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In a previous post, we discussed the origins of the NICE Highly Specialised Technologies (HST) process for the evaluation of therapies for rare diseases. In this blog, we examine the key differences between the Single Technology Appraisal (STA) and the HST processes. There are numerous differences between the two processes; from timescale to individual r...

October 9, 2019

Systematic Reviews – The Problem 


Although considered the gold standard of quality research, systematic literature reviews (SLRs) are very resource intensive (e.g. long timeline, high budget) [1]. This, in combination with the broader issue that “research outpaces understanding,” SLRs get quickly out-of-date [2]. There is a challenge to create reliable ev...

Origins of the Highly Specialised Technologies (HST) process

Treatments for very rare conditions represent a unique challenge to payers. Extremely low patient numbers mean that often only Phase 1/2 trial data are available, and that natural history, quality of life and resource use data are limited. Combined with high unit costs, these evidence challenges...

On the 1st April 2017, the National Institute for Health and Care Excellence (NICE) introduced a fast-track appraisal (FTA) process, with the aim of providing quicker access for patients to the most cost-effective new treatments. Two years on, has this objective been achieved?

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April 10, 2019

Receiving scientific advice from Health Technology Assessment (HTA) bodies is becoming an increasingly important part of the clinical development process. Scientific advice allows for evidence generation more targeted towards the needs of payers, smoothing the reimbursement process and ensuring that new therapies get to market, and ultimately to patients,...

March 21, 2019

Project HERCULES is a unique international multi-stakeholder collaborative project set up by Duchenne UK to develop tools and evidence to support Health Technology Assessments and reimbursement decisions for new treatments for Duchenne Muscular Dystrophy (DMD). It brings together 8 leading pharmaceutical companies, academics, patient organisations and adv...

March 19, 2019

The rate at which future costs and outcomes are discounted has long stood at 3.5% for UK HTA agencies (1,2). The source of this discount rate is the Social Time Preference Rate (STPR), presented in previous versions of the Treasury’s Green Book, which provides central government guidance on appraisal evaluation (3). The Green Book was updated in March 201...

February 27, 2019

The Boston-based Institute for Clinical and Economic Review, otherwise known as ICER, has caught the attention of important stakeholders in the past few years. Between reports for high-impact drugs that have stirred public opinion (such as a review of Orkambi for cystic fribrosis) and its increasing popularity with healthcare payers, ICER is becoming a ke...

February 5, 2019

As described in the Cochrane Collaboration Handbook, a systematic literature review (SLR) attempts to collate all empirical evidence that fits pre-specified eligibility criteria in order to answer a specific research question (primarily to aid in decision-making). It uses explicit, systematic methods that are selected with a view to minimizing bias, thus...

January 28, 2019

The search strategy is the foundation of a well-conducted systematic literature review (SLR) and is the fundamental element that can affect the overall quality of the results of the final review (1, 2). The aim of a search string is to achieve a balance between sensitivity (capturing all relevant articles) and precision (eliminating irrelevant hits); a la...

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