NICE Approval Research Articles

NICE's approval of hepatitis drugs may create cost dilemma

NICE's approval of hepatitis drugs may create cost dilemma

48 Who and When to Refer for Advanced Heart Failure? A Survey of Clinicians Referring to the Freeman Hospital

The Freeman Hospital in Newcastle is one of the leading centres for cardiac transplantation and implantation of ventricular assist devices in the UK, receiving referrals to the Advanced Heart Failure Service predominantly from the North of England, but also Northern Ireland and complex cases from other regions. We were keen to establish whether referring clinicians felt they had sufficient knowledge about the service, its role and which patients may benefit from referral. We designed an online survey and sent a link via email to 127 potential referrers (predominantly colleagues who had referred to the service in the past). 56 of the 127 clinicians responded, giving a response rate of 44% (Table 1). Although 76% of those responding to the survey had previously referred at least one patient, only 70% reported being fully aware of the role of the Advanced Heart Failure Service, 62% felt they had a clear understanding of which patients to refer and 60% were confident in how to refer. Only 21% felt they had sufficient information about the service and guidelines for referral. The uncertainty amongst clinicians about who to refer influences whether patients are referred for advanced heart failure therapies, and therefore impacts on patient care as patients who may benefit from therapies such as transplantation or ventricular assist devices may not be referred for assessment. Factors prompting clinicians to refer patients with heart failure and relatively few co-morbidites, or which deter them from referring, are detailed in Figures 1 and 2. Only 51% would consider referring a patient with more than 1 heart failure hospitalisation in the last year and fewer than 50% would consider referring patients with multiple ICD shocks, new renal impairment, or hypotension requiring reduction or discontinuation of ACE inhibitors/beta blockers, despite all of these being recognised adverse prognostic signs in patients with heart failure. The most common factor deterring clinicians from referring was the presence of multiple co-morbidities (74%). Disappointingly, other reasons for not referring included being unsure of who to refer (14%) or how to refer (10%). 84% of clinicians would be more likely to refer in the future if NICE approved VADs as ‘destination therapy’ rather than only as a ‘bridge to transplant’. Other advances in heart failure management that may increase the likelihood of clinicians referring in the future are summarised in Table 2. There is a clear demand for increased access to information about the Freeman Hospital Advanced Heart Failure Service and guidelines on who to refer, with 84% being “extremely interested” and the remaining 16% “quite interested” in receiving more information. With better education amongst referring clinicians, and NICE approval of destination therapy, referrals will likely increase significantly.

NICE approval of dimethyl fumarate could benefit thousands living with relapsing-remitting multiple sclerosis.

NICE approval of dimethyl fumarate could benefit thousands living with relapsing-remitting multiple sclerosis.

M263 A Quarter Of Ipf Patients Not Eligible For Pirfenidone Treatment Due To The Nice Criteria Significantly Decline Over Time

Introduction Pirfenidone has NICE approval and is recommended for patients with IPF if the FVC is 50–80%. We hypothesised that this would disadvantage a significant cohort of IPF patients who have moderate reduction in transfer factor despite preserved FVC. Methods We present longitudinal data capturing 38 IPF patients who had FVC greater than 80% and not eligible for pirfenidone treatment. Results Since NICE approval in July 2013, 43 patients were eligible for pirfenidone as per the NICE criteria and 38 (47%) patients were outside the NICE criteria. Of those outside the NICE criteria, the average FVC was 98% (81–145) and average DLCO was 58% (21–88). Sixteen (42%) patients had a DLCO This retrospective data demonstrates that the sole use of FVC in the NICE criteria for treating IPF disadvantages patients who demonstrate a significant reduction in transfer factor despite FVC greater than 80%. In this study this reduced transfer factor and preserved FVC can only be attributed to the presence of coexisting emphysema in 9/38 (24%) of patients. Ten (26%) IPF patients not treated with pirfenidone because they did not meet the NICE criteria demonstrate a clinically significant decline in their lung function. Despite this the majority are still not eligible for treatment with pirfenidone. We would therefore advocate following our European partner countries and using both FVC and DLCO as per the CAPACITY criteria when assessing patient suitability for pirfenidone treatment for IPF, as the use of FVC alone with an upper limit of 80% excludes a substantial cohort of IPF patients who have preserved FVC, moderately reduced DLCO with or without the presence of coexisting emphysema and over time a quarter of these patients demonstrate lung function decline. References CAPACITY Study Group. Pirfenidone in patients with idiopathic pulmonary fibrosis (CAPACITY): two randomised trials. Lancet 2011;377:1760–69 http://www.nice.org.uk/nicemedia/live/14156/63713/63713.pdf

