scholarly journals The Impact of Rarity in NICE's Health Technology Appraisals

2021 ◽  
Author(s):  
Sophie Clarke ◽  
Michelle Ellis ◽  
Jack Brownrigg
Keyword(s):  
Access To Medicines ◽  
Decision Makers ◽  
Appraisal Committee ◽  
Technology Evaluation ◽  
Committee Meeting ◽  
Single Technology Appraisal ◽  
Equity In Access ◽  
The Uk ◽  
The Impact ◽  
Mean Time

Abstract In the absence of a framework designed to evaluate medicines for rare diseases in the UK, most orphan medicines are appraised by the National Institute for Health and Care Excellence (NICE) through the Single Technology Appraisal (STA) process. An analysis of STA appraisals of orphan and non-orphan medicines revealed that orphan medicines were subject to a significantly longer mean time in the NICE process than non-orphan medicines (370 days (n=44) vs 277 days (n=118), p=<0.0001). A higher proportion of orphan STAs required more than one Appraisal Committee Meeting (ACM) vs. non-orphan STAs, and orphan STAs were disadvantaged by worse outcomes with respect to positive recommendations than those orphan medicines assessed by Highly Specialised Technology evaluation (HST). The uncertainties inherent to developing orphan medicines may contribute to these disadvantages. Improved understanding of the challenges in drug development for orphan medicines and clearer guidance for decision makers on navigating uncertainty in the HTA process may promote greater equity in access to medicines across rare and common conditions.

scholarly journals The impact of rarity in NICE’s health technology appraisals

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Sophie Clarke ◽  
Michelle Ellis ◽  
Jack Brownrigg
Keyword(s):  
Access To Medicines ◽  
Decision Makers ◽  
Appraisal Committee ◽  
Technology Evaluation ◽  
Committee Meeting ◽  
Single Technology Appraisal ◽  
Equity In Access ◽  
The Uk ◽  
The Impact ◽  
Mean Time

Abstract Background In the absence of a framework designed to evaluate medicines for rare diseases in the UK, most orphan medicines are appraised by the National Institute for Health and Care Excellence (NICE) through the Single Technology Appraisal (STA) process. Results An analysis of STA appraisals of orphan and non-orphan medicines revealed that orphan medicines were subject to a significantly longer mean time in the NICE process than non-orphan medicines [370 days (n = 44) vs. 277 days (n = 118), p =  < 0.0001]. A higher proportion of orphan STAs required more than one Appraisal Committee Meeting (ACM) versus non-orphan STAs, and orphan STAs were disadvantaged by worse outcomes with respect to positive recommendations than those orphan medicines assessed by Highly Specialised Technology evaluation (HST). Conclusions The uncertainties inherent to developing orphan medicines may contribute to these disadvantages. Improved understanding of the challenges in drug development for orphan medicines and clearer guidance for decision makers on navigating uncertainty in the HTA process may promote greater equity in access to medicines across rare and common conditions.

Impact of UK NICE clinical guidelines 168 on referrals to a specialist academic leg ulcer service

2017 ◽  
Vol 33 (2) ◽  
pp. 84-88 ◽  
Author(s):  
Huw OB Davies ◽  
Matthew Popplewell ◽  
Gareth Bate ◽  
Lisa Kelly ◽  
Katy Darvall ◽  
...  
Keyword(s):  
Fold Increase ◽  
Leg Ulcer ◽  
Leg Ulcers ◽  
Below The Knee ◽  
Health And Social Care ◽  
The Uk ◽  
The Impact ◽  
Change In Mean ◽  
Mean Time ◽  
Significant Health

Background Leg ulcers are a common cause of morbidity and disability and result in significant health and social care expenditure. The UK National Institute for Health and Care Excellence (NICE) Clinical Guideline (CG)168, published in July 2013, sought to improve care of patients with leg ulcers, recommending that patients with a break in the skin below the knee that had not healed within two weeks be referred to a specialist vascular service for diagnosis and management. Aim Determine the impact of CG168 on referrals to a leg ulcer service. Methods Patients referred with leg ulceration during an 18-month period prior to CG168 (January 2012–June 2013) and an 18-month period commencing six months after (January 2014–June 2015) publication of CG168 were compared. Results There was a two-fold increase in referrals (181 patients, 220 legs vs. 385 patients, 453 legs) but no change in mean age, gender or median-duration of ulcer at referral (16.6 vs. 16.2 weeks). Mean-time from referral to specialist appointment increased (4.8 vs. 6 weeks, p = 0.0001), as did legs with superficial venous insufficiency (SVI) (36% vs. 44%, p = 0.05). There was a trend towards more SVI endovenous interventions (32% vs. 39%, p = 0.271) with an increase in endothermal (2 vs. 32 legs, p = 0.001) but no change in sclerotherapy (24 vs. 51 legs) treatments. In both groups, 62% legs had compression. There was a reduction in legs treated conservatively with simple dressings (26% vs. 15%, p = 0.0006). Conclusions Since CG168, there has been a considerable increase in leg ulcer referrals. However, patients are still not referred until ulceration has been present for many months. Although many ulcers are multi-factorial and the mainstay of treatment remains compression, there has been an increase in SVI endovenous intervention. Further efforts are required to persuade community practitioners to refer patients earlier, to educate patients and encourage further investment in chronically underfunded leg ulcer services.

