Access in all areas? a round up of developments in market access and health technology assessment: part 4.

In this latest update, we look at recent developments in market access including the pricing agreement of Libmeldy® by the Beneluxa Initiative, the financial impact of managed entry agreements in Italy and the restructuring of Agenzia Italiana del Farmaco (AIFA). We also highlight the collaboration between FINOSE and the New Expensive Drug (NED) section of the Nordic Pharmaceutical Forum.

Access in all areas? A round up of developments in market access and health technology assessment: part 3.

In this latest update, we explore some of the key updates in market access over recent months including the UK's voluntary scheme for branded medicines pricing, access and growth (VPAG), the first drugs funded by the Innovative Medicines Fund in the UK and the Direct Access Scheme in France, and, finally, the new Institute for Clinical and Economic Review (ICER) value assessment framework in the USA.

Access in all areas? A round up of developments in market access and health technology assessment: part 2.

In this latest update, we explore the Inflation Reduction Act (IRA) enacted by the US Congress in August 2022, with the Centers for Medicare and Medicaid Services (CMS) recently releasing the list of the first ten drugs it will negotiate prices on. We also cover the consequences of price controls and rigid value assessment in Germany which have led to the withdrawal of a number of medicines. It will be important to see how the IRA balances cost-saving with holistic value assessment, incentives for innovation and patient access to treatment.

Access in all areas? A round up of developments in market access and health technology assessment: part 1.

In this new series reviewing recent developments in market access, we highlight publications investigating health technology assessment (HTA) guidance, review processes and outcomes across the world and discuss how forthcoming changes in the HTA and regulatory environment in the European Union may allow for more consistency in decision making.

Cost-Effectiveness of Triple Therapy with Budesonide/Glycopyrronium/Formoterol Fumarate Dihydrate versus Dual Therapies in Moderate-to-Very Severe Chronic Obstructive Pulmonary Disease: United Kingdom Analysis Using the ETHOS Study.

Background: In the 52-week ETHOS study (NCT02465567), fixed-dose triple therapy with budesonide/glycopyrronium/formoterol fumarate dihydrate (BGF) reduced moderate or severe chronic obstructive pulmonary disease (COPD) exacerbations versus fixed-dose long-acting muscarinic antagonist (LAMA)/long-acting ß2-agonist (LABA) or inhaled corticosteroid (ICS)/LABA dual therapies. Here, ETHOS data were used to estimate the long-term cost-effectiveness of BGF versus LAMA/LABA and ICS/LABA dual therapies in the United Kingdom. Methods: Costs, exacerbations, quality-adjusted life-years (QALYs), and LYs were extrapolated using a Markov model that considered disease severity progression, risk of moderate and severe exacerbations, adverse events, and treatment discontinuation in patients with moderate-to-very severe COPD receiving BGF 320/14.4/10 µg, the LAMA/LABA glycopyrronium/formoterol fumarate dihydrate 14.4/10 µg (GFF), or the ICS/LABA budesonide/formoterol fumarate dihydrate 320/10 µg (BFF). Utilities for COPD severity states were estimated using EuroQol 5-dimension 5-level data from ETHOS. Exacerbation disutilities were sourced from published literature. Healthcare resource utilization was based on ETHOS data, published literature, key external experts' input, and informed assumptions. Unit costs came from the UK National Health Service Schedule of Reference Costs, Unit Costs of Health and Social Care from the Personal Social Services Research Unit, and published literature. A lifetime horizon was considered, with costs, QALYs, and LYs discounted at 3.5% per annum. Results: The incremental cost-utility ratio (ICUR; per QALY gained) was £9901 for BGF versus GFF and £2164 for BGF versus BFF. The probability of treatments being cost-effective at the conventional UK-adopted willingness-to-pay threshold of ICUR <£20,000 was 85.1% for BGF, 14.3% for GFF, and 0.6% for BFF. Conclusion: Based on ETHOS data, BGF was demonstrated to be cost-effective versus LAMA/LABA and ICS/LABA dual therapies at the conventional UK-adopted willingness-to-pay threshold (ICUR <£20,000). The main cost-effectiveness driver for BGF versus LAMA/LABA and ICS/LABA therapies was reduction in rate of exacerbations, which reduced costs and preserved quality of life.

