The Medicines Intelligence Centre of Research Excellence: Co-creating real-world evidence to support the evidentiary needs of Australian medicines regulators and payers.

Regulators and payers play a pivotal role in facilitating timely and affordable access to safe and efficacious medicines. They use evidence generated from randomised clinical trials (RCTs) to support decisions to register and subsidise medicines. However, at the time of registration and subsidy approval, regulators and payers face uncertainty about how RCT outcomes will translate to real-world clinical practice. In response to this situation, medicines policy agencies worldwide have endorsed the use of real-world data (RWD) to derive novel insights on the use and outcomes of prescribed medicines. Recent reforms around data availability and use in Australia are creating unparalleled data access and opportunities for Australian researchers to undertake large-scale research to generate evidence on the safety and effectiveness of medicines in the real world. Highlighting the critical importance of research in this area, Quality Use of Medicines and Medicine Safety was announced as Australia's 10th National Health Priority in 2019. The National Health and Medical Research Council, Medicines Intelligence Centre of Research Excellence (MI-CRE) has been formed to take advantage of the renewed focus on quality use of medicines and the changing data landscape in Australia. It will generate timely research supporting the evidentiary needs of Australian medicines regulators and payers by accelerating the development and translation of real-world evidence on medicines use and outcomes. MI-CRE is developing a coordinated approach to identify, triage and respond to priority questions where there are significant uncertainties about medicines use, (cost)-effectiveness, and/or safety and creating a data ecosystem that will streamline access to Australian data to enable researchers to generate robust evidence in a timely manner. This paper outlines how MI-CRE will partner with policy makers, clinicians, and consumer advocates to leverage real-world data to co-create real-world evidence, to improve quality use of medicines and reduce medicine-related harm.

Societal preferences for fertility treatment in Australia: a stated preference discrete choice experiment.

Objective: To investigate preferences for fertility treatment from the Australian general population with the aims of calculating the willingness to pay in tax contribution for attributes (characteristics) that make up treatment and for an "ideal" fertility treatment program. We also assessed whether willingness-to-pay varies by the relationship status or sexual orientation of the patient.Methods: A stated preference discrete choice experiment was administered to a panel of 801 individuals representative of the Australian general population. Seven attributes of fertility treatment under three broad categories were included: outcome, process, and cost. Attributes were identified through published literature, focus group discussions, expert knowledge, and a pilot study. A Bayesian fractional experimental design was used, and data analysis was performed using a generalized multinomial logit model. Further analyses included interaction terms and latent class modeling.Results: Six of the seven attributes influenced the choice of a treatment program. Under process attributes, individuals preferred: continuity of care of clinic staff, where patients are seen by the same doctor but different nurses at each visit; "alternative" treatments being offered to all patients; and onsite clinic counseling and peer-support groups. Personalization and tailoring of the treatment journey were not important. Among outcome attributes, the improved success rate of having a baby per cycle and significant side-effects were considered important. Cost of treatment also influenced the choice of treatment program. Individual preferences for fertility treatment were not associated with patients' relationship status or sexual orientation. Latent class modeling revealed sub-groups with distinct fertility treatment preferences.Conclusion: This study provides important insights into the attributes that influence the preferences of fertility treatment in Australia. It also estimates socially-inclusive willingness-to-pay values in tax contributions for an "ideal" package of treatment. The results can inform economic evaluations of fertility treatment programs.

Improving, but could do better: Trends in gestation-specific stillbirth in Australia, 1994-2015.

BACKGROUND: Stillbirth remains a public health concern in high-income countries. Over the past 20 years, stillbirth rates globally have shown little improvement and large disparities. The overall stillbirth rate, which measures risk among births at all gestations, masks diverging trends at different gestations. This study investigates trends over time in gestation-specific risk of stillbirth in Australia. METHODS: Analytical epidemiological study using nationally reported gestational age data for births in Australia, 1994-2015. Average annual change in gestation-specific prospective risk of stillbirth (per 1000 fetuses at risk [FAR]) was calculated among births in 1994-2009 and 2010-2015 at term (37-41 weeks) and for preterm gestational age subgroups: 28-36, 24-27, and 20-23 weeks. RESULTS: The decline in risk of stillbirth at term from 2010 to 2015 from 1.43 to 1.16 per 1000 FAR was more rapid than from 1994 to 2009; for preterm gestations from 24 to 27 weeks, there were no discernible trends; from 28 to 36 weeks, the decline between 1994 and 2009 was not sustained; among births from 20 to 23 weeks, the risk of stillbirth plateaued in 2010-2015, fluctuating around 3.3 per 1000 FAR. CONCLUSIONS: Improvement in the stillbirth rate from 28 weeks' gestation aligns with changes in other high-income countries, but more work is needed in Australia to achieve the levels of reduction seen elsewhere. Gestation-specific risk of stillbirth is more informative than the overall stillbirth rate. The message that the overall risk of stillbirth is not changing disregards gains at different stages of pregnancy.

Assessment of the societal and individual preferences for fertility treatment in Australia: study protocol for stated preference discrete choice experiments.

INTRODUCTION: In Australia, societal and individual preferences for funding fertility treatment remain largely unknown. This has resulted in a lack of evidence about willingness to pay (WTP) for fertility treatment by either the general population (the funders) or infertile individuals (who directly benefit). Using a stated preference discrete choice experiment (SPDCE) approach has been suggested as a more appropriate method to inform economic evaluations of fertility treatment. We outline the protocol for an ongoing study which aims to assess fertility treatment preferences of both the general population and infertile individuals, and indirectly estimate their WTP for fertility treatment. METHODS AND ANALYSIS: Two separate but related SPDCEs will be conducted for two population samples-the general population and infertile individuals-to elicit preferences for fertility treatment to indirectly estimate WTP. We describe the qualitative work to be undertaken to design the SPDCEs. We will use D-efficient fractional experimental designs informed by prior coefficients from the pilot surveys. The mode of administration for the SPDCE is also discussed. The final results will be analysed using mixed logit or latent class model. ETHICS AND DISSEMINATION: This study is being funded by the Australian National Health and Medical Research Council (NHMRC) project grant AP1104543 and has been approved by the University of New South Wales Human Research Ethics Committee (HEC 17255) and a fertility clinic's ethics committee. Findings of the study will be disseminated in peer-reviewed journals and presented at various conferences. A lay summary of the results will be made publicly available on the University of New South Wales National Perinatal Epidemiology and Statistics Unit website. Our results will contribute to the development of an evidence-based policy framework for the provision of cost-effective and patient-centred fertility treatment in Australia.