M264 Health And Economic Impact Of Prescribing Pirfenidone

<h3>Introduction</h3> Pirfenidone is the only licensed drug in Europe for Idiopathic Pulmonary Fibrosis (IPF). Clinical trials (1) have demonstrated efficacy in reducing decline in forced vital capacity (FVC), improving progression free survival and reducing mortality. The translation of clinical trial results to clinical practice is a focus of interest. <h3>Methods</h3> We describe our experiences in prescribing pirfenidone in a single centre observational study of 96 patients from September 2011 to April 2014. <h3>Results</h3> This is an extension of our published work (2). Prior to NICE approval we recruited 49 patients in twenty months. NICE approval resulted in a 140% increase in pirfenidone prescribing. Patient demographics at baseline are shown in Table 1. 54 (56%) patients continued treatment, 19 (20%) discontinued treatments due to adverse effects (AEs), there were 17 (17%) deaths and 4 (4%) patients were transplanted. Patients that died had lower diffusing capacity (DLCO) at baseline compared to those that continued treatment (32.9 vs 47.7 p &lt; 0.0001). Patients that discontinued treatment due to AEs did so within six months and had lower body mass index (25.1 vs 29 p = 0.002) and DLCO (38.8 vs 47.7 p = 0.007). There were a total of 206 AEs in 77 (79%) patients. The majority were gastrointestinal in nature. Of these adverse effects the majority were self-limiting and resolved with simple measures. 44 (21%) resulted in a dose reduction, 23 (11%) resulted in a temporary discontinuation, in 101 (45%) AEs treatment was unchanged and 38 (19%) AEs resulted in drug discontinuation. In selected patients we had one or more lung function results before (34%) and after (50%) pirfenidone treatment. Eighteen months prior to pirfenidone treatment there was an observed reduction in mean% predicted FVC over time. Accepting limitations of missing data, this decline appeared to stabilise over twelve months after commencement of pirfenidone. Based on an annual unit cost of £22, 245.96 for pirfenidone (without undisclosed discount). To date 96 patients have been treated for a total of 876 months at a total cost of £1,623,955 in two and a half years. <h3>Conclusion</h3> This study highlights both the health and economic impacts of pirfenidone over a two and a half year period of prescribing. <h3>References</h3> King TE Jr <i>et al</i>. A phase 3 trial of pirfenidone in patients with idiopathic pulmonary fibrosis. ASCEND Study Group. <i>N Engl J Med.</i> 2014 May 29;370(22):2083-92 Chaudhuri N <i>et al</i>. Real world experiences: pirfenidone is well tolerated in patients with idiopathic pulmonary fibrosis. <i>Respir Med.</i> 2014 Jan;108(1):224-6

What The English Could Learn From The Irish: Making The Nice Approval Process More Cost-Effective.

What The English Could Learn From The Irish: Making The Nice Approval Process More Cost-Effective.

FINGOLIMOD IN NEWCASTLE: THE FIRST 100 PATIENTS' EXPERIENCE

BackgroundFingolimod is prescribed in the UK following NICE approval in 2012. In Newcastle, our regional service, high prevalence and subspecialty structures have allowed us to accumulate a large cohort starting...

AB0441 Abatacept in Rheumatoid Arthritis (RA) Patients: Real-Life Experience in A Subregional Centre in Uk

BackgroundAbatacept is a biologic therapy which suppresses T-cell activation via co-stimulation blockade in rheumatoid arthritis (RA) patients. It became part of our biologic algorithm shortly after initial limited NICE approval...

