Desi Peneva

PURPOSE: This review summarizes the published evidence on the clinical impact of using next-generation sequencing (NGS) tests to guide management of patients with cancer in the United States.METHODS: We performed a comprehensive literature review to identify recent English language publications that presented progression-free survival (PFS) and overall survival (OS) of patients with advanced cancer receiving NGS testing.RESULTS: Among 6,475 publications identified, 31 evaluated PFS and OS among subgroups of patients who received NGS-informed cancer management. PFS and OS were significantly longer among patients who were matched to targeted treatment in 11 and 16 publications across tumor types, respectively.CONCLUSION: Our review indicates that NGS-informed treatment can have an impact on survival across tumor types.

Cost‐effectiveness is traditionally treated as a static estimate driven by clinical trial efficacy and drug price at launch. Prior studies suggest that cost‐effectiveness varies over the drug's lifetime. We examined the impact of “learning by doing,” one of the least studied drivers of changes in cost‐effectiveness across the product life cycle. We combined time‐series trends in effectiveness over time by cancer regimen using the Surveillance, Epidemiology, and End Results‐Medicare database. We estimated the time‐varying effects of treatments in colorectal and pancreatic cancer over their life cycle, including FOLFOX (leucovorin, 5‐fluorouracil, and oxaliplatin) and gemcitabine, on survival of patients. Mean prices over time by strength and dosage form were calculated using historical wholesale acquisition costs. We found consistent downward trends in the mortality hazard ratios, which suggest that effectiveness improves over time. In the case of first‐line FOLFOX for colorectal cancer, the implied incremental cost‐effectiveness ratio based on the observational data fell from $610,000 per life year gained in 2004 to $27,000 per life year gained in 2011. Cost‐effectiveness estimated at launch is unlikely to be representative of cost‐effectiveness over the drug's lifetime. In the drugs studied, the impact of time‐varying clinical effectiveness dominated the impact of changing prices overtime.

Background: Over 6 million Americans have heart failure, and 1 in 8 deaths included heart failure as a contributing cause in 2016. Lifestyle changes and adherence to diet and exercise regimens are important in limiting disease progression. Health coaching and public commitment are two interactive communication strategies that may improve self-management of heart failure. Objective: This study aimed to conduct patient focus groups to gain insight into how best to implement health coaching and public commitment strategies within the heart failure population. Methods: Focus groups were conducted in two locations. We studied 2 patients in Oakland, California, and 5 patients in Los Angeles, California. Patients were referred by local cardiologists and had to have a diagnosis of chronic heart failure. We used a semistructured interview tool to explore several patient-centered themes including medication adherence, exercise habits, dietary habits, goals, accountability, and rewards. We coded focus group data using the a priori coding criteria for these domains. Results: Medication adherence barriers included regimen complexity, forgetfulness, and difficulty coping with side effects. Participants reported that they receive little instruction from care providers on appropriate exercise and dietary habits. They also reported personal and social obstacles to achieving these objectives. Participants were in favor of structured goal setting, use of online social networks, and financial rewards as a means of promoting health lifestyles. Peers were viewed as better motivating agents than family members. Conclusions: An active communication framework involving dissemination of diet- and exercise-related health information, structured goal setting, peer accountability, and financial rewards appears promising in the management of heart failure.

Pediatric multiple sclerosis is associated with challenges in prompt diagnosis and uncertainty regarding optimal treatment. This review aimed to identify treatment guidelines or consensus statements for pediatric patients with multiple sclerosis, US Food and Drug Administration (FDA)–approved treatment options for pediatric multiple sclerosis, and any randomized controlled trials and observational studies examining available pharmacologic treatments in the pediatric multiple sclerosis population. Literature searches were performed in MEDLINE (1946-2016), EMBASE (1974-2016), and the Cochrane Central Register of Controlled Trials to identify treatment guidelines or consensus statements, pediatric multiple sclerosis treatment approvals, and randomized controlled trials and observation studies that examine the safety and effectiveness of available disease-modifying therapies. Only 3 consensus statements provided recommendations for pharmacologic treatments for children, all 3 published before the most recent revisions of the pediatric multiple sclerosis diagnostic guidelines. Despite the changes to the clinical landscape of pediatric multiple sclerosis with the introduction of diagnostic guidelines, fingolimod is the only FDA-approved treatment for pediatric multiple sclerosis in the United States. The effectiveness and safety of other disease-modifying therapies suggested by consensus statements have been reported in relatively small prospective and retrospective observational studies. Clinical evidence from a recently completed randomized controlled trial and future global registries can inform treatment decisions for the pediatric multiple sclerosis population.

We investigated the effect of changes to state AIDS Drug Assistance Programs (ADAP) policies, which govern access to antiretroviral therapy (ART), on clinical and economic outcomes among low-income people living with HIV/AIDS. Retrospective analyses of ART access were conducted on state ADAP policies, using data from ADAP Monitoring Reports and Kaiser Family Foundation from 2006 to 2010. We found stricter eligibility requirements reduce the number of HIV-positive individuals with ART access through ADAP, and decreased ART use increases mortality by 2.67 quality-adjusted life years (QALYs) per beneficiary. If the ADAP income eligibility cutoff were decreased by 50 percentage points in each state, 4,626 individuals would lose ART access nationwide. Based on a $22,143 cost/QALY, this policy would save $274 million in health care expenditures (2012 dollars), but result in 12,352 QALYs lost, valued at $1.2 billion. Therefore, states should exercise caution in restricting programs that increase ART access for low-income people living with HIV/AIDS.