiences. Data systems are critical and should evolve to accommodate the rapidly changing treatment landscape.
More to come in the second half of 2022 to keep reducing the lag between evidence and policy reforms, and improving decision-making in healthcare.
*This was an AstraZeneca organised and funded non-promotional meeting facilitated by OHE.
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“[..] reported problems expressed in methodological aspects of HTA of these technologies also often find their root in a lack of available data, rather than in the complexity of the technology itself."
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A new survey to HTA organisations explores challenges in HTA of complex health technologies | 01 June, 2022
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Complex technologies, including advanced therapeutic medicinal products (ATMPs) and histology-independent therapies, are increasingly being developed leading to a need for new methods to guide HTA decision making. The survey assessed the challenges experienced by European HTA organisation
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for complex health technologies. Authors found that reported problems have their root in a lack of available data, rather than in the complexity of the technology itself. Future attempts to improve data quality could focus on the alignment of data generation with evidentiary requirements early in the development process.
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A new paper led by OHE provides a set of actionable recommendations to policymakers considering the use of supply-side cost-effectiveness thresholds to support decision-making. They include: defining the decision scope, developing an evidence assessment process, maintaining flexibility, and support (local) decision-makers.
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A recent OHE report founds that the Congressional Budget Office (CBO) latest improvements to its simulation model move the estimated innovation impact in the right direction. However, the model still suffers from serious limitations including an oversimplified decision rule regarding pharmaceutical companies’ R&D investment, a narrow conception of the value of biopharmaceutical innovation, and significant uncertainty in the distribution of the estimated impacts.
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Based on a recent pilot between the European MoCA group and a biopharmaceutical company, this blog highlights that the MoCA process, which is voluntary, non-binding, and free-of-charge, provides a valuable opportunity for early exchange of information and perspectives on the value of an orphan medicine while it is at the development stage.
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The privatisation of the NHS in England, through the outsourcing of services to for-profit companies, consistently increased in 2013–20, following the 2012 Health and Social Care Act. A recent study showed that private sector outsourcing corresponded with significantly increased rates of treatable mortality, potentially as a result of a decline in the quality of health-care services.
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A new policy paper released by the UK Department of Health & Social Care announces new funding for digital adoption, a plan to support electronic patient records in all NHS trusts and to use digital tools to manage long-term health conditions. Key pillars of the reform include: improved digital foundations, enhanced national digital channels, and accelerated adoption of proven tech.
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An estimated 37.3 million Americans have Type 2 diabetes, over 40% higher than a decade ago. In this commentary, authors analyse evidence on the impact of two payment models, including those linking physician and hospital reimbursement to the value of the care provided, and value-based insurance design programmes. They found that both have the potential to improve diabetes care but broader alignment of payment reforms is needed to maximize their impact.
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6 October | Hybrid
This year, for the first time, the OHE Annual Lecture is hybrid. You now have the opportunity to join us in London at the Royal College of Physicians or virtually from anywhere in the world on the evening of Thursday 6 October for our 2022 lecture 'Universal Health Coverage: more than just old wine in a new bottle?' presented by Eddy van Doorslaer.
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Our Annual Lecture Series started in 1994 when Sir Douglas Black presented 'A doctor looks at Health Economics'. Throughout the past 28 years we’ve had the privilege of hosting some of the greatest thinkers in the economics of health and health care. The purpose of our Series is to explore urgent and important issues facing health systems around the world and raises awareness of research and evidence that can contribute to addressing these challenges. We hope you can join us, in-person or virtually, for the 2022 instalment.
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Speaker: Eddy van Doorslaer
Eddy has taught and researched in health economics for almost 40 years and has published extensively on the measurement and explanation of inequalities in health and inequities in health care. He holds a joint appointment as Prof. of Health Economics at the Erasmus School of Economics and the Erasmus School of Health Policy and Management, as well as an elected member of the Netherlands Royal Academy of Science since 2014, a consultant for the World Bank, WHO and the OECD, and Editor of JHE and HE for many years.
Register your free ticket today.
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