Scottish Medicines Consortium Research Articles

PRO86 Approaches to the Collection of Utility Values for RARE Diseases: A CROSS-Country Comparison

To further characterise utility data collection methods in (ultra-)orphan indications by identifying published utility studies from the EU5 and Canada, and comparing methodologies reported in submissions to the National Institute for Health and Care Excellence (NICE) with the Scottish Medicines Consortium (SMC) and Canadian Agency for Drugs and Technologies in Health (CADTH). A previously conducted review was expanded to identify utility studies from EU5 countries/Canada published between January 2014–June 2020 and additional highly specialised technology (HST) appraisals published on the NICE website since the original search in May 2019.1 SMC and CADTH websites were then searched for equivalent published appraisals corresponding to all available HST submissions. In total, 126/611 articles from the expanded review were included. 31.0% (39/126) and 16.7% (21/126) collected utilities using observational or interventional studies, respectively. Additional study designs included surveys (13.5% [17/126]), systematic literature reviews (12.7% [16/126]), and/or vignette studies (4.8% [6/126]). Nine SMC and 11 CADTH appraisals corresponding to 17 completed/ongoing HST appraisals were identified, with 7 appraisals being identified on all three websites. In line with previous findings, submissions often reported utility data from interventional studies (15/17 [88.2%] HST, 5/9 [55.6%] SMC and 8/11 [72.7%] CADTH); however, the proportion of appraisals using these data for economic evaluations varied (3/15 [20.0%] HST, 4/5 [80.0%] SMC and 4/8 [50.0%] CADTH). Amongst common alternative methods used to supplement utility data for economic evaluations were vignette studies (4/17 [23.5%] HST, 2/9 [22.2%] SMC and 2/11[18.2%] CADTH). Appraisals for very rare diseases across Canada, England and Scotland rely to different degrees on alternative methods for collection of utilities, corresponding with the variety of published utility study types. There remains a need for further research on the appropriate application of alternative utility collection methods in rare diseases.

PNS145 Access to Medicines in the United Kingdom: A Review of HTA Decisions for New Medicines

To examine Health Technology Assessment (HTA) decisions for new innovative medicines in the UK (England, Scotland, Wales, Northern Ireland). The study examined HTA decisions made between 2017 and 2020 across UK countries for innovative medicines licensed by European Medicines Agency (EMA) in 2017. The analysis was done using published documents. The decisions were classified as “recommend without restrictions,” “recommend with restrictions,” or “not recommended”. EMA approved licenses for 56 new innovative medicines in 2017. From the date of licensing the medicines in 2017 until January 2020, the National Institute for Health and Care Excellence (NICE) in England had completed the appraisal of 28 out of the 56 medicines. Out of these 28, 12 were “recommended without restrictions” 14 were “recommended with restrictions” and 2 were “not recommended”. Majority of the NICE recommendations (i.e. 21 of 26 or 81%) were done through managed access agreements. The Scottish Medicines Consortium (SMC) has assessed 31 out of the 56 medicines licensed in 2017. Out of these 31, 8 were, “recommended without any restriction”, 22 were “recommended with restriction” and 1 was “not recommended”. AWMSG in Wales follows NICE guidance and does not review a medicine if NICE is considering assessing it in the next 12 months. Northern Ireland follows HTA guidance issued by NICE or SMC and adopts a decision shortly after either has made its decision. In Northern Ireland, 37 recommendations were adopted from either NICE or SMC. These 37 include: 10 without restrictions, 21 with restrictions and 6 not recommended decisions. Adoption of HTA recommendations is low and it varies across UK countries. The number of HTA assessments resulting in “not recommended” decision was found consistently low across all UK countries. However, the number of “recommendation with restriction” is relatively high compared to the “recommendation without restrictions”.

