Innovative technologies for reverse total shoulder arthroplasty in Australia: Market access challenges and implications for patients, decision-makers, and manufacturers.

Purpose: The success of reverse total shoulder arthroplasty (RTSA) has expanded its use for a broader range of shoulder indications worldwide. Evidence regarding the relative efficacy and long-term safety of medical technologies used in RTSA is subjected to rigorous assessment. Nonetheless, substantial challenges impede market access for innovative shoulder implant technologies for RTSA in Australia, resulting in delayed patient access. Approach: This paper addresses the key challenges associated with generating evidence for the health technology assessments of innovative medical technologies for RTSA that are required for access to the Australian market. The transition to value-based care requires establishing a benchmarking reference that incorporates patient-reported outcome measures (PROMs) and combines revision outcomes with additional clinical outcomes to increase patient cohort sizes. Establishing the benchmark would require agreement on the outcome measures to be collected for each indication, and investment in reporting patient-reported outcomes for RTSA to the national orthopaedic registry. Implications for practice: The need for increased flexibility in developing evidence for health technology assessment of RTSA medical technologies is required. Optimised approaches for benchmarking RTSA require extensive stakeholder discussions, including the agreement on evidence requirements and follow-up periods, selection of clinical outcomes, as well as pre-operative and post-operative PROMs as a value assessment.

Factors associated with Pharmaceutical Benefits Advisory Committee decisions for listing medicines for diabetes and its associated complications.

Objective To retrospectively analyse the key factors associated with listing decisions by the Pharmaceutical Benefits Advisory Committee (PBAC) for medicines for diabetes and its complications on the Pharmaceutical Benefits Scheme. Methods The clinical and economic evidence were retrieved from public summary documents (PSD) of all major submissions between July 2005 and March 2020. A multivariate binary logit regression analysis was conducted to assess the relationship between the categorical explanatory variables and PBAC recommendations. Results We identified a total of 211 PSD of which 118 (56%) were recommended for listing. Clinical and economic uncertainty were significantly and inversely associated with the PBAC recommendation. Submissions with high clinical and economic uncertainty were less likely to be recommended. Conclusion Our findings will enhance the understanding of medical professionals, pharmaceutical companies, and other stakeholders about the rationale of PBAC reimbursement decisions for these medicines and assist prospective applicant sponsor companies in preparing their submissions.

Cost-Effectiveness of Screening Preschool Children for Hearing Loss in Australia.

OBJECTIVES: While all newborns in Australia are tested for congenital hearing loss through universal newborn hearing screening programs, some children will acquire hearing loss in their first five years of life. Delayed diagnosed or undiagnosed hearing loss in children can have substantial immediate- and long-term consequences. It can significantly reduce school readiness, language and communication development, social and emotional development, and mental health. It can also compromise lifetime educational achievements and employment opportunities and future economic contribution to society through lost productivity. The need for a universal hearing screening program for children entering their first year of primary school has been noted in two separate Australian Government hearing inquiries in the last decade. Sound Scouts is a hearing screening application (app) that tests for hearing loss in children using a tablet or mobile device, supervised by parents at home. It tests for sensorineural or permanent conductive hearing loss and central auditory processing disorder in children. In 2018 the Australian Government funded the roll-out of Sound Scouts to allow up to 600,000 children to test their hearing using Sound Scouts. This study estimated the cost-effectiveness of screening 5-year-old children for hearing loss using Sound Scouts at home, compared with no screening. DESIGN: A decision-analytic model was developed to estimate the incremental costs and quality-adjusted life years (QALYs) of administering Sound Scouts over a 20-year time horizon. Testing accuracy was based on comparing Sound Scouts test results to clinical test results while other parameters were based on published data. Costs were estimated from the perspective of the Australian health care system. Univariate and probabilistic sensitivity analyses were undertaken. RESULTS: Sound Scouts is estimated to result in an average incremental cost of A$61.02 and an average incremental increase in QALYs of 0.01. This resulted in an incremental cost-effectiveness ratio of A$5392 per QALY gained, which is likely to be considered cost-effective by Australian decision makers. Screening with Sound Scouts was found to have a 96.2 per cent probability of being cost-effective using a threshold of A$60,000 per QALY gained. CONCLUSIONS: Using Sound Scouts to screen five-year-old children for hearing loss (at home) is likely to be cost-effective. Screening children using Sound Scouts will result in early identification and intervention in childhood hearing loss, thereby reducing early childhood disadvantage through cumulative gains in quality of life, education, and economic outcomes over their lifetime.

The cost-effectiveness of unilateral cochlear implants in UK adults.

OBJECTIVE: The National Institute for Health and Care Excellence (NICE) updated its eligibility criteria for unilateral cochlear implants (UCIs) in 2019. NICE claimed this would not impact the cost-effectiveness results used within its 2009 technology appraisal guidance. This claim is uncertain given changed clinical practice and increased healthcare unit costs. Our objective was to estimate the cost-effectiveness estimates of UCIs in UK adults with severe to profound hearing loss within the contemporary NHS environment. METHODS: A cost-utility analysis employing a Markov model was undertaken to compare UCIs with hearing aids or no hearing aids for people with severe to profound hearing loss. A clinical pathway was developed to estimate resource use. Health-related quality of life, potential adverse events, device upgrades and device failure were captured. Unit costs were derived mostly from the NHS data. Probabilistic sensitivity analysis further assessed the effect of uncertain model inputs. RESULTS: A UCI is likely to be deemed cost-effective when compared to a hearing aid (£11,946/QALY) or no hearing aid (£10,499/QALY). A UCI has an 93.0% and 98.7% likelihood of being cost-effective within the UK adult population when compared to a hearing aid or no hearing aid, respectively. ICERs were mostly sensitive to the proportion of people eligible for cochlear implant, discount rate, surgery and device costs and processor upgrade cost. CONCLUSION: UCIs remain cost-effective despite changes to clinical practice and increased healthcare unit costs. Updating the NICE criteria to provide better access UCIs is projected to increase annual implants in adults and children by 70% and expenditure by £28.6 million within three years. This increased access to UCIs will further improve quality of life of recipients and overall social welfare.

