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A quantitative assessment of Parkinson’s disease (PD) progression is critical for optimizing clinical trials design. Disease progression model was developed using pooled data from the Progression Marker Initiative study and the Incidence of Cognitive Impairment in Cohorts with Longitudinal Evaluation in Parkinson’s Disease study. Age, gender, concomitant medication, and study arms were predictors of baseline. A mutation in the leucine-rich repeat kinase 2 (LRRK2) encoding gene was associated with the disease progression rate. The progression rate in subjects with PD who carried LRRK2 mutation was slightly slower (~0.170 points/month) than that in PD subjects without the mutation (~0.222 points/month). For a non-enriched placebo-controlled clinical trial, approximately 70 subjects/arm would be required to detect a drug effect of 50% reduction in the progression rate with 80% probability. Whereas, 85, 93 and 100 subjects/arm would be required for an enriched clinical trial with 30%, 50% and 70% subjects with LRRK2 mutations, respectively.

As therapeutic trials target early stages of Parkinson's disease (PD), appropriate patient selection based purely on clinical criteria poses significant challenges. Members of the Critical Path for Parkinson's Consortium formally submitted documentation to the European Medicines Agency (EMA) supporting the use of Dopamine Transporter (DAT) neuroimaging in early PD. Regulatory documents included a comprehensive literature review, a proposed analysis plan of both observational and clinical trial data, and an assessment of biomarker reproducibility and reliability. The research plan included longitudinal analysis of the Parkinson Research Examination of CEP-1347 Trial (PRECEPT) and the Parkinson's Progression Markers Initiative (PPMI) study to estimate the degree of enrichment achieved and impact on future trials in subjects with early motor PD. The presence of reduced striatal DAT binding based on visual reads of single photon emission tomography (SPECT) scans in early motor PD subjects was an independent predictor of faster decline in UPDRS Parts II and III as compared to subjects with scans without evidence of dopaminergic deficit (SWEDD) over 24 months. The EMA issued in 2018 a full Qualification Opinion for the use of DAT as an enrichment biomarker in PD trials targeting subjects with early motor symptoms. Exclusion of SWEDD subjects in future clinical trials targeting early motor PD subjects aims to enrich clinical trial populations with idiopathic PD patients, improve statistical power, and exclude subjects who are unlikely to progress clinically from being exposed to novel test therapeutics.

Objectives: Four in ten women with breast cancer experience high levels of anxiety or depression, despite advances in oncology treatments. The study investigates the role of psychosocial, disease and treatment characteristics, and appraisal processes to better understand factors contributing to this high psychological morbidity. Design: A postal survey was employed to observe psychological morbidity in women 2 and 6 months after initial diagnosis and treatment of breast cancer. The study was conducted as an adjunct to an Australian multi-centre feasibility study of an evidence-based specialist breast nurse (SBN) model of care. Methods: In total, 195 women with a new diagnosis of early or locally advanced breast cancer completed the data collection relating to this study. Psychosocial, disease and treatment information for each woman at diagnosis was recorded in research logs. Women completed the GHQ-12 questionnaire 2 months after diagnosis, and at 6 months they completed the GHQ-12 and an appraisal process questionnaire designed by the National Breast Cancer Centre (NBCC). Bivariate and multiple regression analyses were undertaken to build a statistical model to account for GHQ-12 scores at 6 months. Results: According to the GHQ-12, 43% of women had a likely affective disorder at either 2 or 6 months after diagnosis. Point prevalence decreased from 2 to 6 months yet remained substantial compared with general population statistics. Psychological functioning in women with breast cancer is related to a woman's psychiatric history, grade of tumour, and her appraisal processes. Most importantly, improvedpsychological functioning from 2 to 6 months after diagnosis is related to a woman having a lower primary appraisal of threat and a greatersecondary appraisal of self-efficacy in terms of having confidence in her own ability to cope with concerns associated with the illness. Conclusion: Appraisal processes play a significant role in psychological adjustment to breast cancer. Adjustment may be facilitated by ensuring that the treatment team responds to shortfalls in a woman's appraisal of her illness and her perceived ability to cope, especially where a difficult prognosis is evident.

Psychological morbidity is high for women with breast cancer and is often undetected and untreated. Encouragingly, there are well-evaluated strategies to help improve psychological outcomes, but the challenge remains as to how to transfer these to routine care settings. The National Health and Medical Research Council (NHMRC) National Breast Cancer Centre used evidence-based psychosocial clinical practice guidelines and breast cancer treatment guidelines to develop a comprehensive specialist breast nurse (SBN) model of care and conducted a feasibility study to observe its implementation in diverse clinical conditions. Seven SBNs at four Australian cancer treatment centres implemented the model of care to support 196 women with a new diagnosis of early or locally advanced breast cancer. The SBN role in detecting women's psychological difficulties is reported here. Two months after diagnosis, 36% of women had a likely psychological disorder on the General Health Questionnaire 12-item (GHQ12) version. The SBNs detected as many as 85% and as few as 20% of high GHQ12 scorers, depending upon a woman's psychosocial risk factors and consultation factors. Few women were referred to psychological services, suggesting other barriers to care. More specific communication skills training, psychological symptom screening questionnaires and better access to psychological services may help the implementation of the model of care.

Objective: This study trialled routine measurement of disability, need and outcome in mental health services within Sydney. Method: Fifteen community mental health clinicians with a combined caseload of 283 patients participated in the study. The Health of the Nation Outcome Scales (HoNOS) was used to assess disability and outcome and the patient and staff versions of the Camberwell Assessment of Need (CAN) were used to assess need. Results: The HoNOS and CAN appear to be promising contenders for routine use. Patients receiving assertive case management were rated as having higher levels of disability and need than patients receiving standard case management. Significant change in outcome was demonstrated with the HoNOS. Conclusions: To ensure the continued measurement of consumer outcome, issues such as staff education, training, and the development of computerised information systems should be addressed.