Ranolazine in refractory and chronic stable angina

Aim/Objectives: The current Scottish Intercollegiate Guidelines Network (SIGN) guidelines recommend beta blockers and dihydropiridine calcium channel blockers as first-line agents for refractory angina pectoris. Despite being optimally treated with pharmacotherapy and revascularisation, up to 40% of patients still experience symptoms. Ranolazine, a piperazine derivative, selectively inhibits late sodium currents and is of particular interest as it is currently not recommended routinely by SIGN guidelines and Scottish Medicine Consortium (SMC) but has been prescribed in Tayside, initially through IPTR since 2017 and recently through a Local New Medicine Treatment Protocol and Stable Angina Pathway. Real world experience of ranolazine prescribing in patients with chronic and often refractory angina is not widely reported. We therefore audited its use in Tayside to understand its prescribing pattern within our patient population and assess its effects on angina symptom relief. Methods: Electronic health records and prescribing data between 1st January 2012 and 31st December 2018 were retrospectively analysed. Data on baseline characteristics, prescribing information, past medical history, and angina symptom control were collected. Standard descriptive statistics were used for analysis. Results: 35 patients were identified as suitable for inclusion in the audit. Mean age was 71.4 ± 12.5 years old and 68.6% were male. 23 patients (65.7%) had either a previous percutaneous coronary intervention (PCI) or coronary artery bypass graft surgery (CABG). The most common reason for ranolazine prescription was refractory angina (74.3%) with 375 mg BD being the most common dose. Prescription of guideline-recommended anti anginals was high with 80% of patients being on a beta blocker and a nitrate prior to commencing ranolazine. Encouragingly, 27 patients (77.1%) reported an improvement in Canadian Cardiovascular Society (CCS) angina class and the rate of non-responders was 22.9%. No adverse effects leading to discontinuation of ranolazine was found. Conclusion: Ranolazine may play a role as an additional anti anginal agent with reasonable achievement of symptom control in patients who have refractory angina despite the use of other guideline-recommended anti anginal agents.

Economic Evaluation of Senshio® (Ospemifene) for the Treatment of Vulvovaginal Atrophy in Scotland.

Local oestrogens, the current treatment for vulvar and vaginal atrophy (VVA), are not suitable for all women. Standard of care (SoC) consists of over-the-counter lubricants and moisturisers. Senshio® (ospemifene) provides a treatment option for postmenopausal women who are not candidates for local vaginal oestrogen therapy who would otherwise have an unmet clinical need. The aim of this study was to estimate the cost-effectiveness of ospemifene, a selective oestrogen receptor modulator, for the treatment of moderate to severe symptomatic VVA in postmenopausal women who are not candidates for local vaginal oestrogen therapy. The Scottish Medicines Consortium (SMC) recently evaluated the clinical and cost-effectiveness evidence of ospemifene plus SoC compared with SoC alone. A cost-effectiveness study, from a National Health Service (NHS) Scotland perspective over a lifetime time horizon, was submitted to the SMC. The cohort-based Markov model used robust clinical evidence from two large pivotal phase III randomised controlled studies and included four health states classified by dyspareunia symptom severity: none, mild, moderate and severe. The movement of women between health states was dependent on the effectiveness of treatment in reducing dyspareunia. Extensive sensitivity analyses were undertaken to assess the level of confidence associated with the base-case results. Treatment with ospemifene was associated with an additional cost of £847 per patient and an increase in quality-adjusted life-years (QALY) of 0.06 per patient. Ospemifene had an incremental cost-effectiveness ratio of £14,138 per QALY. In the probabilistic sensitivity analysis, there was a probability of 89% that ospemifene was cost-effective at a threshold of £20,000 per QALY gained. Ospemifene remained cost-effective under all scenario analyses. The SMC reviewed the clinical and economic evidence and judged that the evidence demonstrated a robust case to support prescribing ospemifene in NHS Scotland. Ospemifene is a cost-effective intervention that has recently been accepted by the SMC for the treatment of postmenopausal women with moderate to severe VVA who are not candidates for local oestrogen.

Exploration of the visibility of patient input in final recommendation documentation for three health technology assessment bodies.

