In light of concerns that not all medicines for ultra-rare (also known as ultra-orphan) conditions are appraised under the same NICE process, a new OHE Consulting Report discusses the distinct ethical and economic challenges faced by medicines for ultra-rare conditions, with particular reference to the challenges of HTA in the UK. A failure to consistently consider all ultra-rare disease medicines under the HST process could lead to inequalities in access and health outcomes for patients with ultra-rare conditions.

In 1994 Ben van Hout introduced the concept of the cost effectiveness acceptability curve (CEAC). In the 25 years since, the CEAC has become a standard part of health economic evaluations and incorporated into health technology assessment guidelines. Professors Nancy Devlin and Andrew Briggs spoke at a reception at ISPOR Europe this year to celebrate these contributions. As 2019 comes to a close, OHE reflects on 25 years of the CEAC.

This research by a team of OHE and IHE researchers estimates the value added by second generation antipsychotics over their life-cycle in the UK and Sweden. It concludes that considering the entire life-cycle, the value added by SGAs to the system is higher than the expected value estimated at launch. P&R decisions should consider the value added by medicines over the long-run.

Estimating a cost-effectiveness threshold reflecting the opportunity cost of adopting a new technology in a health system is not easy. A new OHE report provides empirical evidence on the relationship between health outcomes and health expenditures in England. Results suggest that setting a cost-effectiveness criterion for NICE may not be capable of being synthesised using scientific methods alone, but involve political judgements.

OHE’s Koonal Shah gave a presentation on societal values and the provision of end of life treatments at a Manchester Centre for Health Economics seminar.

OHE has submitted a response to the public consultation on updating the principles that guide the development of NICE guidance and standards.

OHE’s Koonal Shah and Oliver Rivero-Arias presented at a NICE Technical Forum on the topic of valuing health in children and adolescents.

When should progression-free survival be used as a surrogate for overall survival in oncology? Uneven reporting of results – different definitions and methods of analysis, lack of rigour in applying methodology – make progress difficult.

This blog post discusses the role of social value judgements in NICE technology appraisals, focusing on the recent appraisal of dinutuximab beta for high-risk neuroblastoma.

A new OHE Research Paper sets out an agenda for the development of methods to estimate cost-effectiveness thresholds in the NHS.


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