Early access for innovative oncology medicines: a different story in each nation.

BACKGROUND: As innovative oncology medicines are rapidly developed, there is increasing pressure on payers to offer patients timely access to life-saving therapies. The uncertainty surrounding these therapies when phase III clinical trials are pending has necessitated new, adapted pathways to market access, with timelines that greatly vary by country. Understanding differences between pathways may identify opportunities to expedite patient access universally. OBJECTIVES: To describe early access pathways for new oncology medicines among selected countries with established health technology assessment (HTA) frameworks and publicly funded health systems, with a special focus on real-world evidence (RWE). METHODS: We reviewed the HTA agency websites of the selected OECD countries: National Institute for Health and Care Excellence (NICE) for England and Wales; Haute Autorité de Santé (HAS) for France; IQWiG and G-BA for Germany; Agenzia Italiana del Farmaco (AIFA) for Italy; Pharmaceutical Benefits Advisory Committee (PBAC) for Australia; and CADTH and Institut National d'Excellence en Santé et Services Sociaux (INESSS) for Canada as the primary source of evidence. RESULTS: Processes for early patient access to innovative oncology therapies varied across selected countries; however, most countries have an established pathway for publicly funded early access (England and Wales, France, Germany, Italy, and Australia). The utilization of RWE to support earlier access (coverage with evidence) also varied by country, with some HTA organizations being actively engaged in these agreements (NICE, AIFA, and HAS) and others having no established processes in place (G-BA and CADTH/INESSS). CONCLUSIONS: This review of early access pathways for novel oncology medicines found substantial variability between countries of interest. Coverage with evidence frameworks may provide a unique opportunity for industry and payers to collaborate on earlier access to innovative cancer therapies with life-saving potential.

Understanding real-world treatment patterns and clinical outcomes in AL amyloidosis patients diagnosed in Canada: A population-based cohort study.

Amyloid light chain (AL) amyloidosis is a rare and chronic bone marrow disorder. Existing claims data can be used to help understand the real-world treatment patterns and outcomes of this patient population. Various population-based administrative databases in Alberta, Canada were queried from 2010 to mid-2019 to identify cases of AL amyloidosis. Baseline patient and disease characteristics, sequencing of pharmacologic therapies, overall survival, and healthcare resource utilization were evaluated. A total of 215 individuals with AL amyloidosis were included. Among patients diagnosed between 2012 and 2019, 149 (85.1%) initiated first-line, 67 (38.3%) initiated second-line, 22 (12.6%) initiated third-line, and 11 (6.3%) initiated fourth-line systemic therapy. In the first-line setting, 99/149 (66.4%) received bortezomib, cyclophosphamide, and dexamethasone (CyBorD) and 21/149 (14.1%) received another bortezomib-based regimen. Survival from time of diagnosis improved over time, with a median overall survival of 25.8 months (95% CI: 9.8, 57.1) for individuals diagnosed in 2010-2011 versus 52.1 months (95% CI: 25.6, NA) for those diagnosed in 2012-2019. Despite this improvement, the proportion of individuals diagnosed in 2012-2019 who survived beyond five-years remained low (5-year survival: 48.4%; 95% CI: 40.9, 57.2) which highlights an unmet need for more efficacious therapies.

An Observational Case Study of Near-peer Teaching in Medical and Pharmacy Experiential Training.

Objective. To compare peer teaching in a medical and a pharmacy clinical teaching unit and to provide suggestions for future research in pharmacy near-peer teaching. Methods. This exploratory observational study used principles of ethnographic methodology for data collection and analysis. Observations were collected in a large downtown teaching hospital. An average of 4-6 hours per day were spent observing a team of medical trainees from the Faculty (School) of Medicine in the general internal medicine (unit for two weeks, followed by a team of pharmacy trainees in an ambulatory hemodialysis (HD) unit for two weeks. Data was collected through field notes and informal interviews that were audiotaped and subsequently transcribed. Data was interpreted by the observer and reviewed weekly by two impartial pharmacists. Results. Five major themes emerged: (1) influence of peer teaching hierarchy; (2) educational distance between peer learners and teachers; (3) effect of the clinical teaching unit size on peer learning; (4) trainees' perception of their teaching role in the clinical teaching unit; and (5) influence of daily schedule and workload on peer teaching. As opposed to pharmacy, a hierarchy and pyramidal structure of peer teaching was observed in medical experiential training. There appeared to be no effect of educational distance on near peer teaching; however, perception of teaching role and influence of daily schedule affected near-peer teaching. Conclusion. Through initial comparisons of medical and pharmacy clinical teaching units, this study provides a reflection of elements that may be necessary to successfully implement near-peer teaching in pharmacy experiential training. Future studies in this area should assess learning outcomes and participant satisfaction, preceptor workload, and impact on patient care.