Value, Affordability, and Decision Making

Besley, S., Henderson, N., Towse, A. & Cole, A. 

Consulting Report
June 2022

Gene therapies represent a new era of medicine, offering the potential for truly transformational health gains, and further benefits for society and health systems. Gene therapy is particularly relevant to rare disease patients, as more than 80 per cent of rare diseases have a known monogenic (single gene) cause. In contrast to traditional small molecule medicines, gene therapies have the potential to correct underlying genetic defects, offering the potential for transformational health gains rather than simply managing symptoms.

OHE and Vifor Pharma present a poster at the last European Conference on Rare Diseases (ECRD) on the Mechanism of Coordinated Access to orphan medicinal products (MoCA) where evidence on avacopan for ANCA-associated vasculitis (AAV) was discussed using the Transparent Value Framework.

In a new paper led by OHE, an international team of co-authors considers the evidence for supply-side cost-effectiveness thresholds, providing a set of recommendations for policymakers. In this blog post, Chris Sampson provides an overview of the findings and key messages.

As part of OHE’s presence at ISPOR 2022 in Washington DC, we hosted an issue panel tackling the hot topic of health technology assessment (HTA) of gene therapies.

The National Institute for Health and Care Excellence (NICE) recently issued its first guidance for a digital therapeutic. NICE recommends the use of Sleepio for treating insomnia symptoms in primary care. OHE played a vital role in developing the evidence to support the guidance. In this blog post, Chris Sampson shares the story of how we got there, and some lessons from the experience.

OHE’s Chris Sampson is currently Chair of the ISPOR Open Source Models Special Interest Group (SIG). The SIG recently published their first journal article in Value in Health and will be delivering a Forum at this week’s ISPOR conference. In this blog post, Chris provides some background to the work and an invitation to get involved.

The NICE methods review consultation included a proposal to consider health inequalities more explicitly in their Health Technology Assessment process. But there is still a long way to go before NICE finds a way to tackle all the complexity of this concept. In the following sections we provide an overall view of the main challenges

As the OHE continues to expand, we have another opportunity to welcome economists at a junior level to our team of talented researchers. Our team get the opportunity to work on a wide variety of policy-relevant projects across the health and life sciences...

Priority setting in health is an important, and sometimes controversial topic. In practice, many health technology assessment agencies give a higher weighting to treatments with ‘desirable’ characteristics. For example, treatments for severe and/or rare diseases. In this blog, we review changes to NICE’s manual to include a new ‘severity modifier’.

We live in a world facing many interrelated challenges, and the choices we make about allocating public sector resources have never been more consequential. The perspective taken in an economic evaluation determines the costs and outcomes included in that evaluation and therefore shapes resource allocation and access to new treatments. In this blog, we review the (lack of) changes to NICE’s manual to include elements of a ‘societal perspective’.


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