Health technology assessment (HTA) recommendations informed by patient concerns are seen to ensure democracy and legitimacy. We explored how written and oral patient involvement in two HTAs was reported on in publicly available final recommendations and discussion summaries of appraisal committees from three HTA bodies. We aimed to gain insights into how patient input was utilized by appraisal committees to better understand the goals of patient involvement and how these are being achieved. In each of the three HTA bodies, templated submission questionnaires provide a formal process for seeking written patient group input. Additionally, the National Institute for Health and Care Excellence (NICE) selects patient experts to provide a templated submission and attend appraisal committee meetings. For Scottish Medicines Consortium (SMC), a patient advocate and clinician combined meeting (PACE) discussed the cancer drug, referred to in the final recommendation. The discussion summaries of all appraisal committees contained references to patient involvement. Where two mechanisms for patient involvement were provided, oral input from the expert patients and PACE were more clearly documented than information from written patient group submissions. NICE reports focused on the perspective of the patient expert. The SMC report highlighted feedback from the PACE throughout. We suggest that the lack of clear reporting on the use of patient group input in deliberations and therefore accountability to patient groups limits progress in patient involvement in HTA. Patient groups may therefore not have a clear understanding of what information they can best provide to inform deliberations, and in reporting back to members.

PBI33 HOW NOVEL IS ICER METHODS GUIDANCE ON HIGH-IMPACT, SINGLE, AND SHORT-TERM THERAPIES?

Innovative cell and gene therapies are often single-administration treatments with high-upfront costs but the potential for long-term health gains and possibly cure. Given the uncertainty in outcomes at launch and the unrecoverable high costs, the US Institute for Clinical and Economic Review (ICER) recently developed novel methods for assessing these treatments. We reviewed these methods and recent health technology assessments (HTAs) by the UK National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) of tisagenlecleucel and voretigene neparvovec, to examine whether ICER’s new methods are already considered in HTAs in the UK. The adapted ICER methods require cure-fraction modeling, discounting of outcomes and costs at the standard 3%, and a threshold analysis of the duration of the beneficial effect. A cost-effectiveness threshold of $150,000 per quality-adjusted life-year (QALY) gained is used to guide evaluation of long-term value for money. ICER will also consider benefits beyond QALYs, including the value of treatment choice and potential benefit or disadvantage of option value. In the HTAs of tisagenlecleucel and voretigene neparvovec, NICE considered cure-fraction models and reduction of the discount rate to 1.5%, and acknowledged value beyond health benefits, such as patients retaining independence and research advancing the broader field of gene therapy. SMC accepted additional value in the single administration, and reduced patient and carer burden. Both NICE and SMC applied higher cost-effectiveness thresholds for ultra-rare diseases. While higher cost-effectiveness thresholds and cure-fraction models are already considered in HTAs for costly, single, or short-term therapies in the UK, ICER’s consideration of the value of treatment choice and option value is a novel approach. ICER plans to implement the adapted methods in January 2020. A review of future ICER assessments will indicate whether the new methodology provides a better way to demonstrate the value of potentially curative treatments.

P191 Real life experience of tocilizumab for the management of giant cell arteritis in Fife, Scotland

Abstract Background Tocilizumab was accepted by the Scottish Medicines Consortium for use in the management of Giant Cell Arteritis (GCA) on 10.09.18. The Fife Rheumatic Diseases Unit is the first Rheumatology unit in Scotland to introduce a structured process for the management of GCA patients with tocilizumab. We describe here our experience of this new service for NHS Fife. Methods All GCA patients considered for tocilizumab are discussed at our weekly Medication Review Meeting by the Consultant Rheumatologists (JSM & SJH) and Rheumatology Pharmacist (SH). By consensus decision, GCA patients deemed appropriate for tocilizumab treatment are then referred to the Vasculitis Nurse Specialist (VNS; PA) for counselling. We have prospectively recorded data on all GCA patients counselled on tocilizumab from 08.11.18 onwards. Patients are followed up closely in a structured manner by the VNS on a fortnightly basis, with both clinical and laboratory assessment at each visit. The VNS ensures that GCA patients follow a rapid steroid taper in line with that used in the GiACTA Trial. Patients also have laboratory assessments performed between each VNS visit at their GP's surgery (to monitor the ESR) to assist in decision making in view of the rapid steroid taper. The VNS also records the cumulative steroid dose along with drug-related side effects (both from steroids and tocilizumab). Results 14 GCA patients have been counselled and commenced on tocilizumab between 08.11.18 and 18.09.19. We have another 2 GCA patients awaiting counselling and due to start tocilizumab soon. Twelve patients have newly diagnosed GCA (5 with unilateral visual loss), 5 with Large vessel vasculitis and 3 patients have refractory GCA. Of the newly diagnosed patients, the reasons for initiating tocilizumab were as follows: poor control of diabetes mellitus on high dose steroids; large vessel vasculitis confirmed on PET scan; previous long-term steroid exposure due to past history of polymyositis; high osteoporotic risk. Conclusion We have successfully introduced a structured process for the use of tocilizumab in Fife GCA patients via a VNS-led GCA clinic. This ensures robust clinical governance around the issues of safety, efficacy and cost. In addition, by collecting real life prospective data, we will add to the existing information available regarding use of this new therapeutic advance for GCA management in routine clinical practice. This initiative is the first of its kind in Scotland and a new service development for NHS Fife. Disclosures P. Allcoat None. S. Hart None. S.J. Hailwood None. J.S. McLaren Honoraria; J.S.M. has received Speaker fees from Roche.

