Developing a framework to incorporate real-world evidence in cancer drug funding decisions : The Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration

Background Oncology therapy is becoming increasingly more expensive and challenging the affordability and sustainability of drug programmes around the world. When new drugs are evaluated, health technology assessment organisations rely on clinical trials to inform funding decisions. However, clinical trials are not able to assess overall survival and generalises evidence in a real-world setting. As a result, policy makers have little information on whether drug funding decisions based on clinical trials ultimately yield the outcomes and value for money that might be expected. Objective The Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration, consisting of researchers, recommendation-makers, decision makers, payers, patients and caregivers, are developing and testing a framework for Canadian provinces to generate and use real-world evidence (RWE) for cancer drug funding in a consistent and integrated manner. Strategy The CanREValue collaboration has established five formal working groups (WGs) to focus on specific processes in the generation and use of RWE for cancer drug funding decisions in Canada. The different RWE WGs are: (1) Planning and Drug Selection; (2) Methods; (3) Data; (4) Reassessment and Uptake; (5) Engagement. These WGs are acting collaboratively to develop a framework for RWE evaluation, validate the framework through the multiprovince RWE projects and help to integrate the final RWE framework into the Canadian healthcare system. Outcomes The framework will enable the reassessment of cancer drugs, refinement of funding recommendations and use of novel funding mechanisms by decision-makers/payers across Canada to ensure the healthcare system is providing clinical benefits and value for money

Discovery of a new Local Group Dwarf Galaxy Candidate in UNIONS: Bo\"otes V

We present the discovery of Bo\"otes V, a new ultra-faint dwarf galaxy candidate. This satellite is detected as a resolved overdensity of stars during an ongoing search for new Local Group dwarf galaxy candidates in the UNIONS photometric dataset. It has a physical half-light radius of 26.9$^{+7.5}_{-5.4}$ pc, a $V$-band magnitude of $-$4.5 $\pm$ 0.4 mag, and resides at a heliocentric distance of approximately 100 kpc. We use Gaia DR3 astrometry to identify member stars, characterize the systemic proper motion, and confirm the reality of this faint stellar system. The brightest star in this system was followed up using Gemini GMOS-N long-slit spectroscopy and is measured to have a metallicity of [Fe/H] $=$ $-$2.85 $\pm$ 0.10 dex and a heliocentric radial velocity of $v_r$ = 5.1 $\pm$ 13.4 km s$^{-1}$. Bo\"otes V is larger (in terms of scale radius), more distant, and more metal-poor than the vast majority of globular clusters. It is likely that Bo\"otes V is an ultra-faint dwarf galaxy, though future spectroscopic studies will be necessary to definitively classify this object.Comment: 13 pages, 7 figures, 3 tables. Accepted for publication in the AAS Journals. Please note that this paper was submitted in coordination with the work of William Cerny et al. 2022. These authors independently discovered this same satellite so our two research groups have coordinated the submission of these discovery paper

The discovery of the faintest known Milky Way satellite using UNIONS

We present the discovery of Ursa Major III/UNIONS 1, the least luminous known satellite of the Milky Way, which is estimated to have an absolute V-band magnitude of $+2.2^{+0.4}_{-0.3}$ mag, equivalent to a total stellar mass of 16$^{+6}_{-5}$ M$_{\odot}$. Ursa Major III/UNIONS 1 was uncovered in the deep, wide-field Ultraviolet Near Infrared Optical Northern Survey (UNIONS) and is consistent with an old ($\tau > 11$ Gyr), metal-poor ([Fe/H] $\sim -2.2$) stellar population at a heliocentric distance of $\sim$ 10 kpc. Despite being compact ($r_{\text{h}} = 3\pm1$ pc) and composed of so few stars, we confirm the reality of Ursa Major III/UNIONS 1 with Keck II/DEIMOS follow-up spectroscopy and identify 11 radial velocity members, 8 of which have full astrometric data from $Gaia$ and are co-moving based on their proper motions. Based on these 11 radial velocity members, we derive an intrinsic velocity dispersion of $3.7^{+1.4}_{-1.0}$ km s$^{-1}$ but some caveats preclude this value from being interpreted as a direct indicator of the underlying gravitational potential at this time. Primarily, the exclusion of the largest velocity outlier from the member list drops the velocity dispersion to $1.9^{+1.4}_{-1.1}$ km s$^{-1}$, and the subsequent removal of an additional outlier star produces an unresolved velocity dispersion. While the presence of binary stars may be inflating the measurement, the possibility of a significant velocity dispersion makes Ursa Major III/UNIONS 1 a high priority candidate for multi-epoch spectroscopic follow-ups to deduce to true nature of this incredibly faint satellite.Comment: 21 pages, 9 figures, 3 tables; Accepted for publication in Ap

Eliminating fees in the Alameda County juvenile justice system meaningfully reduced financial burdens on families

In 2016, the Alameda County Board of Supervisors permanently repealed fees charged to youth in the county’s juvenile justice system. Unlike other types of monetary sanctions, like fines and restitution, fees are not intended to punish defendants or repair survivors. Instead, fees are imposed to recoup administrative costs. Increasingly, advocates are highlighting the harm that monetary sanctions can inflict on justice-involved youth and their families, and are calling for fees to be repealed. This study examines whether removing these fees has an appreciable effect on families’ overall financial burden by applying a rigorous causal-inference approach to data on 2,401 youth placed on probation before and after the fee repeal.This work has been supported, in part, by the University of California Multicampus Research Programs and Initiatives grants MRP-19-600774 and M21PR3278

Safely Increasing Connection to Community-Based Services: A Study of Multidisciplinary Team Decision Making for Child Welfare Referrals.