Economic Value of the Transurethral Resection in Saline System for Treatment of Benign Prostatic Hyperplasia in England and Wales: Systematic Review, Meta-analysis, and Cost-Consequence Model.

CONTEXT: Monopolar transurethral resection of the prostate (M-TURP) is the current UK surgical standard of care for benign prostatic hyperplasia, a condition estimated to affect >2 million men in the United Kingdom. Although M-TURP efficacy in prostate resection is established, potential perioperative complications and associated costs remain a concern. OBJECTIVE: To present up-to-date and robust evidence in support of bipolar transurethral resection in saline (TURis) as an alternative surgical option to M-TURP. EVIDENCE ACQUISITION: A systematic review (SR) of electronic databases (up to 2015) for randomised controlled trials (RCTs) comparing TURis with M-TURP was conducted, followed by evidence synthesis in the form of a meta-analysis of hospital stay, catheterisation time and procedure duration, transurethral resection (TUR) syndrome, blood transfusion, clot retention, and urethral strictures. An economic analysis was subsequently undertaken from the UK National Health Service hospital perspective with costs and resource use data from published sources. EVIDENCE SYNTHESIS: The SR identified 15 good-quality RCTs, of which 11 were used to inform the meta-analysis. TURis was associated with improved safety versus M-TURP, eliminating the risk of TUR syndrome and reducing the risk of blood transfusion and clot retention (relative risks: 0.34 and 0.43, respectively; p<0.05). TURis also reduced hospital stay (mean difference: 0.56 d; p<0.0001). The economic analysis indicated potential cost savings with TURis versus M-TURP of up to £204 per patient, with incremental equipment costs offset by savings from reduced hospital stay and fewer complications. CONCLUSIONS: The TURis system is associated with significant improvements in perioperative safety compared with M-TURP while ensuring equivalent clinical outcomes of prostate resection. The safety benefits identified may translate into cost savings for UK health services. PATIENT SUMMARY: Our review of bipolar transurethral resection in saline, the new prostate resection technique, indicates that it offers equal efficacy while reducing complications and length of hospital stay.

High-dose hemodialysis versus conventional in-center hemodialysis: a cost-utility analysis from a UK payer perspective.

OBJECTIVE: To investigate the cost-effectiveness of high-dose hemodialysis (HD) versus conventional in-center HD (ICHD), over a lifetime time horizon from the UK payer's perspective. METHODS: We used a Markov modeling approach to compare high-dose HD (in-center or at home) with conventional ICHD using current and hypothetical home HD reimbursement tariffs in England. Sensitivity analyses tested the robustness of the results. The main outcome measure was the incremental cost-effectiveness ratio (ICER) expressed as a cost per quality-adjusted life-year (QALY). RESULTS: Over a lifetime, high-dose HD in-center (5 sessions/wk) is associated with higher per-patient costs and QALYs (increases of £108,713 and 0.862, respectively) versus conventional ICHD. The corresponding ICER (£126,106/QALY) indicates that high-dose HD in-center is not cost-effective versus conventional ICHD at a UK willingness-to-pay threshold of £20,000 to £30,000. High-dose HD at home is associated with lower total costs (£522 less per patient) and a per-patient QALY increase of 1.273 compared with ICHD under the current Payment-by Results reimbursement tariff (£456/wk). At an increased home HD tariff (£575/wk), the ICER for high-dose HD at home versus conventional ICHD is £17,404/QALY. High-dose HD at home had a 62% to 84% probability of being cost-effective at a willingness-to-pay threshold of £20,000 to £30,000/QALY. CONCLUSIONS: Although high-dose HD has the potential to offer improved clinical and quality-of-life outcomes over conventional ICHD, under the current UK Payment-by Results reimbursement scheme, it would be considered cost-effective from a UK payer perspective only if conducted at home.