Clinical Effectiveness of Pharmacy-Led versus Conventionally Delivered Antiviral Treatment for Hepatitis C in Patients Receiving Opioid Substitution Therapy: A Pragmatic Cluster Randomised Trial

Background: Highly-effective direct acting antiviral drugs (DAAs) provide the opportunity to eliminate Hepatitis C (HCV) infection. This study examined whether a community pharmacy pathway increased treatment uptake, treatment completion and cure rates for people receiving opioid substitution therapy (OST), compared to conventional care. Methods: This cluster randomised trial was performed in 55 community pharmacies with patients receiving OST. Pharmacists either referred patients with evidence of HCV antibodies to conventional care (27 pharmacies) or assessed them in the pharmacy (28 pharmacies). All pharmacies were trained to offer dried blood spot testing (DBST). In the pharmacy arm, infection with HCV genotype 1 or 3 was confirmed, assessment bloods were taken, and DAAs were prescribed by a pharmacist. In the comparison arm, the patient received care outside the pharmacy. Once prescribed, medication in both arms was delivered as daily modified directly observed therapy alongside OST. The primary outcome was number of patients achieving sustained virologic response 12 weeks (SVR12) after treatment completion compared to total number of people receiving OST treatment at participating pharmacies. Findings: More OST users in the pharmacy arm versus the conventional care arm agreed to DBST (245 vs 145, OR 2∙292, 0∙968-5∙427, p=0∙059); more patients diagnosed with HCV, initiated (112 vs 61, OR 1∙889, 1∙276-2∙789, p=0∙0015) and completed treatment (108 vs 58, OR 1∙928, 1∙321-2∙813, p=0∙0007); more patients achieved the primary endpoint SVR12 in the pharmacy arm compared to the conventional arm (98 vs 43, OR 2∙375, 1∙555-3∙628, p< 0∙0001). Interpretation: Utilising pharmacists to deliver an HCV pathway, made testing and treatment more accessible for patients, whilst ensuring improved reach and maintaining high treatment success rates. Use of this pathway could form a key part on an integrated approach to HCV elimination. Trial Registration: Trial registration: NCT02706223. Funding Statement: Partnership between the Scottish Government, Gilead Sciences Inc. and Bristol Myers-Squib. Declaration of Interests: Dr Radley: Honorariums from Gilead and Abbvie; Research Grants from Gilead and Roche. Prof Donnan: Research grants from Novo Nordisk, GSK, Shire pharmaceuticals, Gilead and Bristol Myers Squibb. Member of the New Drugs Committee of the Scottish Medicines Consortium. Dr Barclay: Speakers fees from Abbvie, Gilead, and advisory board fees from Abbvie, BMS, Gilead, MSD. Professor Dillon: Research Grants, and Honorariums from Abbvie, Bristol Myers Squibb, Gilead, Janssen, Merck Roche Sharp & Dohme and Roche. All other authors declare no competing interests. Ethics Approval Statement: Ethics approval granted by East of Scotland Research Ethics Committee 2 (15/ES/0086), sponsor, research & development and Caldecott approvals by University of Dundee, NHS Tayside, NHS Grampian and NHS Greater Glasgow and Clyde.

