Radiation therapy: Regulatory framework and constraints.

The purpose of this article is to describe the regulatory framework of the radiotherapy practice in France.

Plain Language Summaries of Clinical Trial Results: A Preliminary Study to Assess Availability of Easy-to-Understand Summaries and Approaches to Improving Public Engagement.

BACKGROUND: Easy-to-understand, stand-alone factual summaries of clinical trial results have the potential to improve public understanding of and engagement with pharmaceutical research. The European Clinical Trial Regulation (EU) No. 536/2014 is a major regulatory initiative that will result in a large number of such plain language summaries (PLSs) posted in the public domain. Today, however, little is known about the extent to which PLSs are written and are available to the general public. OBJECTIVES: This preliminary study assessed (i) 20 top pharmaceutical companies' positions on improving transparency and commitment to disclosing trial result summaries in an easy-to-understand format and (ii) the availability of such summaries in the public domain and the ease of locating them via general web searches. METHODS: The availability of PLSs in the public domain was estimated based on the number of EudraCT technical result summaries in four disease areas: chronic obstructive pulmonary disease, asthma, meningitis, and influenza. The likelihood of PLSs being easy to find through internet search engine queries by members of the public was assessed using Google. RESULTS: All 20 sponsors had committed to improve clinical trial transparency, 17 committed to sharing PLSs with trial participants, and 14 had at least one PLS available in the public domain. A total of 99 clinical studies in these four disease areas had technical summaries posted on EudraCT between 1 January 2017 and 30 June 2020. Of these 99, 14 studies had PLSs in the public domain. A total of 12 of 14 PLSs were directly captured by search engine. However, the sponsor trial identifier or EudraCT number had to be included in the search term to locate them. Generic search terms resulted in large volumes of non-relevant results. CONCLUSION: Despite the progressive movement towards clinical trial transparency, easily accessible PLSs on clinical trials are currently scarce. The provision of a European mandate and framework for non-technical result summaries by Regulation (EU) 536/2014 will be a major step to bring about positive change.

More patient and public involvement in healthcare research will help to speed the process of making new medicines. This is known by both the regulators and the healthcare industry. The healthcare industry wants to make more information on human research studies available to patients and the public. One way to help achieve this is to write simple summaries of clinical study results. Here, we use the term plain language summary (PLS) to describe them. The PLS allows people to understand human research studies more clearly. A new law will soon make it necessary to write a PLS for every clinical study done in Europe. But, today, is the PLS being used to inform the public about clinical research studies? And what is its potential? We found only  a few researchers have already begun to write PLSs. PLSs on most studies are not available to the public. Even those PLSs on public websites are very hard to find through a Google search. To better understand the potential of PLSs we are doing more research. This research will look at what the public wants to know about these studies and how they will retrieve this information.

Utilization of Screening Mammograms in the Medicare Population Before and After the Affordable Care Act Implementation.

Objective: This study examined screening mammograms in women aged 65 to 74 years and 75+ years before and after the Affordable Care Act (ACA) implementation. Method: This repeated cross-sectional study of community-dwelling women age 65+ years without a history of breast cancer or mastectomy utilized the Medicare Current Beneficiary Survey and Medicare fee-for-service claims data from 2001 to 2013. We used covariate-adjusted logistic regression with generalized estimating equations, stratified by age group. Results: The adjusted odds of screening mammograms in women aged 65-74 (n = 742) and 75+ years (n = 681) were lower in 2013 (odds ratio [OR]: 0.75, 95% confidence interval [CI]: [0.67, 0.83]; OR: 0.67, 95% CI: [0.60, 0.75], respectively) than the odds of screening mammograms in 2001. Discussion: Annual screening mammograms decreased in women aged 65 to 74 years and 75+ years, despite increased access from the ACA implementation. Future research as to why women are no longer receiving screening mammograms, such as changes in physician specialty guidelines, is warranted.

Reflections on the ISPOR Special Task Force on U.S. Value Frameworks: Implications of a Health Economics Approach for Managed Care Pharmacy.