Does the cancer drugs fund lead to faster uptake of cost-effective drugs? A time-trend analysis comparing England and Wales

Background:The Cancer Drugs Fund (CDF) provides £200 million annually in England for ‘anti-cancer' drugs.Methods:We used a controlled pre-/post-intervention design to compare IMS Health dispensing data for 15 cancer drugs (2007–2012) in England vs Wales, stratified by pre-CDF NICE drug approval status (rejected, mixed recommendations, recommended, not appraised).Results:The CDF was associated with increased prescribing in England for three of five drugs rejected or with mixed NICE recommendations. The prescribing volume ratios (PVR) ranged from 1.29 (95% CI 1.00, 1.67) for sorafenib to 3.28 (2.59, 4.14) for bevacizumab (NICE rejected) and 0.93 (0.81, 1.06) and 1.35 (1.21, 1.49) for sunitinib and imatinib respectively (mixed recommendations). Post CDF prescribing in England increased for both drugs awaiting NICE appraisal pre-CDF (lapatinib PVR=7.44 (5.81, 9.54), panitumumab PVR=5.40 (1.20, 24.42)) and subsequently rejected. The CDF was not associated with increased prescribing in England of NICE-recommended drugs. The three most recently launched, subsequently recommended drugs were adopted faster in Wales (from pazopanib PVR=0.51 (0.28, 0.96) to abiraterone PVR=0.78 (0.61–0.99)).Interpretation:These data indicate that the CDF is used to access drugs deemed not cost-effective by NICE. The CDF did not expedite access to new cost-effective cancer agents prior to NICE approval.

S13 Sole use of forced vital capacity as per national institute of health and care excellence criteria disadvantage 2 in 5 people with idiopathic pulmonary fibrosis

IntroductionPirfenidone has undergone NICE approval and is recommended for patients with IPF if the FVC is 50–80% 1. We hypothesised that the NICE guidance would disadvantage a significant cohort of...

The changing face of oral anticoagulants

Warfarin has been the established oral anticoagulant for the last 50 years, being effective in the prevention and treatment of venous and arterial thromboembolic disorders. However, the frequent requirement for INR monitoring, multiple drug and food interactions have fuelled the need for development of new oral anticoagulants. Dabigatran is the first of a series of new oral anticoagulants that are emerging as the successors to warfarin. This new group of anticoagulants is rapidly gaining FDA and NICE approval and has proven non-inferiority to warfarin and viable alternatives to warfarin in the coming years. Given the obvious impact of this on dental treatment in the primary care and hospital setting this article aims to increase familiarisation with this new medicine group.

Corneal cross-linking service survey in England

Sir, Corneal cross-linking (CXL) describes a procedure whereby topical riboflavin is combined with ultraviolet (UV) light exposure to stabilize corneal ectasias such as keratoconus. Treatment for keratoconus up to now has been supportive only, and as such clinical interest in the procedure is high. Supportive evidence exists in large case series,1, 2, 3 small RTCs3 and large RCTs are underway4, 5 but CXL does not yet have full NICE approval. The aim of our survey was to get an overview of current provision of CXL in England. The Royal College of Ophthalmologists provided a list of corneal consultants, which was cross-checked by telephoning all hospitals in the ophthalmic directory. An online survey was created and sent electronically to all (Figure 1). Figure 1. Eight question online survey sent to all corneal consultants in England. Out of the 115 Ophthalmology units in England, 98 offer a corneal service. The survey response rate was 42% (41 out of 98 units). Of those that responded, 32% currently offer CXL: 10% under the NHS, 12% privately, and 10% both privately and under the NHS. The reported average number of patients being treated is eight per quarter. The reported average length of time CXL has been offered for is 2 years, with NHS CXL being offered on average for a year longer than privately. Regarding units not currently offering CXL, the majority (64%) plan to initiate services in the near future. Rapid CXL, in which UV power is increased and exposure time reduced, is used by 54% whereas 46% used standard CXL (all consultants offering CXL only under the NHS only use standard CXL). Lastly, 77% infuse riboflavin after removing the epithelium, 8% use transepithelial riboflavin infusion and 15% use both methods. In summary, CXL is becoming more commonly available with the majority of departments planning to initiate services. Rapid CXL is becoming the favoured choice; however, the majority still remove the corneal epithelium. The NHS Commissioning Board has added CXL service to its consultation for specialized commissioning. Hence, CXL will probably be limited to specialist centres. Online surveys are easily applied and useful in gauging a snapshot of opinion, but must be used with caution because of difficulty in responses subgroup analysis.

P11 Reduction in Healthcare Resource Utilisation with 2 Years Treatment with Omalizumab in Severe Atopic Asthmatics: A Single Centre Observation

BackgroundWhile asthma is a chronic inflammatory disorder that is managed with inhaled controller and reliever drugs, there remains a large unmet need at the severe end of the disease spectrum.Omalizumab...