Scottish Medicines Consortium accepts nasal spray for depression previously rejected by NICE

Scottish Medicines Consortium accepts nasal spray for depression previously rejected by NICE

Estimating the Relative Treatment Effect and Corresponding Cost-Effectiveness Estimates of Inotuzumab Ozogamicin Vs. Blinatumomab for Adults with Philadelphia Chromosome-Negative (Ph-) Relapsed/Refractory (R/R) B-Cell Acute Lymphoblastic Leukaemia (B-ALL) in the United Kingdom (UK)

Introduction: Inotuzumab ozogamicin (InO), is a novel anti-CD22 antibody-calicheamicin conjugate approved in R/R B-ALL due to its high hematologic remission rate (81%) based on the phase 3 INO-VATE trial comparing to investigators choice (IC). The TOWER trial demonstrated the efficacy and safety of blinatumomab (Blina) for treatment of Ph- B-ALL versus IC. The relative effectiveness of InO versus Blina was investigated by applying indirect treatment comparison (ITC) methods. A UK-based cost-effectiveness model (CEM) submitted to the Scottish Medicines Consortium (SMC) explored the impact of treatment differences with regard to mean life years (LY) gained and quality-adjusted life years (QALY). Methods: As R/R ALL is a terminal disease if left untreated, achievement of complete response/complete response with incomplete count recovery (CR/CRi) in conjunction with stem cell transplant (SCT) is essential for long-term survival. The three most important outcomes related to treatment are thus the level of response determined by CR/CRi, the rate of SCT, and overall survival (OS). Without potentially curative therapy such as SCT, there is no evidence to suggest long-term survival is possible. Therefore, to compare InO to Blina, comparisons of these outcomes were explored using patient-level data from the INO-VATE ALL trial and aggregate data from the TOWER trial. The CEM structure contained four health states categorising patients based on 'No CR/CRi &amp; no SCT', 'CR/CRi and no SCT' and patients receiving SCT ('SCT/Post SCT') - with progression-free survival (PFS) and OS modelled within these states. States were clinically validated as relevant to treatment of the disease. Death was the fourth health state. Different methods were incorporated to allocate Blina patients to the respective health-states. For levels of response (CR/CRi) and SCT a matching-adjusted indirect comparison (MAIC) and a Bucher ITC were explored. As CR/CRi and SCT rates are not mutually exclusive, a multinomial ITC was also conducted. Once allocated into respective health states, OS and PFS were modelled. Three ITC methods were used to compare OS; a simulated treatment comparison (STC), MAIC and a standard network meta-analysis. In the absence of PFS data for Blina, PFS was assumed to have the same relative treatment effect as OS. Quality of life data within the model for the 'No CR/CRi &amp; no SCT' and 'CR/CRi and no SCT' were informed from InO trial data, while SCT quality of life was informed from the literature with time-varying utilities. Costs were incorporated from a UK perspective using 2017 sources and were those submitted to the SMC. Results were annually discounted at 3.5%. Results: Health state proportions for Ph- InO patients were used as the basis to estimate corresponding Blina proportions and show 49.3% of patients treated with InO reach SCT. With higher odds for CR/CRi and SCT for InO, the ITC results consistently indicate Blina leads to lower proportions of patients receiving SCT (19.1-22.5%) and CR/CRi (25.2-33.3%). ITCs comparing OS outcomes for InO versus blinatumomab show negligible differences between treatments, consistently across the three methods. All combinations of the various methods were explored using the list price for both treatments. The results of the CEM ranged from 0.91-1.14 incremental QALYs for InO versus Blina, while LYs ranged from 2.03-2.59 resulting from higher rates of SCT. The incremental cost-effectiveness ratio (ICER) ranged from £3,700 to £7,010 for InO versus Blina. Extensive scenario analysis indicates that InO is a cost-effective option compared to Blina at a willingness to pay threshold of £20,000 per QALY. The SMC recommended InO as a cost-effective use of resources citing an ICER of £6,754 in the CEM when using the MAIC; InO was associated with a mean survival gain of &amp;gt;29 months over Blina corresponding to this ICER. Conclusions: Outcomes from the ITC indicate that InO provides patients with a greater probability of achieving CR/CRi and/or receiving a subsequent SCT versus Blina. As CR/CRi followed by SCT are essential for long-term survival and potential cure, the mean OS gain in the model cited in the SMC recommendation is intuitive as it aligns with the superior CR/CRi and SCT odds ratios associated with InO. Further research is required to determine the long-term PFS and OS following SCT in R/R B-ALL, beyond what can be reliably captured within clinical trials. Disclosures Critchlow: BresMed Health Solutions Ltd.: Consultancy. Cooper:BresMed Health Solutions Ltd.: Consultancy. van Oostrum:Ingress Health: Employment; Pfizer: Consultancy; Merck: Consultancy; Janssen: Consultancy; AstraZeneca: Consultancy. Welch:Pfizer Inc: Employment, Equity Ownership. Russell-Smith:Pfizer: Employment, Equity Ownership.

