Adalimumab, etanercept and ustekinumab for treating plaque psoriasis in children and young people: systematic review and economic evaluation

Background: Psoriasis is a chronic inflammatory disease that predominantly affects the skin. Adalimumab (HUMIRA®, AbbVie, Maidenhead, UK), etanercept (Enbrel®, Pfizer, New York, NY, USA) and ustekinumab (STELARA®, Janssen Biotech, Inc., Titusville, NJ, USA) are the three biological treatments currently licensed for psoriasis in children. Objective: To determine the clinical effectiveness and cost-effectiveness of adalimumab, etanercept and ustekinumab within their respective licensed indications for the treatment of plaque psoriasis in children and young people. Data sources: Searches of the literature and regulatory sources, contact with European psoriasis registries, company submissions and clinical study reports from manufacturers, and previous National Institute for Health and Care Excellence (NICE) technology appraisal documentation. Review methods: Included studies were summarised and subjected to detailed critical appraisal. A network meta-analysis incorporating adult data was developed to connect the effectiveness data in children and young people and populate a de novo decision-analytic model. The model estimated the cost-effectiveness of adalimumab, etanercept and ustekinumab compared with each other and with either methotrexate or best supportive care (BSC), depending on the position of the intervention in the management pathway. Results: Of the 2386 non-duplicate records identified, nine studies (one randomised controlled trial for each drug plus six observational studies) were included in the review of clinical effectiveness and safety. Etanercept and ustekinumab resulted in significantly greater improvements in psoriasis symptoms than placebo at 12 weeks’ follow-up. The magnitude and persistence of the effects beyond 12 weeks is less certain. Adalimumab resulted in significantly greater improvements in psoriasis symptoms than methotrexate for some but not all measures at 16 weeks. Quality-of-life benefits were inconsistent across different measures. There was limited evidence of excess short-term adverse events; however, the possibility of rare events cannot be excluded. The majority of the incremental cost-effectiveness ratios for the use of biologics in children and young people exceeded NICE’s usual threshold for cost-effectiveness and were reduced significantly only when combined assumptions that align with those made in the management of psoriasis in adults were adopted. Limitations: The clinical evidence base for short- and long-term outcomes was limited in terms of total participant numbers, length of follow-up and the absence of young children. Conclusions: The paucity of clinical and economic evidence to inform the cost-effectiveness of biological treatments in children and young people imposed a number of strong assumptions and uncertainties. Health-related quality-of-life (HRQoL) gains associated with treatment and the number of hospitalisations in children and young people are areas of considerable uncertainty. The findings suggest that biological treatments may not be cost-effective for the management of psoriasis in children and young people at a willingness-to-pay threshold of £30,000 per quality-adjusted life-year, unless a number of strong assumptions about HRQoL and the costs of BSC are combined. Registry data on biological treatments would help determine safety, patterns of treatment switching, impact on comorbidities and long-term withdrawal rates. Further research is also needed into the resource use and costs associated with BSC. Adequately powered randomised controlled trials (including comparisons against placebo) could substantially reduce the uncertainty surrounding the effectiveness of biological treatments in biologic-experienced populations of children and young people, particularly in younger children. Such trials should establish the impact of biological therapies on HRQoL in this population, ideally by collecting direct estimates of EuroQol-5 Dimensions for Youth (EQ-5D-Y) utilities. Study registration: This study is registered as PROSPERO CRD42016039494. Funding: The National Institute for Health Research Health Technology Assessment programme

Integrated Personal Health Records: Transformative Tools for Consumer-Centric Care

<p>Abstract</p> <p>Background</p> <p>Integrated personal health records (PHRs) offer significant potential to stimulate transformational changes in health care delivery and self-care by patients. In 2006, an invitational roundtable sponsored by Kaiser Permanente Institute, the American Medical Informatics Association, and the Agency for Healthcare Research and Quality was held to identify the transformative potential of PHRs, as well as barriers to realizing this potential and a framework for action to move them closer to the health care mainstream. This paper highlights and builds on the insights shared during the roundtable.</p> <p>Discussion</p> <p>While there is a spectrum of dominant PHR models, (standalone, tethered, integrated), the authors state that only the integrated model has true transformative potential to strengthen consumers' ability to manage their own health care. Integrated PHRs improve the quality, completeness, depth, and accessibility of health information provided by patients; enable facile communication between patients and providers; provide access to health knowledge for patients; ensure portability of medical records and other personal health information; and incorporate auto-population of content. Numerous factors impede widespread adoption of integrated PHRs: obstacles in the health care system/culture; issues of consumer confidence and trust; lack of technical standards for interoperability; lack of HIT infrastructure; the digital divide; uncertain value realization/ROI; and uncertain market demand. Recent efforts have led to progress on standards for integrated PHRs, and government agencies and private companies are offering different models to consumers, but substantial obstacles remain to be addressed. Immediate steps to advance integrated PHRs should include sharing existing knowledge and expanding knowledge about them, building on existing efforts, and continuing dialogue among public and private sector stakeholders.</p> <p>Summary</p> <p>Integrated PHRs promote active, ongoing patient collaboration in care delivery and decision making. With some exceptions, however, the integrated PHR model is still a theoretical framework for consumer-centric health care. The authors pose questions that need to be answered so that the field can move forward to realize the potential of integrated PHRs. How can integrated PHRs be moved from concept to practical application? Would a coordinating body expedite this progress? How can existing initiatives and policy levers serve as catalysts to advance integrated PHRs?</p

Driving a decade of change: HIV/AIDS, patents and access to medicines for all

Since 2000, access to antiretroviral drugs to treat HIV infection has dramatically increased to reach more than five million people in developing countries. Essential to this achievement was the dramatic reduction in antiretroviral prices, a result of global political mobilization that cleared the way for competitive production of generic versions of widely patented medicines

Accelerating the adoption of preventive health services: building new partnerships and community commitment : proceeding from a conference

Executive summary -- Introduction -- Accelerating the adoption of preventive health services -- Conclusion -- Endnotes -- Appendices.Larry Stepnick, Steven Findlay.Cover title."The National Institute for Health Care Management (NIHCM) Research and Educational Foundation convened the conference on which this publication is based. The Centers for Disease Control and Prevention (CDC) sponsored the conference. It was held September 26-27, 2002 in Washington D.C."--Credits.Also available via the World Wide Web.System requirements: Adobe Acrobat Reader.Includes bibliographical references (p. 31)