Initial child welfare screening decisions, traditionally made by an individual worker, determine if a family will receive further intervention by child protective services. A multi-disciplinary team (MDT) decision-making approach for child welfare referrals aims to provide a more thorough assessment of needs and strengths and to connect families to appropriate community-based providers. This study examined 159 child welfare referrals handled by MDTs compared to 331 referrals handled via the traditional screening approach. The study used a pseudo randomization procedure to assign referrals to the study conditions: Referrals logged on 2.5 days of the week were assigned to the treatment group; all others were assigned to the comparison group. Referrals handled by an MDT were more than four times as likely as those not handled by an MDT to be referred to community-based organizations (OR = 4.32, p < .001). There were no statistically significant differences in families' engagement with community-based organizations or child welfare outcomes. MDTs are a promising step in the initial process of connecting families to services, although they did not affect this study's longer-term outcomes

Safely Increasing Connection to Community-Based Services: A Study of Multidisciplinary Team Decision Making for Child Welfare Referrals.

Initial child welfare screening decisions, traditionally made by an individual worker, determine if a family will receive further intervention by child protective services. A multi-disciplinary team (MDT) decision-making approach for child welfare referrals aims to provide a more thorough assessment of needs and strengths and to connect families to appropriate community-based providers. This study examined 159 child welfare referrals handled by MDTs compared to 331 referrals handled via the traditional screening approach. The study used a pseudo randomization procedure to assign referrals to the study conditions: Referrals logged on 2.5 days of the week were assigned to the treatment group; all others were assigned to the comparison group. Referrals handled by an MDT were more than four times as likely as those not handled by an MDT to be referred to community-based organizations (OR = 4.32, p < .001). There were no statistically significant differences in families' engagement with community-based organizations or child welfare outcomes. MDTs are a promising step in the initial process of connecting families to services, although they did not affect this study's longer-term outcomes

Examining multi-session brief intervention for substance use in primary care: research methods of a randomized controlled trial.

BACKGROUND: Brief interventions such as Screening, a single session of Brief Intervention, and Referral to Treatment (SBIRT) have shown mixed effectiveness in primary care. However, there are indications that multi-session brief interventions may demonstrate more consistently positive outcomes, and perhaps a more intensive approach would be of benefit in addressing substance use in primary care. This study compared the effectiveness of SBIRT with a single BI session (BI/RT) to a multi-session brief-treatment intervention (BI/RT+) in primary care. We also developed easy-to-use, evidence-based materials to assist clinicians in delivering these interventions. METHODS/DESIGN: This study was conducted in three Federally Qualified Healthcare Centers (FQHCs). A total of 10,935 patients were screened, and 600 individuals were recruited. The sample was primarily Black/African American (82 %) with a mean age of 40. Patients who attended a healthcare appointment were screened for substance use via the AUDIT and DAST. Patients were eligible for the study if they scored 8 or higher on the AUDIT, were using only marijuana and scored 2 or higher on the DAST, or were using other illicit drugs and scored 1 or higher on the DAST. Participants were randomly assigned to receive one-session BI/RT, or two to six sessions of brief intervention that incorporated elements of motivational enhancement therapy and cognitive-behavioral therapy (BI/RT+). Both interventions were delivered by behavioral health consultants at the FQHCs. Participants completed follow-up assessments every 3 months for 1 year. Primary outcome variables included substance use treatment sessions attended and days of substance use. Secondary outcomes included measures of health, employment, legal, and psychiatric functioning and HIV risk behaviors. Additionally, we will conduct an economic evaluation examining cost-effectiveness and will analyze outcomes from a process evaluation examining patient and provider experiences. DISCUSSION: The ability of brief interventions to impact substance use has great potential, but research findings have been mixed. By conducting a large-scale randomized controlled trial in real-world health centers, this study will answer important questions about the effectiveness of expanded BIs for patients who screen positive for risky substance use in primary care. Trial registration NCT01751672

Developing a framework to incorporate real-world evidence in cancer drug funding decisions: the Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration.

BackgroundOncology therapy is becoming increasingly more expensive and challenging the affordability and sustainability of drug programmes around the world. When new drugs are evaluated, health technology assessment organisations rely on clinical trials to inform funding decisions. However, clinical trials are not able to assess overall survival and generalises evidence in a real-world setting. As a result, policy makers have little information on whether drug funding decisions based on clinical trials ultimately yield the outcomes and value for money that might be expected.ObjectiveThe Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration, consisting of researchers, recommendation-makers, decision makers, payers, patients and caregivers, are developing and testing a framework for Canadian provinces to generate and use real-world evidence (RWE) for cancer drug funding in a consistent and integrated manner.StrategyThe CanREValue collaboration has established five formal working groups (WGs) to focus on specific processes in the generation and use of RWE for cancer drug funding decisions in Canada. The different RWE WGs are: (1) Planning and Drug Selection; (2) Methods; (3) Data; (4) Reassessment and Uptake; (5) Engagement. These WGs are acting collaboratively to develop a framework for RWE evaluation, validate the framework through the multiprovince RWE projects and help to integrate the final RWE framework into the Canadian healthcare system.OutcomesThe framework will enable the reassessment of cancer drugs, refinement of funding recommendations and use of novel funding mechanisms by decision-makers/payers across Canada to ensure the healthcare system is providing clinical benefits and value for money