In 2016, The Professional Society for Health Economics and Outcomes Research (ISPOR) formed a special task force (STF) to review approaches and methods to support the definition and use of high-quality U.S. value frameworks. As the leadership group of that initiative, we present our perspective, focusing on implications for the managed care pharmacy community. Our reflections are organized by 9 key observations and conclude with a summary recommendation. We begin by emphasizing the importance of distinguishing among "perspectives" and "decision contexts." Possible perspectives include patient, payer, provider, health care sector, and societal. Decision contexts range from formulary inclusion to guideline development to clinical shared decision making, and multiple perspectives can be taken on each of these decisions. The STF focused on value in the context of including a new medicine in a formulary and, thus, health plan, using a health economics approach that compares marginal benefit (gross value) and marginal (opportunity) cost, yielding the net value. Health care is unique compared with other markets. While economists often use market purchases as indicators of value, they also recognize that this does not work well in health care, since most patent-protected drugs are covered by insurance. To assess the likely health and economic impact, health economists often employ cost-effectiveness analysis, using the quality-adjusted life-year (QALY), a metric that combines mortality and morbidity into a single preference-based index. We strongly endorse the STF's recommendation that payers should use the cost-per-QALY metric as a starting point. However, like the STF, and many of those stakeholders who provided input, we recognize that this metric has some limitations in theory and in practice. Nonetheless, the cost-per-QALY metric is a pragmatic tool that can be augmented to address some of its limitations by integrating other elements of value, particularly those related to uncertainty, such as financial risk protection, health risk protection, the value of hope, real option value, and the value of knowing. The resulting adjusted ratio can be compared with a willingness-to-pay threshold or combined in a measure of net monetary benefit. Alternatively, the array of elements can be valued using multi-criteria decision analysis. We end with the key recommendation that further development and testing of these promising approaches is needed to improve the deliberative process of health technology assessment. DISCLOSURES: No outside funding supported the writing of this article. The authors are leaders of the ISPOR Special Task Force on U.S. Value Frameworks. Willke is employed by ISPOR. Garrison and Neumann have nothing to disclose. The opinions expressed in this article should be considered as belonging only to the authors.

Chile's National Advisory Committee on Immunization (CAVEI): Evidence-based recommendations for public policy decision-making on vaccines and immunization.

A National Immunization Technical Advisory Group (NITAG) provides independent, evidence-based recommendations to the Ministry of Health for immunization programmes and policy formulation. In this article, we describe the structure, functioning and work processes of Chile's NITAG (CAVEI) and assess its functionality, quality of work processes and outputs, and integration of the committee into the Ministry of Health policy process using the Assessment tool for National Immunization Technical Advisory Groups. Among its strengths, CAVEI's administrative and work plasticity allows it to respond in a timely manner to the Ministry of Health's requests and proactively raise subjects for review. Representation of multiple areas of expertise within the committee makes CAVEI a robust and balanced entity for the development of evidence-based comprehensive recommendations. High ranking profile of the Secretariat structure furthers CAVEI's competences in policymaking and serves as a bridge between the committee and international initiatives in the field of immunizations.

LeRoy Walters's Legacy of Bioethics in Genetics and Biotechnology Policy.

LeRoy Walters was at the center of public debate about emerging biological technologies, even as "biotechnology" began to take root. He chaired advisory panels on human gene therapy, the human genome project, and patenting DNA for the congressional Office of Technology Assessment. He chaired the subcommittee on Human Gene Therapy for NIH's Recombinant DNA Advisory Committee. He was also a regular advisor to Congress, the executive branch, and academics concerned about policy governing emerging biotechnologies. In large part due to Prof. Walters, the Kennedy Institute of Ethics was one of the primary sources of talent in bioethics, including staff who populated policy and science agencies dealing with reproductive and genetic technologies, such as NIH and OTA. His legacy lies not only in his writings, but in those people, documents, and discussions that guided biotechnology policy in the United States for three decades.

United States Food and Drug Administration Advisory Committee outcomes and agency approval analysis from 2010 to 2015.

OBJECTIVES: This analysis sought to quantify voting behavior and other characteristics of advisory committee (AC) meetings and compare that with the U.S. Food and Drug Administration's (FDA) approval decisions from 2010 to 2015. METHODS: The analysis of the Center for Drug Evaluation and Research AC meetings was conducted using publicly available information from the FDA website and the sponsors' websites. RESULTS: There were 163 voting sessions, 207 votes, and 229 meetings. Voting questions assessed approval (63%), acceptable risk-benefit profile (19%), efficacy (8%), safety and efficacy (7%), and safety (3%). The AC voted in favor of approval 67% of the time and against approval 33% of the time, although it heavily favored one outcome when voting favorably or unfavorably. The FDA approval decision supported the committee's decision in 90% of cases. When such agreement did not occur, it was due to differences in clinical opinion (43%), manufacturing deficiencies (14%), lack of manufacturing data (14%), and a post-AC event (5%). There was insufficient information to determine why there was a differing opinion in 24% of cases. When FDA had a differing opinion, the agency typically did not approve a substance in which the committee recommended approval (81%). CONCLUSION: The results support past research examining the topic from 2001 to 2010. Voting patterns were relatively constant, and they generally heavily favored one outcome. The FDA's ultimate approval decision was in line with the AC vote the vast majority of cases. Any disagreement was usually due to FDA having a differing opinion regarding clinical importance, furthering the notion that AC insight is heavily considered but not the final determinant in agency action. This topic has importance in understanding pharmaceutical approval in the United States, and this has clinical practice implications.

