Sustainable Financing of Innovative Therapies: A Review of Approaches

The process of innovation is inherently complex, and it occurs within an even more complex institutional environment characterized by incomplete information, market power, and externalities. There are therefore different competing approaches to supporting and financing innovation in medical technologies, which bring their own advantages and disadvantages. This article reviews value- and cost-based pricing, as well direct government funding, and cross-cutting institutional structures. It argues that performance-based risk-sharing agreements are likely to have little effect on the sustainability of financing; that there is a role for cost-based pricing models in some situations; and that the push towards longer exclusivity periods is likely contrary to the interests of industry

Implementing Community-Created Self-Management Support Tools in Primary Care Practices: Multimethod Analysis From the INSTTEPP Study

Purpose: With one-half of Americans projected to be living with at least one chronic condition before 2020, enhancing patient self-management support (SMS) may improve health-related behaviors and clinical outcomes. Routine SMS implementation in primary care settings is difficult. Little is known about the practice conditions required for successful implementation of SMS tools. Methods: Four primary care practice-based research networks (PBRNs) recruited 16 practices to participate in a boot camp translation process to adapt patient-centered SMS tools. Boot camp translation sessions were held over a 2-month period with 2 patients, a clinician, and a care manager from each practice. Qualitative case comparison and qualitative comparative analysis were used to examine practice conditions needed to implement SMS tools. The Consolidated Framework for Implementation Research guided data collection and analysis. Results: Four different practice conditions affected the implementation of new SMS tools: functional practice organization; system that enables innovation and change; presence of a visible, activated champion; and synergy and alignment of SMS changes with other work. Qualitative comparative analysis suggested that it was necessary to have an enabling system, a visible champion, and synergy for a practice to at least minimally implement the SMS tools. Sufficiency testing, however, failed to show robust consistency to satisfactorily explain conditions required to implement new SMS tools. Conclusions: To implement tailored self-management support tools relatively rapidly, the minimum necessary conditions include a system that enables innovation and change, presence of a visible champion, and alignment of SMS changes with other work; yet, these alone are insufficient to ensure successful implementation

Ontogenic changes in hematopoietic hierarchy determine pediatric specificity and disease phenotype in fusion oncogene-driven myeloid leukemia

Fusion oncogenes are prevalent in several pediatric cancers, yet little is known about the specific associations between age and phenotype. We observed that fusion oncogenes, such as ETO2–GLIS2, are associated with acute megakaryoblastic or other myeloid leukemia subtypes in an age-dependent manner. Analysis of a novel inducible transgenic mouse model showed that ETO2–GLIS2 expression in fetal hematopoietic stem cells induced rapid megakaryoblastic leukemia whereas expression in adult bone marrow hematopoietic stem cells resulted in a shift toward myeloid transformation with a strikingly delayed in vivo leukemogenic potential. Chromatin accessibility and single-cell transcriptome analyses indicate ontogeny-dependent intrinsic and ETO2–GLIS2-induced differences in the activities of key transcription factors, including ERG, SPI1, GATA1, and CEBPA. Importantly, switching off the fusion oncogene restored terminal differentiation of the leukemic blasts. Together, these data show that aggressiveness and phenotypes in pediatric acute myeloid leukemia result from an ontogeny-related differential susceptibility to transformation by fusion oncogenes. SIGNIFICANCE: This work demonstrates that the clinical phenotype of pediatric acute myeloid leukemia is determined by ontogeny-dependent susceptibility for transformation by oncogenic fusion genes. The phenotype is maintained by potentially reversible alteration of key transcription factors, indicating that targeting of the fusions may overcome the differentiation blockage and revert the leukemic state

The burden of childhood atopic dermatitis in the primary care setting: a report from the Meta-LARC Consortium