PRO111 REIMBURSEMENT OF ULTRA-ORPHAN MEDICINES: A NEW BEST-PRACTICE FRAMEWORK?

Since October 2018, ultra-orphan therapies are subject to a new decision-making pathway in Scotland. Under this pathway, ultra-orphan medicines (definition: prevalence <1:50,000) would be made available for ≥3-years whilst additional evidence is collected to inform a final Scottish Medicines Consortium (SMC) appraisal. Prior to this, the SMC had an ultra-orphan medicine evaluation process, with a broader decision-making framework versus the standard appraisal process. This research aims to evaluate the pre-2018 SMC ultra-orphan medicine evaluation process by identifying all drugs assessed under this process and comparing their outcomes with the National Institute for Health and Care Excellence (NICE) (which has the Highly Specialized Technology [HST] program for evaluation of ultra-rare therapies). Publicly-available SMC guidance was screened (13/10/2014-08/04/2019) and any appraisals under the ultra-orphan process identified (alongside any corresponding NICE guidance) and key information extracted. 51 SMC appraisals under the pre-2018 ultra-orphan pathway were identified (average of 10 per year). 59% were accepted, 22% were optimized, and 20% were not recommended. 65% (33/51) had been assessed by NICE (although only four under the HST), with another five appraisals ongoing. 97% (32/33) NICE final appraisals were either recommended, optimized, or recommended for the Cancer Drugs Fund. Although recommendation rates under the pre-2018 SMC ultra-orphan process were 80%, most of these had also been assessed by NICE, almost all of which received a positive recommendation. Further, very few of these NICE appraisals were under the HST, i.e. these high NICE recommendation rates were achieved via assessment under the standard appraisal pathway. Further research can compare how SMC recommendations of ultra-orphan medicines will evolve according to other countries following the new decision-making pathway.

PCN364 REIMBURSEMENT OF CAR-T CELL THERAPY IN THE UK, A STEP TOWARDS MORE PERSONALISED CANCER TREATMENT

Today’s healthcare system strives for more personalised treatments to improve patient outcomes. CAR-T cell therapy is a novel immunotherapy in which a patient’s own T cells are genetically modified to express chimeric antigen receptor (CAR), which can attach to the surface of cancer cells and destroy them. We reviewed the appraisal of this therapy in the UK. National Institute for Health and Care Excellence (NICE) and Scottish Medicine Consortium (SMC) health technology appraisals for CAR-T cell therapies were reviewed. To date, two CAR-T cell therapies have been appraised by NICE and SMC, tisagenlecleucel and axicabtagene ciloleucel. The appraisal of tisagenlecleucel in relapsed/refractory B-cell acute lymphoblastic leukaemia in people aged ≤25 years faced a number of challenges, including high uncertainty of the data on overall survival, subsequent stem cell transplant rates and immunoglobulin usage, lack of comparator data, and high cost-effectiveness estimates. To mitigate these, tisagenlecleucel has been conditionally approved by both NICE and SMC through the Cancer Drug Fund (CDF) and Patient Access Scheme, respectively. Due to data uncertainties and high cost-effectiveness estimates, both NICE and SMC rejected the use of tisagenlecleucel in relapsed/refractory diffuse large B-cell lymphoma (DLBCL). Additional negotiations lead NICE to use a managed access agreement (MAA) which allows the use of tisagenlecleucel in DLBCL under CDF while additional evidence is being collected. Axicabtagene ciloleucel was also approved by NICE for treating DLBCL and primary mediastinal large B-cell lymphoma after ≥2 systemic therapies through the use of a MAA, but not by the SMC. Despite their potential benefits to patients, CAR-T cell therapies face a number of challenges during HTA appraisal in the UK, including uncertainties in clinical effectiveness and in long-term costs and benefits. Risk sharing between the NHS and manufacturers and the use of innovative payment methodologies should be further investigated.