U.K. experience of sunitinib malate in the treatment of metastatic renal cell carcinoma: Predictors of response from clinical practice.

455 Background: Sunitinib gained NICE approval in March 2009 as first line treatment for metastatic renal cell carcinoma (mRCC) and UK experience of use of Sunitinib is growing. The North of England Cancer Network (NECN) was the first cancer network in the UK to approve the use of Sunitinib in July 2007 and has the longest UK experience with this drug. The clinical outcomes of the NECN patients are presented. Methods: Data was collected and analysed regarding all patients started on Sunitinib from 01/07/2007 to 30/09/2010. Follow up extended to 28/02/2011. Previously published predictors of response were compared to the NECN data to check applicability in this population. Results: 129 patients were identified with a median age of 63 years (range 21-87), 67% were male and WHO performance status was 0, 1 or 2 (33%, 51%, 16%). 80% of patients had nephrectomy (60% radical) and 22% had prior systemic treatment. Median PFS was 10 months (range 0.25-27). Median OS (from Sunitinib initiation) was 17 months (range 0.25 - 47). Performance status was significantly associated with PFS and OS. The median number of treatment cycles was 5 (range 0.25-27). 64% of patients were dose reduced and 9% had a schedule modification to minimise toxicity. 21% of patients had a delay of 4 weeks or more which was not detrimental to median PFS. Dose reduction at treatment initiation was associated with a significant decrease in OS. 11.6% of patients required treatment for hypothyroidism which was associated with a significant improvement in PFS and OS. 15.5% of patients required treatment for hypertension, which was associated with an improvement in OS, but not PFS. Conclusions: PFS in the NECN population is similar to trial data. Strategies to deal with side effects, including dose reduction (after treatment initiation), treatment delay and schedule changes, did not adversely affect PFS. Performance status 0, the development of new hypertension or new hypothyroidism can be regarded as markers of good prognosis in this population. Initial dose reduction should be avoided as this adversely affects prognosis.

First analysis of pharma uptake after NICE approval

First analysis of pharma uptake after NICE approval

New NICE guideline on acute stroke and TIA: need for major changes in delivery of stroke treatment

The recently published NICE clinical guideline on diagnosis and initial management of stroke and transient ischaemic attack (TIA) is a landmark publication for all clinicians involved in stroke care in...

Deconstructing media coverage of trastuzumab (Herceptin): an analysis of national newspaper coverage.

To explore and critically describe the content and main narratives of UK national daily newspaper coverage of trastuzumab (Herceptin). We used the NewsBank database to search eight national daily newspapers, and their Sunday equivalents, retrospectively from 19 February 2006 back to the earliest mention of trastuzumab or Herceptin (19 May 1998). Setting UK national newspapers. To be eligible for inclusion, articles had to contain at least three sentences about trastuzumab. Articles that focused on the financial performance of companies associated with the drug were excluded from the analysis. For each included article, we extracted bibliographic details and data, and independently rated the reporting slant towards trastuzumab and, where relevant, the reporting slant towards access to treatment. We identified 361 articles that met the study inclusion criteria. The proprietary name of Herceptin was always used, with only eight articles mentioning the generic alternative. 294/361 included articles (81.5%) were rated as being positive towards trastuzumab, the remainder rated as neutral. Access to trastuzumab treatment was the main narrative running across included articles and reports of individual patients seeking treatment featured prominently throughout. In 208/361 of included articles (57%) the reporting slant towards access to trastuzumab treatment was rated as negative. 178/361 of included articles (49.3%) mentioned licensing, but rarely mentioned that licensing processes can only occur when the manufacturer applies for a licence. Only a minority of articles mentioned that the drug had to be licensed before it could be subject to the NICE approval process. Newspaper coverage of trastuzumab has been characterized by uncritical reporting. Journalists (and consumers) should be more questioning when confronted with information about new drugs and of the motives of those who seek to set the news agenda.

Arthritis patients get surprise relief

Arthritis sufferers have been given new hope for relief with the unexpected NICE approval of two new drugs to treat the condition on the NHS. The Stormont health committee is now being lobbied to recommend the drug for use by patients in Northern Ireland

The rocky road to the NICE approval of taxanes

The rocky road to the NICE approval of taxanes