Pathways to faster access to medicines

2018 ◽  
Vol 56 (8) ◽  
pp. 93-96 ◽  
Keyword(s):  
Treatment Options ◽  
Clinical Trial Data ◽  
Access To Medicines ◽  
Trial Data ◽  
European Medicines Agency ◽  
The European Union ◽  
Life Threatening ◽  
The Uk ◽  
New Treatments ◽  
The Impact

Before a medicine can be marketed in the UK, marketing authorisation approval is needed from the European Medicines Agency (EMA) or the Medicines and Healthcare products Regulatory Agency (MHRA). However, the time it takes to appraise a medicine is considered by some to delay access to new treatments for people with serious or life-threatening conditions who have no other treatment options. Also, the standard regulatory process may be less suitable for medicines for rare conditions in which it is difficult to gather a large amount of clinical trial data. Here we look at a range of new regulatory and access pathways that have been developed to respond to these challenges and consider some of their potential pitfalls. In a future article we will review the impact that the UK’s departure from the European Union (EU) will have on licensing processes.

scholarly journals Climate change and water in the UK – past changes and future prospects

2015 ◽  
Vol 39 (1) ◽  
pp. 6-28 ◽  
Author(s):  
Glenn Watts ◽  
Richard W. Battarbee ◽  
John P. Bloomfield ◽  
Jill Crossman ◽  
Andre Daccache ◽  
...  
Keyword(s):  
Climate Change ◽  
Water Quality ◽  
Algal Blooms ◽  
Decision Makers ◽  
Climate Forcing ◽  
Important Work ◽  
Lake Water Quality ◽  
Winter Rainfall ◽  
The Uk ◽  
The Impact

Climate change is expected to modify rainfall, temperature and catchment hydrological responses across the world, and adapting to these water-related changes is a pressing challenge. This paper reviews the impact of anthropogenic climate change on water in the UK and looks at projections of future change. The natural variability of the UK climate makes change hard to detect; only historical increases in air temperature can be attributed to anthropogenic climate forcing, but over the last 50 years more winter rainfall has been falling in intense events. Future changes in rainfall and evapotranspiration could lead to changed flow regimes and impacts on water quality, aquatic ecosystems and water availability. Summer flows may decrease on average, but floods may become larger and more frequent. River and lake water quality may decline as a result of higher water temperatures, lower river flows and increased algal blooms in summer, and because of higher flows in the winter. In communicating this important work, researchers should pay particular attention to explaining confidence and uncertainty clearly. Much of the relevant research is either global or highly localized: decision-makers would benefit from more studies that address water and climate change at a spatial and temporal scale appropriate for the decisions they make.

OP303 Do You Get The Message? Making HTA Findings Easier For Decision-Makers To Implement

2021 ◽  
Vol 37 (S1) ◽  
pp. 11-11
Author(s):  
Jess Kandulu ◽  
Ed Clifton ◽  
Iain Robertson ◽  
Neil Smart ◽  
Karen Macpherson
Keyword(s):  
Decision Making ◽  
Technology Assessment ◽  
Health Technology ◽  
Evidence Base ◽  
Decision Makers ◽  
Appraisal Committee ◽  
Economic Modelling ◽  
Health Technologies ◽  
Patient Groups ◽  
The Impact

IntroductionOften health technology assessment (HTA) products developed by the Scottish Health Technologies Group (SHTG) did not reach clear directive conclusions because the evidence base for a technology was weak. Despite being methodologically robust, these products did not meet the needs of decision-makers and may have had negligible impact.MethodsSHTG set out to equip and empower the recommendation-making council (that is, appraisal committee) to reach clear conclusions. SHTG broadened the HTA components and types of evidence that could be considered. The increased breadth of evidence included: clinicians attending council meetings to respond to questions; patient groups making submissions and presenting at council meetings; Scotland-specific economic modelling; and consultation on draft recommendations. SHTG also restructured the council for improved deliberative decision-making.ResultsClear directive conclusions were reached in a substantially higher proportion of HTA products (eighty-eight percent in 2019 compared with eight percent in 2017). It became possible for decision-makers to implement findings. It also became feasible to assess the impact and implementation of recommendations.ConclusionsBroadening SHTG's consideration of HTA components has led to a clearer conclusion being reached and stronger messaging for decision makers. This positions SHTG to increase its influence in the use of health technologies in Scotland.

The Film World’s Cooperative Store: Institutions and Films of the 1930s and 1940s

2018 ◽  
Author(s):  
C. Claire Thomson
Keyword(s):  
World War Ii ◽  
Meat Products ◽  
Public Information ◽  
Early History ◽  
World War ◽  
International Context ◽  
German Occupation ◽  
History Of ◽  
The Uk ◽  
The Impact

This chapter traces the early history of state-sponsored informational filmmaking in Denmark, emphasising its organisation as a ‘cooperative’ of organisations and government agencies. After an account of the establishment and early development of the agency Dansk Kulturfilm in the 1930s, the chapter considers two of its earliest productions, both process films documenting the manufacture of bricks and meat products. The broader context of documentary in Denmark is fleshed out with an account of the production and reception of Poul Henningsen’s seminal film Danmark (1935), and the international context is accounted for with an overview of the development of state-supported filmmaking in the UK, Italy and Germany. Developments in the funding and output of Dansk Kulturfilm up to World War II are outlined, followed by an account of the impact of the German Occupation of Denmark on domestic informational film. The establishment of the Danish Government Film Committee or Ministeriernes Filmudvalg kick-started aprofessionalisation of state-sponsored filmmaking, and two wartime public information films are briefly analysed as examples of its early output. The chapter concludes with an account of the relations between the Danish Resistance and an emerging generation of documentarists.

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