The financial impact of increasing home-based high dose haemodialysis and peritoneal dialysis.

BACKGROUND: Evidence suggests that high dose haemodialysis (HD) may be associated with better health outcomes and even cost savings (if conducted at home) versus conventional in-centre HD (ICHD). Home-based regimens such as peritoneal dialysis (PD) are also associated with significant cost reductions and are more convenient for patients. However, the financial impact of increasing the use of high dose HD at home with an increased tariff is uncertain. A budget impact analysis was performed to investigate the financial impact of increasing the proportion of patients receiving home-based dialysis modalities from the perspective of the England National Health Service (NHS) payer. METHODS: A Markov model was constructed to investigate the 5 year budget impact of increasing the proportion of dialysis patients receiving home-based dialysis, including both high dose HD at home and PD, under the current reimbursement tariff and a hypothetically increased tariff for home HD (£575/week). Five scenarios were compared with the current England dialysis modality distribution (prevalent patients, 14.1% PD, 82.0% ICHD, 3.9% conventional home HD; incident patients, 22.9% PD, 77.1% ICHD) with all increases coming from the ICHD population. RESULTS: Under the current tariff of £456/week, increasing the proportion of dialysis patients receiving high dose HD at home resulted in a saving of £19.6 million. Conducting high dose HD at home under a hypothetical tariff of £575/week was associated with a budget increase (£19.9 million). The costs of high dose HD at home were totally offset by increasing the usage of PD to 20-25%, generating savings of £40.0 million - £94.5 million over 5 years under the increased tariff. Conversely, having all patients treated in-centre resulted in a £172.6 million increase in dialysis costs over 5 years. CONCLUSION: This analysis shows that performing high dose HD at home could allow the UK healthcare system to capture the clinical and humanistic benefits associated with this therapy while limiting the impact on the dialysis budget. Increasing the usage of PD to 20-25%, the levels observed in 2005-2008, will totally offset the additional costs and generate further savings.

Peritoneal dialysis and in-centre haemodialysis: a cost-utility analysis from a UK payer perspective.

BACKGROUND: With limited healthcare resources available, cost-effective provision of dialysis to patients with end-stage renal disease (ESRD) is important. OBJECTIVES: To assess the cost-effectiveness of varying levels of peritoneal dialysis (PD) use versus current practice among incident ESRD patients requiring dialysis. METHODS: A Markov model was developed to investigate the cost-effectiveness of increasing uptake of PD to 39 and 50 % versus current practice of 22 % PD from a UK National Health Service perspective for the year of 2013-2014. A scenario with 5 % PD was also considered. Sensitivity analyses were performed. RESULTS: Five- and 10-year discounted total costs and quality-adjusted life years (QALYs) per patient for the current scenario (22 % PD) were £96,307 and 2.104, and £133,339 and 3.301, respectively. Use of PD in 39 % of patients resulted in 5- and 10-year total per-patient cost savings of £3,180 and £4,102 versus current usage alongside total per-patient QALY increases of 0.017 and 0.020. Use of PD in 50 % of patients resulted in 5- and 10-year per-patient cost savings of £5,238 and £6,758 versus current usage alongside per-patient QALY increases of 0.029 and 0.033. Thus, increasing use of PD was associated with marginally better outcomes and lower costs. Cost savings were driven by lower treatment costs and reduced transport requirements for PD versus haemodialysis. Reducing PD use was associated with higher costs and a small reduction in QALYs. CONCLUSIONS: These findings suggest increasing PD use among incident dialysis patients would be cost-effective, associated with reduced costs and potential modest improvements in quality of life.