PND89 NEGATIVE HTA RECOMMENDATIONS FOR MEDICINES IN NEUROLOGY THERAPEUTIC AREAS – FOURFOLD HIGHER ODDS IN POLAND COMPARED WITH THE UNITED KINGDOM

Technology appraisals (TA) by health technology assessment (HTA) bodies continue to pose a potential hurdle to broad access of innovative medicines in Europe, including Poland. Risk-sharing agreement (RSA) are a potential means of increasing chances of acceptance. The aim of this study was to compare TA outcomes of the Polish HTA body (Agencja Oceny Technologii Medycznych i Taryfikacji; AOTMiT) and UK reimbursement bodies (National Institute for Health and Care Excellence; NICE, Scottish Medicines Consortium; SMC) for medicines in neurology area (F01-G99 of the ICD-10-CM Codes) and analyze RSA trends in Poland. Published outcomes of TAs carried out by AOTMiT between 2015 and 2018 were reviewed and matched to the TAs carried out by NICE/SMC. Fisher's exact test for a 2x2 contingency table was carried out to obtain odds ratio (OR) for a negative HTA outcome. Recommendations on including RSA in positive HTA decisions by AOTMiT were analyzed in two periods (2018 vs. 2015-2017). A total of 76 HTA submissions were identified (47 from AOTMiT and 29 from NICE/SMC). The rejection rate was 40.4 % by AOTMiT and 13.8% by NICE/SMC. The OR for a negative HTA recommendation was 4.24 (95% CI 1.27 to 14.20, p=0.018) between AOTMiT and NICE/SMC. RSA were recommended in 90% of positive TAs by AOTMiT in 2018 compared with 33% in positive TAs in 2015-2017 (OR=2.4; 95%CI 0.47 to 12.61, not significant). Poland remains a challenging market for innovative neurology medicines with fourfold higher odds of HTA rejection compared to the UK. Increasing trend of positive HTA recommendations with suggestions on RSA may positively influence future acceptance rate by AOTMiT for innovative medicines in neurology area.

Difficulties arising in reimbursement recommendations on new medicines due to inadequate reporting of population adjustment indirect comparison methods.

Indirect treatment comparisons are useful to estimate relative treatment effects when head-to-head studies are not conducted. Statisticians at the National Centre for Pharmacoeconomics Ireland (NCPE) and Scottish Medicines Consortium (SMC) assess the clinical and cost-effectiveness of new medicines as part of multidisciplinary teams. We describe some shared observations on areas where reporting of population-adjustment indirect comparison methods is causing uncertainty in our recommendations to decision-making committees when assessing reimbursement of medicines.

Developments in chemotherapy for the treatment of acute myeloid leukemia.

Mark Drummond obtained his medical degree from the University of Glasgow (UK) and since then has trained as a hematologist in the West of Scotland and the Canterbury District Health Board (Christchurch, New Zealand) and back at Glasgow University, where he also gained a PhD in chronic myeloid leukemia. He is currently a consultant hematologist and honorary senior lecturer at the Beatson Cancer Centre (Glasgow, UK) as well as an investigator on multiple UK and international clinical trials.Here he talks to Commissioning Editor Jennifer Straiton, commenting on the recent announcement from the Scottish Medicines Consortium, regarding their acceptance of the use of the combination chemotherapy treatment Vyxeos® (daunorubicin and cytarabine) for the treatment of high-risk acute myeloid leukemia in adults. He then discusses the ongoing and future clinical trials in this disease area and then turns to more personalized treatments.

July 2019 decisions by Scottish Medicines Consortium

July 2019 decisions by Scottish Medicines Consortium

The Cancer Drugs Fund in Practice and Under the New Framework.