Background: Little is known about the burden of AD encountered in U.S. primary care practices and the frequency and type of skin care practices routinely used in children. Objectives: To estimate the prevalence of AD and allergic comorbidities in children 0-5 years attending primary care practices in the U.S. and to describe routine skin care practices used in this population. Design: A cross-sectional survey study of a convenience sample of children under the age of 5 attending primary care practices for any reason. Setting: Ten primary care practices in five U.S. states.Results: Amongst 652 children attending primary care practices, the estimated prevalence of ever having AD was 24 % (95% CI= 21-28) ranging from 15% among those under the age of one to 38% among those aged 4- 5 years. The prevalence of comorbid asthma was higher among AD participants compared to those with no AD, 12% and 4%, respectively (p less than 0.001). Moisturizers with high water:oil ratios were most commonly used (i.e., lotions) in the non-AD population, whereas moisturizers with low water:oil content (i.e. ointments) most common when AD was present. Conclusions: Our study found a large burden of AD in the primary care practice setting in the U.S. The majority of households reported skin care practices in children without AD that may be detrimental to the skin barrier such as frequent bathing and the routine use of moisturizers with high water: oil ratios. Clinical trials are needed to identify which skin care practices are optimal for reducing the significant risk of AD in the community

Collaborative research between clinicians and researchers: a multiple case study of implementation

<p>Abstract</p> <p>Background</p> <p>Bottom-up, clinician-conceived and directed clinical intervention research, coupled with collaboration from researcher experts, is conceptually endorsed by the participatory research movement. This report presents the findings of an evaluation of a program in the Veterans Health Administration meant to encourage clinician-driven research by providing resources believed to be critical. The evaluation focused on the extent to which funded projects: maintained integrity to their original proposals; were methodologically rigorous; were characterized by collaboration between partners; and resulted in sustained clinical impact.</p> <p>Methods</p> <p>Researchers used quantitative (survey and archival) and qualitative (focus group) data to evaluate the implementation, evaluation, and sustainability of four clinical demonstration projects at four sites. Fourteen research center mentors and seventeen clinician researchers evaluated the level of collaboration using a six-dimensional model of participatory research.</p> <p>Results</p> <p>Results yielded mixed findings. Qualitative and quantitative data suggested that although the process was collaborative, clinicians' prior research experience was critical to the quality of the projects. Several challenges were common across sites, including subject recruitment, administrative support and logistics, and subsequent dissemination. Only one intervention achieved lasting clinical effect beyond the active project period. Qualitative analyses identified barriers and facilitators and suggested areas to improve sustainability.</p> <p>Conclusions</p> <p>Evaluation results suggest that this participatory research venture was successful in achieving clinician-directed collaboration, but did not produce sustainable interventions due to such implementation problems as lack of resources and administrative support.</p

How to Translate Self-Management Support Tools Into Clinical Practice

Purpose: Patient self-management is an inevitable part of the work of being a patient, and self-management support (SMS) has become increasingly important in chronic disease management. However, the majority of SMS resources available in the Agency for Healthcare Research and Quality SMS Resource Library were developed without explicit collaboration between clinicians and patients. Methods: Translation of SMS tools derived from the library into primary care practices occurred utilizing boot camp translation in four different practice-based research networks (PBRNs). The typical model of boot camp translation was adapted for the purpose of the Implementing Networks’ Self-management Tools Through Engaging Patients and Practices (INSTTEPP) study to develop SMS tools for implementation in the participating practices. Clinicians, clinic staff members, and patients were involved throughout the translation process. Existing resources from the SMS library were reviewed and adapted by each boot camp translation group to create tools unique to the patients in each network. Results: There was no preexisting resource within the library that was deemed suitable for implementation without modification. Each network adapted tools from the SMS library to create different products. Common themes emerged from each network’s translation process that highlighted the importance of patient engagement in the translation process. Boot camp translation, in conjunction with PBRNs, can be implemented to adapt SMS tools for implementation in member practices. Conclusions: Boot camp translation with a combination of practices and patients can be implemented to facilitate a process of local adaptation that improves the local applicability of SMS tools in primary care clinics

Adapting Boot Camp Translation Methods to Engage Clinician/Patient Research Teams Within Practice-Based Research Networks

