Publication in PharmacoEconomics

A joint effort between the team, the University of Sheffield and Takeda has just been published in PharmacoEconomics. The work investigates and provides practical examples of carer quality of life implementations within HTA. Current guidance appears to be lacking, whilst the approaches being used considerably impact cost-effectiveness estimates.

Approaches to Selecting “Time Zero” in External Control Arms with Multiple Potential Entry Points: A Simulation Study of 8 Approaches

As a spinoff from answering a methodological question for a client, the team have led on a paper (now in press) conducting a simulation of how to handle multiple potential start points being available for real world data comparisons. This forms part of a special issue of MDM.

Article published in Nature

Our director, Anthony Hatswell, has had a paper published in Nature on putting together a good CV for scientific jobs. Hopefully it is of interest to those currently considering their options, and a chance to share some of our experience in reviewing applications.

Propensity scoring in aHUS

Anthony, Kevin and Emma are coauthors on a paper just published piece of analysis using propensity scoring to investigate comparative effectiveness inatypical hemolytic uremic syndrome.

Simulation study regarding analysis methods for quality of life data

Anthony Hatswell & Ash Bullement are coauthors on a recently published simulation study looking at the analysis of utilities as 'time to death'. The work looks at the circumstances under which either progression, or time to death based utilities can be preferred.

Summarising salient information on historical controls

Anthony Hatswell and co-authors from a range of institutions ahve published a paper suggesting how historical controls can be summarised when used to supplement contemporary trials (for example with single arm trials). The paper also signposts the various analytical methods that can be used to then perform unbiased companions.

An Evidence Review Group Perspective of a NICE Highly Specialised Technology Appraisal: VN for RPE65-mediated IRD

In collaboration with the Peninsula Technology Assessment Group (PenTAG, University of Exeter), Delta Hat’s Ash Bullement and Anthony Hatswell are co-authors on a recent article published in Pharmacoeconomics. The article summarises the Evidence Review Group’s perspective on a recent Highly Specialised Technology (HST) appraisal conducted by NICE of voretigene neparvovec (a gene therapy for people with a very rare inherited eye disorder).

The history of evidence based medicine, relating to COVID-19

Anthony Hatswell has had an article published on some of the seminal moments in the history of evidence-based medicine, and how these can inform the current COVID-19 crisis. Specifically, how many of the issues faced may be more acute with COVID-19, but are not new.

The Effects of Model Misspecification in Unanchored Matching-Adjusted Indirect Comparison: Results of a Simulation Study

Anthony Hatswell has had an article published in Value in Health, on the effects of model misspecification. The article shows where analysts should be wary of using the technique, but also that in the majority of cases it remains both accurate and unbiased.

Evaluation of survival extrapolation in immuno-oncology using multiple pre-planned data cuts: learnings to aid in model selection

Ash Bullement and Anthony Hatswell have had an original research article published in BMC Medical Research Methodology, concerning the use of multiple data cuts from a trial to iteratively fit and compare extrapolations as the data mature. The research found that within the context of an immune-oncology treatment, more flexible parametric modelling approaches tended to yield more realistic extrapolations, and could be reasonably justified based on interim data.

NICE, in Confidence: An Assessment of Redaction to Obscure Confidential Information in Single Technology Appraisals by the National Institute for Health and Care Excellence

In collaboration with YHEC, Delta Hat has co-authored an article on the topic of redacting information in Single Technology Appraisals conducted by NICE. The study found that a definitive incremental cost-effectiveness ratio was presented within final NICE guidance in only 20 of 110 appraisals where a simple patient access scheme was considered; illustrating a lack of consensus regarding presentation of results, and the importance of appropriate redaction in justifying decision making.

A Review of Recent Decision-Analytic Models Used to Evaluate the Economic Value of Cancer Treatments

In collaboration with Holly Cranmer (Takeda) and Gemma Shields (Centre for Health Economics, University of Manchester), Ash Bullement has had a review article published in Applied Health Economics and Health Policy. The review summarises the key modelling approaches considered in oncology, alongside their advantages and limitations. The review found that partitioned-survival and Markov state-transition models were most commonly used in cancer. In addition, the review found that justification of model structures is often limited, and should be improved upon in future studies.

Cost-Effectiveness Analysis of Recombinant Factor VIII Fc-Fusion Protein (rFVIIIFc) for the Treatment of Severe Hemophilia A in Italy Incorporating Real-World Dosing and Joint Health Data

A cost-effectiveness analysis co-authored by Ash Bullement, Samuel McMordie, and Anthony Hatswell in collaboration with Swedish Orphan Biovitrum AB (publ) and Bioverativ, a Sanofi Company has been published in PharmacoEconomics – Open. This analysis builds upon a previous study by Henry et al. to incorporate data concerning joint health outcomes, as well as real-world dosing data.

Health Technology Assessment of Curative Interventions—an Old Problem With New Issues

An article co-authored by Ash Bullement and Anthony Hatswell has been published in the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) bimonthly new publication Value & Outcomes Spotlight. The article provides a summary of the key challenges faced when assessing curative interventions, alongside initiatives undertaken and illustrative case studies.

Survival Extrapolation in Cancer Immunotherapy: A Validation-Based Case Study

Ash Bullement has recently had an article published in Value in Health regarding the survival extrapolation techniques for patients treated with cancer immunotherapy. The study considers a suite of candidate survival extrapolation methods that may be suitable, and concludes that those techniques that make use of external data appear to provide more accurate longer-term survival estimates.

Novel approaches for creating historical controls from existing data

Anthony is the lead author on a paper in Statistical Methods in medical Research, which is now online.

Cost-effectiveness of avelumab for metastatic Merkel cell carcinoma

An article authored by Ash Bullement and Anthony Hatswell has been published in Pharmacoeconomics Open regarding the cost-effectiveness of avelumab for the treatment of metastatic Merkel cell carcinoma. The analysis provides a summary of the cost-effectiveness model submitted as part of NICE’s technology appraisal of avelumab, including the use of spline-based survival models and time-to-death based utilities.

Review and validation of survival extrapolation in NICE appraisals of cancer immunotherapies

An article authored by Ash Bullement has been published in the Journal of Medical Economics on the topic of survival extrapolation for patients treated with cancer immunotherapies. The article presents a review of extrapolation methods presented in company submissions to NICE, and assesses the accuracy of these extrapolations compared with later data.

Still running against the odds: revisiting the London Marathon ballot

While quite different to our usual publication work, Anthony has had an article published in Significance, the magazine of the Royal Statistical Society (RSS) on the probability of getting a London Marathon place through the public ballot. The article discusses the increasing popularity of the London Marathon, and the potential reasons for the recent surge in popularity.

Probabilistic Sensitivity Analysis in Cost‑Effectiveness Models: Determining Model Convergence in Cohort Models

A tutorial co-authored by Delta Hat’s Anthony Hatswell and Ash Bullement on the topic of undertaking probabilistic sensitivity analysis has now been published in Pharmacoeconomics.

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