The Cancer Drugs Fund (CDF) was established in 2010 to improve access to treatments not routinely available. Having widely overspent, stricter budgeting rules were introduced in 2016. The CDF can now only include treatments with potential to be cost effective once sufficient data are collected. Our objective was to explore the process and criteria used for consideration of treatments under the new CDF framework and to describe the extent of evidence collection. We identified CDF list, UK National Institute for Health and Care Excellence (NICE) and Scottish Medicines Consortium documents (10 May 2018). Data were collected on drugs and indications, reasons for inclusion in the CDF, data collection, incremental cost-effectiveness ratios (ICERs), and corresponding recommendations for Scotland. In total, 12 drugs were listed on the CDF in 17 indications, 12 of which were considered end-of-life care. The most common cancers were non-small-cell lung (n = 4), urothelial (n = 3), lymphocytic leukaemia (n = 2) and multiple myeloma (n = 2). The companies' ICERs were generally lower than those from the committee and the evidence review group. Drugs were included in the CDF for 6-42 months, with the majority included for ≥18 months. Data were frequently collected on overall survival (n = 16) and progression-free survival (n = 5) using NHS systems and, frequently, ongoing trials. Data collection frequently included overall survival and exceeded the 2 years recommended in the CDF strategy. It appears the CDF is allowing patients access to drugs long before they may become available for routine use. Our results are limited by the availability of published information and the small dataset.

Three new medicines accepted by Scottish Medicines Consortium

Three new medicines accepted by Scottish Medicines Consortium

To HTA or Not to HTA: Identifying the Factors Influencing the Rapid Review Outcome in Ireland

ObjectivesReimbursement systems are evolving and endeavor to balance access and affordability. One such evolution in Ireland is the compulsory rapid review (RR) process, the outcome from which is a recommendation for a health technology assessment (HTA) or no HTA. For drugs that avoid an HTA, evaluation times are shorter, lengthy price negotiations are avoided, and access is faster. In the absence of formal decision-making criteria around the requirement of an HTA, this study examines the factors influencing the outcome of the RR process in Ireland. MethodsA database was developed combining data from publicly available sources for drug evaluations conducted by the National Centre for PharmacoEconomics (NCPE) (January 2010-June 2017, n = 296). Because Irish cost data were not publicly available for all drugs, cost data from the Scottish Medicines Consortium were employed as a proxy. Employing logistic regressions, the factors influencing the RR outcome are revealed. ResultsAfter an RR, an HTA was recommended for 55% of drugs. The regression results revealed therapeutic area (endocrine, musculoskeletal, and neoplasm), first-in-class and orphan disease increased the probability of an HTA. Furthermore, when proxy costs were included, results revealed that every €1000 increase in annual drug costs per patient increased the probability of an HTA being required by 1% and that an HTA was more likely than no HTA when annual drug costs exceeded €15 000. ConclusionGiven the current focus on access and affordability, this study identifies the factors influencing the requirement of an HTA in Ireland.

OP79 Improving Public Understanding Of Scottish Medicines Consortium Advice

IntroductionTransparency of processes and decision making is important to the Scottish Medicines Consortium (SMC). An independent review of access to new medicines in Scotland in 2016 recommended that SMC should review its communication of decisions with a view to achieving greater transparency. SMC therefore began to develop plain English summaries of advice on each new medicine.MethodsA multi-stakeholder approach was adopted to develop the summary documents, with patient groups involved. Firstly, a review of communications for the public from other HTA organizations was conducted. The public involvement team then held a workshop to find out what patient groups felt would be important to include when explaining SMC decisions to patients and the public. The process was also informed by reviewing examples of good practice from other parts of NHSScotland, including patient versions of Scottish Intercollegiate Guidelines Network (SIGN) clinical guidelines. Exemplar documents were then developed and feedback sought from the Public Involvement Network Advisory Group.ResultsA format was developed for the SMC ‘Decision Explained’ summaries consisting of a question and answer format for each medicine decision in a two page document. The summaries were piloted internally over a six month period, during which the development process and layout were finalized. Since September 2018 these summaries have been published on the website alongside the technical advice.ConclusionsPartnership working between SMC and patient groups has helped to develop a new way of communicating SMC's decisions to patients and the public in a clear way, helping to improve transparency and understanding. Evaluation of the summaries will be undertaken from six months of publication.