Purpose: Boot camp translation is a proven process to engage community members and health professionals in translating and disseminating evidence-based “best practices” models for health prevention and chronic illness care. Primary care practice improvement studies, particularly involving patient-driven change, as seen with self-management support (SMS), require engaged practice teams that include patients. Models of engagement such as boot camp translation may be effective. Methods: Four geographically dispersed practice-based research networks (PBRNs) from the Meta-LARC consortium engaged 16 practices to form SMS implementation teams involving a clinician, care manager, and 2 patients in each team. Our study adapted the boot camp translation model to engage the implementation teams in describing patient SMS, studying the Agency for Healthcare Research and Quality’s SMS Resource Library, and adapting and implementing self-management tools at each practice site. Testimonials and quotes were collected across the 4 PBRNs through a facilitated brainstorming discussion and consensus model at each PBRN kickoff meeting to address the focused question, “What do patients want and need in order to self-manage their chronic illnesses?” Results: Testimonials collected across the 4 PBRNs and participation levels indicated there was a high degree of engagement in the boot camp translation process across the PBRNs and the practices. Each PBRN developed themes expressed by patients and the practices regarding what patients want and need to self-manage their illnesses. Each practice selected, adapted, and implemented an SMS tool. Conclusions: Results suggest that adapted boot camp translation was effective in guiding multiple practices to implement self-management support tools for the INSTTEPP trial. Additional study of the adapted boot camp translation process in practice quality improvement and practice redesign studies is needed

Impact of a Boot Camp Translation Intervention on Self-Management Support in Primary Care

Purpose: Self-management support (SMS) is a pillar of the well-established chronic care model and a key component of improving outcomes for patients with chronic illnesses. The Implementing Networks’ Self-management Tools Through Engaging Patients and Practices (INSTTEPP) trial sought to determine whether a boot camp translation process could assist small to medium-sized primary care practices with care managers implement SMS tools. Methods: INSTTEPP used a stepped-wedge design across 16 practices from 4 practice-based research networks over 12 months. Each network completed a 2-month boot camp translation for creating SMS tools with 16 participants (2 patients, a clinician, and a care manager from each of 4 practices) and subsequent implementation. Outcome measures for patients were the Patient Activation Measure (PAM), self-rated health, and Patient Assessment of Chronic Illness Care (PACIC) process-of-care items at baseline, 1 and 2 months. Clinician Support for Patient Activation Measure (CS-PAM) and theory of planned behavior outcomes were assessed at 5 points over 10 months for clinicians and staff. Results: A total of 297 patients and 89 practice staff and clinicians completed surveys during the study. Over successive 2-month sampling periods, intervention patients experienced greater improvement in PACIC process of care and self-rated health compared to control patients (P 0.10 for all) were not significantly different. Conclusions: Significant effects on process of care and self-rated health are evidence that the boot camp translation intervention impacted SMS. A larger trial with a typical 6-month boot camp intervention may show significant effects on other outcomes

Influence of patient symptoms and physical findings on general practitioners' treatment of respiratory tract infections: a direct observation study

BACKGROUND: The high rate of antibiotic prescriptions general practitioners (GPs) make for respiratory tract infections (RTI) are often explained by non-medical reasons e.g. an effort to meet patient expectations. Additionally, it is known that GPs to some extent believe in the necessity of antibiotic treatment in patients with assumed bacterial infections and therefore attempt to distinguish between viral and bacterial infections by history taking and physical examination. The influence of patient complaints and physical examination findings on GPs' prescribing behaviour was mostly investigated by indirect methods such as questionnaires. METHODS: Direct, structured observation during a winter "cough an cold period" in 30 (single handed) general practices. All 273 patients with symptoms of RTI (age above 14, median 37 years, 51% female) were included. RESULTS: The most frequent diagnoses were 'uncomplicated upper RTI/common cold' (43%) followed by 'bronchitis' (26%). On average, 1.8 (95%-confidence interval (CI): 1.7–2.0) medicines per patient were prescribed (cough-and-cold preparations in 88% of the patients, antibiotics in 49%). Medical predictors of antibiotic prescribing were pathological findings in physical examination such as coated tonsils (odds ratio (OR) 15.4, 95%-CI: 3.6–66.2) and unspecific symptoms like fatigue (OR 3.1, 95%-CI 1.4–6.7), fever (OR 2.2, 95%-CI: 1.1–4.5) and yellow sputum (OR 2.1, 95%-CI: 1.1–4.1). Analysed predictors explained 70% of the variance of antibiotic prescribing (R(2 )= 0,696). Efforts to reduce antibiotic prescribing, e.g. recommendations for self-medication, counselling on home remedies or delayed antibiotic prescribing were rare. CONCLUSIONS: Patient complaints and pathological results in physical examination were strong predictors of antibiotic prescribing. Efforts to reduce antibiotic prescribing should account for GPs' beliefs in those (non evidence based) predictors. The method of direct observation was shown to be accepted both by patients and GPs and offered detailed insights into the GP-patient-interaction