The cost-effectiveness of Cochlear implants in Swedish adults.

BACKGROUND: Research has shown unilateral cochlear implants (CIs) significantly improve clinical outcomes and quality of life in adults. However, only 13% of eligible Swedish adults currently use a unilateral CI. The objective was to estimate the cost-effectiveness of unilateral CIs compared to a hearing aid for Swedish adults with severe to profound hearing loss. METHODS: A Markov model with a lifetime horizon and six-month cycle length was developed to estimate the benefits and costs of unilateral CIs from the Swedish health system perspective. A treatment pathway was developed through consultation with clinical experts to estimate resource use and costs. Unit costs were derived from the Swedish National Board of Health and Welfare and the Swedish Association of Local Authorities and Regions. Health outcomes were reported in terms of Quality Adjusted Life Years (QALYs). RESULTS: Unilateral CIs for Swedish adults with severe to profound hearing loss are likely to be deemed cost-effective when compared to a hearing aid (SEK 140,474 per QALY gained). The results were most sensitive to the age when patients are implanted with a CI and the proportion of patients eligible for CIs after triage. CONCLUSIONS: An increase in the prevalence of Swedish adults with severe to profound hearing loss is expected as the population ages. Earlier implantation of unilateral CIs improves the cost-effectiveness among people eligible for CIs. Unilateral CIs are an efficacious and cost-effective option to improve hearing and quality of life in Swedish adults with severe to profound hearing loss.

Is Reclassification of the Oral Contraceptive Pill from Prescription to Pharmacist-Only Cost Effective? Application of an Economic Evaluation Approach to Regulatory Decisions.

BACKGROUND AND OBJECTIVE: Unplanned pregnancies can lead to poorer maternal and child health outcomes. The Australian Therapeutic Goods Administration committee rejected reclassifying a range of oral contraceptive pills (OCPs) from prescription to pharmacist-only medicines in 2015, mainly based on safety concerns. Improving access to OCPs may encourage some women to use contraceptives or switch from other contraceptive methods. However, some adverse events may increase and some women may stop using condoms, increasing their risk of sexually transmitted infections. This study aimed to estimate the cost effectiveness of reclassifying OCPs from prescription to pharmacist-only. PERSPECTIVE: Healthcare system. SETTING: Australian primary care. METHODS: A Markov model was used to synthesise data from a variety of sources. The model included all Australian women aged 15-49 years (N = 5,644,701). The time horizon was 35 years. Contraceptive use before reclassification was estimated using data from the Household, Income and Labour Dynamics in Australia (HILDA) survey, while survey data informed use after reclassification. Health outcomes included pregnancies, pregnancy outcomes (live birth, miscarriage, stillbirth, ectopic pregnancy and abortion), sexually transmitted infections, adverse events (venous thromboembolism, depression, myocardial infarction and stroke), ovarian cancer cases and quality-adjusted life-years. Costs included those related to general practitioner and specialist consultations, contraceptives and other medicines, pharmacist time, hospitalisations and adverse events. All costs were reported in 2016 Australian Dollars. A 5% discount rate was applied to health outcomes and costs. RESULTS: Reclassifying OCPs resulted in 85.70 million quality-adjusted life-years experienced and costs of $46,910.14 million over 35 years, vs. 85.68 million quality-adjusted life-years experienced and costs of $50,274.95 million with OCPs remaining prescription-only. Thus, reclassifying OCPs was more effective and cost saving. However, a sensitivity analysis found that more research on the probability of pregnancy in women not using contraception and not trying to conceive is needed. CONCLUSION: Reclassifying OCPs is likely to be considered cost effective by Australian decision makers.

Cost-Effectiveness of Reclassifying Triptans in Australia: Application of an Economic Evaluation Approach to Regulatory Decisions.

BACKGROUND: Migraine is a common, chronic, disabling headache disorder. Triptans, used as an acute treatment for migraine, are available via prescription in Australia. An Australian Therapeutic Goods Administration (TGA) committee rejected reclassifying sumatriptan and zolmitriptan from prescription medicine to pharmacist-only between 2005 and 2009, largely on the basis of concerns about patient risk. Nevertheless, pharmacist-only triptans may reduce migraine duration and free up healthcare resources. OBJECTIVES: To estimate the cost-effectiveness of reclassifying triptans from prescription-only to pharmacist-only in Australia. METHODS: The study design included decision-analytic modeling combining data from various sources. Behavior before and after reclassification was estimated using medical practitioner and patient surveys and also administrative data. Health outcomes included migraine frequency and duration as well as adverse events (AEs) discussed by the TGA committee. Efficacy and AEs were estimated using randomized controlled trials and observational studies. RESULTS: Reclassifying triptans will reduce migraine duration but increase AEs. This will result in 337 quality-adjusted life-years gained at an increased cost of A$5.9 million over 10 years for all Australian adults older than 15 years (19.6 million). The incremental cost-effectiveness ratio was estimated to be A$17 412/quality-adjusted life-year gained. CONCLUSIONS: The incremental cost-effectiveness ratio is likely to be considered cost-effective by Australian decision makers. Serotonin syndrome, a key concern of the TGA committee, had little impact on the results. Further research is needed regarding pharmacist-only triptan use by migraineurs currently using over-the-counter medicines and by nonmigraineurs, the efficacy of triptans, and the risk of cardiovascular and cerebrovascular AEs and chronic headaches with triptans.