Clinic, courtroom or (specialist) committee: in the best interests of the critically Ill child?

Law's processes are likely always to be needed when particularly intractable conflicts arise in relation to the care of a critically ill child like Charlie Gard. Recourse to law has its merits, but it also imposes costs, and the courts' decisions about the best interests of such children appear to suffer from uncertainty, unpredictability and insufficiency. The insufficiency arises from the courts' apparent reluctance to enter into the ethical dimensions of such cases. Presuming that such reflection is warranted, this article explores alternatives to the courts, and in particular the merits of specialist ethics support services, which appear to be on the rise in the UK. Such specialist services show promise, as they are less formal and adversarial than the courts and they appear capable of offering expert ethical advice. However, further research is needed into such services - and into generalist ethics support services - in order to gauge whether this is indeed a promising development.

A comparison of decisions to discharge committed psychiatric patients between treating physicians and district psychiatric committees: an outcome study.

BACKGROUND: The Israel Mental Health Act of 1991 stipulates a process for involuntary psychiatric hospitalization (IPH). A patient thus hospitalized may be discharged by either the treating psychiatrist (TP) or the district psychiatric committee (DPC). The decision rendered by the DPC is often at odds with the recommendation of the TP. Although much has been written about the ethical issues of restricting patients' rights and limiting their freedom, far less attention has been devoted to the psychiatric, medical, and social outcome of legal patient discharge against the doctor's recommendation. METHODS: In our study we examined the outcomes of the decisions made by the DPC using readmission data, an internationally recognized indicator of the quality of hospital care, and compared them to the outcomes of patients discharged by the TP. All IPH discharges resulting from the DPC's determination for the year 2013 (N = 972) were extracted from the Israel national register. We also collected all IPH discharges owing to the TP's decision for 2013 (N = 5788). We defined "failure" as readmission in less than 30 days, involuntary civil readmission in less than 180 days, and involuntary readmission under court order in less than 1 year. RESULTS: The rehospitalization pattern was compared in the two groups of patients discharged from their psychiatric hospitalization during 2013 (index discharges) and followed up individually for a year. We found a statistically significant difference between the DPC and the TP group for each of the time frames, with the DPC group returning to IPH much more frequently than the TP group. Using cross-sectional comparison with logistic regression adjusted for age, gender, diagnosis and length of hospitalization, we found the probability of a decision failure in the TP group was significantly less with an OR of 0.7 (95% CI .586-.863), representing a 30% adjusted decrease in the probability for failure in the TP group. CONCLUSIONS: The results we present show that the probability of decision "failure" (readmission) was found to be significantly higher in the DPC group than in the TP group. It is often assumed that IPH patients will fare better at home in their communities than in a protracted hospitalization. This is frequently the rationale for early discharge by the DPC (30.1 days vs. 75.9 DPC and TP groups, respectively). Our results demonstrate that this rationale may well be a faulty generalization.

The Australian Royal Commission into Institutional Responses to Child Sexual Abuse and the Roman Catholic Church.

The Australian Royal Commission into Institutional Responses to Child Sexual Abuse received more reports of sexual abuse of minors from victims of personnel from the Catholic Church than from any other source. It looked beyond the circumstances of the individual reports, to the response of Church leaders. It then took the inquiry to the more fundamental issue of the elements of the Church's structure and its unique culture that enabled sexual abuse and supported the hierarchy's counter-productive responses. This commentary looks at the structural and cultural aspects of the institutional Church most directly connected to sexual abuse by clerics and the ensuing cover-up and it examines their theological and historical foundations. The reality that sexual abuse by clerics was not only known but condoned and covered up cannot be justified but it can be explained in great part by the Church's justification for its own structure and the role of its clerics.

The impact of Australia's Royal Commission on child- and youth-serving organizations.

This commentary considers the impact to date of Australia's Royal Commission into Institutional Responses to Child Sexual Abuse on child- and youth-serving organizations, particularly its influence on organizations' efforts to create and maintain 'child safe, child friendly' cultures, policies and practices. Opportunities and challenges for organizational leaders are outlined. The commentary calls for more involvement by researchers in empirical research that is relevant to the causes and prevention of abuse in organizations, and for findings to be disseminated in ways that are useful to organizations.